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Epilepsy, one of the most widespread neurological diseases in the world, characterized by recurrent seizures, affects approximately 50 million people globally. In Europe, up to six million people are estimated to be living with the disease. Whilst treatment options have come a long way over the last few decades, clinical practice still shows significant unmet needs for people living with epilepsy.
With an ultimate goal of preventing epilepsy or at least, modifying the disease or achieving seizure freedom for every patient with epilepsy, Angelini Pharma is dedicated to advancing epilepsy treatment, education on the biology of the disease and complexity of the different types and syndromes by working directly with patients, healthcare professionals and multidisciplinary stakeholders to understand the challenges they face.
In this article, I will provide an overview of the current treatment landscape for people living with epilepsy, the research and development into new and innovative solutions for them to potentially achieve seizure freedom, and the challenges faced when adopting scientific innovation into clinical practice. It is important to understand how these challenges create further barriers for people living with epilepsy, the impact they have on their quality of life and what can and is being done to address these specific challenges.
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Leading a strong movement to diversify epilepsy research
Over the past 10 years we have seen scientific breakthroughs in understanding epilepsy genetics, and in the development of novel tools for target-driven approaches. Building on these results, it’s inspiring to see how epilepsy research and development continues to diversify to tackle the unmet need that still persist. From structural to genetic, infectious to metabolic and immune or developmental epilepsies, researchers are driving innovation in many different areas. Just last month, the manuscript that I co-authored, ‘New epilepsy therapies in development,’ published in Nature Reviews Drug Discovery highlighted that there is currently a encouraging pre-clinical and clinical treatment pipeline, with more than 200 projects at different stages for different types of epilepsies and diseases with seizures, with the principles of precision medicine being applied to epilepsy.
Looking ahead, the future of epilepsy treatment holds promising innovations. Gene therapies, while still far ahead on the horizon, offer the potential to be curative for certain rare genetic epilepsies and could be revolutionary when they arrive. These therapies represent a transformative approach, directly addressing the underlying causes of specific epilepsy syndromes. Additionally, novel therapeutic modalities, such as antisense oligonucleotides which could be positioned between small molecules and gene therapies, are already in clinical development and may soon provide new, precise treatment options.
At Angelini Pharma, we will continue to maintain our position at the forefront of epilepsy research, with strategic investments in small molecules, biologics, and innovative therapeutic approaches to find solutions for people living with epilepsy. We focus on areas where we can make a significant impact, leveraging our capabilities and partnerships to push further into previously unexplored areas to elevate our understanding of epilepsies and develop potentially groundbreaking therapies. For instance, our collaborations with JCR Pharmaceuticals, the Mario Negri Institute for Pharmacological Research (IRCSS) and initiatives including the Open Innovation Challenge remain top priorities to ensure that we are engaging, supporting and inspiring the patient and physician communities to advance epilepsy care and improve patient outcomes.
Understanding challenges in adopting scientific innovation into clinical practice
Despite the advancements to date, a gap persists between scientific innovation and clinical practice. Real-world settings often present challenges that hinder the adoption of new treatments. One significant issue is the reluctance to embrace new therapies due to a historical nihilism—stemming from the belief that new drugs may not significantly improve patient outcomes. This skepticism is compounded by the complexities of polytherapy, where patients are treated with multiple drugs, leading to cumulative side effects, which all create a poor quality of life for those living with epilepsy. Optimised selection of modern treatment options and reduction of the overall drug load could potentially improve efficacy and limit side effects at the same time.
To better understand patient needs and improve patient care, we partner and collaborate with patients and their communities, patient groups and organizations, academic collaborators, and other leading biotech companies. One example is the Headway initiative which we have been running together with The European House – Ambrosetti since 2017. The program regularly maps the European epilepsy care landscape and, importantly, highlights where the healthcare system does not yet fully meet patient needs. Headway therefore provides crucial information to help uncover barriers and elevate clinical practice.
Adopting patient-centric approaches to improve quality of life
Epilepsy significantly impacts patients’ daily lives, and particularly their quality of life. A recent cross-sectional study published by Siebenbrodt and collaborators shows that quality of life is affected by a variety of factors. Disease severity, seizure frequency and tolerability of anti-seizure medications as well as the presence of depression, stigma, and worry about new seizures are all strongly associated with poor quality of life.
A patient-centric approach, emphasizing education and communication between healthcare providers and patients, is vital for optimizing treatment outcomes. Patients play an increasingly important role in treatment decisions —they know their experiences and needs best. Therefore, empowering them with knowledge about their condition and treatment options available to them enables them to make informed decisions alongside their doctors. Healthcare providers can also benefit from informed patients and leverage their experiences to determine new treatment regimens and inspire fresh approaches to research and development.
At Angelini Pharma, we champion a patient-centric approach and are committed to listening to and amplifying the voices of people with epilepsy for improving their quality of life. One of the ways we are doing this is by encouraging the implementation of Patient-Reported Outcome Measures (PROMs) and Patient-Reported Experience Measures (PREMs) into clinical practice. Our initiative, “PROMs and PREMs in Epilepsy Women of Childbearing Age”, seeks to foster a patient-centered approach to epilepsy care that prioritizes the real experiences of people living with epilepsy. The initiative is specifically designed for women who experience drug-resistant epilepsy in childbearing age and aims to create and validate PROMs and PREMs instruments in epilepsy treatment. Starting from 2025, we will be able to start gathering insights from the PROMs and PREMs using validated tools, hence significantly contributing to advancing better care and to enhancing quality of life for people with epilepsy.
Conclusion: Pushing the boundaries for patient care in epilepsy
The journey towards better epilepsy care and ultimately, seizure freedom, is one of continuous innovation and collaboration. By engaging with the community and fostering professional partnerships, we can advance epilepsy treatment and close the gap between clinical practice and scientific innovation. However, as with all ambitions, strength comes from unity, and we invite our industry peers, clinician and patient communities to come together to share feedback and insights and work together to improve epilepsy care worldwide. Let’s continue to push the boundaries of what’s possible and strive for a future where every patient with epilepsy can achieve seizure freedom and a better quality of life.
If you would like to learn more about pre-clinical and clinical treatment pipelines for different types of epilepsies and diseases with seizures, visit Nature Reviews Drug Discovery to read the article, ‘New epilepsy therapies in development.’