200th Participant Enrolled in Precision Medicine Program for ALS

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ALS Therapy Development Institute reaches 2/3 milestone towards goal of 300

Kevin Gosnell (center) with his wife, Kathy, and three sons, Scott, Jake and Joe, before their Ice Bucket Challenge during T&K Asphalt’s Family Day in August 2015.

Enrolling in the Precision Medicine Program [at the ALS Therapy Development Institute] gives me hope today that I can contribute to research that might lead to treatments and a cure for ALS.

The ALS Therapy Development Institute announced today that it has enrolled the 200th participant in its Precision Medicine Program (PMP). This program is the first of its kind to be created for ALS and includes multiple aspects unique to the field of precision medicine specifically aimed to discover and develop treatments for ALS. This milestone meets two-thirds of the program’s goal for enrollment of 300 participants, which was made possible by the donations received from the 2014 social media phenomenon, the ALS Ice Bucket Challenge.

The ALS Therapy Development Institute is executing the world’s first precision medicine program specifically focused on ALS. The goal of the program is to identify biomarkers and outcome measures to stratify subtypes of ALS patients. Accurate stratification of ALS patients based on disease progression rate will facilitate more rapid clinical trial enrollment and shorter endpoints, thus enabling more efficient clinical outcomes. The Institute is providing all participants with access to the data via a secure online portal, where they can monitor the progression of the program overall and explore data created from their own genome sequencing, iPS cell line development and drug screening. Participants can also monitor changes in their disease progression as measured from accelerometer data, the ALSFRS-R score and soon to be analyzed speech recording data collected from each participant. Data obtained by the Precision Medicine Program will be instrumental for identification of the subtypes of ALS, as well as for the discovery and clinical development of therapies for ALS.

“Every person enrolled in our Precision Medicine Program is an active participant in research aimed to find effective treatments and a cure for ALS,” said Steve Perrin, Ph.D., CEO and CSO of the ALS Therapy Development Institute. “For the first time, our scientists have access to an unprecedented amount of data in ALS that we are using expeditiously towards discovering therapies for people living with this disease.”

The 200th participant in the Institute’s Precision Medicine Program is 47 year-old Kevin Gosnell, who was diagnosed with ALS in May. Gosnell is from Hanover, MA, and is the CEO and Owner of T&K Asphalt Services, Inc.

“ALS is a disease that has no cure, no real treatment, and it’s fatal. As of right now, I have no use of my right leg, and picking up a cup with my right arm is incredibly difficult,” said Gosnell, who enrolled in the Precision Medicine Program this morning. “Enrolling in the Precision Medicine Program gives me hope today that I can contribute to research that might lead to treatments and a cure for ALS.”

For more information on the 200th participant and others in the Precision Medicine Program, please visit http://www.alstdi.org/ips. A presentation on the data produced from the program thus far will be made by Matvey Lukashev, Ph.D., during the Institute’s public research symposium on November 13 in Boston. To register for that event, visit http://www.alstdi.org/summit.

About The Precision Medicine Program at the ALS Therapy Development Institute
Precision medicine is an emerging field of biomedical research that aims to leverage patients’ genomic and other molecular or cellular data together with their clinical information to more rapidly identify potential therapies. The Institute’s Precision Medicine Program seeks to gain critical new insight into the mechanisms of ALS through integrative analysis of each participating patient’s genetic data, obtained by full genome sequencing, and their clinical data including a combination of monthly self-reporting questionnaires, motion tracking, and voice recordings. This information will be linked to data obtained by analyzing patient-derived cells that are differentiated from induced pluripotent stem cells (iPSC). These patient-derived cells will facilitate identification and development of better-focused ALS drug discovery screens.

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 ALS patients 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, patients and their families. For more information, visit http://www.alstdi.org.

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