ALS ONE – Massachusetts ALS Partnership (MAP)

The ALS Association Commitment

The ALS Association and the ALS Finding a Cure® are working together with ALS ONE to develop and fund appropriate clinical and research initiatives to maximize synergies that will make a significant impact on the disease. This collaboration was founded in January 2016 and solidified in November 2016. Each partner is contributing $2 million to this strategic initiative, for a total commitment of $6 million this year.

Goals

Kevin Gosnell brought together the top leaders, researchers, doctors and caretakers in ALS within Massachusetts, to join forces to bring the strengths of their prestigious institutions to an ambitious plan: to bring a treatment to persons with ALS by 2020.

By working together to fund and develop a treatment, they will demonstrate their shared commitment to fighting ALS while also continuing to provide high-level care for those who have already been diagnosed. By unifying resources, expertise and dedicated teams into a large coordinated team, individual goals will be aligned to move ideas and products quickly from inception to trial and from idea into care practice.

How ALS ONE advances the search for a treatment for ALS

• Leverages the expertise from each research institution though sharing ideas and responsibilities to advance each of the strongest therapeutic opportunities identified forward.
• Eliminates unnecessary duplication and expedites the path to find an ALS treatment.
• Improves care for people living with ALS by creating more access to clinical trials through education and transportation, as well as providing technology, equipment and support services.

Key Players

Kevin Gosnell late founder and CEO of ALS ONE was diagnosed with ALS in spring 2015 and passed away August 8, 2016 from complications of the disease. He founded ALS ONE in January 2016 with a vision to bring the best minds in the ALS community to work together.

The ALS ONE leadership team is comprised of many of the top ALS experts in the world, including:

• Nazem Atassi, M.D., James Berry, M.D. and Merit Cudkowicz, M.D., MSc. of Massachusetts General Hospital in Boston
• Building a regional ALS clinical trials network to rapidly and efficiently bring the most promising new therapeutic ideas to trials in people living with ALS
• Initiating a new clinical trials to test promising treatments which targets inflammation
• Building a translational pipeline that includes developing new lab tests and imaging tools for testing treatment efficiency
• Ron Hoffman, Founder and Director CCALS
• In partnership with Drs. Atassi, Berry and Cudkowicz, CCALS is improving patient access to multidisciplinary ALS care and research studies using novel technology and nursing resources.
• Steven Perrin, Ph.D., ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass.
• Developing a CD40 ligand as a therapeutic approach for ALS based on preliminary data generated at ALS TDI in collaboration with academic and industry partners
• Integrating preclinical testing (mechanistic, pharmacodynamics and pharmacokinetic data) of promising therapeutics with the regional clinical trials network
• Robert Brown Jr., M.D., Ph.D., University of Massachusetts Medical School in Worcester, Mass.
• Developing strategies to silence the production of toxic RNA and proteins produced from the expanded C9orf72 gene in mouse models, with the intent to move studies into clinical trials
• Augmenting the clinical infrastructure to enhance integration with the regional trials network

Lucie Bruin, Ph.D., M.B.A., Chief Scientist of The ALS Association and Stephen Winthrop, a member of The Association’s National Board of Trustees, both serve on ALS ONE’s Board of Directors.

Partners in the initiative include The ALS Association, ALS Finding a Cure®, ALS Therapy Development Institute, Massachusetts General Hospital, University of Massachusetts Medical School and Compassionate Care ALS (CCALS) and ALS ONE.

Website:

www.alsone.org