Posts belonging to Category Clinical Trials
“That our cells and method of delivery are safe in ALS patients bodes very well for expanding to other indications. We expect to commence our FDA-approved Phase I trial in chronic spinal cord injury later this year using the same methodology. We want to thank the surgeons at Emory, who developed these techniques, as well as the patients and their families who have taken part in the trial.”
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 [...]
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.
Data Indicate Initial Clinical Benefit in Overall Clinical and Respiratory Function
NEW YORK, NY and PETACH TIKVAH, ISRAEL, March 21, 2013 — BrainStorm Cell Therapeutics, a leading developer of adult stem cell technologies for neurodegenerative diseases, today reported some of the final results from a clinical study evaluating the company’s NurOwn™ technology in 12 ALS patients. NurOwn [...]
Lithium in patients with amyotrophic lateral sclerosis (LiCALS): a phase 3 multicentre, randomised, double-blind, placebo-controlled trial
From the UKMND-LiCALS Study Group
Summary article posted online in The Lancet Neurology, February 27, 2013 •
Lithium has neuroprotective effects in cell and animal models of amyotrophic lateral sclerosis (ALS), and a small pilot study in patients with ALS showed a [...]
Because of the relentless effects of ALS, a diverse group of ALS voices — patients, families, organizations and the community — came together to speak about their experiences with ALS and to urge the FDA to work with them to overcome some of the obstacles faced in ALS research, clinical trials and standards of care.
“I believe happiness is a choice. Some days it is a very difficult choice,” he says. “This is true for everyone. I look at the human life like an experiment. Every new moment, every new experience, tragic or otherwise, is an opportunity to gain a more accurate perspective and helps lead me to clarity.”
“Some may look at recent failures and be discouraged,” wrote Richard Bedlack of the Duke ALS Clinic in Durham, North Carolina, in an e-mail to Alzforum. Thanks to advances in genetics and biomarkers, and a list of potential target pathways, “I think this is a time of unprecedented hope and excitement in ALS research”
Today Biogen Idec (BIIB) reported top-line results of EMPOWER, a Phase 3 trial investigating dexpramipexole in people with amyotrophic lateral sclerosis (ALS). The trial did not meet its primary endpoint, a joint rank analysis of function and survival, and no efficacy was seen in the individual components of function or survival. The trial also failed to show efficacy in its key secondary endpoints. Additional analyses of multiple subpopulations failed to demonstrate any efficacy among these groups. Based on these results, Biogen Idec will discontinue development of dexpramipexole in ALS.
THIS IS OUR YEAR.
THIS IS OUR MOMENT.
THIS IS THE TIME TO STEP UP AGAINST ALS.
Researchers continue to make breakthroughs in understanding how ALS functions.
There’s also been some promising news about potential treatments.
That’s why now is the time to swing for the fences.
So please dig in, dig deep, and make a donation to the RASCALS Foundation.