The ALS Association is proud to be the first investor in antisense technology, dating back to 2004 when antisense was just an idea in Dr. Don Cleveland’s lab at University of California San Diego (UCSD). Fast forward to 2018 and we are seeing promising results in antisense drugs targeting the two most common causes of inherited ALS, mutations in the SOD1 and C9orf72 genes.

Dr. Brian Wainger from the Massachusetts General Hospital (MGH) presented initial top-level results from a recently completed phase II clinical trial of ezogabine (retigabine) on motor neuron excitability (NCT02450552). The study, supported by The ALS Association, met its main goal of quantifying a reduction in motor neuron excitability in people with ALS following treatment. Results were presented during the 29th International Symposium on ALS/MND in Glasgow, Scotland, last week.

In a promising new study by Drs. Robert Brown and Christian Mueller at the University of Massachusetts Medical School report that a type of viral gene therapy using synthetic microRNAs (miRNAs) targeting the ALS SOD1 gene is safe and effective in nonhuman primate macaques (monkeys). The ALS Association provided $1.7 million in funding for this study, which demonstrated an efficient reduction of the SOD1 protein without side effects. This paves the way forward for further development of this potential therapy.

Mitsubishi Tanabe Pharma America (MTPA) will present initial data on efforts to create an oral version of edaravone, a key drug in the treatment of ALS that is currently only available intravenously, during the International Symposium on ALS/MND in Glasgow, Scotland. The symposium will be held Dec. 7-9. MTPA is expected to present results that demonstrate that oral edaravone is processed in the body in the same manner as the infused formulation.

Why motor neurons die in ALS largely remains a mystery. In a study funded by The ALS Association, Dr. Anne Hart, professor of neuroscience at the Carney Institute for Brain Science at Brown University, and her colleagues gained insight into why some types of motor neurons die, while other don’t. A paper about the study was recently published in PLOS Genetics.

Meet Dr. James Shorter, professor of Biochemistry and Biophysics at the Perelman School of Medicine at the University of Pennsylvania in Philadelphia. He and his team work tirelessly in the lab to better understand the causes of ALS, so those causes can be translated into potential therapeutic targets. The ALS Association has proudly funded him since 2014.

Dr. Emily Plowman, one of our Clinical Management Grant awardees, recently published a promising case report on a person with early-stage ALS who has been participating in a mild-intensity respiratory strength training program. The individual has been doing the strength training program with Dr. Plowman and her team at the University of Florida in Gainesville, Florida, over the past 24 months.

Yesterday, MediciNova, Inc. announced that the U.S. Food and Drug Administration (FDA) relayed positive feedback regarding its phase III clinical trial plan to test MN-166 (ibudilast) in a broad population of people with ALS. No safety issues were raised by the FDA and safety will be revisited when results are available in the phase III trial.

Yesterday, ITF Pharma, Inc. announced that Tiglutik™, the first and only thickened liquid form of riluzole, was approved by the FDA for the treatment of ALS. This formulation contrasts with the oral pill form of riluzole that has been on the market for ALS for more than 20 years.

By the beginning of 2019, three trials are slated to be underway to help develop antisense therapy for people with ALS, dividends on a bold investment The ALS Association made in 2004, when the technology was new. We sat down with Dr. Don Cleveland, a pioneer in the field, for the second part of our series highlighting antisense technology.

When Dr. Timothy Miller and his colleagues from Washington University in St. Louis published preclinical data in The Journal of Clinical Investigation last month, showing how second-generation antisense drugs were effective in ALS mouse and rat models, it served as a vivid reminder that every research investment and discovery adds up.

The ALS Association and Teva Pharmaceutical Industries Ltd. (“Teva”) have announced the recipients of the TEVA CNS Target Identification Crowdsourcing Challenge awards for their outstanding proposals to identify novel ALS targets. The two awards will be granted to Dr. Philip Wong, along with Dr. Jonathan C. Grima and Dr. Jeffrey D. Rothstein, all from Johns Hopkins University School of Medicine in Baltimore, Maryland.

Biopharmaceutical company Orphazyme A/S launched a phase III clinical trial of arimoclomol, giving a dose to a person with ALS for the first time, the company announced recently. The randomized and placebo-controlled trial will enroll 231 people with ALS in North America and Europe to determine efficacy and safety of the oral drug.

Dr. Rahul Desikan is incredible. He’s a prominent researcher of neurodegenerative diseases, including ALS, as well as a loyal husband, father, son, and friend. And on February 17, 2017, in a cruel twist of fate, he became a person with ALS.

We recently announced that we’re providing new funding to allow GNS Healthcare to use artificial intelligence (AI) to create a comprehensive disease model to advance research into ALS. GNS Healthcare will use its powerful machine learning platform, called REFS, in conjunction with the rich Answer ALS patient datasets, which are accessible to clinicians and scientists throughout the ALS research community. The project will be led by Dr. Iya Khalil, chief commercial officer and co-founder of GNS Healthcare.

Research funded by The ALS Association helped develop the first mouse model that specifically expresses poly(GR), a type of dipeptide repeat protein associated with C9orf72, which uncovered a new ALS disease pathway. Dr. Leonard Petrucelli’s group at the Mayo Clinic in Jacksonville, Florida, recently published the research in Nature Medicine.

Dr. Marka Van Blitterswijk from the Mayo Clinic Jacksonville is a scientist and one of our former Milton Safenowitz postdoctoral fellows. Since moving on from the program, she has established her own ALS lab as an assistant professor. We recently awarded her a prestigious multi-year grant surrounding her biomarker work.

The ALS Association is proud to have supported the development of bright, young scientists through the Milton Safenowitz Postdoctoral Fellowship program since 2004. The Safenowitz family, through our Greater New York Chapter, founded the program in memory of Milton Safenowitz, who died of ALS in 1998.

Since our founding in 1985, we have put collaboration at the forefront of everything we do. The gift of the ALS Ice Bucket Challenge in August 2014 gave us the unique opportunity to up our game in establishing innovative research partnerships around the world.

In the May 2018 issue of Frontiers in Neuroscience, current The ALS Association-funded researchers, Drs. J. Paul Taylor and Maria Purice of St. Jude Children’s Research Hospital in Memphis, published a comprehensive mini-review describing disease pathways that cause ALS, with a focus on mutations in RNA-binding proteins.