ALS Therapy Development Institute to Explore New Clinical Trial Endpoints with Denali Therapeutics

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Research Collaboration will Utilize Data from the Institute’s Precision Medicine Program

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The discovery and development of sensitive endpoints of disease progression may help accelerate all ALS clinical trials.

The ALS Therapy Development Institute announced today a new research collaboration with Denali Therapeutics to investigate potential new endpoints for use in ALS clinical trials. The Institute and Denali will evaluate and analyze data sets collected by the Institute through the first-of-its kind Precision Medicine Program.

More than 250 people from 40 US states and 6 countries are already enrolled in the Program. In addition to completing health and other surveys, participants provide ongoing disease progression data from home using three measures: the revised ALS Functional Rating Scale (ALSFRS-R), speech recordings and accelerometers.

“Translating potential treatments for ALS from the lab to clinical trials is a crucial step in solving the huge unmet patient need in ALS. The discovery and development of sensitive endpoints of disease progression may help accelerate all ALS clinical trials. This collaboration is a great example of how we are working with leading companies to do so, while at the same time identifying the most exciting treatments to put into clinical trials,” said Steve Perrin, PhD, CEO & CSO of the ALS Therapy Development Institute.

Denali’s Chief Medical Officer Carole Ho, MD, commented: “Denali is committed to developing therapeutics for neurodegenerative diseases. We are excited to collaborate with the ALS Therapy Development Institute, a leader in the field, to develop wearable technology endpoints and internet based tools. They will enhance our ability to track disease progression and response to therapy in clinical trials.”

Clinical trial endpoints are measures used in clinical trials to determine whether or not the treatment being investigated affects disease progression. The discovery of new endpoints to measure disease progression may help to reduce the number of people or the amount of time a trial needs to run in order to determine statistically significant efficacy of a treatment.

Currently, most ALS clinical trials use a scale known as the ALSFRS-R, a questionnaire utilized by clinicians to evaluate the pace at which a person’s disease is advancing. However, ALS is a heterogeneous disease in which progression rates vary greatly among individuals with ALS. Due in large part to this heterogeneity, researchers have determined that upwards of 450 people with ALS are required to be tracked for more than a year in a clinical trial using ALSFRS-R to measure a statistically significant 30% effect of a potential therapeutic on disease progression.

“I’m grateful to be a part of ALS TDI’s promising Precision Medicine Program. I believe that their work with Denali Therapeutics will speed up the discovery process and get us closer to treatments and a cure for ALS,” said Sarah Coglianese of San Francisco, a person with ALS and National ALS.net Ambassador.

About the Precision Medicine Program
The Precision Medicine Program at the ALS Therapy Development Institute is a key part of the Institute’s efforts to discover and develop effective treatments for ALS. The program became the first ALS-focused precision medicine program in the world when it enrolled its first participant in late 2014. Today, there are more than 250 people enrolled from 40 US states and 6 countries and more are being enrolled each week. Uniquely, data collected in the Precision Medicine Program is shared via a secure online portal with participants directly, making it one of the most open research programs ever attempted in ALS. Participants provide ongoing disease progression data from home using three measures: ALSFRS-R, speech recordings and accelerometers. They also provide rich demographic and epidemiological data through the completion of 15 different surveys covering everything from where they have lived to what type of medications and supplements they are taking, among other things. Blood and skin samples are collected at enrollment from which whole genome sequencing and induced pluripotent stem cell lines may be created. The data created through these and other experiments are actively being mined by scientists at the Institute and its research partners to identify potential clinical trial endpoints, subgroups of people with ALS and to inform drug screening. For more information, visit http://www.als.net/ips.

About the ALS Therapy Development Institute
The ALS Therapy Development Institute and its scientists actively discover and develop treatments for ALS. The Institute is the world’s first and largest nonprofit biotech focused 100 percent on ALS research. Led by people with ALS and their families, the charity understands the urgent need to slow and stop this horrible disease. Based in Cambridge, MA, the Institute has served as one of the leaders in sharing data and information with academic and ALS research organizations, people with ALS and their families. For more information, visit http://www.alstdi.org.

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