Minnesota gene therapy research saves the life of Michigan boy with rare terminal illness ALD
MINNEAPOLIS – There has been a breakthrough for boys who have a terminal illness called adrenoleukodystrophy, or ALD - and it's all thanks to the work of some dedicated Minnesota researchers.
One in 20,000 boys in the U.S. is born with ALD, a disease that causes them to slowly lose sight, mobility and speech.
Priscilla Veneklause knows all about ALD. The Grand Rapids, Michigan, resident has been through a lot.
"It was horrific, being a teenager and watching your dad deteriorate," Priscilla said.
He had the genetic brain disease that affects males. It took her father's life, and then her precious baby boy Landen was diagnosed, too.
"That was devastating, because I automatically, your brain goes to the worst case scenario, so I thought of my father," she said.
Priscilla got proactive, and traveled from their home in Michigan to Minnesota. M Health Fairview Masonic Children's was doing a clinical trial.
"We were thinking, 'What do we have to lose,'" she said.
Dr. Paul Orchard, a transplant specialist, led the trial at Masonic. Instead of a traditional bone marrow transplant, Landen got gene therapy with a stem cell transplant.
"The idea then with gene therapy, rather then using somebody else's cells, engineer your own cells to do that," Dr. Orchard said.
Landen took the transplant like a champ, but it wasn't easy.
"It was just a hot mess, it sucked," Landen said.
It was worth the pain he says, because the therapy worked. Dr. Orchard says it's pretty amazing.
"It seems to have stopped the progression of the disease," he said.
Landen says he is quite grateful.
"I'm living right now, I know how to eat, I know how to walk, I know how to talk," Landen said.
"There's hope in the future for these boys to live long, healthy lives," Priscilla said.
The FDA just licensed the gene therapy, which is offered at Masonic and three other places in the world.
The hope is this gene therapy could also help stop other terminal diseases.
