Gene replacement therapy that works in dogs to be tested in children

by: Dave Wagner

SEATTLE – KIRO 7 has learned of a revolutionary treatment, here in Seattle, that has saved the lives of dogs and is just months away from being tested in children. In video obtained by KIRO 7, dogs that were destined to die show no signs of the disease after a single infusion of gene-replacement therapy.  The disease is so deadly in children, 50-percent of them die before their second birthday.

“This is a huge deal.  This is probably the most important thing we’ll ever work on,” said Dr. Casey Childers of U.W. Medicine.

For the past eight years, Childers has been focused on finding a cure for a form of muscular dystrophy called Myotubular Myopathy or MTM. It is a rare disease that affects the skeletal muscles.

“Patients are unable to walk. They’re unable to speak, unable to swallow and unable to breathe without assistance. It’s a childhood disease. It affects baby boys and it’s universally fatal. So it’s a bad, bad disease,” said Childers.

Myotubular Myopathy affects dogs, too.  In U.W. Medicine video, never seen by the public until now, gene-replacement therapy has resulted in a remarkable transformation in dogs and a possible cure for MTM. 

>>Videos of children, dogs with Myotubular Myopathy  

The search for a cure began with the search for a dog by a mother in Jacksonville, Florida.  The son of Alison and Paul Frase was born with Myotubular Myopathy.   Joshua Frase was born on February 2, 1995 with a devastating prognosis. 

 “My doctor came to me and she said, Alison, I really believe Joshua has a severe disorder and he’s not gonna make it through the day.” 

Joshua continued to defy the odds, but only had the strength to move his right hand.  

 “Joshua cognitively was a normal little boy.  He was just locked in a body,” said Paul Frase.

With 50 percent of children with MTM dying before their second birthday, Alison was determined to find a cure for her son Joshua. Alison was told by their geneticist at Boston Children’s Hospital that if she could find a dog with MTM, doctors could try gene-replacement therapy that could possibly lead to a treatment for her son. 

 “That’s when her wheels started spinning and she said, we’ve got to figure something out,” said Paul Frase.

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