MEMPHIS, Tenn. – For children born with rare inherited bone marrow disorders, treatment can often mean chemotherapy, a bone marrow transplant, and weeks in isolation.<\/p>\n
Researchers at St. Jude Children\u2019s Research Hospital hope to one day change that.<\/p>\n
A team led by Dr. Shengdar Tsai has been awarded up to $28.5 million through the Advanced Research Projects Agency for Health\u2019s 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.<\/p>\n
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.<\/p>\n
\u201cThese patients live with this idea of almost like an invisible sword over their heads,\u201d Tsai said. \u201cIt\u2019s a progressive disorder, and we believe that if we can edit them at an early stage, it could really improve their quality of life.\u201d<\/p>\n
Unlike some existing gene therapies that require removing a patient\u2019s cells, editing them in a laboratory, and returning them to the body, researchers hope to develop an \u201cin vivo\u201d approach that delivers gene-editing medicine directly to patients.<\/p>\n
The project will initially focus on rare disorders, including GATA2 deficiency and Shwachman-Diamond syndrome.<\/p>\n
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.<\/p>\n
\u201cToday, the only cure for bone marrow failure syndromes is bone marrow transplant,\u201d Dr. Wlodarksi said.<\/p>\n
He said the procedure often requires chemotherapy and weeks of hospitalization and isolation while also exposing children to risks such as infection.<\/p>\n
Researchers hope the technology they\u2019re developing could eventually offer a less invasive option.<\/p>\n
\u201cWith this new approach, with a simple IV infusion, we could potentially cure the disease,\u201d Dr. Wlodarksi said.<\/p>\n
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.<\/p>\n
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.<\/p>\n
\u201cWe think these technologies could be really broadly applicable,\u201d Tsai said. \u201cWe think they could be used to treat many different rare genetic diseases.\u201d<\/p>\n
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