UW begins new clinical trial to treat fatal blood diseases in children, young adults

A clinical trial has launched at the UW School of Medicine and Public Health to test the safety of a new type of stem cell transplantation to treat a variety of deadly blood disorders in children and young adults.

The trial is being sponsored by the UW Program for Advanced Cell Therapy (PACT) in partnership with the Department of Pediatrics, Division of Pediatric Hematology, Oncology and Bone Marrow Transplant.

The Food and Drug Administration-approved trial will use a form of transplant that replaces a patient’s bone marrow with alpha-beta T-cell depleted peripheral blood stem cells from closely matched unrelated donors or family members.

Inga Hofmann, MD
Inga Hofmann, MD

The potential for this form of transplantation is the ability to cure many non-cancerous blood disorders, like sickle cell disease, severe congenital neutropenia and some anemias, among others, according to Inga Hofmann, MD, principal investigator of the study, UW Health oncologist, and assistant professor of pediatrics and the director of Pediatric Bone Marrow Transplant Program at the UW School of Medicine and Public Health.

“Proving safety is the first step, of course, but as our research progresses, we are very excited about what this type of transplantation could become,” she said.  Full story.