Upcoming and current ALS Clinical Trials at UCSD ALS Center
1.) Antisense Oligonucleotide Therapy for SOD1 Related Familial ALS
What: A novel therapy for SOD1 related ALS that will target and decrease levels of the mutated protein. The therapy is infusion of an antisense oligonucleotide (DNA like chemical) into the cerebral spinal fluid, the fluid that surrounds the brain and spinal cord.
Who: Patients who have ALS that runs in their families AND have a documented mutation in the SOD1 gene (Superoxide dismutase 1) AND are early in the course of their disease.
SOD1 mutations: For the vast majority (90%), ALS is a “sporadic” disease that does not run in their family. However, about 10% of people who have ALS, have a strong family history of ALS. Of those with ALS that runs in their family, about 1/5 of these families have mutations in SOD1 and roughly 50% of the family members will carry the mutation and will be affected by ALS. Typically, this is well known to families. A genetic test is available to determine if an individual carries the SOD1 mutation. This specialized test can be requested by any physician. We do not yet understand how SOD1 causes ALS.
How many patients? 16
Participating Centers: UCSD, Harvard, Johns Hopkins, Methodist Neurological Institute, University of Wisconsin.
When: Currently in toxicology testing. Anticipate start of Phase I trial Spring of 2008.
Whom to contact? If you have a documented mutation in SOD1 and would like to enroll in the trial, please contact Timothy Miller, MD, PhD at timiller@ucsd.edu. Patients without documented SOD1 mutations will not be eligible for the trial.
What’s all the buzz about? Drs. Miller and Cleveland, at the University of California, San Diego and Dr. Smith at the Center for Neurologic Study, working with Isis Pharmaceuticals, have demonstrated that these antisense oligonucleotides distribute widely in the brain and spinal cord, decrease their intended target (SOD1), and delay disease in the animal model of ALS. For detailed information, see (link to JCI paper). While the first trial will only be for those patients with SOD1 mutations, we hope to apply this same strategy to sporadic AL
Links:
More information about UCSD ALS Clinic
More information about UCSD Department of Neurosciences
More information about ALS Research in San Diego
General information about ALS clinical trials
General information about ALS
The ALS Association
The San Diego Chapter of the ALS Association
Muscular Dystrophy Association
National Institute of Neurological Disorders and Stroke
UCSD Neurosciences
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