FDA grants humanitarian use for Miltenyi's stem cell transplant device

Miltenyi Biotec announced today that the FDA had approved its device for humanitarian use in patients with acute myeloid leukemia (AML) who are getting allogeneic stem cell transplants from matched, related donors. The device, called the CliniMACS CD34 Reagent System, is designed to lower the risk of graft-versus-host disease (GVHD), which is a common complication of this type of transplant.

GVHD occurs when immune cells known as T cells from the donor attack the recipient's tissues--a life-threatening complication. Germany-based Miltenyi's device uses antibodies that are connected to magnetic beads to remove donor T cells from the graft, according to the company's press release. They do that by enriching CD34+ blood stem cells, which then repopulate the recipient's immune system, while also removing other cells that can cause GVHD.

The FDA cleared Miltenyi's system for humanitarian use after a Phase II study conducted by the National Heart, Lung, and Blood Institute and the National Cancer Institute showed that when it was used, the incidence of GVHD at two years post-transplantation was just 19%, according to the release. The system did not negatively affect relapse, engraftment or survival, the company says.

This was a single-arm study, so Miltenyi did not provide data comparing use of CliniMACS CD34 to the standard of care. But according to the Leukemia & Lymphoma Society, patients are typically given immune-suppressing drugs such as cyclosporine and methotrexate to prevent GVHD. "The low incidence of acute and chronic GVHD, in the absence of additional immunosuppression are distinct advantages in using the CliniMACS CD34 Reagent System," said Dr. Robert Soiffer, chief of the Division Hematologic Malignancies at the Dana-Farber Cancer Institute, in a statement included in Miltenyi's release.

Several companies and oncology research institutions have long been searching for ways to prevent GVHD. Last year, Dana-Farber was among a group of academic institutions that identified a biomarker to spot stem cell transplant patients who are at high risk of GVHD.

- read the press release here