U.S. federal health regulators have approved a new drug for amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. The drug called Radicava or edaravone, is the second drug to be greenlighted by the FDA for the condition after more than 20 years.
Radicava is manufactured by MT Pharma America, a subsidiary of Mitsubishi Tanabe Pharma Corporation in Japan. It is given by intravenous infusion and will cost more than $145,000 per year.
ALS is a progressive neurodegenerative disease that affects the nerve cells in the brain and the spinal cord that are responsible for making muscles work. The condition causes the nerve cells to lose their ability to initiate and control muscle movement leading to paralysis and death.
Most of those with the condition die from respiratory failure, usually between three to five years after the symptoms started to appear.
Cure Remains Elusive
While the approval of a new ALS treatment offers new option for those with the condition, it does not provide a cure for the disease, which affects about 12,000 to 15,000 Americans, according to figures from the Centers for Disease Control and Prevention.
In a six-month study conducted in Japan, the drug was found to slow down the progression of the disease. The first drug approved by the FDA for ALS, Riluzole can increase survival of patients by two or three months but it is not yet clear if Radicava has an effect on survival.
The new drug was approved not because it extended survival, but because it was found that the symptoms in patients who use it deteriorate more slowly, which means that for those given the new treatment, their condition will still get worse albeit this would happen more slowly.
In the study, 137 patients were randomly assigned to receive Radivaca or placebo. After six months, the conditions of those who were given the drug declined less than those who were given placebo.
At the end of the study period, those in the Radicava group scored about 2.5 points better on a 48-point scale that measure the disease's severity. MT Pharma vice president of medical affairs Jean Hubble, said that the treatment appears most beneficial in patients who were still at the earliest stage of their disease.
Researchers are not certain how the medicine works, but it seems to sop up the free radicals that are partly responsible for the progress of the condition.
Hope For Better Treatment
The ALS Association, a national non-profit organization fighting Lou Gehrig's Disease, applauded the approval and expressed its optimism for the availability of more therapies for those suffering from ALS in the near future.
The money raised from the ALS Ice Bucket Challenge that spread on social media in 2014 has helped fund important developments in the research about the disease, which could eventually lead to better treatments. Research funded by the proceeds of the ice bucket challenge, for instance, has identified a gene responsible for ALS.
"We hope today's announcement signals the beginning of a new chapter in the fight against this terrible disease," the ALS association said. "There are several drugs to treat ALS currently in clinical trials and we are hopeful that people living with ALS have even more therapies available to them sooner rather than later."