Researchers at the University of Washington School of Medicine in Seattle are excited over an experimental cancer drug that slowed down muscular dystrophy in mice. Although still preliminary, he researchers called it "very dramatic and impressive.”

 

There are a number of muscle-wasting diseases which are all incurable including Duchenne muscular dystrophy - the most common form found children. In Duchenne muscular dystrophy, the lack of a muscle protein known as dystrophin causes muscle weakness that lead to breathing problems. Patients usually die in their teens or early twenties.

The drug called trichostatin A (TSA) was being tested on skin cancer in mice when researchers noted that it affected the production of a molecule known as follistatin. Follistatin can indirectly cause muscle growth and counteract the deterioration caused by faulty dystrophin.

One advantage of TSA is that it can be taken orally in simple pill form unlike gene therapy. However, unlike gene therapy, TSA must be taken continuously. Right now, the potential side effects of long-term TSA use are still unknown.

Muscular dystrophy comes in various forms, but sadly, there is no known cure for any of them. The Duchenne form of this disease is the most common, particularly among children, which involves a mutation fora muscle protein known as dystrophin. The muscles weaken when copies of this protein cease to function. Corollarily, sufferers develop breathing problems, and ultimately, death in their teens or early twenties.

Now, light seems to be at hand at the end of the tunnel as researchers may have found a probable cure for the said disease. Administering an experimental cancer drug in mice with the disease, they saw that the muscular dystrophy actually slowed down.

Pier Puri of the Burnham Institute in La Jolla, California, along with colleagues, attempted to increase muscle function in mice carrying a mutation in the dystrophin gene. What they did was to treat the animals with the cancer drug called trichostatin A (TSA). This compound supposedly causes a change in certain proteins. According to Puri, the changes in the proteins somehow affect the production of a molecule known as follistatin, which can indirectly cause muscle growth and counteract the deterioration caused by faulty dystrophin.

The experiment on our little rodent friends involve the mutant mice being given a daily dose of TSA for three months. After that period, they were then subjected to a fitness test on a treadmill. Those on TSA managed to last 20 minutes on the treadmill, while the control group just lasted 12.5 minutes.

Although these experiments are not conclusive, being in the preliminary stage, hopes are raised that the approach might offer advantages over other medicines for muscular dystrophy which are currently being practiced in clinical trials. The researchers believe the drug could be manufactured in pill form. However, the patient would have to be continuously administered with it. Potential side effects of long-term TSA use are still unknown.