“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”
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).
Results of the first stage showed that receiving dexpramipexole appeared to slow the progression of symptoms measured both by the ALS Functional Rating Scale and by pulmonary capacity. The second stage had similar results, with slower disease progression and a reduced risk of death in participants receiving the higher dosage.