Trial Tests 'Master Regulator' in ALS

A phase 2 clinical trial to test the experimental drug GM604 in people with amyotrophic lateral sclerosis (ALS) has opened at two trial sites in the United States. Enrollment is expected to begin soon.

GM604 technology, developed by Genervon Biopharmaceuticals in Pasadena, Calif., is based on "master regulators," which are molecules found in early-stage human development that control the development of the human nervous system.

According to Genervon, the GM604 molecule's structure is identical to one of nine active parts of a neurotrophin called motoneuronotorphic factor (MNTF). The company says it confers neuroprotection via control and regulation of numerous ALS-associated genes and pathways.

The study, which is expected to enroll 12 adults with ALS, is designed to test the safety and tolerability of GM604. Additional study objectives include determining preliminary effects of GM604 on ALS disease biomarkers and other measures of disease progression.

How to participate in the phase 2 trial

Prospective trial participants must be at least 18 years old and meet the El Escorial criteria for a definite diagnosis of ALS. In addition, they must:

  • be able to comply with study procedures;
  • be medically cleared to undergo lumbar (lower back) puncture for the collection of cerebrospinal fluid;
  • not have a history of liver disease, severe renal (kidney) failure, diabetes, coronary heart disease or cancer; and
  • meet additional criteria.

Study site locations are:

Massachusetts General Hospital (not yet recruiting)
Boston, Mass.
Principal investigator: Nazem Atassi
Sub-investigator: Merit Cudkowicz
Contact: Nazem Atassi, 617-726-2000,

New York
Columbia Medical Center (not yet recruiting)
Principal investigator: Hiroshi Mitsumoto
Contact: Hiroshi Mitsumoto, 212-305-2940,    

To learn more, see GM604 Phase 2A Randomized Double-Blind Placebo-Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS) (GALS), or search for trial ID NCT01854294 at

About Clinical Trials

About Clinical Trials

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 Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to

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