Ibrutinib for treatment of graft-versus-host disease

[Neil Cuadra]

In the article At Last, a Brightening Outlook for Chronic GVHD (July 2017), Dr. Steven Pavletic comments on the status of research on graft-versus-host disease (GVHD) treatment. Dr. Pavletic is senior clinician in the Experimental Transplantation and Immunology Branch of the NCI Center for Cancer Research. (I've met him.)

The news is that studies of the use of ibrutinib (trade name Imbruvica) to treat GVHD have been positive. Imbruvica is used to treat chronic lymphocytic leukemia (CLL) and other diseases.

Here's an excerpt from the article:

At the American Society of Hematology annual meeting in December 2016, researchers released findings of a small clinical trial of ibrutinib treatment for 42 patients with chronic GVHD.

After treatment with ibrutinib, two-thirds of the patients experienced improvements in GVHD symptoms. Importantly, the symptoms completely resolved in 21 percent of these patients, and the symptom relief lasted for 5 months or longer in many of them. Based on these findings, the FDA issued Breakthrough Therapy status for ibrutinib in June 2016.

Researchers are now conducting a phase III clinical trial of ibrutinib to treat GVHD, with focus on its potential use as initial therapy rather than for patients who already have developed treatment resistance. According to published reports, the companies plan to file for regulatory approval of a GVHD indication for ibrutinib this year.