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FDA approves new ALS drug treatment
The U.S. Food and Drug Administration approved a new medication for treatment of amyotrophic lateral sclerosis, commonly known as ALS. The new medication, Radicava (edaravone) is the first treatment to be approved since 1995 and is now the second FDA approved treatment for ALS. Riluzole, the only other approved treatment for ALS was approved because it appeared to slow the progress of the disease but was never expected to be a cure for ALS.
History of ALS
ALS or Lou Gehrig’s disease (named after a famous baseball player diagnosed with the disease in the 1930’s) affects 12,000-15,000 people in the United States, with about 5,000 new diagnoses each year. The exact cause of ALS is still unknown but it is thought to be similar to multiple sclerosis (MS). ALS causes the gradual breakdown of special nerves in brain and spinal cord. As the disease gets worse the brain is unable to communicate with the rest of the body. When the brain is unable to communicate with the spinal cord people lose the ability to do simple tasks such as walking and eating, and it can eventually lead to the inability to breathe. Hence, the most common cause of death for people with ALS is respiratory failure.
ALS might sound vaguely familiar as it was brought to the spotlight on social media through the ice bucket challenge in 2014. To participate in the ice bucket challenge, participants had a large bucket full of ice water dumped over their head in order to promote ALS awareness. See how it all started here. Over an 8 week period $115 million were raised for ALS research through the ice bucket challenge. As a direct consequence of the ice bucket challenge 3 new genes were identified, over 150 new research projects started, and 9 global research collaborations were initiated.
Currently, treatment options (Riluzole and now Radicava) are not aimed at curing ALS but slowing the advancement of the disease. Other medications and treatments are attempting to increase independence and comfort while dealing with the symptoms of ALS. Symptoms that physicians can help to treat or provide relief for include:
- Muscle cramps and spasms
- Stiff muscles
- Excessive saliva
- Sleep problems
- Uncontrolled laughing or crying
Treatment for these symptoms include breathing care, physical therapy, occupational therapy, speech therapy, nutritional support, and psychological/social support. Physicians and healthcare professionals help to increase independence for as long as possible and help provide comfort measures, such as pain control and anti-nausea agents that can minimize the symptoms of ALS. Radicava provides a new treatment option in a setting where few options are available.
Radicava was used in a research trial in Japan where 137 individuals were given either Radicava or a placebo (a substance thought to be Radicava but really not). After 24 weeks of treatment, the patients who had received Radicava showed less decline in symptoms based off of clinical assessment of daily functioning when compared to those who received the placebo treatment. Radicava is administered intravenously. The initial cycle is daily IV infusions for 14 straight days followed by two weeks with no medication infusions. From there, infusions are for 10 consecutive days followed by a two-week drug free period. During infusions, most patients will need to either go to a local infusion center or have a home health nurse come to their house to perform the infusion.
Although not a cure for ALS, Radicava may have a significant impact on people who suffer from ALS. Since there is only one medication currently available for treatment, having an alternative medication offers significant hope for people with ALS. Many factors go into deciding if someone can safely take a medication or not. More options provide a better chance for treating ALS. Furthermore, there is hope that Radicava will be more effective in dealing with ALS than Riluzole.
There is speculation over the current use of Riluzole and the benefit it has for ALS patients. Currently it is estimated that between 50-75% of people with ALS are taking Riluzole. Edward Kasarskis, M.D., Ph.D., Director of the multidisciplinary ALS Center at the University of Kentucky Neuroscience Center, recommends that a comprehensive treatment plan be utilized. This would including proper nutrition, multiple different therapies, and Riluzole. One question that physicians like Nathan Staff, Director of the ALS Clinic at the Mayo Clinic, is asking about is the cost benefit relationship of the new medication Radicava. Staff note that it can be a difficult decision for patients and families alike to consider; paying hundreds of thousands of dollars for the possibility of three, four, or maybe five months of life. Riluzole prolongs life on average for three months. It has yet to be known the true effects of Radicava but there is strong hope that it will have a positive impact on people who have ALS.
Physicians at Mayo Clinic expect people will be lining up for the medication, but only time will tell how Radicava will impact the lives of the ALS community.