FDA approves Radicava, first new ALS treatment in 20 yearsMay 6 – The U.S. Food and Drug Administration on Friday approved the first new drug in decades to treat the fatal neurological disease ALS. The FDA approved edaravone, sold under the drug name Radicava, produced by the drug company Mitsubishi Tanabe Pharma America. Clinical trials showed the drug slowed down the symptoms of ALS, also known as Lou Gehrig’s Disease. There is no known cure for ALS, a disease that slowly paralyzes people and is 100 percent fatal.
The FDA issued its approval after a Japanese clinical trial of a small pool of 137 ALS patients who were administered edaravone, declined less in terms of daily functioning compared to those who did not receive the drug. Radicava is expected to be available in the United States by August. FDA officials said they urged the Japanese drug company to apply to make the treatment available as quickly as possible. That drug helps heal nerve cells attacked by the disease, but generally only extends a patient’s life expectancy by a few months. It interacts with the nerve-damaging compounds created as ALS progresses.
It is administered intravenously daily for intervals of two weeks, followed by a two-week break over the course of a year. The treatment is not without expense for health insurance companies. The cost per infusion is $1,000 and a full course of the medication comes in at more than $145,000. Its makers said it intends to work with patients whose health insurance plans that require significant copayments to lower the out-of-pocket cost.
MS vs. ALS: How Do They Differ?Both MS and ALS are neurodegenerative diseases that attack the central nervous system and affect the nerves and muscles, impacting a person’s ability to move. In early stages, several symptoms of ALS and MS can be similar, like muscle stiffness, muscle spasms, difficulty walking, and fatigue. In contrast, ALS is a motor neuron disease that mainly affects the actual motor neuron cells in the brain and spinal cord. In ALS, sclerosis damages the motor neurons, causing the myelin sheath to become harder. Unlike ALS, the course of MS is unpredictable and differs from person to person.
In the late stages of ALS, patients are completely debilitated and paralyzed. Unlike ALS, which affects only the nerves involved in movement, MS can also affect nerves that control the senses and the bladder. MS patients tend to experience greater mental impairment, while ALS patients typically exhibit physical difficulties. In contrast, ALS is a fatal disease with an average life expectancy of about three to five years post-diagnosis. MS is more common in women and ALS is more common in men.
MS is usually found earlier in life than ALS. MS is most commonly diagnosed in people ages 20 to 50, while ALS is most commonly diagnosed in people ages 40 to 70. MS is not thought to be a genetically inherited disease, although people usually have a higher risk of developing the disease if a close relative has MS. In contrast, around 10 percent of ALS cases are caused by mutated genes passed down directly from the parents.
ALS Intervention, Treatment and Goal WritingMany ideas and examples were given as well as goal writing options which can be difficult when a patient has ALS. by Christie on December 31, 2017. Amber is extremely knowledgeable and provided excellent treatment strategies and ideas for creative adaptive equipment. For me as a COTA I have gained more insight of how you can use simple low cost material or just simple piece of foam or rubber band to adjust anything to help a patient improve their comfort level, not just patients with ALS but any patients with great needs. As a therapist working with clients who have ALS I thought it was a great overview and I found myself nodding in agreement throughout her presentation.
I loved how it gave me great ideas on how to help pts with ALS ! It made me excited about being a OT again and why we do what we do. The ideas shared for goals and treatment were very creative and relevant to my practice of having a monthly ALS clinic. Great speaker and so many great examples of treatment ideas and adaptive equipment. Full of great ideas to use with not only our ALS pt’s , but with all our pt’s.
Great examples of intervention techniques and new ideas for adaptive equipment. I had no idea OT could be so impactful for ALS clients! Very good information and treatment strategies. Excellent treatment strategies for ALS and good treatment ideas. Very concise and effective treatment ideas that can be accomplished in the home or clinic….
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Petition · Brainstorm: Pursue accelerated approval of NurOwn treatment for ALS patients · Change.orgMy name is Bobby Forster and I have ALS, also known as Lou Gehrig’s disease. Most people with ALS live 2-5 years after their first signs of disease and our treatment options are limited. Recently, I was a participant in the US trial of a new treatment called NurOwn. After two weeks of treatment I went from barely able to stand for more than ten seconds, to being able to walk with a walker, to being able to walk unassisted. This therapy is one that will require multiple treatments every couple months.
The company that makes NurOwn, Brainstorm, is hesitant to pursue accelerated approval of this treatment with the FDA and has opted to conduct a Phase III trial which could delay approval for another 5 years.&nbs p;Without accelerated approval thousands of ALS patients could die over the next several years waiting for the traditional process for approval to be completed. Another challenge Brainstorm faces is raising enough money to conduct an expensive Phase III trial. If the money is not raised then Brainstorm and NurOwn could be dead in the water and the treatment will never make it patients. This treatment has now been in multiple trials both in the US and Israel.
It is time for all ALS patients to gain access to this ground breaking treatment. Please sign this petition and ask Brainstorm to pursue accelerated approval of NurOwn with the FDA. I am sick and tired of seeing my friends die while there exists a safe and effective treatment.