In a new study published in Nature Genetics, University of Pennsylvania researchers and colleagues have made inroads into the mechanism by which ALS acts. Working with a powerful fruit fly model of the disease, they found a way of reducing disease toxicity that slows the dysfunction of neurons and showing that a parallel mechanism can reduce toxicity in mammalian cells. Their discoveries offer the possibility of a new strategy for treating ALS.
Mouse engineers presented the latest models overexpressing the amyotrophic lateral sclerosis gene fused in sarcoma (FUS) at “New Frontiers in Neurodegenerative Disease Research,” a Keystone Symposium held 4-7 February 2013 in Santa Fe, New Mexico. These animals have been four years in the making. This is a long time even for mice (see ARF related [...]
Globs of protein clustered in the neurons that control muscles have long been the hallmark of amyotrophic lateral sclerosis (ALS), the fatal neurodegenerative disease also commonly known as Lou Gehrig’s disease. Now, a study of the most commonly found mutant gene in people with ALS reveals an unexpected origin of some of those sticky masses, a finding that may offer drug developers a new target for treatments.
The ALS Therapy Development Institute (ALS TDI) and the Gladstone Institutes today announced the formation of a research collaboration to speed the discovery of potential treatments for ALS through the preclinical drug development process.
A key challenge to the development of new drugs based upon the genetic information derived from familial ALS, as well as genes associated with sporadic ALS, is to understand how mutations in the various genes produce a similar clinical and pathological phenotype.
Scientists hope that by creating models of disease in different animals—from flies to reprogrammed human stem cells—research will converge on an answer, or multiple answers. “I think you have to use all these models, with different mutations, in the hopes that there will be one that really does provide the breakthrough,” says Maniatis. For patients and families, that breakthrough can’t come soon enough.