Antibiotic Helps Mice with OPMD

The antibiotic doxycycline, on the market to treat infections, has shown promise in treating oculopharyngeal muscular dystrophy (OPMD) in mice with an OPMD-like disease, says a British research group that published its findings online May 1 in Nature Medicine.

When Janet Davies at Addenbrooke’s Hospital in Cambridge, UK, bred mice with an abnormally expanded section of the gene for PABPN1, the same genetic defect that causes OPMD in humans, they found that the OPMD-affected mice treated with doxycycline developed weakness later and stayed stronger than did untreated mice.

The treated mice had fewer abnormal clumps (aggregates) in their muscle cells, and they lost fewer cells. The researchers say both these mechanisms are probably involved in lessening disease severity and delaying disease onset.

Guy Rouleau, an MDA-supported physician-scientist at the University of Montreal whose research group identified the OPMD gene defect in 1998, says he would advise “cautious optimism” in interpreting these results, because the translation from mice to humans in drug studies is often not straightforward. “It is a very important first step,” he said, “but should not be overinterpreted.”