US firm Isis Pharmaceuticals has initiated a Phase I study of the antisense drug ISIS-SOD1Rx in patients with an inherited and aggressive form of Lou Gehrig's disease, also known as familial amyotrophic lateral sclerosis (ALS).
ISIS-SOD1Rx is designed to selectively inhibit the production of superoxide dismutase (SOD1) gene, a mutant form of which has been found to cause nearly 20% of all familial ALS cases.
The placebo-controlled, dose-escalation study to assess the drug's safety, tolerability and pharmacokinetic profile will be carried out by administering the drug directly into the central nervous system.
According to ALS Association chief scientist Lucie Bruijn, the therapy will be the first treatment for ALS specifically aimed at SOD1.
"The development of new treatments for ALS is an extremely challenging and costly process," Bruijn said.
The drug's development is being funded by the ALS Association and the Muscular Dystrophy Association.
