Queen’s University professor breaking ground on Hemophilia

For upwards of 20 years Dr. David Lillicrap, a professor in the department of Pathology and Molecular Medicine at Queen’s University, has dedicated himself to research aimed at curing hemophilia, an inherited bleeding disorder that affects a person’s ability to clot blood and can lead to serious bleeding episodes.

“We’re working with something called gene therapy for hemophilia. We’ve been doing this now for actually over 20 years and in the last ten or so years we’ve actually made some very good progress,” he said.

Lillicrap, flanked by his Ph.D. Candidate, has worked day in and day out on treating dogs like Croissant, who suffers from Hemophilia, producing some very encouraging results.

“It will be like they never had hemophilia,” said Ph.D. candidate Fateme Babaha.

“We’ve basically cured a significant number of dogs now with this therapy,” added Lillicrap.

But how are they doing it?

“We engineer what we call a delivery system to deliver into the dogs a copy of the gene that’s gone wrong and is producing the bleeding problem,” he said.

This, he said, has prevented bleeding episodes for over 10 years in the same dogs, all from a simple blood infusion.

Few people understand the plight of living with an inherited bleeding disease like Brock Powell, a Kingstonian and patient of Dr. Lillicrap who suffers from a very similar condition called von Willebrand disease.

“It’s a bleeding disorder but essentially my platelets are defective,” said Powell.

Medical advancements and research like the very work Dr. Lillicrap is doing have had direct impacts on Powell’s life, and may soon be available people he knows who suffer from hemophilia.

“Just not having to worry is the biggest thing. We try to live the most normal life we can but, ultimately, it’s always in the back of your head,” he added.

Lillicrap says there have been gene therapy products that have received regulatory patient approval in the past year.

He says he and his colleagues will continue to work to help make hemophilia treatment accessible, and affordable.

“So eventually, this is going to be quality of life improving and also economically beneficial, but that’s complicated and it’s going to take a little while longer before that’s happening,” he said.