FUS — a protein mutated in some amyotrophic lateral sclerosis patients — plays a key role in the mechanism that repairs oxidative damage in DNA molecules in motor neurons, a study found. The findings suggest a mechanism through which FUS defects cause amyotrophic lateral sclerosis (ALS), and add to the…
Medicines used for ovarian and breast cancer, called PARP inhibitors, might be repurposed to treat people with amyotrophic lateral sclerosis (ALS), a study in cells suggests. This suggestion came from researchers’ work with veliparib (ABT-888), an investigational PARP inhibitor, that found it effectively reduced TDP-43 aggregates — a hallmark of…
The U.S. Food and Drug Administration has given positive feedback to MediciNova’s Phase 3 developmental plan for its investigational therapy ibudilast (MN-166) for amyotrophic lateral sclerosis (ALS). Following the FDA’s guidance and suggestions, the company will design a trial that determines the maximum benefit of the treatment,…
In the Ken Burns documentary, “Baseball,” during the episode titled “Eighth Inning: A Whole New Ballgame,” comedian Billy Crystal mentions in passing the (I assume fictional) name Al Smenglevitz. Years later, I co-opted the character as a coping mechanism — an alter ego. Only, I simply refer to…
Treatment with EHP-102, an investigational cannabinoid-derived medicine, reduced weight loss, preserved motor neurons of the spinal cord, and lowered the abnormal reactivity of astroglial cells in a mouse model of amyotrophic lateral sclerosis (ALS), new research shows. Findings were recently published in the study, “Neuroprotective effects of…
The pharmaceutical development company Noxopharm and its majority-owned subsidiary, U.S. biotechnology firm Nyrada, have discovered a potent inhibitor of the protein IRAK4, a “master switch” critical to the development of inflammatory autoimmune diseases, such as amytrophic lateral sclerosis (ALS). IRAK4 has emerged as a key switch in the…
My ALS diagnosis was in 2010. In the months following, I tried to learn as much as possible about the condition and how it would affect my life going forward. I discovered that medical experts knew almost everything about the physical progression of ALS and what to expect…
Specific patterns of a misfolded protein — superoxide dismutase 1 (SOD1) — are found in the brain and spinal cord of patients with sporadic amyotrophic lateral sclerosis (ALS), according to a new study, supporting research suggesting that misfolded SOD1 plays a role in this ALS subtype. The research, “…
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
Adding Azilect (rasagiline) — a medication already approved to treat Parkinson’s disease — to Rilutek (riluzole) may be able to slow the advancement of fast-progressing amyotrophic lateral sclerosis (ALS), a Phase 2 trial suggests. However, its positive safety results are being challenged by other physicians, who raise concerns that…
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