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.

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).

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.

The exciting finding that reduction of a single protein, SUPT4H1, specifically reduces toxic gene products associated with C9orf72 long repeat expansions, gives researchers a new potential therapeutic target.

Kevin Gosnell, founder of ALS ONE, passed away on Monday because of complications from the disease. Gosnell was diagnosed with ALS in spring 2015 and immediately put his business acumen and CEO leadership skills to work, convening the best minds in the ALS community. He founded ALS ONE in January 2016 and brought together leading neurology experts and care specialists from Massachusetts in partnership to leverage their institutions’ strengths to expedite progress toward finding a treatment for ALS within the next four years.

Over the last day, The ALS Association has received multiple questions surrounding the NEK1 gene discovery and how it affects people living with ALS. Below are some common questions and our answers, along with places to read more information.

The main goal is to evaluate safety and tolerability. Despite the small size of the study, researchers conducted pre-efficacy measurements to preliminarily understand whether NurOwn has a beneficial effect in people living with ALS or not.

I encountered my first ALS reversal in July 2011, more than a decade into my career in ALS, while leading an ALSUntangled review of Dean Kraft, an energy healer. On Kraft’s website was a video about a person named Nelda Buss, who was reported to have been diagnosed with ALS at a top hospital and progressed to where she had lost nearly all movement and was near death. After two years of Kraft’s treatments, she was said to have recovered most or all of her motor function. I was skeptical.

“The results in the study are encouraging providing important data on safety and dosing of the stem cells in study participants,” stated Lucie Bruijn, Ph.D., M.B.A, Chief Scientist of The ALS Association.

Stem cells are cells that are capable of developing into different cell types, including neurons (brain cells) and glia (brain support cells). Not all stem cells are the same and choosing stem cell populations with specific desirable properties could in fact improve the therapeutic potential of stem cell therapy.

On June 10, 2016, The ALS Association brought together the current and past awardees of its Milton Safenowitz Postdoctoral Fellowship to participate in a day-long research symposium and awards ceremony. The workshop was funded by The Greater New York Chapter of The ALS Association.

Edaravone is believed to act as a free radical scavenger, a compound that works by getting rid of toxic waste generated as a normal by-product the cells function. In ALS it is thought that these by-products are not as effectively removed and the compound may be neuroprotective by relieving the effects of this oxidative stress. The increase in oxidative stress is thought to damage motor neurons (cells that die in ALS).