

CIBMTR Receives Largest Grant in College History

The Center for International Blood & Marrow Transplant Research (CIBMTR) was awarded MCW's largest grant ever, just under $45 million in federal money over six years, to fund and analyze large clinical research trials. Dr. Mary Horowitz, Interim Chief, Division of Hematology and Oncology is the PI of the grant titled: Blood and Marrow Transplant Clinical Trials Network - Data Coordinating Center.

In the fight against blood cancers - leukemia, lymphomas and myelomas - transplants of bone marrow and umbilical cord blood allow doctors to kill off rogue cells with chemotherapy and then rebuild a patient's immune system. About 20,000 blood and bone marrow transplants take place in the United States annually, and the number is rising about 5% to 10% a year.

Still, slightly fewer than half of the people who could benefit from such a transplant actually end up receiving one. Often the problem is the lack of a perfect or very close match, a difficulty that a potentially revolutionary approach in one of the clinical trials could remedy.

At present, doctors use a system of matching to ensure that donor marrow is compatible with the recipient's. Eight proteins are compared; at least seven must match. Four of the proteins come from a person's mother; four from the father.

If scientists can develop methods to safely transplant marrow that matches only on four, rather than seven, of the proteins, then mother, father and children all would be compatible, significantly reducing the problem of not being able to find donors.

"For the first time, we're able potentially to have donors for everyone, which is the Holy Grail in transplantation," said Richard J. Jones, director of bone marrow transplantation at Johns Hopkins, which has been developing the so-called half-match technique.

In a late-stage clinical trial about to begin, these half-matched transplants will be measured against transplants of umbilical cord blood. Like bone marrow, umbilical cord blood has the ability to rebuild our immune systems. But the cells, which come from the umbilical cords of newborns, usually require less precise matching because they are less mature.

Other major trials are expected to examine variations in drug metabolism and to search for biomarkers, sometimes-small genetic differences that may explain why a therapy succeeds with one patient and fails with another.

The grant comes from the National Heart, Lung and Blood Institute, part of the National Institutes of Health.

"Without this grant, large multicenter clinical trials (in bone marrow transplantation) would largely come to a grinding halt," said Mary Horowitz, principal investigator for the project and chief scientific director of the Center for International Blood and Marrow Transplant Research at the college. "There are some really important studies that just wouldn't get done if we didn't have this network."

The Blood and Marrow Transplant Clinical Trials Network, a collaboration between Horowitz's center and transplant centers across the country, performs the trials.

First launched with federal money in 2001, the consortium has coordinated the protocols and organization of more than 25 trials involving about 4,000 patients. "A big grant over six years - that seems to be a tremendous opportunity for them," said Laura L. Douglass, president and chief executive officer of Next Generation Clinical Research Consulting Inc. "It is hopefully going to be one of the most coordinated and better efforts in this area, and it's at an institution that's got a lot of experience working statistically with clinical data."

In the area of transplants, Douglass added, "Wisconsin is a front-runner."

Horowitz said a major reason her center at the Medical College received the $45 million grant was that it collects data from 200 American transplant centers and about as many international centers detailing the outcomes of bone marrow and blood transplants. This information is valuable in selecting and designing clinical trials.

While individual institutions can carry out trials on their own, modern medicine increasingly depends on large, multisite trials involving large numbers of patients. The NIH grant places one group, the consortium, in the position of managing the details involved in coordinating such a large study.

"Institutions can be very isolated in the outcomes they have," Douglass explained. "Anytime we can have one group that can analyze a large amount of data from many different institutions, it really does provide opportunity to determine some of the best practices and approaches for treatment."
At Johns Hopkins, Jones put it this way: "You can do studies in your own institution, but the only way that you can show that it works is in a larger trial."


Source: Milwaukee Journal/Sentinel, August 19, 2011:
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