The ALS ONE Massachusetts ALS Partnership (MAP) Will Accelerate Treatment and Care in ALS

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which leads to total paralysis and death, usually within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA), which modestly extends survival.

"The collaboration among these distinguished centers of ALS research will help accelerate the development of meaningful new approaches to treatment," said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association. "We are gratified to be able to help these partnerships bring their expertise to bear on this important goal, and are excited by the potential they represent." Dr. Bruijn serves on the Board of ALS ONE.

Kevin Gosnell, who passed away in August 2016 due to complications from ALS, founded ALS ONE in January 2016, to expedite progress toward finding a treatment for ALS by 2020 while improving care now. His vision continues with these new funding commitments. The ALS ONE partnership has already placed resources, expertise and dedicated teams into a powerful coordinated team with aligned goals and the ability to move ideas and products quickly from inception to trial and from idea into care practice.

Drs. Nazem Atassi, James Berry and Merit Cudkowicz of MGH will build a regional ALS clinical trials network for rapidly and efficiently bringing the most promising new therapeutic ideas to trials in people with ALS. They will also build a translational pipeline that includes the development of new lab tests and imaging tools for testing treatment efficacy and initiate clinical trials of new potential treatments which target inflammation in ALS.  

Finally, the group will expand their efforts to move clinical care beyond the walls of the clinic to improve patient access to multidisciplinary ALS care at home and access to research studies, in order to provide improved care and resources for all ALS patients. Ron Hoffman, Founder and Executive Director of CCALS says the group will work with ALS ONE partners and other providers to ensure coordinated communication between all ALS care professionals and ALS clinics to ensure that up-to-date technology, timely research information and nursing resources are available to ALS patients. By integrating the CCALS care model with the multidisciplinary clinics at UMass and MGH, and partnering the model with research outreach efforts, the already dramatic impact on the lives of people with ALS can extend even further.

Under the leadership of Steven Perrin, Ph.D., ALS TDI will spearhead integration of preclinical testing of promising therapeutics with the regional clinical trials network. Rigorous development of preclinical data, including mechanistic, pharmacodynamics and pharmacokinetic data, is essential for improving the chances of success once a drug reaches clinical testing. Preclinical pipeline development at ALS TDI has made them a leader in the field.

Robert Brown, M.D., of University of Massachusetts Medical School, will develop two strategies to silence the production of toxic RNA and proteins from the mutant gene C9orf72, the most common cause of inherited ALS. In the first strategy, his group will use a viral vector to deliver an RNA to reduce expression of the gene. In the second strategy, they will employ a new method, called CRISPR/Cas9, to digest the offending gene enzymatically. These studies will be performed initially in mice and will then be moved to trials in larger animals as a prelude to human application. In parallel, they will augment their clinical infrastructure to enhance integration with the regional trials network.

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig's Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org.

About ALS Finding a Cure®
ALS Finding a Cure® is a new research organization dedicated to being a game changer in discovering a cure for this fatal disease. Founded in 2014 by noted philanthropist and Conair Founder and Chairman, Leandro (Lee) Rizzuto, as a tribute to Christie Rizzuto, Lee's daughter-in-law who was diagnosed with ALS in 2009 at the age of 41, and under the joint leadership of Lee Rizzuto and Peter N. Foss, ALS Finding a Cure® is focused on identifying the gaps in the scientific understanding of ALS that are preventing the development of a cure. The organization, a program of The Leandro P. Rizzuto Foundation, collaborates with a wide range of companies, ALS organizations, other disease non-profits, and ALS patients and families to ensure research efforts are non-duplicative, synergistic, and focused on the ultimate goal: getting to a cure. For more information about ALS Finding a Cure®, visit http://www.alsfindingacure.org.

About ALS ONE
ALS ONE is a partnership of the top ALS experts from leading Massachusetts institutions, including ALS Therapy Development Institute, UMass Medical Center, Massachusetts General Hospital and Compassionate Care ALS founded by the late Kevin Gosnell. These venerable institutions are combining forces with the goal of finding a treatment for ALS within the next four years, while simultaneously working to improve the care model for persons living with ALS and their families. ALS ONE is a d/b/a of the non-profit 501c3 ALS Knights. For more information, visit www.ALSONE.org.

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SOURCE The ALS Association

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http://www.alsa.org