Study will test drug to treat illness after stem cell transplants

Vijaya Bhatt, M.B.B.S.

Stem cell transplants have saved countless lives of patients with blood cancers and other diseases. But, a side effect of the life-saving treatment also can rob patients of quality of life and in some cases, their life.

Vijaya Bhatt, M.B.B.S., UNMC assistant professor of internal medicine and oncology/hematology, has received a grant for up to $1.2 million to determine if a drug called ruxolitinib, is effective for treating a certain type of graft versus host disease (GVHD) called sclerotic. The grant is funded by Incyte, a global biopharmaceutical company.

UNMC and its clinical partner, Nebraska Medicine, are world-renowned for their expertise in transplantation and are only one of the few centers nationwide testing the drug for the purpose of treating GVHD.

The drug, which reduces inflammation, has proven effective for other uses, and shows promise as a treatment for patients who develop GVHD after receiving an allogeneic transplant. An allogeneic transplant uses a matching donor’s cancer-free stem cells, which are then infused into the cancer patient.

Sometimes donor cells attack the recipient’s healthy tissues and organs, which can impair function and is a significant cause of medical problems, including death.

Dr. Bhatt, principal investigator of the study, said GVHD is difficult to treat since first line treatments often fail.

Finding a treatment is important because an estimated 40,000 patients undergo allogeneic stem cell transplants every year, and about 30 to 50 percent of these patients develop acute GVHD and about 30 to 70 percent develop chronic GVHD. The research team will focus on the chronic GVHD, which comes on slowly and continues sometimes for years.

Dr. Bhatt and co-principal investigator Stephanie Lee, M.D., at Fred Hutchinson Cancer Research Center in Seattle, will recruit 47 study participants beginning this month, at five centers, including UNMC and Fred Hutchinson.

The UNMC study site also will involve Ram Mahato, Ph.D., professor and chairperson in the UNMC College of Pharmacy Department of Pharmaceutical Science, and Lynette Smith, Ph.D., assistant professor in the UNMC College of Public Health Department of Biostatistics.

“When I joined UNMC three years ago, I felt the need to work on improving care of patients with GVHD,” Dr. Bhatt said. “Since then, we’ve established a multidisciplinary team in which we work with other providers who are instrumental in helping treat our patients.”

He said patients who’ve been treated with the drug have experienced good results so far. “It seems to be effective and doesn’t have as many side effects as some of the other medications,” he said. “Our hope is that it will improve how patients feel and improve the quality of their life.”

2 comments

  1. Carol Russell says:

    Nice work!

    Carol Russell
    SRC Member

  2. Hanh says:

    Congrats, VJ!

Comments are closed.