Request for Proposals
Human Gene Therapy Clinical Trials for ALS

Summary

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, is a fatal neurodegenerative disease that strikes 1 in 100,000 Americans every year. ALS is a disease of nerve cells located in the brain and spinal cord that cause action in muscles. Defects in these nerve cells called motor neurons result in the loss of the ability to activate and control muscles. Generally, ALS progresses rapidly and the disease runs its course in an average of 2 to 5 years. Over the years a number of measures have been clinically tested to treat ALS; however, to date there is no treatment that significantly arrests the progressive nature of the disease. There is a growing body of evidence that suggests that gene therapy may be a promising intervention in the treatment of ALS.

The Project

A number of recent publications have reported promising results of gene therapy in the laboratory using various vectors in combination with genes such as VEGF, IGF-1 and RNAi. Most of these studies have been conducted using the SOD1 mouse model of ALS. In an effort to seek wider application of gene therapy technology and explore its potential as a therapy in sporadic ALS, Muscular Dystrophy Association is inviting proposals to advance ALS gene therapy clinical trials. Competitive applicants are those who have demonstrated expertise in ALS and in gene therapy technology independently or in collaboration with other scientific investigators. The deadline for requesting the pre-application form in response to this RFA is December 1, 2005. You may submit the request for the grant application online through the following link to the MDA Web site: http://www.mda.org/research/rrga-form.html. Indicate in the “project title field” of the pre-application form that your request is in response to the RFA entitled “Human Studies in gene therapy for ALS.”