The Sant Pau Research Institute (IR Sant Pau) will lead the innovative SPAiN project (Spanish Partnership for Autoimmune Neuropathies), a national initiative aimed at transforming the knowledge and care of autoimmune neuropathies (ANs), a group of rare diseases affecting the peripheral nervous system.

For its development, the project has been awarded €1,634,710.00 through the “PMPER-24 – Research Projects on Rare Diseases” call by the Carlos III Health Institute, as part of the Joint Missions of the Ministry of Health and the Ministry of Science, Innovation and Universities. A total of €20 million has been allocated to rare disease research projects, resulting in the approval of eight research projects across Spain, two of which are based in Catalonia: the IR Sant Pau project and another led by the Germans Trias i Pujol Research Institute (IGTP). This last project aims to create a national network to tackle the challenges of type 1 myotonic dystrophy, in collaboration with the INCLIVA Health Research Institute at the University Clinical Hospital of Valencia.

Under the leadership of Dr Luis Querol, researcher of the Neuromuscular Diseases Group at IR Sant Pau and neurologist at the Neuromuscular Unit of the Neurology Department at Sant Pau. The IR Sant Pau plays a central role in this national network. It brings together 17 reference centres across 9 autonomous communities. This leadership reflects the institute’s excellence as a neuromuscular reference centre in Spain and its commitment to translational research.

ANs, a group of rare diseases affecting the peripheral nervous system, pose a significant medical challenge due to their difficult diagnosis and the limitations of current treatments. These conditions include Guillain-Barré syndrome, chronic inflammatory demyelinating polyneuropathy (CIDP), and multifocal motor neuropathy. Although treatable, these diseases present diagnostic and therapeutic challenges that can compromise patients’ quality of life.

“These diseases pose numerous challenges. Their causes remain largely unknown, diagnosis is difficult, and while therapeutic options exist, they are limited and urgently need updating. SPAiN was created with the aim of transforming our understanding of these conditions and improving the lives of those affected,” explains Dr Querol.

The project will leverage cutting-edge technologies such as genomics, proteomics, and antigenomics to identify more precise biomarkers and explore new therapeutic pathways. These tools will enable personalised treatments and improved patient monitoring, thereby reducing the impact of these diseases on patients’ lives.

A direct impact on healthcare

One of SPAiN’s key pillars is ensuring that scientific advancements reach patients equitably across the entire country. Thanks to the network led by IR Sant Pau, any patient, regardless of their location, will be able to benefit from the expertise and resources of the country’s leading reference centres.

“Autoimmune neuropathies impact a small but dispersed population, making the creation of a national network crucial. At Sant Pau, we have worked to ensure that SPAiN reaches every corner of Spain, providing more equitable care and opportunities for patients to participate in cutting-edge research,” emphasises Dr Querol.

IR Sant Pau’s role as a national and international leader

IR Sant Pau’s leadership in this project is built on its long-standing role as a reference centre for autoimmune neuropathies. For years, the institute has been involved in international networks such as IGOS (International Guillain-Barré Syndrome Outcome Study) and INCBase (International CIDP Database). Now, by coordinating SPAiN, it seeks to drive advancements that will establish Spain as a global reference in this field.

“This project is a unique opportunity to showcase the expertise accumulated at Sant Pau and to expand our impact through a national network that is also connected to major international consortia. This demonstrates our institute’s commitment to scientific excellence and to improving patients’ lives,” states Dr Querol.

An investment in the future of personalised medicine

SPAiN will not only lay the foundations for new therapies and better diagnostics, but it will also enhance our understanding of the mechanisms behind these rare diseases. The project will run over the next two years, making use of existing resources and expanding the research and healthcare network.

“Although autoimmune neuropathies are rare diseases, their consequences can be devastating. SPAiN aims to break down barriers, creating tools that not only enhance diagnosis but also enable the development of more effective and accessible treatments,” concludes Dr Querol.

The Hypatia Mars association presented, on 28 January at the Modernist Building of Hospital Sant Pau, the second mission of female researchers of different ages and disciplines, which will take place from 2 to 15 February at the Mars Research Desert Station (MDRS) in the Utah desert, United States. During this mission, a new crew of analogue astronauts will live in isolation conditions, coinciding with the International Day of Women and Girls in Science, celebrated on 11 February. The project will once again have the collaboration of the Sant Pau Research Institute (IR Sant Pau) and Hospital Sant Pau, both of which already participated in the first mission.

What is the Hypatia II Project?

The Hypatia II project encompasses a series of initiatives aimed at exploring and overcoming the challenges posed by space exploration in terms of health, well-being, and sustainability from a female perspective. Within this framework, a project focused on menstrual health stands out, led by Dr Marina Martínez, a postdoctoral researcher in the Department of Geology at the Autonomous University of Barcelona (UAB), supported by a Margarita Salas grant. She is also a member of the Center for Advanced Sample Analysis of Astromaterials from the Moon and Beyond (Chip Shearer, PI) at SSERVI (NASA). In this project, she will collaborate with Dr Joaquim Calaf and Dr Josep Perelló, researchers from the Reproductive Health Group at IR Sant Pau and the Gynaecology and Obstetrics Service at Hospital Sant Pau.

This project addresses an often-overlooked issue: how to manage menstrual cyclicity during space missions, especially long-duration ones, such as the colonisation of the Moon or Mars. Currently, the suppression of the menstrual cycle through hormonal methods is a common practice among astronauts. However, this solution can lead to gender inequalities and negative implications for the female body.

Objectives of the Menstrual Health Project

The project is structured around three main areas of action aimed at integrating the perspective of female cyclicity in space exploration.

First, it seeks to make the female hormonal cycle visible and respected by incorporating this knowledge into the organisation of space missions. This includes adapting tasks, work rhythms, and environmental conditions to the specific needs of each phase of the crew members’ cycles.

Second, the project prioritises research and the implementation of sustainable solutions for menstrual management. Among these solutions, the use of the menstrual cup, developed in collaboration with AstroCup, stands out as an alternative that helps reduce waste, improve hygiene, and ensure greater comfort in microgravity environments.

Finally, the project explores the potential of menstrual blood as fertiliser through experiments with plants grown in space conditions. The goal is to assess its viability as a useful resource within an efficient recycling system aligned with the zero-waste principle.

An Initiative Within a Global Programme

This project is just one of the multiple research lines being developed within the Hypatia II programme, a multidisciplinary initiative that promotes a more inclusive, sustainable, and innovative approach to space exploration. The knowledge gained from these investigations will not only benefit space missions but also have direct applications on Earth in areas such as health, sustainability, and gender equality.

A study recently published by researchers from the Sant Pau Research Institute (IR Sant Pau) and the Stroke Unit of Sant Pau Hospital in Journal of Lipid Research provides new evidence on the essential role of the qualitative properties of lipoproteins, such as LDL and HDL, in the pathophysiology of cardiovascular diseases, including ischaemic stroke. The findings underscore the importance of going beyond traditional quantitative cholesterol levels to evaluate the risk of these pathologies.

Dr Sonia Benítez, a researcher in the Cardiovascular Biochemistry Research Group at IR Sant Pau and one of the study’s authors, emphasised that “it is not so much the amount of LDL or HDL as their quality that determines the residual risk of ischaemic stroke. This study confirms that some qualitative alterations in lipoproteins, such as the increased negative electric charge in LDL and HDL (LDL(-) or HDL(-)), could play a causal role in the progression of cardiovascular diseases.”

The relationship between ischaemic stroke and lipoproteins

Ischaemic stroke, one of the leading causes of mortality and disability worldwide, is often linked to carotid atherosclerosis. Approximately 20% of strokes are directly associated with the presence of atheromatous plaques in the carotid arteries, significantly increasing the risk of severe vascular events. Traditionally, the clinical management of these patients has focused on reducing LDL and HDL cholesterol levels. However, this new study from IR Sant Pau highlights that the qualitative characteristics of lipoproteins are also crucial in the development and progression of these diseases. This new perspective creates opportunities to innovatively address lipoprotein alterations and associated risks.

The study was conducted as an observational cohort study at Sant Pau Hospital between January 2016 and March 2019. The population studied included adult patients who had experienced anterior circulation ischaemic stroke and recently diagnosed carotid atherosclerosis, as well as a control group of healthy subjects. Lipoproteins were isolated from blood samples of 27 healthy subjects and 64 patients with carotid atherosclerosis seven days and one year after the stroke.

Alterations in qualitative properties

Seven days after the first stroke, LDL showed an increase in negative charge, an elevation of pro-inflammatory ceramides and triacylglycerols, and a decrease in phospholipids and cholesterol. A comprehensive lipidomic study of LDL was conducted in collaboration with Dr Öörni’s group at the Wihuri Research Institute in Helsinki. These LDL modifications are associated with inflammatory and atherogenic processes that increase the vulnerability of carotid plaques. Regarding HDL, protein composition alterations were identified, such as a reduction in apoA-I levels and an increase in apoA-II and apoC-III, impairing their antioxidant and anti-inflammatory properties and compromising their ability to prevent LDL modification and its inflammatory effect.

A notable aspect is that these qualitative alterations persist despite the early introduction of medications like statins. “This suggests that these modifications are deeply rooted in the patient’s pathophysiology. However, one year after the stroke, thanks to therapeutic interventions such as statins and antiplatelet agents, as well as possible lifestyle improvements, lipoproteins showed significant improvement,” adds Dr Sonia Benítez.

Thus, LDL became less prone to oxidation and aggregation, while HDL partially recovered its protective properties. Additionally, a reduction in LDL(-) and HDL(-) levels—electronegative lipoprotein subtypes with high atherogenic potential—was observed. “This may mean that therapeutic interventions can partially reverse the harmful effects of these alterations,” adds Dr Núria Puig, also a researcher in the Cardiovascular Biochemistry Group and the first author of the publication.

A paradigm shift

This research introduces a paradigm shift in understanding the functional role of lipoproteins in cardiovascular diseases, particularly in ischaemic stroke associated with carotid atherosclerosis. The findings underscore the need for an integrative approach to explore the qualitative properties of lipoproteins to identify patients at high risk of complications, even when their quantitative lipid levels appear normal.

This can help personalise treatments based on the specific characteristics of each patient and better tailor pharmacological and non-pharmacological interventions. The researchers also point towards developing therapeutic strategies aimed at modifying lipoprotein composition, with a potential positive impact on preventing cardiovascular complications. This includes reducing LDL(-) and HDL(-) through dietary changes, physical exercise, and more consistent adherence to statin treatments.

Clinical implications and future directions

However, the study has some limitations, such as the small sample size, which makes it difficult to generalise the results. This highlights the need for studies with larger cohorts to confirm the findings and analyse specific subgroups, such as by sex or type of therapeutic intervention. In this regard, Dr Pol Camps from the Stroke Unit, second author of the publication, and Dr Benítez have recently formed, along with other European groups, a consortium called BioStroke focused on stroke biomarker research, aiming to establish collaborations that will allow for expanding studies to larger patient cohorts.

According to Dr Benítez, “translational research in this field is key to bringing these findings into clinical practice. Our future goal is to develop tools to identify patients at higher risk and eventually design therapeutic strategies aimed at reversing these lipoprotein alterations.” This research opens a promising avenue for future clinical approaches that focus not only on traditional lipid levels but also on the qualitative properties of lipoproteins, thereby complementing current therapeutic strategies and reducing the risk of stroke recurrence and other cardiovascular complications.

Reference article:

Puig, N., Camps-Renom, P., Hermansson, M., Aguilera-Simón, A., Marín, R., Bautista, O., … Benitez, S. (2024). Alterations in LDL and HDL after an ischemic stroke associated with carotid atherosclerosis are reversed after 1 year. Journal of Lipid Research, (100739), 100739. doi:10.1016/j.jlr.2024.100739

The Sant Pau Research Institute (IR Sant Pau) has been selected to coordinate the ambitious European project Synapsing, an unprecedented initiative funded with seven million euros by the European Union through the Horizon-Europe programme. This project, involving thirteen leading institutions in clinical research, neuroimaging, and social sciences from nine European countries, aims to revolutionise the diagnosis and management of psychiatric and neurodegenerative diseases through blood biomarkers, socioeconomic data, and innovative tools.

The coordination of the project will fall to Dr Olivia Belbin, head of the Molecular Neurodegeneration Group at IR Sant Pau, who will bring her expertise in translational research to advance understanding of neurodegenerative diseases. Dr Maria J. Portella, head of the Mental Health Research Group, will also play a key role, contributing her specialised knowledge in mental health to ensure effective integration between both fields of study. This joint leadership reinforces the multidisciplinary and collaborative character of the project, highlighting the synergy between two traditionally separate areas of research.

Synapsing not only promises scientific advances but also seeks to promote greater equity in medical care. By integrating knowledge from different disciplines and geographical areas, the project aims to create a diagnostic and therapeutic model applicable across Europe. “IR Sant Pau is coordinating, for the first time, a European project of this scale, marking a historic event in our trajectory and consolidating our position as a reference centre in clinical and translational research,” highlighted Dr Belbin.

Unprecedented Innovation

In the field of mental health and neurodegenerative research, the lack of objective diagnostic tools and personalised treatments remains a significant challenge. Synapsing represents a paradigm shift, seeking practical and scientific solutions to these problems.

“Psychiatric disorders, such as schizophrenia, major depressive disorder, and bipolar disorder, often share symptoms with neurodegenerative diseases like Alzheimer’s and Parkinson’s. This overlap causes delays in diagnoses and complicates the administration of appropriate treatments,” explained Dr Portella, who added that this new European project will attempt to address this issue through an innovative approach based on biomarkers and transdiagnostic data.

Among Synapsing’s primary objectives is the integration of advanced technologies, multimodal data, and biological samples from patients with psychiatric and neurodegenerative diseases into unified and easily accessible collections. This will overcome the lack of transdiagnostic research resources and establish the foundations for a better understanding, diagnosis, and measurement of psychiatric symptoms.

The project will also seek to understand the shared pathophysiological mechanisms of synaptic degeneration that lead to clinical symptoms common to both types of disorders. This knowledge will guide the development of more effective and specific therapeutic strategies. “Another key aspect is the development of blood biomarkers to improve diagnosis. Synapsing seeks to establish tools that allow for faster and more objective diagnoses of disorders such as major depressive disorder, bipolar disorder, and schizophrenia, reducing errors and confusion with neurodegenerative diseases,” commented Dr Belbin.

Additionally, the project aims to better manage patients with psychiatric symptoms through biomarkers that enable the measurement of treatment efficacy and the prediction of therapeutic outcomes. “This will help personalise therapies and ensure more rigorous monitoring of patient progress,” added Dr Portella.

Analysing Sociodemographic Risk Factors

Another fundamental pillar is the analysis of modifiable sociodemographic risk factors that may be associated with biological changes in patients with psychiatric and neurodegenerative diseases. The findings from this research will provide evidence for designing public policies aimed at preventing the debilitating symptoms of these diseases.

Finally, Synapsing focuses on engaging the research community, clinicians, and patients in a collective effort to bridge the gap between research, clinical practice, and governmental policies. This objective aims to ensure that scientific advances translate into tangible benefits for patients and society.

A Collaborative and Multidisciplinary Approach

Synapsing will bring together experts from various disciplines, including neurology, psychiatry, biology, social sciences, and ethics. This approach is essential to addressing the complexity of these diseases, which are often studied separately but share underlying mechanisms. As Dr Belbin commented, “For the first time, a project of this magnitude addresses both psychiatric disorders and neurodegenerative diseases, recognising their overlaps and leveraging a transdisciplinary approach to better understand these pathologies.”

IR Sant Pau will play a central role in all phases of the project thanks to collaboration between its Memory Unit and its Mental Health Unit. As Dr Portella highlighted, “This collaboration marks a turning point in integrating the efforts of two traditionally separate areas. It is an example of how breaking down barriers between disciplines can generate significant advances in patient care.” This synergy is expected to enable the integration of clinical, neurobiological, and sociodemographic data for a more in-depth understanding of these conditions.

Creation of a Large Cohort

One of the main innovations will also be the generation of an unprecedented cohort, including more than 3,000 patients from various European countries, encompassing both psychiatric and neurodegenerative diseases. By unifying clinical, neurobiological, and sociodemographic information in a single database, the project aims to provide a unique tool for future research.

The cohort will not only allow for the identification of blood biomarkers distinguishing these pathologies but will also facilitate the analysis of sociodemographic risk factors. “For the first time, we will have a database integrating multidimensional information from patients across the psychiatric and neurodegenerative spectrum. This will be crucial for understanding the connections between these diseases and how they evolve in different contexts,” said Dr Belbin.

Furthermore, this joint effort establishes a new way of addressing challenges in mental health and neurodegenerative research. The quality and scale of this data will help outline more effective clinical guidelines and design personalised treatments, opening new doors in translational medicine. “This integrated and accessible database will have a lasting impact, allowing researchers and clinicians across Europe to advance together towards more equitable and personalised medicine,” emphasised Dr Portella.

Expected Results

The Synapsing project will span five years, aiming to achieve significant advances in the diagnosis, treatment, and prevention of psychiatric and neurodegenerative diseases. Among the most anticipated results is the identification of key blood biomarkers in 2025, marking a historic milestone for personalised medicine. Additionally, the first scientific articles reflecting the impact of this project on understanding these complex diseases are expected to be published in 2026.

Beyond scientific advances, the project seeks to transform the clinical and social approach to these conditions. The collected data will be used to update clinical guidelines, enabling more precise diagnoses and treatments tailored to the specific needs of each patient. Similarly, evidence-based recommendations will be developed for public policies aimed at mitigating the risk factors associated with these diseases.

The social focus of Synapsing is also essential. Through a specially designed questionnaire, sociodemographic factors influencing the progression and development of these diseases will be identified. “This project not only seeks biomedical results but also aims to improve patients’ living conditions by understanding how factors such as unemployment or socioeconomic inequality affect their health,” pointed out Dr Belbin. These findings will enable governments to implement more effective preventive measures and reduce inequalities in healthcare.

L’Institut de Recerca Sant Pau (IR Sant Pau) ha estat beneficiat amb una ajuda d’1.406.625 euros per part de l’Institut de Salut Carlos III de Madrid (ISCIII). Aquest suport econòmic es destinarà a modernitzar i millorar la Unitat d’Investigació Clínica del centre, reforçant la seva capacitat per dur a terme estudis d’alta qualitat i donar resposta a les necessitats dels pacients. L’ajuda s’emmarca en un projecte de l’ISCIII per impulsar la investigació a Espanya, tant mitjançant la millora de les unitats ja existents com amb la creació de noves.

En el cas de l’IR Sant Pau, els fons permetran optimitzar els espais i adquirir equipaments d’última generació, amb especial atenció a les tecnologies informàtiques i la telemedicina. Aquestes millores beneficiaran especialment els pacients amb patologies neurodegeneratives i malalties complexes, consolidant l’IR Sant Pau com un referent en recerca biomèdica.

“Aquesta ajuda ens brinda l’oportunitat de fer un salt qualitatiu en les nostres capacitats de recerca. A l’IR Sant Pau estem compromesos a oferir als nostres pacients els tractaments més innovadors, i aquesta millora ens permet avançar en aquesta direcció”, va explicar la Dra. Rosa Antonijoan, directora de Farmacologia Clínica del Centre d’Investigació del Medicament de l’IR Sant Pau i responsable del projecte.

“Volem ser un referent en assaigs clínics per a pacients fràgils, especialment aquells amb patologies neurodegeneratives. Aquesta inversió ens permetrà garantir que els nostres pacients tinguin accés a investigacions capdavanteres en un entorn segur i adequat”, va afegir la Dra. Antonijoan.

Adaptació a les noves necessitats dels pacients

La unitat d’investigació clínica de l’IR Sant Pau ja compta amb una trajectòria consolidada, però aquest projecte potenciarà les capacitats del centre per tal d’adaptar una zona amplia per pacients amb dificultat d’accés a instal·lacions i garantir l’atenció del pacient fràgil en un entorn controlat i proper al personal clínic. Aquesta zona inicialment estava dissenyada per a la participació de pacients sans. “La recerca en neurociències i els assaigs oncològics de fase 1 són àrees prioritàries per a l’IR Sant Pau, però els pacients que hi participen són fràgils i necessiten cures especials que ara, amb l’ampliació, esperem poder oferir de manera més adequada”, va apuntar la Dra. Antonijoan.

Entre les principals millores hi ha la redistribució dels espais per maximitzar l’ús de les instal·lacions i atendre un major nombre de pacients. Es preveu ampliar l’espai disponible per a la investigació clínica a la planta 1 de l’institut, passant dels 1.371,12 m² actuals als 1.612,10 m². La reforma inclourà l’accessibilitat plena per als participants, amb zones de circulació àmplies, sales d’espera confortables, espais que garanteixin la privacitat i una senyalització clara. També es treballarà per millorar el benestar dels acompanyants, humanitzant l’entorn.

Una part de l’ajuda es destinarà a l’adquisició d’equipaments avançats, amb la incorporació de tecnologies de telemedicina i sistemes informàtics per gestionar els assaigs. Es busca implementar un model de recerca clínica descentralitzada, amb l’objectiu d’incrementar la participació de col·lectius vulnerables en els estudis, incloent-hi persones grans, amb malalties neurològiques o minoritàries.

També està prevista l’adequació de les instal·lacions per garantir la comoditat i seguretat dels pacients amb malalties com Alzheimer, Parkinson, Huntington o ELA. Aquestes adaptacions ampliaran la capacitat dels investigadors per dur a terme estudis més complexos i ambiciosos, incrementant l’impacte dels resultats tant en la comunitat científica com en l’atenció als pacients.

Impuls als assaigs clínics en fases inicials

Un dels objectius principals del projecte és enfortir els assaigs clínics en fases 1B i 2, àrees on l’IR Sant Pau ja ha demostrat un gran potencial. Aquestes fases són essencials per avaluar la seguretat i els primers indicis d’eficàcia dels tractaments. A més, es contempla la possibilitat d’integrar assaigs de fase 3 quan sigui necessari, posicionant l’IR Sant Pau com a líder en recerca translacional.

S’espera augmentar tant el nombre de pacients que participen en els assaigs com la qualitat i humanitat de la seva experiència. Les mesures proposades fomentaran l’equitat i la justícia en la distribució de la recerca, alhora que afavoriran el reclutament i la permanència dels pacients en els estudis. Així, la unitat es consolidarà com un pol d’atracció per a la recerca clínica pública i privada, amb especial atenció als pacients fràgils, en particular aquells amb malalties neurològiques o rares. Tot això es farà minimitzant els desplaçaments, amb un impacte ambiental positiu gràcies a la reducció d’emissions de CO₂.

Interoperabilitat per sistemes informàtics

L’ajut també és destinarà a desenvolupar i implementar un model d’interoperabilitat per sistemes informàtics, cosa que resulta imprescindible per situar al pacient al centre de la informació sanitària. Aquest model permetrà la coordinació i l’accés eficient a la documentació clínica. Amb un sistema interoperable, els professionals de la salut podran accedir ràpidament a la informació rellevant, millorant així la presa de decisions clíniques i optimitzant l’atenció al pacient.

Aquest enfocament també facilitarà la integració de dades provinents de diverses fonts, amb la qual cosa es promourà una atenció més coordinada i efectiva i s’asseuran d’aquesta manera les bases per a una col·laboració en xarxa per a la realització d’assajos clínics descentralitzats i preparar la informació per a possibles usos secundaris en xarxa.

Calendari d’implementació

El desenvolupament del projecte està planificat per a un període de dos anys. Durant aquest temps, s’adquiriran els equipaments i es completaran les obres necessàries abans de finals de 2026. Per al 2027, l’IR Sant Pau espera tenir totes les millores plenament operatives, consolidant-se com un centre de referència en investigació clínica avançada.

A més, es proporcionarà formació especialitzada al personal del centre per garantir l’ús òptim dels nous recursos i tecnologies, assegurant una atenció de màxima qualitat als pacients que participin en els estudis.

As part of the annual meeting of the American Society of Haematology (ASH) 2024, one of the foremost global events in this field, Dr Ana Caballero, researcher in the Cellular Immunotherapy and Gene Therapy group at the Sant Pau Research Institute (IR Sant Pau) and associate in the Haematology Service led by Dr Javier Briones, delivered an oral presentation on the results of the phase I/II clinical trial of the HSP-CAR30 treatment for patients with refractory or relapsed Hodgkin lymphoma and CD30+ T-cell lymphoma. This innovative cellular therapy, a pioneering development in Europe, was entirely designed and manufactured at the IR Sant Pau and Hospital. It has drawn significant interest due to its safety and efficacy in a patient population with unmet therapeutic needs.

To date, 32 patients have been treated with HSP-CAR30, and plans are in place to enrol an additional 10 patients to further bolster the findings. According to Dr Caballero, this expansion will help confirm the robustness of the preliminary results and establish a firmer basis for the treatment’s future development. The results presented indicate that 55% of patients treated with HSP-CAR30 achieved complete remission of the disease—an impressive figure considering the severity of the studied population. Additionally, it was estimated that 41% of patients would remain progression-free two years post-treatment, an encouraging indicator of the therapy’s potential to provide long-term cancer control.

Dr Ana Caballero highlighted the significance of these results: “What is most encouraging is that the responses are not only effective but also durable, representing a genuine advance for a population with very limited options,” she explained.

An innovative treatment developed entirely at Sant Pau

The HSP-CAR30 treatment is based on genetically modified CAR T-cell technology, designed to specifically recognise and attack the CD30 antigen present in tumour cells. “We designed a treatment combining a second-generation CAR T-cell with an enriched population of memory T-cells, optimised to deliver a potent and long-lasting response,” explained Dr Laura Escribá, coordinator of CAR T-cell production and quality control within the Cellular Immunotherapy group of the Haematology Service. She added, “The results we are seeing are promising and reflect the team’s effort to offer a solution to patients who have no other therapeutic options.”

A safe and long-lasting drug

One of the most notable features of HSP-CAR30 is its manageable safety profile. During the trial, side effects such as cytokine release syndrome were mostly mild and controllable with available treatments. The incidence of neurotoxicity was minimal, underscoring the therapy’s potential as not only effective but also a safe option. “Our goal was always not just to develop a treatment that works, but also one that is tolerable for patients. We know that quality of life is just as important as survival,” Dr Caballero emphasised.

The initial results also demonstrate significant persistence of the therapeutic effect. According to the data presented, the modified CAR T-cells remain active in the patient’s body for up to 24 months post-infusion, reinforcing their ability to offer lasting tumour control. Dr Caballero explained that the focus on memory T-cells is key to extending the treatment’s benefits: “Our product is designed to last and to continue combating cancer over time. This is particularly important for patients facing aggressive and refractory tumours.”

Commitment to research

Hodgkin lymphoma (HL) is known for its high cure rate in the early stages of treatment. However, up to 30% of patients experience recurrence or fail to respond to initial therapies, leaving them in a critical situation with few alternatives. In her presentation at ASH, Dr Caballero stressed the importance of addressing this unmet need: “Hodgkin lymphoma, although treatable in most cases, presents a major challenge when patients fail to respond to conventional treatments. It is precisely in this population that HSP-CAR30 is showing its transformative potential.”

The ASH Congress, recognised as the leading international forum in haematology, was the ideal platform to showcase the potential impact of HSP-CAR30. The presentation generated widespread interest among specialists and experts, who emphasised the importance of further advancing these innovative therapies. Dr Caballero underlined the urgency of continuing research and development of the treatment: “Although the results are encouraging, we know there is still work to be done. These preliminary data are a significant step, but we remain committed to validating and improving this treatment for future patients.”

The development of HSP-CAR30 exemplifies Sant Pau’s leadership in advanced immunotherapy. This project demonstrates how interdisciplinary collaboration and academic research can have a direct and meaningful impact on patients’ lives. “We are proud of what we have achieved so far, but our ultimate goal is for treatments like HSP-CAR30 to become an accessible therapeutic option for patients facing this type of cancer,” concluded Dr Caballero.

Researchers from the Neurobiology of Dementia group at the Sant Pau Research Institute (IR Sant Pau) have published two pioneering studies in the journals Alzheimer’s & Dementia and Neurology, revealing key advances in understanding Alzheimer’s disease in individuals with Down syndrome (DS). They have demonstrated the relationship between white matter hyperintensities and cerebral microhemorrhages with the development of this disease in this population.

Studies in individuals with DS are crucial because this population has a highly determined genetic risk for developing Alzheimer’s due to the triplication of chromosome 21. This genetic condition leads to the overproduction of beta-amyloid, one of the primary biological characteristics of the disease. As a result, almost all individuals with DS show biological signs of this condition from the age of forty, although clinical symptoms may appear later.

This phenomenon makes DS a unique model for studying the early mechanisms of Alzheimer’s disease and its interactions with cerebrovascular factors, providing data that may also be applicable to the general population. “The life expectancy of individuals with DS has significantly increased in recent decades, but this success has highlighted the need to address the growing impact of Alzheimer’s in this population,” says Dr. Alexander Bejanin, researcher in the Neurobiology of Dementia group at IR Sant Pau, who coordinated the two studies now published. “Our studies allow us to identify key factors that could improve early detection and prognosis of the disease, not only in individuals with DS but also in the general population.”

White matter hyperintensities

White matter hyperintensities (WMHs) are detected through magnetic resonance imaging (MRI) as bright areas in the brain. In individuals with DS, these lesions appear earlier and with greater severity than in the general population. They are key indicators of cerebrovascular issues and are strongly linked to the progression of Alzheimer’s disease.

According to Alejandra Morcillo-Nieto, the lead author of the study published in Alzheimer’s & Dementia, “WMHs are crucial for understanding how cerebrovascular disease and Alzheimer’s disease are related in individuals with DS. These lesions are particularly prevalent in periventricular, frontal, parietal, and occipital regions, and this is the first study to identify the areas where they are most common in this population.”

The study, which analysed 261 adults with DS and 131 adults without this condition, shows that “WMHs increase with age and are correlated with biomarkers such as beta-amyloid and phosphorylated tau, suggesting a direct connection with the pathophysiology of Alzheimer’s disease, independent of vascular risks,” says Morcillo-Nieto. Moreover, these anomalies are related to clinical status, suggesting that they may be directly influencing functional deterioration.

Cerebral microhemorrhages

Cerebral microhemorrhages are small deposits of blood in the brain that may indicate vascular damage and are associated with Alzheimer’s disease. In individuals with DS, these lesions are more frequent and primarily concentrate in the posterior regions of the brain, such as the cerebellum and occipital areas. This is one of the findings of the study published in Neurology, and although they are not exactly the same areas as those for WMHs, they are strongly related.

According to Sàra Zsadanyi, the lead author of the study, “microhemorrhages are a key piece of understanding the risk of complications in anti-amyloid therapies. Their increasing prevalence with age allows us to better identify key moments for intervention.” The study identified microhemorrhages in 20% of individuals with DS, compared to only 8.9% in healthy controls.

These lesions increase not only with age but also with clinical severity, rising from 12% in early stages to 35% in advanced stages of dementia. The results also show a correlation with Alzheimer’s disease biomarkers, highlighting their relevance for monitoring and treating this vulnerable population.

Overall assessment of advances

According to Dr. Alexander Bejanin, “with our studies, we want to highlight the crucial role of characterising these two vascular lesions in the development of new therapies for Alzheimer’s disease.” Dr. Bejanin also emphasises that the findings “are not only relevant for Down syndrome but have broader implications for understanding Alzheimer’s disease in general.”

These discoveries have a direct impact on the management and treatment of the disease. “With the arrival of new anti-amyloid therapies, these lesions are a crucial factor to consider, as they may increase the risk of adverse effects,” warns Dr. Bejanin. This underscores the importance of using MRI to monitor disease progression and personalise treatments.

Future perspectives

The research also opens the door to exploring new preventive and therapeutic strategies. According to Dr. Bejanin, “studies in DS allow us to better understand the relationship between vascular lesions and Alzheimer’s pathology, offering a unique opportunity to apply this knowledge to the general population.” The researchers highlight that these findings could improve understanding of the role of vascular lesions in Alzheimer’s, both as a cause and a consequence of the disease.

Sant Pau Hospital is an international reference centre for neurodegenerative diseases associated with DS. Its Alzheimer-Down Unit, the only one in Spain, continues to lead research redefining the future of diagnosis and treatment for these conditions. This unit works in a multidisciplinary way, combining clinical and translational research with a personalised approach to meet the specific needs of individuals with DS.

Reference articles:

• Morcillo-Nieto AO, Zsadanyi SE, Arriola-Infante JE, (…) Bejanin A. Characterization of white matter hyperintensities in Down Syndrome. Alzheimer’s Dement. 2024; 1-15. https://doi.org/10.1002/alz.14146
• Zsadanyi SE, Morcillo-Nieto AO, R. Aranha M, (…), Bejanin A. Associations of Microbleeds and Their Topography With Imaging and CSF Biomarkers of Alzheimer Pathology in Individuals With Down Syndrome. Neurology 2024; 103:e209676. https://doi.org/10.1212/WNL.00000000002096

Researchers from the Sant Pau Research Institute (IR Sant Pau) have collaborated with an international team to develop a tool that promises to transform how small vessel disease associated with the NOTCH3 gene is assessed and managed, including CADASIL, the most severe presentation of this condition. Published in the prestigious journal JAMA Neurology, the study introduces a staging scale that classifies the progression of this disease into five main stages and nine subgroups, offering for the first time a clear model to understand its evolution.

Dr Israel Fernández-Cadenas, head of the Pharmacogenomics and Neurovascular Genetics Group at IR Sant Pau and one of the researchers involved in developing this classification, explains that this advancement represents a fundamental shift in the management of these diseases. “Until now, it was known that CADASIL progressed over time, but physicians lacked tools to classify its progression systematically. The new scale, however, provides a framework that allows doctors to place patients in a specific stage, which not only helps provide them with a clearer understanding of their prognosis but also reduces the uncertainty that characterises many rare diseases like this one.”

Easy Application to Clinical Practice and Research

The scale has been designed to integrate data from magnetic resonance imaging (MRI) and functional assessments, tools already part of routine medical practice. According to Dr Elena Muiño, a researcher in Dr Fernández-Cadenas’ group and another contributor to the development of the classification, this means that its implementation will not require additional investments or complex technologies, a key factor for its adoption in diverse medical settings, including those with limited resources.

One of the major advantages of this tool is that it not only facilitates a better understanding of the disease’s progression but also improves the way experimental treatments are designed and evaluated. “By classifying patients according to the stage of the disease, clinical trials will be able to identify more precisely which therapies are effective for each stage,” explains Dr Muiño.

Application in Other Contexts

The impact of this scale is not limited to CADASIL. Although this study focused on patients with genetic variants associated with the NOTCH3 gene, the model could also be applied to other forms of small vessel disease, including sporadic forms such as those caused by hypertension. Dr Muiño emphasises that “CADASIL is considered the genetic model par excellence for small vessel disease. Therefore, it seems logical to think that this classification system will be applicable to other causes of small vessel disease, facilitating their study, which also lack a specific treatment.”

The development of the scale initially involved a cohort of 195 patients with CADASIL and was later validated in a sample of over 1,700 individuals from various regions worldwide, confirming its international applicability. Additionally, a longitudinal follow-up of up to 18 years allowed researchers to observe how the disease progresses in most patients, particularly over long periods. This knowledge not only helps set clearer expectations for patients and their families but also provides a solid foundation for designing more specific therapeutic interventions.

A Challenge that Still Remains

Despite these advances, Dr Muiño acknowledges that treating CADASIL remains a challenge. “Currently, there are no specific therapies for this disease beyond general measures to manage cardiovascular risk factors. However, implementing this scale represents an essential step towards developing targeted treatments.”

The publication of this study in a high-impact journal such as JAMA Neurology underscores its scientific and clinical significance, highlighting the importance of a multidisciplinary and international approach to tackling complex diseases like this. “This is a crucial step forward in shedding light on an area that, until now, was filled with uncertainty,” concludes Dr Muiño. “Not only are we giving patients a better understanding of what they face, but we are also laying the groundwork for future treatments that could change their lives.”

About CADASIL and the NOTCH3 Gene

CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy) is a genetic condition that affects the brain’s small blood vessels, causing an alteration in blood flow distribution to various brain regions. This condition is caused by genetic variants in the NOTCH3 gene, located on chromosome 19, whose alteration leads to the aggregation of the Notch3 protein and other proteins, ultimately resulting in damage to the blood vessel wall.

The progressive damage to these small vessels leads to clinical manifestations including recurrent strokes, migraines, mood disorders, and, in advanced stages, vascular dementia. Most cases typically manifest between the ages of 45 and 60, although earlier and later forms have been described depending on the location of the genetic variant and individual factors such as sex and cardiovascular risk factors.

Although CADASIL has historically been considered a rare disease, recent studies suggest its prevalence could be much higher than previously estimated. It is believed that up to 1 in 300 people carries a pathogenic genetic variant in the NOTCH3 gene. Indeed, many individuals may not develop severe symptoms or may present with milder forms of the disease depending on the location of the genetic variant. This observation has generated growing interest in research, as it could explain a significant proportion of undiagnosed cerebrovascular diseases, particularly in older adults.

Reference Article:

Gravesteijn G, Rutten JW, Cerfontaine MN, Hack RJ, Liao Y-C, Jolly AA, et al. Disease severity staging system for NOTCH3-associated small vessel disease, including CADASIL. JAMA Neurol [Internet]. 2024; Disponible en: http://dx.doi.org/10.1001/jamaneurol.2024.4487

One more year, the Sant Pau Research Institute (IR Sant Pau) celebrated a new edition of its Research Day, an event aimed at reaffirming the center’s position as a leader in biomedical research and scientific innovation. The gathering brought together research leaders, healthcare professionals, and technology experts to discuss the latest advancements transforming healthcare.

During the event, attended by more than a hundred professionals from the institute, various roundtables were held to address topics such as cutting-edge research in major hospitals, the challenges of collaboration between research and healthcare, the benefits of research and technological innovation for patients, and the latest developments in the fields of neuroscience and mental health—an area where IR Sant Pau stands out as one of the leading centers.

Dr Jordi Surrallés, director of IR Sant Pau, opened the event by welcoming attendees and highlighting achievements accomplished through the strategic and sustainability plans, both concluding this year. “We have solidified our position as a reference center and achieved financial stability, which will be crucial in the coming years,” emphasized Dr. Surrallés.

The presentation detailed key accomplishments, such as an increase in publications in high-impact journals and the number of theses supervised by IR Sant Pau. One area that has shown significant growth is the dissemination of the institute’s activities. “This was established as a priority because it’s important not only to conduct research but also to be accountable to society for the achievements reached,” said Dr Surrallés.

Looking ahead, Dr Surrallés expressed optimism and outlined the five pillars of the new strategic plan for IR Sant Pau for the 2025–2030 period. These include the need to continue transforming the institution, embracing change without fear; striving for excellence in both scientific output and management; continuing to grow and meeting new demands to remain competitive in an increasingly globalized world; generating a positive impact on health and adding value to society; and strengthening the internationalization of IR Sant Pau, reflecting the organization’s potential and quality.

Dr Valentín Fuster and the Importance of Early Intervention

The event also featured Dr Valentín Fuster, General Director of the Spanish National Center for Cardiovascular Research (CNIC), under the Carlos III Health Institute (ISCIII), and Director of the Cardiovascular Institute at Mount Sinai Hospital in New York, recently renamed Mount Sinai Fuster Heart Hospital in his honor.

In his lecture, Dr Fuster discussed a shift in his research focus from studying cardiovascular disease to understanding how health works. He emphasized the importance of early intervention. “Our research shows that between the ages of 20 and 40, cardiovascular risk factors such as hypertension or high cholesterol have a greater impact than when people reach 65,” he explained.

Dr Fuster also highlighted how recent technological advancements have enabled the identification of subclinical arterial disease in individuals as young as 30, which can later develop into more serious conditions. He stressed the need for educational and training initiatives at an early age, referencing his work with the children’s television program Sesame Street, where a character inspired by him, Dr Ruster, was created. “While our studies indicate initial improvement, we’ve found that bad habits tend to return over time, necessitating new interventions to reinforce the messages we aim to convey,” he added.

A Day Rich in Content

Before Dr Fuster’s lecture, several scientific sessions and panels took place. The first panel featured Adrià Comella, CEO of Sant Pau Hospital; Josep Maria Campistol, CEO of Hospital Clínic de Barcelona; Anna Arán, CEO of Hospital Parc Taulí; and Manel del Castillo, CEO of Hospital Sant Joan de Déu. This panel was a novelty in the event, which traditionally had a more purely scientific focus. This year, the inclusion of perspectives from these leading university hospitals aimed to analyze the challenges and complexities of conducting research in large hospitals.

All participants emphasized the importance of research in improving healthcare. They highlighted that the work carried out in hospitals must be complemented by the research conducted in institutes, which, in turn, benefits from close collaboration with clinical practice. This creates what is known as a virtuous circle. “It is essential to maintain a commitment between the hospital and the research institute, working together to address the challenges posed by our patients,” said Adrià Comella.

Other panels focused on more scientific content, such as research and technological innovation, and the latest developments in neuroscience—a field in which IR Sant Pau is a reference center. Notable advancements include the discovery earlier this year of a genetic form of Alzheimer’s disease linked to the APOE4 gene.

The 2024 Sant Pau Research Day reaffirmed Sant Pau’s commitment to scientific excellence and the continuous improvement of healthcare, solidifying its position as a benchmark in biomedical research. The event also served as an opportunity to strengthen collaboration between institutions and share knowledge driving the medicine of the future.

Dr Maria Torrens, a researcher in the Intensive Care Medicine research group at the Sant Pau Research Institute (IR Sant Pau) and an intensivist at the Intensive Care Unit (ICU) of Sant Pau Hospital, has been honoured with one of the research grants awarded by the Spanish Society of Intensive Care, Critical Care and Coronary Units (SEMICYUC). This grant, worth €10,000, acknowledges the scientific and clinical value of the research project titled “Quantification of systemic congestion using ultrasonography as a predictor of weaning failure from mechanical ventilation”. The project is coordinated by Dr Lluís Zapata and Dr Carles Subirà, members of the same Intensive Care Medicine research group.

The term weaning refers to the gradual process of disconnecting a patient from mechanical ventilation, with the goal of restoring independent breathing capacity. This is a critical moment in the management of patients who have required ventilatory support, as failure in this process can lead to significant complications and prolonged ICU stays.

This prospective, observational, and multicentre study primarily aims to identify, through ultrasound, patients at risk of failing the disconnection from mechanical ventilation before initiating spontaneous breathing trials. Ultrasound evaluation of systemic venous congestion is presented as an innovative, non-invasive tool that could significantly enhance the weaning process, reducing dependency on mechanical ventilation and thereby improving the prognosis for critically ill patients.

The awarding of this grant highlights the clinical research excellence at IR Sant Pau and its commitment to innovation as a fundamental pillar of progress in intensive care medicine. These recognitions strengthen the institute’s dedication to generating scientific knowledge that directly contributes to better patient care.

We at IR Sant Pau extend our congratulations to Dr Maria Torrens and her team for this achievement and their contribution to advancing high-impact clinical research projects. We will continue to support the efforts of our professionals to remain at the forefront of biomedical research in service of society.

Today, the HUB Alzheimer Barcelona was launched, a pioneering alliance that brings together the leading research centres and public hospitals in the city to strengthen Barcelona’s leadership in Alzheimer’s disease research and care. Among the founding members of this initiative, promoted by the Pasqual Maragall Foundation with the support of the Barcelona City Council, is the Hospital de Sant Pau, recognised as one of the leading institutions in Alzheimer’s research and care in Spain and internationally.

Dr Alberto Lleó, director of the Neurology Department at Hospital de Sant Pau and researcher with the Neurobiology of Dementias Group at the Sant Pau Research Institute (IR Sant Pau), highlighted that this alliance will “create synergies among leading centres to implement new biological therapies and strengthen clinical research”.

What actions will Sant Pau undertake?

Hospital de Sant Pau will play a prominent role by leading actions such as training researchers through collaborative PhD programmes and the implementation of new treatments in clinical practice.

Through this initiative, Sant Pau reaffirms its commitment to research and the development of innovative solutions to improve the lives of people affected by Alzheimer’s, consolidating Barcelona as a global hub in this field.

Which entities are part of this alliance?

The HUB Alzheimer Barcelona is spearheaded by the Pasqual Maragall Foundation, the founding centre, with the support of the Barcelona City Council. It also includes Hospital de Sant Pau, Hospital Clínic, Hospital del Mar, and the Vall d’Hebron University Hospital, as well as two leading institutions specialising in Alzheimer’s research: the Barcelonaβeta Brain Research Centre (BBRC) and the Ace Alzheimer Center Barcelona.

The presentation event held today featured the Mayor of Barcelona, Mr Jaume Collboni, Dr Arcadi Navarro, Director General of the Pasqual Maragall Foundation and its research centre, the BBRC; Dr Mercè Boada, representing the member centres of the HUB Alzheimer Barcelona; and Cristina Sáez, a science journalist. The event was closed by the Minister of Health, Ms Olga Pané.

Researchers at the Sant Pau Research Institute (IR Sant Pau) have taken part in a pioneering study that has demonstrated, for the first time, that gene therapy can be an effective and safe therapeutic option for patients with Fanconi anaemia, a rare condition characterised by progressive bone marrow failure and a high predisposition to cancer. The results, published in the prestigious journal The Lancet, are the culmination of over two decades of preclinical research and seven years of patient follow-up.

Fanconi anaemia, a genetic disease often diagnosed in childhood, leads to the gradual loss of blood cells, causing severe infections, fatigue, and bleeding. Until now, the only definitive treatment has been a bone marrow transplant from a compatible donor, a procedure requiring prior chemotherapy and associated with significant risks, including prolonged hospitalisation.

No clinical studies conducted to date had succeeded in making gene therapy effective for this complex disease. However, the recently published work has demonstrated that, even without chemotherapy conditioning, the autotransfusion of haematopoietic stem cells corrected for the genetic defect leads to a progressive increase in corrected cells in most patients. In two treated patients, the proportion of corrected cells exceeded 90%, effectively halting the disease’s natural progression, which would otherwise involve the steady depletion of blood cells.

The Phase I/II clinical trial was led by the Fundación Hospital Niño Jesús in Madrid, with the long-term follow-up study overseen by Rocket Pharmaceuticals Inc. Dr Julián Sevilla, supported by Dr Josune Zubicaray—both haematologists at the Advanced Therapies Unit of the Niño Jesús University Children’s Hospital and members of the CIBER for Rare Diseases (CIBERER)—served as principal investigators under the scientific direction of Professor Juan Bueren. Dr Paula Río, the study’s lead author, and Dr Susana Navarro, both researchers at CIEMAT—a body under the Ministry of Science, Innovation and Universities—along with the Rare Diseases Area of CIBERER and the Fundación Jiménez Díaz Health Research Institute (IIS-FJD), were also key collaborators. Additionally, the study was coordinated with the National Advanced Therapies Network (TERAV), promoted by the Carlos III Health Institute.

Until now, bone marrow transplants from healthy, compatible donors were the sole definitive treatment for bone marrow failure in these patients. While this therapy has improved significantly over recent years, it still requires chemotherapy conditioning to prevent rejection and is linked to both short- and long-term risks, often necessitating extended hospital stays.

Sant Pau’s Contribution

Professor Jordi Surrallés, director of IR Sant Pau and CIBERER group leader, was among the researchers involved in the study. “The teamwork within the National Fanconi Anaemia Research Network has positioned Spain as a global leader in advanced therapies for this disease. This research marks an unprecedented milestone in the treatment of Fanconi anaemia,” he stated.

The gene therapy detailed in The Lancet was developed with contributions from numerous teams responsible for the various stages of its application. The collection and purification of stem cells mobilised from patients’ blood were carried out at Niño Jesús University Children’s Hospital in Madrid, Vall d’Hebron University Hospital in Barcelona, and the Catalan Blood and Tissue Bank, coordinated by Dr Cristina Díaz de Heredia. These cells were then corrected for the genetic defect ex vivo at the CliniStem Cleanroom of CIEMAT using a genetically modified virus (a lentiviral vector carrying the FANCA gene) developed earlier by the research team.

Once corrected for the genetic defect, the cells were reinfused into patients at the Paediatric Haematology Service of the Niño Jesús Hospital as an autotransfusion, without requiring chemotherapy. This allowed patients to be discharged just 72 hours after the infusion.

Over 20 Years of Research

The investigation that led to these results began more than two decades ago with preclinical studies at CIEMAT laboratories, where a lentiviral vector carrying the FANCA gene was developed to correct the genetic defect in stem cells. This vector, recognised as an Orphan Drug in Europe and the United States, paved the way for the clinical trials culminating in this publication in The Lancet.

The study confirms that chemotherapy can be avoided in patients with Fanconi anaemia—a remarkable achievement given the fragility of these patients’ stem cells. Researchers have shown that an autotransfusion of corrected cells is sufficient to halt, and in some cases even reverse, bone marrow failure.

Towards a Consolidated Therapy

Rocket Pharmaceuticals is currently working with the European Medicines Agency and the US Food and Drug Administration to obtain marketing authorisation for this therapy. Meanwhile, preclinical studies are underway to expand this therapeutic approach to other subtypes of Fanconi anaemia, potentially increasing the number of patients who could benefit from this innovation.

This breakthrough offers new hope for patients and their families. Alicia de las Heras, president of the Fanconi Anaemia Foundation, expressed: “A few years ago, this advancement seemed unimaginable. Now, thanks to science and the tireless efforts of our specialists, we have real hope for our children.”

This study reinforces Spain’s leadership in developing innovative therapies for rare diseases, consolidating biomedical research as a driver of global health progress.

Article de referència:

Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from an open-label phase I/II and long-term clinical trials. Río, P., Zubicaray, J., Navarro, S., Gálvez, E., Sánchez-Domínguez, R., Nicoletti, E., Sebastián, E., Rothe, M., Pujol, R., Bogliolo, M., John-Neek, P., Bastone, A. L.,Schambach, A., Wang, W., Schmidt, M., Larcher, L., Segovia, J.C., Yáñez, R. M., Alberquilla, O., Díez, B., Fernández-García, M., García-García, L.,  Ramírez, M., Galy, A., Lefrere, F., Cavazzana, M., Leblanc, T., García de Andoin, N., López-Almaraz, R., Catalá, A., Barquinero, J., Rodríguez-Perales, S., Rao, G., Surrallés, J., Soulier, J., Díaz-de-Heredia, C., Schwartz, J. D., Sevilla, J., Bueren, J.A.,& on behalf of the FANCOLEN-1 gene therapy investigators. The Lancet, 2024, Vol 404. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(24)01880-4/fulltext

With the collaboration of:

The Sant Pau Research Institute (IR Sant Pau) continues to establish itself as a benchmark in the field of biomedical research. Three of its outstanding researchers, Dr José M. Guerra, Dr Gemma Vilahur, and Dr José Martínez González, have been selected to join the new board of directors of the Networked Biomedical Research Centre on Cardiovascular Diseases (CIBERCV). This network, which brings together the leading cardiovascular research groups in Spain, aims to advance the research, prevention, diagnosis, and treatment of these diseases, which are among the leading causes of mortality worldwide.

Dr José M. Guerra, head of the Clinical and Translational Cardiology Research Group at IR Sant Pau, will co-lead the Electrophysiology and Arrhythmias Programme with Dr Eva Delpón, from Universidad Complutense de Madrid (UCM). The programme’s goal is to promote research into the mechanisms and factors involved in the onset and progression of cardiac arrhythmias, as well as their treatment, prevention, and measures to mitigate their negative consequences. It aims to develop collaborative and high-impact research projects, fostering interaction between basic, clinical, and epidemiological groups within CIBERCV, other areas of CIBER knowledge, and national and international centres of excellence. The programme’s findings will contribute to scientific understanding and translate these discoveries into practical applications that improve patients’ quality of life.

Dr Gemma Vilahur, head of the Molecular Pathology and Therapeutics of Atherothrombotic and Ischaemic Diseases Research Group at IR Sant Pau, will co-lead the Atherosclerotic Cardiovascular Disease (ECVA) Programme alongside Dr Borja Ibáñez from the Spanish National Centre for Cardiovascular Research (CNIC). This programme encompasses the study and treatment of atherosclerotic disease and its clinical complications, including acute and chronic coronary syndrome, abdominal aortic aneurysm, stroke, and ischaemia-reperfusion processes. The ECVA Programme will adopt a comprehensive and multidisciplinary approach, fostering collaboration among CIBER groups and stimulating basic, translational, and clinical research with the aim of improving cardiovascular health.

Finally, Dr José Martínez González, a CSIC researcher within the Cardiovascular Remodelling Regulatory Mechanisms Group at IR Sant Pau, will head the Training and Mobility Programme. This cross-cutting initiative seeks to equip researchers with tools and competencies in both clinical and basic research to address current challenges in cardiovascular diseases. It will also promote knowledge exchange among CIBERCV groups, fostering a collaborative environment that drives the generation of new ideas and innovative solutions.

These appointments not only recognise the talent and scientific excellence of IR Sant Pau but also reinforce the role of its researchers as thought leaders in the cardiovascular field. Their experience and prestige endorse their ability to lead strategic programmes within CIBERCV, setting the agenda in key areas of biomedical research.

With their participation in the new board of directors, significant progress is expected in understanding and treating cardiovascular diseases, as well as inspiring and guiding the next generation of researchers. These appointments consolidate IR Sant Pau’s status as an international leader in scientific innovation and development, highlighting the global impact of its work on improving cardiovascular health and patient quality of life.

Researchers from the Institut de Recerca Sant Pau (IR Sant Pau), in collaboration with the Headache and Neuralgia Unit at Hospital de Sant Pau, the Universitat Autònoma de Barcelona, and the Universitat Pompeu Fabra, have published a pioneering study in the journal Headache on the use of deep brain stimulation (DBS) for treating chronic refractory cluster headache (CRCH). This research, the first of its kind to analyse the mechanisms of action through connectomics—an emerging field studying networks and connections between brain areas to understand how they interrelate and function together—offers new findings that could mark a breakthrough in the treatment of this highly debilitating disease.

Chronic cluster headache is a rare condition with severe symptoms that are difficult to treat. It is estimated that only 10% of patients with cluster headache—already an uncommon condition affecting approximately 1 in 1,000 people—develop a chronic form. Of this small group, only 10% are unresponsive to any available treatment, becoming “refractory” patients. Those affected typically experience multiple daily episodes of intense pain, which interfere with their personal and professional lives, causing significant economic and emotional impact for both the patients and the healthcare system.

The study included 14 patients with an average age of 42 years, all impacted by CRCH. DBS therapy was applied to the ventral area of the midbrain, a strategic stimulation point located in the lateral region of the ventral tegmental area (VTA), identified in the study as a “sweet spot” for generating a positive response. Clinical outcomes were monitored at intervals of 3, 6, 12, and 24 months, providing detailed data on the long-term efficacy of the intervention.

Clinical outcomes and headache burden reduction

The study used the “headache burden” (HAL) as the primary success indicator, a composite index considering the frequency, duration, and severity of pain attacks. The results showed a significant reduction in HAL levels among the 14 patients: from an average of 424 points at the start of the study, decreasing to 146 points at 24 months, representing an average reduction of 65% in the total burden of the disease.

Within the studied group, more than half (58%) of the patients achieved a reduction of over 50% in their HAL, experiencing considerable improvements in their quality of life, with the number of crises decreasing from five or six daily to just one or two crises per week. An additional 21% of patients showed a partial response to treatment, reducing their burden by 30–50%, while another 21% achieved minimal improvement (less than 30%).

Innovation in treatment and advances in brain connectomics

In addition to the notable clinical success, this research stands out for its pioneering analysis of the brain pathways involved in the improvement of patients treated with DBS. Using connectomics, the team identified the possible involvement of corticorubral tracts, a network of connections between the cerebral cortex, the red nucleus—located in the midbrain—and the cerebellum, which appear to play a key role in the beneficial effects of DBS. This analysis provides a better understanding of how and why some patients respond to the treatment and lays the groundwork for future research aimed at optimising candidate selection and improving clinical outcomes.

Dr Juan Ángel Aibar, from the Neurosurgery Research Group at IR Sant Pau and co-author of the study, explains that “DBS offers hope for those CRCH patients who find no relief in any other therapy. Advancing our understanding of the brain pathways activated by this intervention helps us comprehend the mechanisms behind its effectiveness, which could, in the future, allow us to identify the most suitable patients for this type of treatment.”

A path to excellence in treatments for refractory diseases

Hospital Sant Pau is currently the only centre in Spain with an established DBS programme for treating refractory cluster headache. This advance reflects the commitment of the hospital and IR Sant Pau to positioning themselves as a reference centre for treating refractory diseases—those for which all conventional therapies have failed.

The authors of the study highlight that while the treatment has shown high effectiveness in many patients, it is important to maintain realistic expectations, as this therapy does not cure the disease but significantly reduces its impact. The positive results observed in this research, particularly in male patients, also underline the need for further studies investigating how biological differences might affect the response to therapy in men and women.

Future perspectives

The Sant Pau team will continue its research, seeking to refine the therapies available for patients with chronic refractory cluster headache and deepen the understanding of DBS mechanisms of action. According to their estimates, there are still individuals in Spain who could benefit from this therapy but are not currently receiving appropriate treatment due to a lack of awareness about this innovative option.

This study reinforces the importance of reference centres and underscores the role of dissemination and education within the medical and scientific communities to ensure that patients with extreme and unmet needs can access cutting-edge care.

Reference article:

Aibar-Durán, J. Á., González, N., Mirapeix, R. M., Sánchez-Mateos, N. M., Arsequell, C. R., Pichot, M. B., … Rodríguez Rodríguez, R. (2024). Deep brain stimulation for chronic refractory cluster headache: A case series about long-term outcomes and connectivity analysis. Headache. doi:10.1111/head.14875

The Sant Pau Research Institute (IR Sant Pau) and the Fundació Amics Joan Petit Nens amb Càncer have launched an innovative project aimed at improving the quality of life for children with cancer who need to undergo a bone marrow transplant. This initiative seeks to demonstrate that physical exercise prior to the procedure, tailored to their functional abilities, can be safe and provide significant benefits during the treatment process.

With a budget of €100,000, the project will run for three years. The necessary funds are being raised through various charitable activities organised by the Fundació Joan Petit. Among these is the campaign “Let’s Fill the Roller Hockey Rinks with the Joan Petit Bracelet,” driven by the Catalan Skating Federation, which encouraged athletes and fans to purchase a solidarity bracelet to support young patients and their families. This campaign is set to be repeated early next year. Funds already raised have been delivered to kick-start the project, and future initiatives are expected to help complete the total budget.

Charity Event and Fund Handover

On Tuesday, 26th November, a ceremony was held in the lobby of the Hospital Sant Pau, where the Fundació Joan Petit delivered funds raised through the Catalan Skating Federation. The event also featured a symbolic hockey match played by children from different teams, held in the corridors of the hospital’s Paediatrics ward. Players from F.C. Barcelona, Xavier Barroso and Sergi Aragonès, participated alongside coach David Cáceres, and handed out gifts to the hospitalised children.

During the event, Joan Torner, president of the Fundació Joan Petit Nens amb Càncer, highlighted the resilience shown by children facing cancer, drawing a parallel with roller hockey: “In hockey, when you fall, you must get up and keep playing. Similarly, these children teach us every day that despite setbacks, you must rise and move forward with strength and hope.”

The Value of Prehabilitation Exercise

The project, named HIIT-FIT-KIDS-ALLO-T-TRIAL, is a pilot programme combining supervised physical exercise for children and adolescents preparing for an allogeneic haematopoietic progenitor transplant (allo-HPT). This medical procedure replaces diseased bone marrow with healthy marrow and is used to treat conditions such as leukaemia, lymphoma, and certain hereditary syndromes. While this treatment significantly increases survival rates, it also raises the risk of cardiovascular complications, exacerbated by the side effects of chemotherapy and radiotherapy.

The project’s innovative approach involves implementing a prehabilitation programme —planned and supervised exercise before the transplant— to strengthen the patients’ physical condition. According to Dr Roser Álvarez-Pérez, principal investigator of the project and researcher in the Paediatrics group at IR Sant Pau, “although the concept of prehabilitation has been successfully tested in adults, it remains an unexplored field in paediatrics. This project aims to improve patients’ functional capacity and cardiovascular function, preparing them to face the transplant in better physical condition while minimising the impact of prolonged hospitalisation.”

The study comprises three key phases: before, during, and after the transplant. The first phase, known as prehabilitation, begins four to six weeks before the procedure and includes a tailored exercise programme based on the patient’s functional capacity, assessed through tests such as blood analysis, electrocardiogram, echocardiogram, and an exercise stress test.

During the hospitalisation phase, supervised exercises will be performed daily in the patient’s room, conditions permitting. Post-hospitalisation rehabilitation will continue for six months, combining in-hospital sessions with activities that patients can carry out at home.

The intervention group will participate in a structured programme incorporating high-intensity interval training (HIIT), a method alternating short periods of maximum effort with rest, optimising results even with limited time. In contrast, the control group will receive conventional care, including standard physical activity recommendations for these patients, but with less structured exercise.

Ensuring the safety of the programme is a priority, with evaluations to rule out contraindications such as fever or low platelet counts. “The primary goals of the study are to determine the programme’s feasibility —whether children can consistently participate given treatment limitations— and its safety, ensuring it does not cause additional complications,” Dr Álvarez adds.

Future Prospects

In addition to immediate benefits, the project aims to promote long-term healthy habits, integrating exercise as a key component of medical treatment. According to Dr Álvarez, “just as pre-season training improves fitness to face competition, prehabilitation with exercise prepares patients to tackle the challenge of the transplant with more energy and strength.”

With a planned duration of three years and an estimated annual participation of 15 patients, this innovative programme could lay the groundwork for future multi-centre research and establish prehabilitation exercise as a fundamental tool in paediatric cancer treatment.

This project not only represents progress in paediatric medicine but also serves as an example of how collaboration between institutions and community support can profoundly impact the lives of children facing severe illnesses.

With the collaboration of:

IR Sant Pau has been recognised with the award for best oral presentation at the 15th National Congress of Paediatric Cardiology and Congenital Heart Disease (SECPCC), recently held in Murcia, for a study exploring the genetic basis of paediatric myocarditis. This accolade, granted at the main scientific event for paediatric cardiologists in Spain, highlights the significance and impact of this discovery in the field of cardiovascular medicine.

Myocarditis, an inflammation of the heart muscle, has traditionally been considered an acquired condition, generally caused by viral infections. However, recent research suggests that some cases of myocarditis may have an underlying genetic component. The study, led by Dr Roger Esmel, a member of the Paediatrics research group at IR Sant Pau and a paediatric cardiologist at Hospital Sant Pau, has delved into this hypothesis, focusing on paediatric patients, a group that has received limited attention in this area.

“The incidence of myocarditis is far lower than that of inflammatory conditions in other organs, such as gastroenteritis, bronchitis, or meningitis, raising the question of why some individuals develop myocarditis while others do not, even when exposed to the same infections. Our study seeks answers in genetics,” explained Dr Esmel.

Methodology and Findings

The study, conducted over eight years in collaboration with multiple medical centres, analysed paediatric patients with uncomplicated myocarditis—those who did not experience severe heart failure or arrhythmias. Using state-of-the-art Next Generation Sequencing (NGS) technologies, the researchers identified pathogenic or likely pathogenic genetic variants in 23% of cases.

These findings represent a significant breakthrough, demonstrating that nearly one in four paediatric patients with myocarditis could have an underlying genetic predisposition. This discovery challenges the perception of myocarditis as purely an acquired disease and opens the door to new strategies for prevention and treatment.

A Shift in Clinical Management

Until now, many patients with uncomplicated myocarditis have been discharged quickly without thorough follow-up. However, the findings indicate that these patients may have a hidden risk of developing cardiomyopathies in the future, underscoring the importance of genetic evaluations in specific subgroups. “The aim is to identify at-risk patients from the first episode and provide them with more rigorous follow-up. This will not only improve their prognosis but also prevent severe complications in adulthood,” added Dr Esmel.

In addition to immediate clinical implications, the study also highlights the need to advance towards a personalised medicine model. Identifying clinical and radiological factors associated with genetic variants will enable the optimisation of medical resources, focusing genetic testing on patients with a higher likelihood of genetic predisposition. Despite the promising findings, Dr Esmel cautions that this is only the beginning. “Broader studies are needed to confirm our observations and establish robust recommendations that can be applied in clinical practice,” he noted.

Researchers from the Institute of Biomedical Research of Barcelona (IIBB-CSIC) and the Sant Pau Research Institute (IR Sant Pau), led by Dr Vicenta Llorente Cortes, in collaboration with teams from the CIBER areas of Cardiovascular Diseases (CIBERCV) and Diabetes and Associated Metabolic Diseases (CIBERDEM), have developed peptides that inhibit the aggregation of LDL cholesterol (low-density lipoproteins), thereby preventing the formation of atherosclerotic plaques in an experimental model of hypercholesterolaemia.

The study, recently published in the journal Atherosclerosis, also involved teams from Miguel Servet Hospital in Zaragoza and the University of Basilicata in Italy.

Atherosclerosis, one of the leading causes of heart attacks and strokes, is triggered by the accumulation of LDL cholesterol in the arterial walls. Once trapped in the arterial wall, LDL particles undergo modifications that promote the progression of plaques, which can rupture and obstruct normal blood flow by inducing the formation of clots responsible for acute myocardial infarction.

These newly developed peptides represent a promising and innovative advance in the treatment of this disease. Their potential is especially significant for patients genetically predisposed to atherosclerosis, such as those with familial hypercholesterolaemia, for whom current therapeutic options are limited and insufficient to reduce cardiovascular risk. This underscores the urgent need for new solutions.

Preventing Plaque Formation

As explained by Dr Vicenta Llorente Cortes, who leads the Lipids and Cardiovascular Pathology research group at IIBB-CSIC, “In this study, we developed peptides that bind to and stabilise the structure of LDL particles. Furthermore, in a humanised murine model for lipoproteins, we demonstrated that administering these peptides inhibits atherosclerosis by preserving the structural integrity of LDL.”

This effect, Dr Llorente elaborates, is achieved by stabilising the conformation of ApoB100, a protein found in LDL particles that is crucial for maintaining their integrity and preventing their modification within the arteries. “We also demonstrated the efficacy of this treatment in LDL samples isolated from patients with familial hypercholesterolaemia,” she adds.

The relevance of this entirely novel treatment lies in its unique ability to stabilise the structure of ApoB100 and preserve the integrity of LDL particles in the vascular wall, preventing their aggregation—a key process in the development of atherosclerosis.

Reference Article:

Benitez-Amaro, A., Garcia, E., La Chica Lhoëst, M. T., Martínez, A., Borràs, C., Tondo, M., … Llorente-Cortés, V. (2024). Targeting LDL aggregation decreases atherosclerotic lipid burden in a humanised mouse model of familial hypercholesterolaemia: Crucial role of ApoB100 conformational stabilisation. Atherosclerosis, (118630), 118630. doi:10.1016/j.atherosclerosis.2024.118630

L’Institut de Recerca Sant Pau (IR Sant Pau) ha estat reconegut en la primera edició de les beques de recerca Miquel Rutllant, atorgades per la Fundació Investigació Salut i Progrés (FISP), que tenen com a objectiu promoure projectes liderats per investigadors emergents per accelerar el desenvolupament de solucions terapèutiques innovadores en teràpies avançades, medicina personalitzada i medicina de precisió en malalties oncohematològiques. En aquest marc, el Grup d’Oncogènesi i Antitumorals ha rebut finançament per al projecte Nanoinmunoteràpia de precisió contra cèl·lules mare leucèmiques CXCR4+ en Leucèmia Mieloide Aguda, liderat pel Dr. Ugutz Unzueta, investigador Miguel Servet de l’IR Sant Pau.

Aquest projecte se centra en el desenvolupament d’una nova estratègia terapèutica personalitzada basada en nanoinmunoteràpia per al tractament de la leucèmia mieloide aguda (LMA). La innovadora aproximació combina una nanomedicina de precisió, dissenyada pel grup en col·laboració amb el Grup de Nanobiotecnologia de la Universitat Autònoma de Barcelona (UAB) i patentada conjuntament per la UAB i l’IR Sant Pau, que permet l’eliminació selectiva de cèl·lules mare leucèmiques CXCR4+. A més, aquesta estratègia pretén activar el sistema immunitari contra les cèl·lules malignes mitjançant la inducció d’una mort cel·lular immunogènica, alhora que integrarà bloquejadors de punts de control immunitari per potenciar encara més la resposta del sistema immune.

L’impacte potencial d’aquest projecte és significatiu, ja que, si es demostra l’eficàcia del nanoconjugat desenvolupat, la leucèmia mieloide aguda podria incloure’s en els assajos clínics de Fase I que l’equip està preparant amb la spin-off biotecnològica Nanoligent. Aquesta empresa, fundada per investigadors de la UAB i l’IR Sant Pau, és llicenciatària de les patents relacionades amb aquestes nanomedicines i preveu iniciar aquests assajos en els pròxims anys.

El projecte compta amb la participació d’un equip multidisciplinari format pel Dr. Ugutz Unzueta, la Dra. Isolda Casanova, la Dra. Lorena Alba, Annabel Garcia i Luis Carlos Navas, tots ells membres del Grup d’Oncogènesi i Antitumorals i del CIBER en Bioenginyeria, Biomaterials i Nanomedicina (CIBER-BBN), que treballen conjuntament per oferir noves perspectives terapèutiques per a una malaltia tan complexa com la leucèmia mieloide aguda. Aquesta beca suposa un pas més en el compromís de l’IR Sant Pau amb la recerca biomèdica d’excel·lència, posant el focus en solucions innovadores que puguin millorar la qualitat de vida dels pacients.

The 3cat Foundation has announced the research projects awarded in the latest edition of La Marató de TV3 and Catalunya Ràdio 2023, dedicated to sexual and reproductive health. A total of 26 research projects, led by 52 research teams, will receive funding. The Sant Pau Research Institute (IR Sant Pau) has been recognised with two projects coordinated by the centre and participation in a third. These scientific works address some of the most critical challenges in sexual and reproductive health. The selected studies aim to improve the treatment of preeclampsia, optimise the follow-up of patients with endometriosis, and advance the prevention and management of sexually transmitted infections (STIs).

Dr Elisa Llurba, head of the Perinatal and Women’s Medicine Research Group at IR Sant Pau, was one of the researchers participating in the presentation event held this morning at TV3’s facilities. In her speech, she highlighted the importance of this recognition for research on diseases that have traditionally been overlooked and underfunded, especially those affecting women and pregnancy. She emphasised that “true health cannot exist without women’s health” and underscored the gender perspective as a fundamental element to advance the health of society as a whole.

During the same event, Carme Figueras, Vice President of the Board of Trustees of the La Marató de 3Cat Foundation, highlighted the significance of this initiative, stating that “these initiatives not only drive research but also shed light on the social impact of often-silenced diseases.” Figueras also emphasised the critical role of La Marató in raising public awareness and fostering collective commitment, which translates into resources dedicated to scientific progress. These actions are key to reducing health inequalities and ensuring a future with more opportunities for everyone.

Innovative Treatment for Preeclampsia

The first project, led by Dr Elisa Llurba, addresses preeclampsia, a condition that affects one in ten pregnant women and often has severe consequences for both the mother and the baby. This condition is the second leading cause of maternal death and is linked to one in three premature births, as well as an increased risk of future cardiovascular diseases in both mothers and newborns.

“Our study explores the use of statins, a drug used to treat cholesterol, to improve placental function, prolong complicated pregnancies, and reduce the risk of severe complications,” explained Dr Llurba. The multicentre clinical trial, which will include 150 patients, involves collaboration with Hospital Clínic, Vall d’Hebron Hospital, and the Autonomous University of Barcelona. This approach aims to transform the management of a condition that, to date, has no cure, providing new solutions to improve outcomes for both mothers and babies.

Monitoring Patients with Endometriosis

The second project, led by Dr Rocío Luna, a researcher in the Gynaecological and Peritoneal Oncology Research Group at IR Sant Pau, analyses the progression and quality of life of patients with endometriosis, a chronic gynaecological condition affecting one in ten women of reproductive age. Endometriosis profoundly impacts patients’ quality of life and their families, but it often takes between 8 and 15 years to diagnose, and there are still no specific or effective treatments.

This project seeks to identify pharmacological and non-pharmacological factors that improve quality of life and establish guidelines for more effective clinical and ultrasound monitoring. “The goal is to generate new guidelines for comprehensive and personalised treatment that reduces complications and provides evidence-based solutions,” noted Dr Luna.

Breaking Barriers in STIs Among Young People

The third project, involving Dr Cristina Rius, head of the Communicable Diseases Research Group at IR Sant Pau, focuses on the prevention, early diagnosis, and management of STIs among young people in Catalonia. This study, led by Dr Cristina Agustí from the Health Sciences Research Institute of the Germans Trias i Pujol Hospital, also works on partner notification to reduce the spread of these infections. The goal is not only to curb infections, but also to break the stigma associated with these conditions and provide accessible tools for their prevention and treatment.

Recognition for Transformative Research

These three projects are part of the 26 selected by La Marató 2023, an edition focused on sexual and reproductive health, contributing to raising awareness and support for conditions affecting millions of people worldwide, especially women and young people. These often-overlooked and insufficiently addressed conditions have a profound impact on people’s quality of life and represent a crucial challenge for healthcare systems.

This recognition reinforces IR Sant Pau’s commitment to scientific innovation, public health improvement, and knowledge transfer to address historically neglected issues. Sant Pau reaffirms its role as a leader in biomedical research and as an advocate for the importance of applying a gender perspective in scientific research.

Dr Pablo Alonso, Director of the Epidemiology, Public Health, and Primary Care Area at the Sant Pau Research Institute and researcher at the Iberoamerican Cochrane Centre, has once again been recognised as one of the most cited scientists on the prestigious Highly Cited Researchers 2024 list, published by Clarivate Analytics. This achievement places him among the select group of researchers whose scientific output ranks in the top 1% most referenced globally in their respective fields.

Dr Alonso focuses on the development and application of scientific tools that guide clinical practice and health policy decisions based on the best available evidence. His commitment to high-quality research and innovation has led him to stand out not only nationally but also internationally.

“This recognition as a highly cited author is, above all, a reflection of the collective work and dedication of the GRADE group to high-quality scientific evidence and its application to clinical practice. This award is not only an individual acknowledgement but also a tribute to the interdisciplinary collaboration and commitment of everyone in the GRADE group who work tirelessly to improve clinical guidelines and health policies on a global scale,” explained Dr Pablo Alonso.

The Sant Pau Research Institute celebrates this significant recognition, which highlights the scientific excellence of its researchers and reinforces its commitment to generating knowledge for the benefit of society. Sant Pau congratulates Dr Alonso on this well-deserved achievement, which once again emphasises the importance of his contribution to scientific advancement.

Clarivate’s Highly Cited Researchers List

Since 2001, Clarivate has published an annual list recognising the elite group of international scientists and social researchers who have demonstrated outstanding global influence in their respective fields of study.

This prestigious selection is based on data from Web of Science™ and an in-depth analysis conducted by experts from the Institute for Scientific Information (ISI) at Clarivate. Researchers included in the list have authored multiple Highly Cited Papers™, articles ranked in the top 1% by citation count in their research field and year of publication over the past decade in Web of Science™. The Clarivate list is therefore an indicator of scientific excellence and highlights those leading the progress of knowledge in their disciplines.

Access the 2024 Clarivate Highly Cited Researchers list by clicking here.