An anonymous donor has given $155,000 to help a McKinney boy beat an ultra-rare illness.
Will Woleben, 6, has Leigh Syndrome, a fatal genetic disease that has cost him the ability to walk, talk and eat. Despite these obstacles, he continues to play indoor hockey, root for his favorite sports teams and attend first grade.
Researchers at UT Southwestern Medical Center are pioneering an approach that could help Will and thousands of children with similar genetic defects. The donation will allow UT Southwestern to kick off initial lab testing of the technique, known as custom gene therapy.
"We couldn't be more thankful," said Will's father, Doug Woleben. "You get to a point where you're not used to good news happening."
The donor reached out to the Wolebens after their story ran on the front page of The Dallas Morning News on Sept. 2.
The Wolebens and UT Southwestern are moving quickly, because Will's disease is progressing. Few children with his condition live past the age of 10. While the donor helped the family reach its initial funding milestone of $300,000, UT Southwestern scientists will need around $3 million more to manufacture and test the experimental drug before they'll be able to administer it to children.
Steven Gray, a gene therapy expert who joined the UT Southwestern faculty in December and is part of the hospital's new gene therapy center, is working on treatments for several ultra-rare diseases like Will's. Because these conditions affect so few people, and because there is little data about which course of study might be best, federal and foundation grants are hard to come by, says Gray. So he partners with families to raise funds and move the research forward faster.
He said the new donation makes it much more likely that he and his colleagues will be able to develop a treatment in time to help Will.
"I really don't see or hear about things like this happening elsewhere. It just blows my mind," said Gray.
The Wolebens have received other help and support from North Texans. A McKinney woman who read their story convinced restaurants in and around Dallas to donate a portion of their proceeds to the Wolebens' Foundation, Cure Surf1, which is named for Will's defective gene.
"As a grandmother who is expecting her third grandson this hits really close to home," said Lori Busa. Her weekly "Dine 4 Will" series kicks off Monday at Grub Burger Bar in Plano. The restaurant will donate 20 percent of receipts placed in a special Cure Surf1 bucket from Sept. 17 through Sept. 21.
"If I can take one ounce of burden off of them, then I will do everything in my power to do so," she said in an email.
This Sunday, the Wolebens are also hosting a "Golf for the Cure" fundraiser at Topgolf at The Colony.
"We are incredibly lucky, but we still have a long way to go," said Doug.
