US Approves New Cell Therapy for Blood Cancer: Progression-Free Survival Rates Reach 78% Without Severe Complications

The US Food and Drug Administration (FDA) has approved Tregzi, the first cell-based immunotherapy utilizing regulatory T-cells (Treg). It is designed to improve survival without chronic graft-versus-host disease (GVHD) in adult patients with blood cancers undergoing allogeneic hematopoietic stem cell transplantation.

The treatment represents a new approach to bone marrow transplantation, where the primary challenge is not only destroying the tumor but also preventing severe complications caused by donor cells attacking the patient's body.

Tregzi is manufactured from the blood cells of a compatible donor and comprises three distinct components: purified hematopoietic stem cells, regulatory T-cells (Tregs) that suppress excessive immune responses, and conventional T-cells (Tcons) involved in fighting tumor cells. This precise formulation makes it possible to maintain the anti-tumor effect while simultaneously mitigating the risk of chronic GVHD.

The efficacy and safety of the therapy were validated by the PRECISION-T clinical trial, which enrolled 187 adult patients with acute leukemia and myelodysplastic syndrome. Patients were randomized to receive either Tregzi or a standard transplant.

One year post-procedure, 78% of patients treated with Tregzi survived without developing severe chronic GVHD, compared to 38.4% in the standard transplant group. The incidence of severe chronic GVHD during the first year was also significantly lower in the Tregzi group, standing at 12.6% versus 44% in the control group.

The FDA noted that the trial results demonstrate a clear clinical benefit, with the potential rewards outweighing the risks. The most common side effects reported were infections, which aligns with expectations for patients undergoing post-transplant recovery. No severe infusion-related reactions or cases of graft rejection were reported.

Tregzi has been granted orphan drug and advanced regenerative medicine therapy designations. The therapy was developed and received approval by Orca Biosystems, Inc.

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