Research Update — from ALSA’s National Office
June 29, 2004

In the current issue of Proceedings for the National Academy of Sciences (PNAS), scientists demonstrate that Copaxone protects neurons in a model of Parkinson’s disease. These studies together with a published study of Copaxone in a mouse model of ALS (PNAS 2003, 100 (8): 4790-5) has prompted an initial safety study in ALS patients.

Study Rationale:

The immunization strategy in mice induces T cells (of the immune system) from the periphery to enter the damaged central nervous system, slowing down local inflammation in the brain and increasing neurotrophic factor production surrounding the neurons. Copaxone is an amino acid polymer that generates T cell production. The T cells secrete ant-inflammatory cytokines such as IL-4, IL-10 and transforming growth factor-? (TGF?). In addition, in the Parkinson’s mouse model, levels of glial cell line derived neurotrophic factor (GDNF) was shown to be increased.

Copaxone is used in the clinic for multiple sclerosis. Although ALS and MS have little in common, some studies suggest an immune component may contribute to ALS. Although it is not clear that this therapeutic approach will have an effect in ALS and other laboratories studying the effects of this compound in mouse models of ALS showed less dramatic changes in survival as compared with last year’s publication, a small pilot study for ALS is certainly warranted.

Questions and Answers

Question: How could a vaccination help people with ALS?

Answer: The study of glatiramer acetate is a small trial of 30 people with ALS to test the safety of this drug, evaluate side effects or adverse events, and to determine if this drug has an impact on the body’s immune system. There is no evidence that glatiramer will stop or prevent ALS, but it may reduce inflammation around the motor neurons and slow disease and/or boost neurotrophic factors in the motor neuron environment.

Unlike more familiar immunizations for childhood diseases, glatiramer would not prevent ALS. Other most common vaccines (chicken pox, measles) are the main types of immunization that stimulate or bolster the body’s immune system to prevent specific diseases.

Question: Who can enroll in the current study of glatiramer acetate?

Answer: This small study of 30 patients with ALS is being conducted by the ALS Center at Columbia University in New York City, NY. It is a six month study. Anyone can be enrolled who meets the enrollment criteria and can travel to the study site in New York City at the start of the study and each month for the next six months. There is no charge to patients for participating in the study; however study participants are responsible for their own travel expenses for the seven visits to the study site.

For more information on the study and for details on enrollment criteria, contact Carolyn Doorish at (212) 305-2027 or cd2141@columbia.edu

Question: Can my doctor prescribe glatiramer acetate for me?

Answer: The research on glatiramer acetate was done on mouse models of Parkinson’s and ALS. There is no laboratory or clinical data yet demonstrating safety or benefit for people with ALS. Without such data showing safety and benefit, it is not advisable for physician to prescribe glatiramer acetate "off label."

Glatiramer acetate is FDA-approved and is a treatment for multiple sclerosis with a brand name of Copaxone.