Neurology & Therapy

Advancements in Epilepsy Research

Andrea_Puletto — Thu, 01 Aug 2024 11:25:23 +0000

Advancements in Epilepsy Research

Liesbeth Fran莽ois, Data and Translational Sciences

Andrea_Puletto Thu 01/08/2024 - 13:25

Although the field of epilepsy research is well-established, we have only just begun to scratch the surface of our understanding of more complex epilepsies. We are entering a period of disruption, where new breakthroughs and the integration of innovative technologies into drug discovery and development show promise in transforming epilepsy care. One area is 鈥�molecular hallmarks鈥� 鈥� molecular signatures that contribute to the presentation of complex epilepsies.

Discovering 鈥榤olecular hallmarks鈥�

At 金禾娱乐城, a key area of our research is focused on broadening targets for treatments. In a study published earlier this year, we identified well-known and new gene regulatory networks shared across different refractory epilepsies; we called these 鈥榤olecular hallmarks鈥�.i This research was the culmination of five years of collaborative work that has enabled us to start to advance our understanding of hard-to-treat epilepsies and opened new possibilities for drug-target discovery and development.

鈥楳olecular hallmarks鈥� are currently under study by our Early Development team who have mapped out a strategy for further research to potentially make the hallmarks targetable. The research targets include neuronal support and myelination, neuroinflammation and immune response, energy metabolism, and the brain extracellular matrix (ECM).

Targeting complex epilepsies

Epilepsy is more than seizures and we are continuing to uncover the vast complexities of the condition. 鈥楳olecular hallmarks鈥�, therefore, offer potential additional targets for epilepsy treatments that go beyond seizure suppression, enabling us to target the underlying mechanisms of the most challenging seizure disorders. This shift represents a move away from conventional treatment methods, and into research that focuses potentially disease modifying therapies.

This 鈥榤olecular hallmark鈥� research is an important part of our epilepsy strategy and provides a data-driven framework for the further identification and intervention of further dysregulated biological pathways.

Ultimately, our goal at 金禾娱乐城 is to develop new treatment options, based on these scientific discoveries, that can attempt to enhance patient outcomes.

The role of computational biology

Epilepsy is not a one-size-fits-all condition. Each person鈥檚 experience of living with epilepsy is unique and given what we know so far about the complexity of epilepsies, there is a clear role for computational biology in epilepsy research. Through analysis of the expression profiles of refractory epilepsies, we can start to understand global biological mechanisms that are impacted in refractory epilepsies. Our research continues to drive our understanding of the human pathobiology of different refractory epilepsies to support our aim to identify new disease modifying treatments for patients. This research aims to help us comprehend the mechanisms underpinning epilepsy and enable the development of new therapies for refractory epilepsies.

The future of research

Everything we do starts with people and families living with epilepsies, helping them achieve their ideal and maximize their life opportunities. Looking to the future as we advance our epilepsy research, we recognize that those living with complex epilepsies will continue to rely on chronic, symptomatic anti-seizure medicines, and we continue our research to help improve outcomes with the symptomatic approaches available to patients today. However, we constantly need to push our approaches to secure the best outcome for those we serve. Molecular hallmarks and master regulators provide invaluable insights and open new avenues for precision medicine and new treatments that address the unmet needs of people living with epilepsies.

innovation

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Innovating in Epilepsy

Andrea_Puletto — Fri, 14 Jun 2024 11:19:04 +0000

Innovating in Epilepsy

Mike Davis, Epilepsy & Rare Syndromes

Andrea_Puletto Fri 14/06/2024 - 13:19

In recent years, epilepsy research has evolved from focusing solely on symptomatic control of seizures to investigating the underlying pathologies, aiming to develop disease-modifying and ultimately curative therapies.

At 金禾娱乐城, we understand the importance of staying at the forefront of innovation, and prioritizing research and development within the dynamic category of epilepsy - principles that have built our 30-year heritage in this area. To ensure we are a part of this evolution, we need to advance and progress. Our people-first approach focuses on three areas of development:

Driving innovation in science
Partnering with patients to advance the ecosystem
Elevating the standard of care for all people living with epilepsies

We are entering a rapid period of disruption where we need to address multiple unmet needs of specific patient populations through evolving modalities, targets, and care delivery. To achieve this, we need to adopt new approaches that employ emerging technologies and require capabilities beyond anything currently present in the market.

We are focused on the development of an extensive future epilepsy portfolio, made up of targeted, precision medicines, focusing on the entire spectrum of epilepsies. As well as continually improving the outcomes of the symptomatic approaches, available to patients today.

Advancing symptomatic care

Our research focuses on developing a better understanding of the prevention of seizures and the underlying mechanisms of the most challenging seizure disorders. We are advancing distinct solutions across many different epilepsies for specific patient populations, and have several early discovery phase research projects, involving both small molecules and gene therapies, that are currently active.

Our goal is to disrupt this space by advancing solutions across many different domains and dimensions of the epilepsies. This starts with our current products where we continue to research their impact on non-seizure outcomes relating to cognition, behavior, emotional development, and sleep architecture; neurodevelopment delays; corollary links to other neurodegenerative diseases; Sudden Unexpected Death in Epilepsy (SUDEP) and all-cause mortality.

Disease modification

Teams from across 金禾娱乐城 are envisioning a next generation of therapies that will take epilepsy care beyond symptomatic treatments by targeting the underlying mechanisms that lead to both seizure and non-seizure effects of the diseases and syndromes we aim to treat and opening the door to disease modifying and curative solutions. Working together, we are exploring fundamental disease etiologies across the epilepsies, uncovering root causes of monogenic epilepsies, and targeting the pathobiology of structural epilepsies, such as the role neuronal support and myelination, neuroinflammation and immune response, energy metabolism, and the brain extracellular matrix (ECM) play in the refractory complex epilepsies.

The role of AI

Technology and AI are having an increasingly significant role in epilepsy research and development - for instance, to help patients and families better characterize and manage their seizures. Our technology-based partnerships with Eysz and NextSense introduce new ways to track brain activity outside traditional EEG monitoring. Furthermore, our R&D teams continually harness 金禾娱乐城鈥檚 deep institutional knowledge of the pathobiology of epilepsies. Using advanced analytics, we leverage AI resources to strengthen our discovery and development efforts. This includes identifying mechanisms and developing disease models through to prioritizing potential targets.

Broadening access

At 金禾娱乐城, we believe that all patients who need our treatments should have access to them now and in the future. Health inequities are a challenge in bringing innovations to people living with epilepsies and elevating their standard of care. We鈥檙e working to address this through sustainable approaches to reach the historically underserved people of lower-middle-income countries and address their unmet needs. For instance, in Rwanda, we have recently worked with the government to enable those living with epilepsy to have access to 金禾娱乐城 treatment.

Health equity is also about deepening our local connections in the communities where we operate. This starts with understanding how diverse patient groups experience care and treatment, to better understand where social determinants of health may have exacerbated health inequities among historically underserved communities and define targeted approaches.

Future thinking

Looking ahead, we expect to see the continuing emergence of treatment modalities, delivery technologies, and hidden insights into underlying pathobiology. This has the potential to advance the development of disease-modifying solutions and support the progress toward curative medicine options for those living with epilepsies. Ultimately, our goal at 金禾娱乐城 is to continue to innovate across the entire spectrum of epilepsy care 鈥� we want to treat the treatable and cure the curable.

innovation

Neurology & Therapy epilepsy neurology

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World Parkinson鈥檚 Day: Why the urgency for innovation grows greater every year.

Andrea_Puletto — Thu, 11 Apr 2024 07:27:26 +0000

World Parkinson鈥檚 Day: Why the urgency for innovation grows greater every year.

Thomas Morel, Patient-Centered Outcomes Research

Andrea_Puletto Thu 11/04/2024 - 09:27

As we mark another World Parkinson鈥檚 Day, my experience in patient-centered research has taught me one thing: the urgency for innovation in this disease area grows greater every year.

The vision of the Parkinson鈥檚 Community is to reach the goal of disease modification 鈥� to alter the course of Parkinson鈥檚 using interventions that target the underlying cause of disease. However, for me, the term 鈥榙isease modification鈥� can be a bit of a banana skin, likely to trip us up, and raise more questions than it answers. People with Parkinson鈥檚 currently rely on symptomatic treatments, interventions that were assessed in late-stage Parkinson鈥檚 with established outcome measures, such as the MDS-Unified Parkinson's Disease Rating Scale (MDS-UPDRS). In contrast, disease modification will require early intervention in people with early-stage Parkinson鈥檚, who experience a variety of motor and non-motor symptoms that cannot be assessed in a sufficient granular way using existing outcome measures. This, in a field without valid biomarkers and where even the definition of early-stage Parkinson鈥檚 disease can be unclear!

Our recent collaboration with people with Parkinson鈥檚 to co-create patient-reported outcomes (PROs) sought to answer some of these questions. We worked collaboratively with people living with Parkinson鈥檚 as joint investigators alongside clinical, regulatory, and outcome measurements experts. The group was involved across all phases of research; firstly, identifying the symptoms that are cardinal and important to the experience of living with early-stage Parkinson鈥檚, and latterly, in the development of PRO instruments to better assess these symptoms. People with Parkinson鈥檚 informed every stage of our program, from qualitative study protocol design, conceptual model development, and subsequent co-creation of two PRO instruments.

This collaboration confirmed two important theories for me; firstly, the perspectives of people living with Parkinson鈥檚 are invaluable in research. Secondly, for the kind of deep, co-creation relationships needed to understand and fight this disease, it鈥檚 essential to foster a culture of collaboration, trust, and respect with people living with Parkinson鈥檚.

As a disease area, Parkinson鈥檚 has a lot of catching up to do. For me, we will continue to fail as an industry if we continue to rely on legacy measures of treatment success that are not fit for purpose. If we continue to view Parkinson鈥檚 as simply a movement disorder, and if we think of people living with this condition as an afterthought in our research journey. Patient experiences in early-stage Parkinson鈥檚 are complex and wide-ranging, currently available outcome measures do not capture this and new, innovative approaches are urgently required. I can only commend Critical Path Parkinson鈥檚 pre-competitive collaboration initiative in this regard to address this community-level unmet need on outcomes measures and clinical study endpoints to accelerate drug development in Parkinson鈥檚.

My hope for the years ahead is that we鈥檒l get that much closer to the goal of really tackling the underlying cause of this disease, working with people with Parkinson鈥檚 on a solution that improves their lives now and in the future.

innovation

World Parkinson鈥檚 Day Parkinson Neurology & Therapy Neurology

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Unique patient-led study into life with myasthenia gravis published in Neurology and Therapy

eCMSadmin — Thu, 18 Nov 2021 09:00:00 +0000

Unique patient-led study into life with myasthenia gravis published in Neurology and Therapy

Kenza Seddik, Patient Value Neurology & Europe/International Solutions

eCMSadmin Thu 18/11/2021 - 10:00

Myasthenia gravis (MG) is a chronic neuromuscular condition, with an annual incidence of 8 鈥� 10 cases for every 1 million, which can occur at any age and in any race. , As a rare, yet severe disease, working directly with the patient community to better understand their lived experience is so important, to help us identify unmet needs and any gaps in access to care.

Recently, a patient-led and co-authored study into the real-life experience of MG was published in Neurology and Therapy, bringing to life the true impact of the disease on patients鈥� everyday lives. Contributing to this unique analysis has been insightful in so many ways, and collaborating with people living with MG was a valuable opportunity to understand first-hand the challenges they experience.

This collaboration was led by a Patient Council; nine patient advocates across Europe and the United States, all living with MG. Strong partnerships like these between patient advocates and 金禾娱乐城 are one step forward in our mission to support innovation in MG and help facilitate more open dialogue between patients and doctors.

Throughout the writing of this report, five key themes were uncovered that help shine a light on the reality of living with MG and emphasise what it means to live with a disease with such varying, fluctuating symptoms, that are not widely understood. We learned from patients the difficulty of adapting their everyday life to manage their symptoms, the challenges of communicating their experience to their physicians, and the emotional toll this takes.

You can read the full patient-led report in Neurology and Therapy and find out more about these key themes here.

We鈥檙e proud that this analysis can sit alongside the growing body of valuable literature in this space. Through listening to patients and learning from their insights, we hope to elevate the patient voice, gain a deeper understanding of their experience, and ultimately work together to fulfil unmet needs.

The development of 鈥楾he Lived Experience of Myasthenia Gravis: A Patient-led Analysis鈥� manuscript was led by two patient advocates in MG: Nancy Law from Colorado, USA and Kelly Davio from London, UK. The study has been published posthumously following Nancy鈥檚 death in September 2021 and her invaluable contribution to this paper and leadership of the MG patient advocacy community is hugely appreciated by all involved.

solutions

Neurology & Therapy MG

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Neurology & Therapy

Advancements in Epilepsy Research

Discovering 鈥榤olecular hallmarks鈥�

Targeting complex epilepsies

The role of computational biology

The future of research

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Innovating in Epilepsy

Advancing symptomatic care

Disease modification

The role of AI

Broadening access

Future thinking

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World Parkinson鈥檚 Day: Why the urgency for innovation grows greater every year.

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Unique patient-led study into life with myasthenia gravis published in Neurology and Therapy

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