Research into a therapy for amyotrophic lateral sclerosis (ALS), which is funded by The ALS Association, is producing what scientists hope will be groundbreaking results.
ALS is a rare, fatal disease marked by the degeneration of the body's motor neurons. Victims of the disease lose muscle function and become completely paralyzed. Most die within two to five years of diagnosis.
Biogen, a partner company of Isis Pharmaceuticals, has commenced a Phase 1/2 clinical study of ISIS-SOD1Rx, which could be used to treat patients with ALS. Funding for research into the drug began in 2003.
ISIS-SOD1Rx is what scientists believe to be the genetic cause of familial ALS, a gene mutation. Presently, Biogen researchers are conducting a randomized, placebo-controlled, dose escalation Phase 1/2 clinical study to gauge the safety and effectiveness of the drug, which is administered via injection into the cerebral spinal fluid.
“It is extremely exciting to see The ALS Association’s early investment in the development of antisense technology to treat ALS moving forward into the clinic,” Dr. Lucie Bruijn, chief scientist for The ALS Association, said. “One of the core strategies of our research program is to link academic science closer with industry to promote and de-risk these therapeutic approaches to help facilitate the development of potential treatments for people living with this disease. We are also enthusiastic about the opportunity this technology has brought for not only ALS but other neurodegenerative diseases."
Dr. C. Frank Bennett, senior vice president of research at Isis Pharmaceuticals, agreed.
“We are grateful to The ALS Association for providing some of the initial funding which supported the initial concept of targeting ALS with antisense technology that lead us to this moment,” he said.
