ALS patient seeks FDA fast track for new drug

Denise Dador Image
Sunday, March 8, 2015
ALS patient seeks FDA fast track for new drug
A man diagnosed with ALS is asking the public to join the campaign to get new medication for his disease approved as quickly as possible.

Needing a cane to walk is unknown territory for a man who considers himself a modern day cowboy. But, last summer, Rob Donahue had to retire from riding his horses when a mysterious muscle weakness worsened.

"I've lost muscle tone. It's just dead," Donahue said.

By December, doctors diagnosed Donahue with Amyotrophic Lateral Sclerosis, or ALS, a disorder that involves the death of motor neurons, eventually robbing patients of all movement, including the ability to breathe. The survival rate is two to five years. No viable treatment exists.

"My son is graduating college in June and I want to still be able to walk," Donahue said.

Donahue's wife, Silvia, heard about an experimental drug called GM-604 made by Pasadena pharmaceutical company Genervon. Experts describe it as a master controller, an agent that can biologically modify thousands of genes and increase the preservation of motor neurons.

While studies appear promising, they're still preliminary. In February, Genervon asked the Federal Food and Drug Administration for accelerated approval.

One ALS patient even started an online petition in hopes of speeding up the process.

"People can't wait five, ten, 15 years for the clinical trial process. Things need to happen much quicker," said Nick Grillo, who also suffers from ALS.

Grillo's petition has amassed nearly 250,000 signatures, but will it convince the FDA?

One expert says drugs that treat life threatening conditions are given immediate consideration.

"This drug, if it's good, if it's safe, if it works, it will be approved," said Dr. Frances Richmond, of the USC International Center for Regulatory Science.

Richmond said only those who see study results -- the FDA and the drug maker -- can judge if the risks outweigh the benefits.

"There will be concern about whether those trials were really extensive enough to do more than give patients false hope," Richmond said.

But hope is the word working for Donahue. As the debate on benefits versus risks goes on, the real risk many patients say are those who die waiting.

Donahue says his full time job is to exercise, eat right and do all he can to fight the disease.