Thursday, January 22, 2015

Support GM1 Research

GM1 research has been going on for many years, Auburn University has had success in cats using gene treatment therapy. More funding is needed to move onto the next phase.

A friend who's daughter also has GM1 type 2, recently produced this video: Eli and Evan's pictures are displayed at the end of the movie

A friend in the GM1 Gangliosidosis facebook group posted the following:

Just to clarify a little bit, the $500,000 is needed to finish the pre-clinical gene therapy work and initiate an investigational new drug (IND) application with the FDA (the application required for a clinical trial). The gene therapy studies at Auburn in the GM1 cats were started many years ago and Dr. Martin's success has been tremendous with gene therapy treated GM1 cats to date living over 4 years of age and still doing very well (untreated cats live ~8 months, this work was published last year in the journal Science Translational Medicine). This work was all supported by grants, but now safety and toxicity studies must be conducted before starting the IND process. So at this point it has been shown that the therapy works in GM1 mice and cats, but now it has to be proven to be safe. It is hard to get federal funding for safety and toxicity studies (bc they are not all that exciting) yet they are necessary. So the $500,000 is needed to support the final safety studies and go through the IND process in order to get to a clinical trial. I hope that helps. Here's a little more information