Groundbreaking new studies at a North Texas hospital are bringing new hope to families with little left.
The studies are aimed at helping children battling ultra-rare, deadly illnesses, like the one 6-year-old Will Woleben are battling.
The McKinney first grader was diagnosed at age two with Leigh Syndrome, a genetic disorder affecting the brain.
“We didn't know if we'd get to this point to be honest. The disease, when we first found out about it, we learned that the average age of survival is only two-and-a-half,” said Will’s father Doug Woleben.
Leigh Syndrome has no cure or treatment.
Over the past few years, parents Doug and Kasey Woleben have watched Will go from being a happy, seemingly-healthy little boy, to a child unable to walk or eat on his own.
“You can't comprehend that your child is dying, right, that they would die so young and so I quickly think we realized that we only had one choice which was to fight,” said Doug Woleben.
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The fight brought them to UT Southwestern where Dr. Steven Gray is pioneering new gene therapy treatment for children with ultra-rare diseases.
“Why aren’t pharmaceutical companies doing more of this?” asked NBC 5 reporter Meredith Yeomans.
“So the pharmaceutical interest, it’s tough. You know, gene therapy is a disruptive technology. It's a one-time treatment for potentially a life-long benefit which it’s hard to build a business model around that,” said Dr. Gray.
During his first and only clinical trial, Dr. Gray says each patient saw progress or the spread of their disease slow.
More than 20 clinical trials are now in the works including one for Will Woleben.
“We don't even know if he's going to be accepted but you know we don't care. We're still going to work towards finding a cure,” said mom Kasey Woleben.
The biggest question now is whether the health of their hockey-loving, soon-to-be first grader can hold out until his trial gets the go-ahead.
In the meantime, the Wolebens find victory knowing they're not alone in the fight to save their son.
Click here for more on the Wobelen's mission to find a cure for Surf 1 Leigh Syndrome.
