- The U.S. Food and Drug Administration approved Radicava (edaravone), a drug that will help treat patients who have ALS.
- Studies conducted in mice have shown that weekly doses of glucocorticoid steroids like prednisone help speed up recovery in muscle injuries.
- Researchers at the National Institute of Health's National Center for Advancing Translational Sciences, along with the University of Nevada, Reno School of Medicine, have shown a drug that has been known to unsuccessfully treat cancer as a potential treatment for Duchenne muscular dystrophy.
Muscular dystrophy is a critical condition that must be treated immediately to lessen the severity of its symptoms. Though the condition is rare, it's still crucial to become aware of the illness and the recent studies that have been done for you to become more knowledgeable about what you can do to prevent or manage the disease, whether you or a family member has it. There is much news and many updates to follow regarding the discovery of treatment and other scientific facts about the condition. But what are these news pieces you need to catch up on? Read on as we discuss the latest news in muscular dystrophy and other neuromuscular diseases.
The latest news on muscular dystrophy and neuromuscular disease
Here are a few of the most recent discoveries and studies made on muscular dystrophy during the past few months:
FDA approves a drug for treatment of ALS: May 9, 2017
The U.S. Food and Drug Administration approved Radicava (edaravone), a drug that will help treat patients who have ALS. This drug has been used in Japan, and upon learning of its effects, the FDA has engaged with the drug's developers on filing market applications across the U.S. It's a new treatment for ALS that was approved by the FDA for many years. And medical professionals in the FDA are pleased that patients now have an additional option for treatment. The drug, Radicava, is an intravenous infusion, administered with a treatment cycle of dosing for 14 days every day as a start, then followed with a drug-free period of two weeks. Then, the cycle will repeat.
In a clinical trial in Japan, 137 patients were randomized, receiving either edaravone or placebo. The trial lasted for six months, and by the end of week 24, those who had received edavarone declined less as compared to the other patients who had been mediated with a placebo. Adverse reactions for those who took edaravone included bruising and gait disturbance. There have also been serious risks that require medical care, such as swelling, shortness of breath, hives, or allergic reactions to sodium bisulfite, which is found in edaravone. Edaravone has been granted an orphan drug designation, providing incentives to both assist and encourage the drug development for rare diseases such as ALS. FDA granted approval of the drug to Mitsubishi Tanabe Pharma America, Inc.
Taking steroids weekly strengthens and repairs muscles: May 18, 2017
Studies conducted in mice have shown that weekly doses of glucocorticoid steroids like prednisone help speed up recovery in muscle injuries. These steroids have also helped repair damaged muscles from muscular dystrophy. One primary problem of using steroids is that they tend to cause muscle wasting and weakness when taken for a long period of time. This is a significant issue for those who have chronic conditions, especially ALS. It's the reason why patients are forced to stop taking steroid treatments. But after the study with mice, it has proven that steroids taken in weekly doses rather than daily doses have established to promote muscle repair. While there is no human data yet, the findings strongly suggest alternative ways of giving the drug that won't harm but promote the muscle repair that those with chronic conditions need.
This study on weekly steroids has been published in the Journal of Clinical investigation. It shows that prednisone directs the production of annexing, which is a protein that stimulated muscle healing. Weekly doses of the steroid stimulate KL15, a molecule that improves muscle performance. While daily doses result to muscle wasting, healthy mice with muscle injuries who have had two weekly doses of the steroid performed better than those who have received placebo. Mice that received steroids daily performed poorly as compared to mice with placebo. Mice with muscular dystrophy have also performed better on the treadmill when given a weekly dosage of steroids, as compared to mice dosed with placebo. When taken every day, the steroid would adversely affect the mouse's muscles, wasting and damaging it in the long run. The study was conducted to know exactly how prednisone prolongs the patient's ability to walk independently. Since the adverse reactions of steroids affect the mobility of people with chronic conditions, it has been a medical curiosity on how to treat conditions correctly. Hopefully, the study would be done on humans to see how it would react on patients with ALS. But for now, steroid treatment is still not usually offered due to its side effects.
Researchers aim to repurpose therapy for muscular dystrophy treatment
Researchers at the National Institute of Health's National Center for Advancing Translational Sciences, along with the University of Nevada, Reno School of Medicine, have shown a drug that has been known to unsuccessfully treat cancer as a potential treatment for Duchenne muscular dystrophy. This drug is called SU9516, which improves the muscle repair process and muscle structure. The team screened over 350,000 compounds to find SU9516, a previously developed drug to treat leukemia. It showed that the compound helped in improving muscle function in lab and animal DMD models. It was published in Molecular Therapy, giving a promising approach in treating ALS and other muscle-wasting diseases.
People with DMD have less dystrophin than usual, which is a protein that keeps muscle cells intact. Through boosting the a7B1 integrin, a cell structural protein, it can help alleviate the symptoms of DMD while slowing down its progress. The screen revealed that SU9516 helped raise integrin production, as well as promote muscle cell and finger formation that indicate a potential as a drug to treat the disease. The drug can be used alone or in combination with therapies that still need to be worked on. It can also be used for other conditions that damage the muscle, such as cachexia. Once researchers find a way to increase the production of integrin with drugs artificially, it can help protect muscle cells from further damage. The research team from NCATS and UNR plan to work with medicinal chemists to make a molecule more accurate for DMD, also to remove the anti-cancer components for better treatment and testing for patients.
When it comes to learning more about muscular dystrophy, you can only learn so much from outdated books and websites. What you need to do is also read about the latest news and updates on how the disease is being studied and what new treatments are available for patients to use. Many people around the world will benefit from learning the many updates on muscular dystrophy, because it helps raise the necessary awareness to raise funds that contribute to the studies in finding a cure.
We hope that this article on the latest news regarding muscular dystrophy helped you become more knowledgeable on the progress made by medical professionals in the field. If you want to learn more about muscular dystrophy, then navigate our website for more information on what it is and how you can treat the condition. And if you would like to share more points on muscular dystrophy, then comment down below.