It has been over two decades since the Food and Drug Administration (FDA) has approved a new drug for amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease. However, the FDA recently broke this cycle and approved the first new drug to treat ALS, giving those with the disease hope, where there was none.
There is no cure for ALS, but a drug approved in 1995, Riluzole, has been effective in slowing the progression of the disease. ALS is a neurodegenerative disease that progresses very quickly, attacking the nerve cells in the spinal cord and brain.According to the Centers for Disease Control (CDC), between 12,000 and 15,000 people in the United States have ALS. The most common cause of death in people with ALS is respiratory failure, which can occur within three to five years of the initial diagnosis.
After a successful clinical trial in Japan, the FDA approved Radicava to treat ALS in May. The drug is capable of slowing the decline in physical function in patients who have been diagnosed with ALS. When researchers administered Radicava to animal models, it successfully prevented nerve cell death by decreasing oxidative stress. The nerve damage caused by ALS is not clearly understood.
Some of the health community has concerns about the clinical study conducted by Japanese researchers, because the volunteers were in the early stages of the disease, exhibiting less severe disability and slightly slower progression. The findings may not apply to everyone with ALS and other researchers believe the FDA approved Radicava because the results of the study were strong enough to prompt approval.
Radicava was approved as an “orphan drug,” which means it is intended to treat a rare condition or disease that affects 200,000 or less. The orphan status grants
specific financial incentives for the development process of the drug, including extended patent protection and tax credits.
The cause of ALS is still unknown, but medical experts have a better understanding of the biology of ALS than they did a decade ago. The knowledge deficit has contributed to the lack of beneficial drug development. Many people who are diagnosed with ALS have already experienced a considerable loss in nerve cells. So, drugs that have already been tested may not be effective in treating the disease, because it has progressed too far to treat.
The side effects of Radicava include constipation, bruising, rash, skin changes, insomnia and dysphagia, difficulty swallowing. When comparing the side effects of the placebo group with the group that was given Radicava, the side effects are essentially the same, but the drug appears to be safe, according to Dr. Justin Kwan, an assistant professor of neurology at the University of Maryland School of Medicine.