In 2016, a significant number of ALS research discoveries, advances in clinical trials, collaborations and strategic initiatives all accelerated the pace of discovery in finding treatments and a cure for ALS.
Earlier this month, The ALS Association was happy to travel to Dublin to participate in the 27th International Symposium on ALS/MND. The Symposium, the largest ever, brought together top ALS researchers and clinicians from all over the world. People in attendance, including those from The ALS Association, used the hashtag #alssymp during the event. Here are some of our favorites!
After over a year in the making, The ALS Association in partnership with Prize4Life awarded the ALS Assistive Technology prize in Dublin during the ALS/MND International Alliance Meeting. We are thrilled to award one of the top prizes to Dexter Ang and David Cipoletta, two young entrepreneurs that founded Pison Technology based out of Massachusetts. They blew the judges away with their easy-to-use, self-contained communication system based on muscle EMG signals.
You may have heard that IBM's supercomputer, Watson, competed on Jeopardy! and is now being used to solve everything from business problems to diagnosing cancer. Today, we were thrilled to hear the latest Watson news: Working with a team at the Barrow Neurological Institute in Phoenix, headed by Dr. Robert Bowser, Watson helped researchers discover 5 new ALS genes.
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.
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.
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.
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.
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.
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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.
Our friend Ted Harada passed away this week. Many of you may have known him. Ted was first diagnosed with ALS in August 2010 and immediately became a tireless advocate, volunteer, and voice of the ALS community.
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.
In August 2015, The Association committed $3.5 million from money raised through the ALS Ice Bucket Challenge to the Genomic Translation for ALS Care (GTAC), a collaborative nationwide precision medicine initiative through Columbia University Medical Center (CUMC) and Biogen, which is being led by Dr. Matthew Harms. GTAC is actively enrolling now (scroll to the bottom for more details) with the ultimate goal of ensuring the genetic characterization of all people living with ALS.
Today, a new disease pathway for C9orf72-related ALS was revealed in journal Neuron in a study led by principal investigator Dr. Fen-Biao Gao from the University of Massachusetts Medical School in Worcester, Mass. The research team found that the C9orf72 expansion leads to DNA damage, signifying another potential ALS therapeutic target. The ALS Association supported this encouraging study that also included current and past Milton Safenowitz Postdoctoral Fellows Dr. Dejun Yang (2015 recipient) and Dr. Helene Tran (2012 recipient).
Deborah Silver, an accomplished vocalist and performer, has been active in the fight against ALS since 2009 when her sister was diagnosed with the disease. My sister, Marjie Block, was diagnosed with ALS in 2009. I was by her side visiting doctor after doctor hoping to get any diagnosis other than ALS. We even prayed for Lyme Disease….anything but ALS! Ultimately, this unfortunate diagnosis stayed the same and Marjie’s courageous battle began.
Following up on our June 2016 blog post about edaravone, an intravenous drug therapy produced by Mitsubishi Tanabe Pharma Corporation, locally based in Jersey City, N.J., with a head office in Osaka, Japan, there has been recent news as to the status of the drug in the U.S.
In July 2016, The ALS Association invited members of the ALS community (defined as people living with ALS, family members, and caregivers) to participate in a community care services survey. This survey included 15 questions and was shared via several of The Association’s communication channels. Nearly 800 members of the community responded.
My mom was diagnosed with ALS in May 2011. She literally lost her speech within 6 weeks of slurring her words. She was semi-retired but enjoyed her job and co-workers, so she still worked part time at a hospital as an insurance biller. Bravery!