Researchers Identify a New Therapeutic Target for C9orf72-Associated ALS

Researchers supported by The ALS Association announced that they had identified a new protein called SUPT4H1 that has potential to be used in therapy development for people whose ALS is caused by the C9orf72 gene. This research was published in the journal Science by Drs. Aaron Gitler at Stanford University in Stanford, California and Leonard Petrucelli at the Mayo Clinic in Jacksonville, Florida. Read on to learn more about this latest discovery.

Why SUPT4H1?

Drs. Gitler and Petrucelli targeted SUPT4H1 since it was shown that reducing its presence decreases the production of proteins containing long repeat expansions associated with another neurodegenerative disease, Huntington’s disease. However, reduction of SUPT4H1 did not affect expression of normal, non-expanded proteins. Continue reading Researchers Identify a New Therapeutic Target for C9orf72-Associated ALS

Essey Award Winner Discusses Progress in ALS Genetics

Since 1996, the Sheila Essey Award for ALS Research has been presented by Dick Essey at the American Academy of Neurology Annual Meeting, in memory of his wife Sheila, who battled ALS for 10 years and passed in 2004. This year, the award was presented to Ammar Al-Chalabi, Ph.D., FRCP, DipStat from King’s College London. Find out how Dr. Al-Chalabi’s work is driving progress in ALS research forward.

This year, The ALS Association and the American Academy of Neurology (AAN) were very pleased to honor Dr. Ammar Al-Chalabi with the Sheila Essey Award for ALS Research for his major contributions to the understanding of ALS and the search for new therapies to treat the disease. Dr. Al-Chalabi was presented the 2016 Sheila Essey Award by Dick Essey at the 68th AAN Annual Meeting in Vancouver, B.C. on April 20, 2016. The $50,000 prize was given to fuel his promising ALS research projects and to fund talented young scientists on his research team.

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Dr. Ammar Al-Chalabi

Dr. Al-Chalabi is currently Professor of Neurology and Complex Disease Genetics at King’s College London and Director of King’s MND Care and Research Center. He has made significant contributions to the ALS field. Dr. Al-Chalabi helped identify many known genetic causes of ALS, including C9orf72; developed the ALSoD database, an important tool for researchers funded by The ALS Association, MNDA UK and Therapy Alliance; and has made significant contributions to understanding disease staging, which impacts ALS care and clinical trial design. For more information regarding his illustrious research career click here.

Continue reading Essey Award Winner Discusses Progress in ALS Genetics

The Neuro Collaborative: An Engine for ALS Therapeutics

In 2014, The ALS Association Golden West Chapter and patient advocate Jim Barber partnered to build the Neuro Collaborative concept. That year, following the amazing outpouring of support from the ALS Ice Bucket Challenge, The ALS Association committed $5 million dollars to the project. With additional help from The ALS Association Orange County and Wisconsin Chapters, the Neuro Collaborative has become an engine for ALS therapeutics. Learn more about the progress of each partner in the Neuro Collaborative below.

Early development of potential therapeutics is a major bottleneck in ALS therapy development. Eliminating this bottleneck is a significant opportunity for accelerating new treatments. That’s where the Neuro Collaborative comes in.

The Neuro Collaborative is a partnership between three leading laboratories in California: Clive Svendsen, Ph.D. at Cedars Sinai in Los Angeles; Steven Finkbeiner, M.D., Ph.D. at Gladstone Institutes, affiliated with University of California San Francisco; and Don Cleveland, Ph.D. at University of California San Diego.

The goal of the Collaborative is to establish and invest in a leading team of experts to efficiently advance ALS drug development together with industry partners. This complements other programs including the Drug Development Contract Program in the TREAT™ ALS portfolio investing in academic and industry partnerships. This synergetic model leverages open dialogue and the scientific expertise of leading researchers to achieve therapeutic milestones as quickly as possible. It also reduces risk in the drug discovery process and attracts pharmaceutical companies to invest in drug development and clinical trials for ALS. With success, potential therapies for ALS will move more quickly than ever toward FDA approval and the open market. Continue reading The Neuro Collaborative: An Engine for ALS Therapeutics