Here’s the latest news from the University of Buffalo (UB) about an experimental therapy called GsMTx4. Based on a molecule found in tarantula venom, this “protein” can prevent muscle mass loss and muscle damage in an animal model of Duchenne Muscular Dystrophy (DMD). Enjoy your reading and thank you for sharing these articles in the DMD community.
This research from UB, published last July in the magazine “Neuromuscular Disorders,” shows that this new drug significantly reduces the loss of muscle mass.
What Is GsMTx4?
GsMTx4 is a small protein, a peptide*, present in the venom of the tarantula. This protein works by preventing some ion channel activity in muscles without affecting the communication between nerve cells and muscle cells.
Frederick Sachs: “GsMTx4 represents an out-of-the-box treatment to slow the progression of DMD.”
Thomas Suchyna noted in a previous study that in addition to protecting muscles, GsMTx4 protected against cardiomyopathy, a common cause of death in patients with DMD.
Frederick Sachs: “Remarkably, we did not observe any side effects in mice in this study. The drug also has a long life so that subcutaneous injection may be needed only once a week.
The researchers concluded that GsMTx4 could also be complementary to other therapies, such as the anti-inflammatory agents and gene replacement strategies prescribed or studied in DMD.
GsMTx4 has already been licensed to Tonus Therapeutics and Akashi Therapeutics as a sublicence for further development.
Status:
By the spring of 2019, a new investigatory new drug application will be filed with the United States Food and Drug Administration (FDA). If successful, by 2020, Phase I/II studies in humans will be followed.
Manufactured by chemical synthesis, GsMTx4 is considered an ‘orphan drug,’ a designation that the FDA grants to promising therapies for rare diseases.
About DMD
Duchenne muscular dystrophy (DMD) is a disease that affects boys almost exclusively and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. Duchenne muscular dystrophy (DMD)—for which no treatment is currently available—directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.
More Links
University of Buffalo:: Promising new therapy spares muscle loss in Duchenne muscular dystrophy
These results were published in the study entitled, “GsMTx4-D provides protection to the D2.mdx mouse,” in Neuromuscular Disorders.
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