In 11 independent studies, a consortium of ALS researchers shows that transplanting neural stem cells into the spinal cord of an ALS mouse model slows disease onset and progression, improves motor function, and significantly prolongs survival.
While some progress has been made in the treatment of ALS, combination therapies with drugs that target other pathways, alongside stem cell technology, are sorely needed
Cytokinetics Announces Opening of BENEFIT-ALS, a Phase IIb Clinical Trial of Tirasemtiv (CK-2017357)
“Patients who suffer from this devastating disease are in critical need of a novel therapy that addresses the functional deficits that limit their activities of daily living,” stated Jeremy M. Shefner, MD, PhD, Professor and Chair of the Department of Neurology at the Upstate Medical University at the State University of New York and Principal Investigator of BENEFIT-ALS. “If successful, this novel mechanism therapy could improve the lives of many patients living with ALS.”
“That is a type of therapy that will apply broadly to all ALS. All of the data would suggest that if ALS is caused by a particular gene, and you shut down that gene, that you should stop the progression of ALS,” Dr. Miller says.
With regard to the development of an efficacious treatment for ALS, we are still in its infancy. There is much more to be done—and much more quickly than we’ve seen.
Many patients with Lou Gehrig’s disease, a fatal disorder formally known as amyotrophic lateral sclerosis, may want to try the drug on their own—even though there’s no evidence that it is safe or effective in patients with the disease.
The ALS Therapy Development Institute (ALS TDI) will launch a Phase II clinical trial on TDI 132 (aka:fingolimod/Gilenya®) as a potential treatment for ALS (Lou Gehrig’s disease).