Drug prevents major bone marrow transplant complication

The Food and Drug Administration (FDA) recently approved Orencia for use in the prevention of graft versus host disease (GVHD). The drug was originally created to treat rheumatoid arthritis and has been approved for that use in the U.S. since 2005.

GVHD is a dangerous health condition that can occur after a stem cell transplant, also known as a bone marrow transplant (BMT). In GVHD, the immune system cells from the donor, or “the graft,” attack the person receiving the cells, or “host,” causing a range of symptoms from mild to severe.

Persons with rare primary immunodeficiencies (PI) benefit greatly from BMT. The treatment is common in PIs such as severe combined immune deficiency (SCID), chronic granulomatous disease (CGD), and Wiskott-Aldrich Syndrome (WAS). In many cases, the procedure is lifesaving for these persons.

However, BMT is not without its complications, a major one being GVHD. In GVHD, the new immune system cells, specifically the T cells, from the donor see the existing organs and tissues in the recipient as “foreign.” 

The transplant recipient experiences attacks from the donor cells on their skin, and in their gastrointestinal tract, mucous membranes, and certain organs like the liver and lungs. GVHD can be acute or long-term and is potentially fatal. Doctors treat GVHD with steroids and other medications to weaken the new immune system so the donor cells don’t attack the recipient’s body.

Orencia works by preventing T cells from being activated. When T cell activation is interrupted, GVHD is halted.

The drug is potentially useful in adults and children ages 2 and up undergoing BMT from an unrelated donor, and is the first drug approved to prevent GVHD.

“Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication,” said Dr. Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in a press release.

“By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”

Studies conducted on the use of Orencia found that the drug, used in combination with standard immunosuppressive drugs, increased the overall GVHD-free survival rate of persons undergoing BMT from a mismatched unrelated donor.

The FDA’s Orphan Products Grants Program partially supported the studies. The Orphan Products Grants Program provides funding for clinical studies on the safety and efficacy of products for use in rare diseases and conditions.