Mark Sands identifies combination treatment in mouse model of Batten disease

Gene therapy combined with bone marrow transplant synergizes to provide benefit in mouse model of this fatal childhood disease. Read More

From the WUSTL Newsroom

Infants with Batten disease, a rare but fatal neurological disorder, appear healthy at birth. But within a few short years, the illness takes a heavy toll, leaving children blind, speechless and paralyzed. Most die by age 5.

There are no effective treatments for the disease, which can also strike older children. And several therapeutic approaches, evaluated in mouse models and in young children, have produced disappointing results.

But now, working in mice with the infantile form of Batten disease, scientists at Washington University School of Medicine in St. Louis and Kings College London have discovered dramatic improvements in life span and motor function by treating the animals with gene therapy and bone marrow transplants.

The results are surprising, the researchers say, because the combination therapy is far more effective than either treatment alone. Gene therapy was moderately effective in the mice, and bone marrow transplants provided no benefit, but together the two treatments created a striking synergy.

The research is online in the Annals of Neurology.

“Until now, this disease has been refractory to every therapy that has been thrown at it,” says senior author Mark Sands, PhD, professor of medicine and of genetics at the School of Medicine. “The results are the most hopeful to date, and they open up a new avenue of research to find effective therapies to fight this devastating disease.”

The combination therapy did not cure the disease, the scientists caution, but mice that received both treatments experienced significant, lasting benefits.

For more from Caroline Arbanas of the WUSTL Newsroom, click here.

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Posted on April 18, 2012
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