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. The portal allows the patients to monitor their health status as reflected by the 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 Institute’s
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,
Greta Mae Hart (see photo) and others in the Precision Medicine Program, please
visit www.alstdi.org.
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 (ALS.net) 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. 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 www.alstdi.org.
Media Contact: Mari Cody, ALS Therapy
Development Institute, 617-441-7220, mcody@als.net