Articles from April 2013



110% Gratitude to All Our Volunteers

April marks National Volunteer Month. This observance holds very special meaning for all of us here at the Robert A. Stehlin Campaign for ALS (R.A.S.C.A.L.S.). That’s because we are a 100% all-volunteer 501(c)(3) non-profit charity. There are no administrative costs.
That means no salaries, payments, or perks for anyone. Out-of-pocket expenses truly means out of our [...]

NEALS SOD1 Research is First Antisense Oligonucleotide Trial for a Central Nervous System Disorder

Antisense Oligonucleotides May be Feasible ALS Therapeutic Strategy

BOSTON, Massachusetts, March 29, 2013 — Published online today in Lancet Neurology, an SOD1-related familial ALS trial under the direction of NEALS researchers Timothy Miller, MD, PhD (Washington University School of Medicine) and Merit Cudkowicz (Massachusetts General Hospital) indicates that antisense oligonucleotide delivery to the central nervous system may [...]

‘RNA Sponge’ Mechanism May Cause ALS/FTD Neurodegeneration

Researchers at Emory University School of Medicine have demonstrated that this ALS/FTD mutation may be harmful because it creates an “RNA sponge,” soaking up an important regulatory protein that binds RNA.

Johns Hopkins Researchers Discover New Clues About How ALS Develops

The researchers also found, to their surprise, that suppressing an ALS-causing gene in oligodendrocytes of mice bred with the disease—while still allowing the gene to remain in the motor neurons—profoundly delayed the onset of ALS. It also prolonged survival of these mice by more than three months, a long time in the life span of a mouse. These observations suggest that oligodendrocytes play a very significant role in the early stage of the disease.

Cytokinetics Inc. Announces Initiation of First-Time-in-Humans, Phase I Clinical Trial of CK-2127107

Cytokinetics, Incorporated announced today the initiation of a first-time-in-humans, Phase I clinical trial of CK-2127107 in healthy male volunteers. Cytokinetics is developing CK-2127107, a novel small molecule activator of the fast skeletal muscle troponin complex, for the potential improvement of skeletal muscle function in diseases and medical conditions associated with neuromuscular dysfunction, muscular weakness, and/or muscle fatigue. Like tirasemtiv, the lead drug candidate from the company’s skeletal muscle activator program, CK-2127107 slows the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers, which sensitizes the sarcomere to calcium.