Muscular Dystrophy Treatments May Be On The Horizon

Several possible new treatments for muscular dystrophy have been discovered in recent months, some rather surprising. For example a molecule in cola, when injected into laboratory animals with analogous conditions, alleviated the symptoms and arrested the progress of the degenerative disease. The substance, called THI, is found in caramelized sugar and brown sugar as well as cola; it boosts levels of a protein ordinarily responsible for muscle maintenance, which malfunctions in people with the form of muscular dystrophy referred to as Duchenne. THI was both injected into the experimental subjects and added to their drinking water—mimicking the way most humans who consume it do so.

Duchenne muscular dystrophy is only one form of the disease, but it is the most common, accounting for half of all cases. With muscular dystrophy, the muscles are not properly repaired after damage, even the wear and tear of ordinary life. In healthy people, muscles can be permanently weakened by severe injury, but more prosaic damage is repaired by cellular processes. In muscular dystrophy patients, these processes don’t work. This means the limbs slowly lose function, followed by the muscles responsible for respiration—most patients eventually need to rely on a ventilator to breathe.

There is no cure for muscular dystrophy currently available. There are medications to slow its progress, but these drugs cannot reverse the damage, or even stop it on a long-term basis, and so the focus is generally on management. Physical therapy can help maintain as much mobility as possible, and surgical treatments can minimize the effects of muscular contractures and curvature of the spine. In addition, a pacemaker may be used to maintain a regular heartbeat. One experimental treatment is looking at a slightly different approach to protecting the muscle-maintenance protein, by deactivating a quality control mechanism that destroys it in muscular dystrophy patients.

A third approach being studied looks at the genetics behind the disease. Muscular dystrophy is hereditary, with a complex inheritance pattern that means carriers of the mutations responsible may have no reason to be aware of it. Most Duchenne patients are male. Genetic editing techniques are being developed that may be able to fix the mutated genes early on, preventing proteins from being damaged and the disease from developing.

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