LGS Foundation launches accelerator to jump-start rare epilepsy research
Connecting industry, researchers, advocates to speed treatment development
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The LGS Foundation has launched a research accelerator program to break down clinical trial roadblocks and fast-track better treatments for rare epilepsies. (Photo from iStock)
The Lennox-Gastaut Syndrome (LGS) Foundation has launched a collaborative research accelerator to unite experts, dismantle research roadblocks, and speed up the development of new treatments for LGS and related developmental and epileptic encephalopathies (DEEs).
The newly formed LGS and Associated DEE Research Accelerator brings together pharmaceutical companies, academic scientists, and patient advocates to solve systemic issues that hinder research and slow down clinical trials.
“Progress in LGS has been slowed by problems no single organization can solve alone, including inconsistent [clinical trial] endpoints and the lack of a shared evidence base,” Tracy Dixon-Salazar, PhD, president and CEO of the LGS Foundation, said in a press release. “The Accelerator gives the community a place to fix those barriers together, so the science moves faster and patients see the benefit sooner.”
LGS is a severe form of epilepsy marked by multiple, treatment-resistant seizure types, along with a range of developmental issues, including intellectual disabilities, behavioral challenges, and movement difficulties. It belongs to a broader group of rare genetic conditions known as DEEs, which are all associated with seizures and developmental problems.
Addressing roadblocks in therapeutic development
A combination of available therapies is often insufficient for controlling seizures and other symptoms in people with these disorders. However, disconnected research efforts, redundancy, and slow data sharing have hindered progress in developing new treatments, according to the foundation.
The organization hopes that the Accelerator will provide a coordinated space for researchers and clinicians to align on research priorities, troubleshoot roadblocks, and pool resources to facilitate faster therapeutic development.
Participants will gather at an annual meeting and in smaller working groups throughout the year. All members will work together to set the research agenda, focusing on the most important unmet needs in LGS and associated DEEs.
Among the initial focus areas will be the lack of consistent clinical trial endpoints for determining whether an experimental treatment is effective. A priority of the program will be to identify biomarkers for monitoring treatment responses across clinical studies.
Another goal is to incorporate shared natural history data as a real-world evidence arm in clinical trials. Natural history studies are observational studies that follow people with a disease who are receiving standard care. This allows scientists to understand the disease’s natural course and thus how an investigational therapy might alter it.
The LGS Foundation aims to ensure that all Accelerator participants have access to data from its LGS natural history study, LGS-CORE, for use in their research. Through annual surveys completed by families, the registry collects data on LGS symptoms and their progression, medications, and quality of life. Families who wish to participate can register online.
Other pressing needs include identifying risk factors for death in LGS and establishing family-approved outcomes measures for monitoring LGS. Additional focus areas can be proposed and voted on by members each year.
