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

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.

This type of stem cell therapy has been difficult in the past because it also depletes a patient’s natural immune protection response to infectious agents that their body would otherwise be able to protect against, like dormant viruses.

Hofmann and her PACT cell processing partners are utilizing an FDA-approved technology called the Miltenyi CliniMACS system, which selectively eliminate types of cells in the transplantation process that cause two common post-transplant disorders, graft-versus-host disease and Epstein-Barr Virus infection.

“PACT has established cell manufacturing competencies within UW Health that meet exacting FDA regulatory standards for this study,” said Ross Meyers, PhD, director of PACT cell manufacturing.

The Phase 1 study seeks to enroll 12 people ages 3 months to 40 years old. It is the fourth clinical trial sponsored by PACT.

The trial will be conducted at UW Health’s University Hospital in Madison, in collaboration with the UW School of Medicine and Public Health and the UW Carbone Cancer Center.