John Kissel at Ohio State University in Columbus has MDA funding to start a full-scale trial of albuterol, a beta receptor agonist, in facioscapulohumeral dystrophy . A recent small pilot study of albuterol in FSHD showed promise. These drugs seem to increase the amount of muscle by changing muscle cell biochemistry. The exact mechanisms aren't understood. The companies also plan to submit an application for full approval of the drug this summer.
Neurotrophic factors seem to protect nerve cells from damage under a number of conditions.
MDA has long supported research in these substances, and clinical trials were conducted at MDA-supported clinics. The cell-containing capsule is inserted at the base of the spinal cord, where it leaks CNTF into the spinal fluid. The drug will be given orally for about six months. The Massachusetts General researchers say a previously planned trial of procysteine, an antioxidant, has been canceled. All patients tolerated the procedure well.