MEMPHIS, Tenn. – For children born with rare inherited bone marrow disorders, treatment can often mean chemotherapy, a bone marrow transplant, and weeks in isolation.
Researchers at St. Jude Children’s Research Hospital hope to one day change that.
A team led by Dr. Shengdar Tsai has been awarded up to $28.5 million through the Advanced Research Projects Agency for Health’s Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) program. The federal initiative is investing more than $160 million to accelerate the development of precision genetic medicines for rare inherited diseases.
The funding will support a project called DRIVE, or Delivering Rare Disease In Vivo Editors, which aims to develop a new gene-editing platform that could one day treat inherited bone marrow disorders by correcting the genetic mutation causing the disease.
“These patients live with this idea of almost like an invisible sword over their heads,” Tsai said. “It’s a progressive disorder, and we believe that if we can edit them at an early stage, it could really improve their quality of life.”
Unlike some existing gene therapies that require removing a patient’s cells, editing them in a laboratory, and returning them to the body, researchers hope to develop an “in vivo” approach that delivers gene-editing medicine directly to patients.
The project will initially focus on rare disorders, including GATA2 deficiency and Shwachman-Diamond syndrome.
Today, the only curative treatment for many inherited bone marrow failure syndromes is a bone marrow transplant, according to Dr. Marcin Wlodarski, director of the Bone Marrow Failure Program at St. Jude.
“Today, the only cure for bone marrow failure syndromes is bone marrow transplant,” Dr. Wlodarksi said.
He said the procedure often requires chemotherapy and weeks of hospitalization and isolation while also exposing children to risks such as infection.
Researchers hope the technology they’re developing could eventually offer a less invasive option.
“With this new approach, with a simple IV infusion, we could potentially cure the disease,” Dr. Wlodarksi said.
The $28.5 million award will help researchers develop the gene-editing platform, complete the studies needed before testing in patients, and prepare future therapies for clinical trials.
While the work is still in its early stages, researchers believe the technology could eventually be adapted to treat many other rare inherited diseases beyond bone marrow disorders.
“We think these technologies could be really broadly applicable,” Tsai said. “We think they could be used to treat many different rare genetic diseases.”
Download the FOX13 Memphis app to receive alerts from breaking news in your neighborhood.
Trending stories: