100th Patient Enrolled in Worlds First Precision Medicine Program for ALS

Cambridge, MA (PRWEB) April 14, 2015

The ALS Therapy Development Institute announced today that it has enrolled the 100th patient in its Precision Medicine Program (PMP). This milestone marks a significant step in the program, which after today includes over 500 people interested in participating and 280 prescreened for enrollment. Nearly 200 additional people living with ALS (PALS) or healthy volunteers have been scheduled for participation before the end of the year. 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.

The Institute is providing all participants with access to the data via a secure online portal, where they can monitor their health status by viewing changes in motion tracker and speech recording data, and track the data generated from the biological samples. 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.

Each of the people enrolled in this program are true trailblazers in my opinion. Their effort through the Precision Medicine Program adds in a huge way to our already hyper-focused and data-driven efforts to develop ALS treatments. The patients and volunteers in the Precision Medicine Program are standing right there on the edge of scientific discovery together with us at the Institute as we share the goal of urgently finding ways to get at this disease in a meaningful way, said Steve Perrin, Ph.D., Chief Executive and Scientific Officer of the ALS Therapy Development Institute.

The Institute began planning its Precision Medicine Program in 2013, and announced a call for volunteers this past summer. The Institutes enrollment was boosted by the social media phenomenon, the ALS Ice Bucket Challenge. Nearly $4 million was donated directly to the Institute, and every dollar was assigned directly to ALS research, including $1 million to the Precision Medicine Program, allowing it to expand enrollment from 25 to 300 people.

The Institute is currently working to expand the program further to include more patients and volunteers, and expects to make additional announcements regarding that in the coming months.

For more information on the 100th patient and others in the Precision Medicine Program, please visit http://www.als.net.

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 Institutes Precision Medicine Program seeks to gain critical new insight into the mechanisms of ALS through integrative analysis of each participating patients 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 (ALS.net) and its scientists actively discover and develop treatments for ALS. The Institute is the worlds 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. The ALS Therapy Development Institute, based in Cambridge, MA, 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.als.net.

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100th Patient Enrolled in Worlds First Precision Medicine Program for ALS