When the ice bucket challenge, being filmed with ice water poured over your head in sympathy with victims, went viral in 2014, world awareness of ALS dramatically increased as well as research funds to fight the illness. Now, the FDA has approved a new drug made by Mitsubishi Tanabe Pharma America to treat the illness. It is only the second drug approved to treat ALS and the first in twenty-two years.
ALS, or amyotrophic lateral sclerosis, is a particularly cruel disease that targets motor neurons. Persons with ALS have an increasingly difficult time controlling the involuntary signals between the brain and individual muscles. As the condition progresses, muscles atrophy and die, ultimately leaving the body without the ability to induce breathing. Survival after diagnoses is generally no more than two to four years.
Although ALS has been known since the 19th century, Lou Gehrig’s announcement that he had ALS in 1938 was the first time most people ever became aware of the rare disease. Its causes are still being investigated, but both genetics and a history of traumatic brain injuries appear to play a role in some people. A recent study linked the cause to genetic mutations that prevent the creation of protein that repairs nerve cells.
The new drug, called Radicava, will be available by August and administered intravenously at regular intervals. Though not a cure, Radicava was shown to slow the progress of ALS in clinical trials. About 2 in 100,000 people are diagnosed with ALS.
The new treatment will cost about $146,000 for those without insurance, a sum sure to continue the debate about high drug prices in the United States. In Japan, where the drug is already in use, its cost is $35,000.