According to a story from reuters.com, the U.S.-Israeli company BrainStorm Therapeutics will not provide its investigational stem cell treatment under the recently passed U.S. Right to Try law. The company cited concerns about making the treatment affordable for patients. The stem cell therapy is under development for the treatment of rare, neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS). Instead, the company plans to continue its clinical trial of the drug, which is being tested as a treatment for ALS, in order to gain official FDA approval.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis is a neurodegenerative disease in which the the neurons that control voluntary muscle movement gradually die. The causes of this disease are not known, though a small percentage of cases may be inherited from one’s parents. Symptoms include muscle stiffening and twitching, muscle atrophy, and difficulty, swallowing, speaking, and breathing. Patients are also affected mentally, with many showing changes to behavior that can degenerate into dementia. Eventually, patients become unable to move, and require support to continue breathing. Most patients eventually die in their sleep when their respiratory muscles fail. There are few treatments available, and only one, riluzole, can delay progression by a couple of months. To learn more about amyotrophic lateral sclerosis, click here.
The Fundamental Flaws of Right to Try
The decision from BrainStorm is completely legal, as the Right to Try law does not require companies to provide experimental drugs per a request from a patient or doctor. The concern that the company had about making the drug affordable is probably compounded by the fact that Right to Try also does not require insurers to cover the drugs, nor does it require doctors to prescribe them.
The situation highlights the reality that Right to Try is essentially a “placebo” law, that has no concrete requirements that guarantee that patients who are seeking experimental treatments will actually ever get them. Unfortunately, the law can provide false hope to desperate patients, who are probably better off attempting to get experimental drugs through the FDA’s compassionate access program.
Only one patient will get to try the drug under Right to Try: Matt Bellina, a naval pilot with amyotrophic lateral sclerosis who worked to get the new law passed. The CEO of BrainStorm, Chaim Lebovits, has agreed to personally sponsor his treatment.
