UVA to Expand Bone Marrow Transplant and Immunotherapy Offerings

At a Glance

  • UVA Cancer Center has renewed its NCI designation
  • Two new clinician researchers are expanding the UVA bone marrow transplant program
  • Karen Ballen, MD, will focus on increasing transplant options for adult members of minority groups through ongoing research efforts
  • Daniel “Trey” Lee, MD, will be launching the pediatric transplant program at UVA
  • Lee is working to develop new CAR T cell therapies for deadly forms of brain cancer that affect children

UVA Cancer Center has successfully renewed its NCI designation for another five years, a testament to the center’s ongoing efforts to expand and optimize its clinical research infrastructure and provide patients access to the most advanced cancer treatment and prevention strategies available. At the heart of this mission is UVA’s ability to recruit world-class clinicians and researchers to lead the way. Two recent additions to the UVA bone marrow transplant program are proving up to the challenge.

Karen Ballen, MD, comes to UVA from Boston, where she was director of the leukemia program at Massachusetts General Hospital and professor of medicine at Harvard Medical School. As section chief for hematologic malignancies, Ballen will be overseeing UVA’s bone marrow transplant program, which she notes has substantial advantages.

“We are one of two programs in Virginia that can offer the whole complement of transplants, autologous as well allogeneic donor transplants,” she says. In addition, UVA is approved by both the Foundation for Accreditation of Cellular Therapy (FACT) and is a National Marrow Donor Program-approved institution. This is the highest level of accreditation and reflects UVA’s excellent quality of care and patient outcomes.

A prolific researcher, Ballen was impressed by UVA Cancer Center’s extensive clinical trials program, which gives patients access to investigational treatments. Ballen will continue her own research efforts at UVA, which focus on expanding options for members of minority groups and those of non-Western European descent.

“Based on the research that I and other people have conducted, we are designing new methods that make haploidentical and umbilical cord blood transplants safer and reduce the risk of infection,” she says. Both these techniques allow a wider range of matches than traditional methods. In fact, based on this research, in 2017 almost every patient who needs a transplant can find a donor.

Finally, Ballen notes that UVA has structured its bone marrow transplant program to meet the needs of its patients, who often must travel for great distances to receive treatment. “UVA has facilities for patients and their families to stay overnight,” she says. “In addition, through our population health and telehealth initiatives, we are trying to move as much care as possible into local communities.”

New Pediatric Bone Marrow Transplantation Program

While Ballen focuses on the adult bone marrow transplant program, Daniel “Trey” Lee, MD, is charged with launching UVA’s transplant program for pediatric patients, which he hopes to be ready by the fourth quarter of the year. “We believe that Virginians of any age who need a bone marrow transplant should not have to leave the state,” says Lee.

Lee’s arrival at UVA also means that pediatric patients will have access to a promising form of investigational immunotherapy that has shown to be particularly efficacious for childhood cancers. “The success of antibody-based immunotherapy for adult cancers has not translated well for children,” he says. “We have had more success modifying patients’ T cells.”

Lee was among the second group of researchers worldwide to infuse children with multiply relapsed or refractory leukemia with CD19 chimeric antigen receptor T (CAR T) cells, and the results so far are very encouraging. “Almost five years after starting the trial, our leukemia-free survival rate is 60 percent, which is significantly higher than the 10 to 20 percent seen in similar patients given conventional therapies,” he says. “We now have the CD19 CAR T cell trial open here for children ages 2 to 21 years with refractory or relapsed acute lymphoblastic leukemia.”

UVA is the only place in the Commonwealth where this investigational therapy is offered. Lee is also developing new CAR T cell therapies for meduloblastoma, ependymoma, and other deadly forms of brain cancer that affect children.

“The best cancer institutions in the country combine excellent clinical care, clinical research and basic science research and invest in all the support infrastructure needed to run these programs well and treat very complicated patients,” Lee says. “In my view, UVA is part of that group.”

Learn more about cancer clinical trials now enrolling at UVA.