Recently, Dr. Charles Thornton from the University of Rochester Medical Center, who leading the research team our fund-raising efforts support, shared some exciting news about the team’s efforts to find a cure for myotonic muscular dystrophy. Paraphrasing a bit he explained: Earlier this year we applied for a large grant of around $5.6 million over 4 years. The scientists who reviewed the project gave it high marks and it had to go through administrative review at NIH (National Institutes of Health). In late September we learned the grant will be funded. The goal of the project is to optimize a drug that can be taken forward into clinical trials, and to do all of the safety testing that is needed to obtain an “IND” – permission by the FDA to give a new drug to people. The money goes mostly to Isis Pharmaceuticals to cover the costs of purchasing raw materials and, most expensively, to subcontract with the laboratories that do the animal safety testing, which is very extensive. The optimization parts are done jointly at Isis and University of Rochester. Having this co-investment from the NIH, (about 40-50% of their development costs over the next 4 years) helps to make sure that the work will go forward.
Shortly after learning the news, Dr. Thornton was the first researcher to speak at a joint NIH/FDA conference called “Antisense Oligonucleotides in Neuromuscular Disease.” It was the first meeting of its kind. He was glad to acknowledge that their work was supported by NIH and went on to acknowledge “that a small group called Run America Foundation helped get the whole thing launched.”
We’re very hopeful this work will lead to a breakthrough medication that can slow down, stop or even reverse the debilitating effects of myotonic muscular dystrophy. Thanks to everyone who contributes financial and moral support to our cause. Happy Holidays!
