- An interactive webinar about the MDA-supported ISIS-SOD1-Rx trial for people with SOD1-related familial ALS took place March 2, 2011, via the Isis Pharmaceuticals website.
- ISIS-SOD1-Rx is an "antisense" compound designed to reduce production of toxic SOD1 protein molecules.
- A replay of the webinar will be available for a limited time after March 2, 2011.
Isis Pharmaceuticals hosted an interactive webinar Wednesday, March 2, 2011, at 12 noon Eastern Standard Time, to help prospective participants understand its MDA-supported, phase 1 study of ISIS-SOD1-Rx for people with familial amyotrophic lateral sclerosis (ALS) due to mutations in the SOD1 gene.
For those unable to participate during the live webinar, a replay will be available for a limited time on the Isis website.
About the ISIS-SOD1-Rx trial
ISIS-SOD1-Rx is an experimental "antisense" drug designed to interfere with production of toxic SOD1 protein. Abnormal, toxic SOD1 protein, produced from mutated SOD1 genes, is the root cause of ALS in approximately 1 percent to 3 percent of people with the disease.
The trial of ISIS-SOD1-Rx is designed to evaluate the safety, tolerability and phamacokinetics of ISIS-SOD1-Rx. ("Pharmacokinetics" is the science of how the body absorbs, distributes, metabolizes and excretes a substance.)
The experimental Isis drug is administered into the spinal fluid through a temporary tube via a pump. In this study, the drug will be given as a single, 12-hour infusion.
Four dosage levels will each include eight participants. At each level, six participants will be randomly assigned to receive ISIS-SOD1-Rx, and two will receive a placebo.
Study participants may be enrolled in more than one dosage group, if they qualify.
Genetic confirmation of an SOD1 mutation is required to enroll in the study. Genetic testing may be available for ALS patients if they have a family history of the disease and if other qualifications are met. For more information about the study and the U.S. study centers, see Safety, Tolerability and Activity Study of ISIS SOD1-Rx. Some travel assistance may be available.
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur. MDA has no ability to influence who is chosen to participate in a clinical trial. To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials.
Or, contact: Patricia Andres, NEALS Coordinating Center, Boston, (617) 724-8995, email@example.com.
About the ISIS-SOD1-Rx webinar
Merit Cudkowicz, co-chair of the Northeast ALS Consortium and a professor of neurology at Massachusetts General Hospital; and Timothy Miller, assistant professor of neurology at Washington University School of Medicine, was featured on the webinar.
Registration was through Patients on the Isis site. For technical support related to the webinar, contact: Brigitte Porte, Isis Pharma, Calsbad, Calif., (760) 603-4660, firstname.lastname@example.org
Meaning for people with ALS
The MDA-supported ISIS-SOD1-Rx trial represents a new direction in ALS treatment; namely, the targeting of a specific cause of one of the familial forms of the disease, the SOD1-related form.
If ISIS-SOD1-Rx proves safe, well tolerated and successful at reducing levels of toxic SOD1 protein production, the prospects for people with SOD1-related ALS may markedly improve.
See also ALS SOD1 Trial: A Watershed Moment for more about the ISIS-SOD1-Rx study.
Editor's note: This story was updated Feb. 7, 2011, to reflect the fact that the official name of this experimental drug is ISIS-SOD1-Rx, not SOD1-Rx.
The article was updated March 2 to reflect that the webinar had occurred.