2004 Research Highlights

Therapeutics

Drug Discovery

• The most promising drugs from last year’s NIH-sponsored screening have gone through basic and animal testing. The top one—also verified by 1,000 Center assays—now has NIH approval for large clinical trials. Those will begin after regulatory agencies sign off.
• We’ve forged new networks with other major nonprofits to screen even more drugs.
• Within the Center, drug development continues alongside basic research. We’re starting tests of a new drug to enhance benefits of others.
• Celebrex trials are finished, awaiting data analysis.

 

Gene Therapy

• With support and leadership from Project ALS, Jeff Rothstein and colleagues have advanced studies of a gene therapy, using IGF-1, to extend the life of motor neurons for breathing. If success continues—so far, animal results are exciting—human testing could be a year away.

Nerve Repair

• Douglas Kerr has shown that stem cells morph into motor neurons when injected into paralyzed rat spinal cords. He’s pushed the neurons into taking early steps on the road to muscle targets.
• The Center, with Project ALS support, is bent on promoting motor nerve growth. Our scientists are at work on the underlying biology. Drugs to test have surfaced, based on that biology, and animal studies are well under way.

Basic Research

Center scientists now actively explore major cell pathways that go wrong in all types of ALS.

The earliest damage strikes the cell’s energy-makers, the mitochondria, Don Cleveland found. They become clogged with abnormal protein. Motor neuron mitochondria deal with the protein in an unusual way; that’s why ALS targets those cells. Blocking toxic reactions that injured mitochondria trigger may halt the disease.

Jonathan Glass has shown that gross problems in motor neurons begin at the close-to-muscle end. The major find suggests we could buy time to save neurons before injury reaches the vital cell nucleus.

Genetics Research

David Cornblath and John Griffin have helped pinpoint the ALS4 gene—yet another inherited ALS. Center scientists believe the ALS4 animal model they’re engineering will highlight similarities and differences in the various types of the disease, telling more surely which pathways to target for a cure.

FYI

This year our scientists have published more than 85 ALS-related papers in respected journals, including Science, Nature, Proceedings of the National Academy of Sciences, New England Journal of Medicine, Journal of Cell Biology and Nature Genetics.

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