The world of pharmaceuticals and biotechnology is ever-growing and changing, but seemingly only behind closed doors.
Companies such as BrainStorm develop new treatments and medications that could change, or possibly even save, the lives of patients with conditions deemed incurable by the medical community. The problem is that these new treatments and medications take between seven and 10 years to reach the market because of the stringent standards and practices that must be shouldered before obtaining the Food and Drug Administration's approval. Without FDA approval, no patients may undergo these new treatments.
The concept that there are people dying of illnesses when there is a chance of recovery was not acceptable to people like Matt Bellina, who was diagnosed with amyotrophic lateral sclerosis (ALS) a few years ago. BrainStorm, a biotechnology company, has developed an experimental therapy named NurOwn that has given ALS patients new hope in surviving the disease, which has a prognosis of two to five years from diagnosis.
The treatment, though, is still in the throes of clinical trials. BrainStorm has a “double-blind placebo-controlled phase 3 trial evaluating the efficacy and safety of repeated doses of the investigational therapy,” and “NurOwn is actively recruiting eligible participants at all 6 U.S. sites,” said the company.
With the help of his family and others like him, Bellina appealed to lawmakers with the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act (Right to Try), which was signed by the president on May 30, 2018.
With the Right to Try Act in effect, BrainStorm is allowed to and has decided to give Bellina the NurOwn treatments outside of the clinical trials. Without the proper research completed, there is no solid evidence of the treatment’s efficacy (or lack thereof), which means it might not even work, but Bellina felt he had to at least try. “Given my options even a slight chance at efficacy is better than not trying at all,” Bellina said.
