By Mel Reichman, Ph.D.
Certain genetic changes in super oxide dismutase-1 (SOD1), the second most common form of inherited ALS, cause this protein composed of two parts (a dimer) to fall apart into individual pieces (monomers). These SOD1 monomers can abnormally clump together in motor neurons during ALS disease. Join me at The ALS Association December webinar to learn about my research to develop new ways to discover novel ALS drugs that stabilize SOD1 dimers. This includes a high-throughput screening technique, which could reveal unexpected, safe combinations of FDA-approved drugs to treat ALS.
Continue reading A Two-Pronged Approach for SOD1-linked ALS Drug Discovery
Over 30,300 neuroscientists from around the world gathered in San Diego in November for the Annual Meeting of the Society for Neuroscience (SfN), the largest annual meeting of scientists in the world. ALS was strongly represented at the meeting, with the presentation of approximately two hundred new research studies on genes, models, disease mechanisms and therapy development. These included many supported by The ALS Association under our TREAT ALSTM global research program that I highlight below.
Continue reading The ALS Association Joined Thousands of Neuroscientists at the Annual Society for Neuroscience Meeting in San Diego
The ALS Association, in partnership with ALS Finding a Cure and the Northeast ALS Consortium (NEALS) is giving $1 million in total funding to support a new ALS clinical trial to test the efficacy of the experimental drug RNS60 in reducing inflammation that may slow the disease process. The Association is contributing $500,000 out of the total award. The trial is currently being setup and is slated to begin in the New Year. The study will be led by Drs. Ettore Beghi, at the IRCCS Mario Negri Institute for Pharmacological Research in Milan, Letizia Mazzini, at the University Hospital of Novara in Novar, Italy and Sabrina Paganoni, at Massachusetts General Hospital in Boston.
Continue reading The ALS Association Partners with ALS Finding a Cure and NEALS to Support New Phase II RNS60 Trial Targeting Neuroinflammation
Your research donations make a difference! ALS Association funded Dr. Mervyn Monteiro, Professor at the University of Maryland School of Medicine, developed novel ALS mouse models expressing mutant ubiquilin 2 (UBQLN2), a cause of inherited ALS-FTD that was published this week in the journal Proceedings of the National Academy of Sciences (PNAS). Importantly, these mouse models demonstrate many characteristics of ALS observed in humans, thereby providing valuable tools for identifying disease pathways in ALS and for investigating therapeutic strategies to treat ALS.
Continue reading UBQLN2 Mouse Models Replicate Key Human ALS Disease Features
Dr. Aaron Gitler and his colleagues recently published a paper in the August 12th issue of Science uncovering a potential new therapeutic target aimed at C9orf72 ALS, supported by The ALS Association. He found that inhibiting just one protein, called Spt4, significantly reduced toxicity caused by the C9orf72 repeat expansion. Learn more about how he used a simple model, yeast, to make this discovery and its therapeutic potential to treat ALS. And join us on November 7th to hear from Dr. Gitler during The ALS Association research webinar series (see details below).
Continue reading Novel Potential Therapeutic Targeting C9orf72 ALS Discovered – Interview with Dr. Gitler
The ALS Association Research Department is pleased to show their research dollars in action. Two new papers that were recently published in high impact scientific journals Proceedings of the National Academy of Sciences (PNAS) and Cell were supported by The ALS Association. The first paper by Drs. Zuoshang Xu and John Landers from University of Massachusetts Medical School in Worcester, Mass., demonstrates a novel profilin 1 (PFN1) mouse model that displays disease characteristics similar to human disease. This paper underscores the importance of developing novel animal models to understand ALS disease processes and to discover new therapeutic targets. The second paper by Dr. J. Paul Taylor and colleagues from St. Jude Children’s Hospital in Memphis, Tenn. reveals a new disease mechanism involving the C9ORF72 expansion mutation that will be targeted for ALS therapy.
Continue reading Novel ALS PFN1 mouse model developed and new C9ORF72 disease mechanism uncovered
Center for Genomics of Neurodegenerative Disease, New York Genome Center – By Dr. Hemali Phatnani
Through donations from the ALS Ice Bucket Challenge, The ALS Association is supporting strategic initiatives that emphasize collaboration, data sharing and state-of-the-art scientific methods. Here we feature the Center for Genomics of Neurodegenerative Disease (CGND) at the New York Genome Center (NYGC). Meet Dr. Hemali Phatnani and learn about how the CGND at the NYGC is making a huge impact on ALS research.
Continue reading NYGC’s Research Program Probes the Genetics and Genomics of ALS