Veteran Affairs Locations Looking for A Few Good Men and Women with ALS to Enlist in Clinical Trial
The Department of Veterans Affairs (VA) has an immediate need for military veterans with ALS who are interested in participating in the National Veterans Affairs Collaborative Amyotrophic Lateral Sclerosis (ALS) Clinical Safety and Tolerability Trial of Sodium Phenylbutyrate at six nationwide VA sites.
During the 20-week clinical trial, which began in April, all study participants will be given increasing doses of the oral medication sodium phenylbutyrate (NaPB). Participants in the clinical trial, jointly coordinated by the Massachusetts General Hospital Neurology Clinical Trials Unit and the Bedford VA, also are required to be at least 18 years of age and have no other neurological condition, and women who are able to have children must utilize birth control.
Although it is known that nerve cells die in the brains and spinal cords of patients who have ALS, the cause of the cell death is not well established. There is evidence that this cell death may be caused by altered changes in messenger RNA, made from DNA, the body’s genetic material. Drugs such as NaPB can improve the expression of genes, block how the motor nerve cells in ALS die, and may prove to be an effective therapy for ALS.
A study performed at the Edith Nourse Rogers Veterans Memorial Hospital in Bedford, Mass., demonstrated that NaPB improved the survival in mouse models that have symptoms similar to patients with ALS. The study was directed by doctors Robert J. Ferrante of the VA, along with Merit E. Cudkowicz of the Massachusetts General Hospital and the VA and Robert H. Brown Jr. from the Massachusetts General Hospital. The findings were reported in the Journal of Neurochemistry.
“Over the past 15 years, doctors Brown, Cudkowicz, and myself have been colleagues in developing a ‘bench to bedside’ approach to better characterize the disease pathogenesis and identify mechanisms to slow the neurodegenerative process, with the hope of finding a cure for ALS,” said Ferrante. “This approach has brought together a strong research program for basic and molecular neuroscience, a preclinical drug program using both in vitro and in vivo models of ALS, and a national clinical trial consortium of centers to conduct therapeutic trials in patients with ALS. They have combined their individual expertise in ALS into a unique translational initiative to help end this dreaded disease.”
Additional support for two non-VA sites is sponsored by the Muscular Dystrophy Association. The non-VA sites include Massachusetts General Hospital and Johns Hopkins University.
The following VA sites are currently recruiting veterans for this study:
Edith Nourse Rogers Memorial Veterans Hospital, Bedford, MA
Durham VA Medical Center, Durham, NC
Michael E. DeBakey VA Medical Center, Houston, TX
Iowa City VAMC, Iowa City, IA
VA Medical Center, Lexington, KY
VA Medical Center, Syracuse, NY
For information about this study please contact Patricia Loya at (781) 687-2884, Patricia.Loya@med.va.gov or Sally MacDonald, RN, (781) 687-2963, Sally.MacDonald@med.va.gov
For a full list of study locations, please visit The ALS Association web site at www.alsa.org/patient/drug.cfm?id=629. The ALS Association national Web site contains up-to-date information on ALS-related clinical trials. Many trials still have open enrollment. For details, visit www.alsa.org/patient/drug.cfm.
