The ALS Association

ALS Ice Bucket Challenge Progress
Brian Frederick
The ALS Association
(202) 255-9443



Grand Challenge Winners Poised to Develop Crucial Imaging Marker for ALS Therapy Development

Washington, D.C. (March 13, 2017)— The ALS Association and ALS Finding A Cure® (ALSFAC) are pleased to announce the recipient of their Grand Challenge award for development of an imaging marker for TDP-43, a protein found in almost all cases of ALS. The $1 million Grand Challenge was awarded to a team of researchers led by Timothy Miller, M.D., Ph.D., Paul Kotzbauer, M.D., Ph.D., Vijay Sharma, M.D., Ph.D., and Nigel Cairns, M.D., Ph.D. of Washington University in Saint Louis, Missouri and Yuna Ayala, M.D., Ph.D. of Saint Louis University.

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.

Almost all cases of ALS are characterized by formation of clumps, or aggregates, of the protein TDP-43 within motor neurons, the cells that die in the disease. These protein aggregates form during the disease and may offer clues to its causes and progression, but there is currently no way to detect them in people living with ALS. The ability to track TDP-43 aggregation over the course of disease and in response to therapy should accelerate the development of new treatments, just as imaging amyloid plaques has accelerated therapy development in Alzheimer's disease.

Recognizing this unmet need in the ALS field, The ALS Association and ALSFAC through the ALS Accelerated Therapeutics (ALS ACT) partnered to offer the Grand Challenge award. ALS ACT is a novel academic-foundation-industry collaboration with ALSFAC and The Association, initiated with researchers from General Electric (GE) Healthcare and four academic Northeast ALS Consortium (NEALS) sites, which is designed to accelerate treatments for people living with ALS.

The winning team will develop a so-called PET tracer, a molecule that is safe, crosses the blood brain barrier, binds to TDP-43 aggregates, and can be detected by positron emission tomography (PET) imaging, a type of medical neuroimaging widely used for studying the brain. The winning team has recently developed methods to generate large quantities of aggregated TDP-43 and techniques to assess whether a potential tracer compound will bind to these aggregates. They also identified an initial compound that does bind to aggregated TDP-43, setting the stage for identification of further compounds and refinement of the best compounds for use in people.

“We have seen in other fields that development of neuroimaging markers has greatly accelerated the development of new treatments,” commented ALS Association Chief Scientist Lucie Bruijn, Ph.D., MBA. “Being able to detect TDP-43 in the nervous system will allow us to quickly and accurately gauge the effects of potential therapies on this important protein. Thus, we are excited to support the development of this new PET marker by this expert team of scientists.”

“We are very excited to support this great team to develop an important imaging marker for ALS. Development of this marker could help with diagnosis and as a marker of treatment effect for new therapies. It is also likely that this imaging tool would be helpful to people with other TPD-43 related neurodegenerative disorders,” commented ALSFAC Chief Medical Officer, Merit Cudkowicz, M.D., Chief of Neurology at Massachusetts General Hospital.

For more information about the Grand Challenge click here.

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

About ALS Finding a Cure®
ALS Finding A Cure® is 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 leadership of Peter N. Foss and a team of renowned ALS researchers, 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 research organizations, and ALS patients and families to ensure research efforts are non-duplicative, synergistic, and focused on the ultimate goal: finding a cure. For more information about ALS Finding a Cure®, visit

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