The ALS Association (ALSA) today announced renewed funding of a continuing effort to create a model of ALS in a laboratory animal of growing popularity among neurobiologists, the zebrafish. The ALSA-initiated project by investigators at Ohio State University in Columbus, Christine Beattie, Ph.D., and Anthony Burghes, Ph.D., is showing progress and is an important adjunct to the TREAT ALS initiative that will bring advances in ALS research rapidly to clinical trials.

Drs. Beattie and Burghes have already begun to create transgenic zebrafish as a model system for investigating ALS therapies. An additional two years of funding should allow the team to produce and characterize sets of fish that show symptoms of ALS to understand the biology of the disease process and to help design new treatments.

“I am encouraged by the progress made in creating a new model system to study ALS,” said ALSA’s Science Director Lucie Bruijn, Ph.D. “The development of a zebrafish model will be an invaluable tool to identify new targets and screen potential compounds for ALS.”

The co-investigators are well along in the process of creating the different fish mutants, in which each carries an introduced, altered gene coding for copper-zinc superoxide dismutase (SOD1). This is the gene that is mutated to produce some of the inherited forms of ALS in people. Many different mutations in SOD1 can produce the disease. The researchers will be investigating the effects of three particular SOD1 mutations in the fish.

Zebrafish are increasingly popular among developmental and neurobiologists, as the species has easily manipulated genetics, the basic vertebrate genome is present, and the larvae are transparent. One can literally watch as the fish develop their nervous systems and internal organs.

The researchers will see if the altered fish show problems with swimming and shorter life spans, as is expected for mutations that are present in people with inherited ALS. They are developing lab tests that will enable these investigations. Funding will also provide the means to examine in detail the connections between nerve and muscle in the relatively simple neuromuscular system of the fish and see if there are disruptions induced by the mutations in SOD1. In short, a powerful new tool will be developed with the additional funding supplied by ALSA in this targeted approach to solving the disease.