A recent study found that a synthetic compound known as a bottlebrush block copolymer (BB polymer) can prevent the onset of Duchenne muscular dystrophy (DMD) in mice.
“These findings provide evidence of a highly potent and efficacious candidate DMD therapy, either as a stand-alone therapy or potentially as a combinatorial drug approach,” the authors wrote.
Read more about DMD therapies
As early diagnosis of DMD has improved with time, there has been a growing focus on treating the disease before it progresses. In their study, the researchers explored whether a type of BB polymer could stabilize muscles and restore function.
The authors first tested the compound in single skeletal muscle fibers from DMD mice. They found that the ability of the fibers to contract improved immediately. Furthermore, the BB polymer was 150,000 times more potent than other polymers that have been previously studied.
Next, the researchers administered the BB polymer to DMD mice after birth, continuing dosage every four days. Findings revealed that treated mice had no increase in serum creatine kinase levels, while untreated mice had significantly elevated levels.
What is creatine kinase?
Creatine kinase (CK) is an enzyme found in muscles. When muscles become damaged, CK leaks out of the muscles and into the bloodstream. In diagnosing DMD, CK levels are checked in a blood sample and if they are unusually high, this is a key indicator of DMD.
Additionally, muscles in the diaphragm and leg had no signs of damage upon visual inspection. Muscle cell surface area was also significantly greater in the treated versus untreated mice.
The study examined cardiac muscles as well, since heart complications are fairly prevalent in DMD. Once again, the results showed that the BB polymer effectively prevented muscle damage and protected the heart from injury.
The researchers didn’t find signs that BB polymer treatment was unsafe: It did not change any tested biomarkers of kidney and liver health.
Though much more research is needed, the authors noted BB polymer treatment has potential to preserve muscle function in DMD. “If muscle membrane instability and muscle tissue necrosis could be prevented in the long term by synthetic polymers, the preserved form and function of skeletal muscle, diaphragm, and heart would then be expected to markedly enhance the health and lifespan of DMD patients,” the authors concluded.
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