The transplant program at WCMC is dedicated to pursuing clinical and translational research while providing superior patient care. Areas of research interest include:
1) Development of novel conditioning regimens for patients with advanced hematologic malignancies.
The concept of reduced intensity transplantation has not entirely fulfilled its promise. We now routinely offer transplantation to patients well into their 70s, but toxicity, chronic GVHD and recurrence remain major problems. WCMC transplant program is focusing on the development of new regimens with increased efficacy, but limited chronic GVHD. We have studied various modifications of current regimens as well as novel methods of GVHD prophylaxis. Examples of ongoing studies include the combination of CPX-351, a novel anti leukemic drug as a "bridge to transplant" (U.Gergis PI) or the use of clofarabine conditioning regimens. New studies under development will involve the incorporation of hypomethylating agents before transplant as a method of chemotherapy sensitization as well as post-transplant administration of hypomethylating agents. In the near future we hope to be able to assign treatment based on sophisticated detection of minimal residual disease.
Thanks to the introduction of targeted treatment approaches, the outcome for patients with lymphoma has improved dramatically for most, but not all patients.We are particularly focused on improving outcomes for patients with lymphoma who failed initial treatments. We are devising novel treatment combinations and conditioning regimens particularly suited for such patients.
Outcomes for patients with myeloma have much improved. Patients live longer, healthier and more productive lives than ever before. This has in large part occurred because of the introduction of autologous transplant which remains a mainstay of the treatment for myeloma. In induces longer and better remissions than any other treatment and patients often can be without treatment (treatment holidays) for prolonged periods of time. But continued research is needed to reach the goal of cure. Two ongoing studies evaluate the incorporation of high dose lenalidomide and high dose Bendamustine in transplant conditioning regimens. The Principal Investigators for these studies are Dr. Mark and Dr. Shore respectively. Protocols for allogeneic transplantation are under development and may be suitable for patients with high risk disease.
2) Alternative donor Transplant –the Haplo Cord transplant concept.
Only approximately 60% of Caucasians and a much smaller percentage of people of other ethnic groups. New treatments that allow safe and effective transplants for such patients are urgently needed.
We developed the concept of haplo-cord transplant where we transplant two very different stem cell products: a related haplo-identical and an umbilical cord blood graft. The initial results were recently reported and are extremely encouraging. We are now planning randomized studies comparing haplo-cord with double cord transplantation as well as phase II studies for high risk patients.
Dr. Gergis was the first to report the efficacy of tocilizumab an anti-IL6 Receptor antibody for the treatment of GVHD. Follow up studies are planned. If the activity of this drug can be confirmed, it could revolutionize the treatment of graft vs host disease.
4) National and Multi-Center Studies
Our membership in the Alliance, the largest NIH funded cancer clinical trials network, allows us access to national studies of novel interventions by the alliance and by BMT-CTN (Clinical Trials Network dedicated to transplant studies)
We recently joined the Chronic GVHD consortium as an affiliate member. This is an NIH supported cooperative group and our membership provides access to new drugs and interventions for chronic GVHD.
We also participate in two important industry sponsored studies including a randomized study of photopheresis for chronic GVHD, which should better define the role of this important treatment modality. The second study evaluates the role of the drug Fresenius ATG in the prevention of graft vs host disease. Institutional Collaboration
Our protocol design and treatment approaches are developed and inspired by our close collaboration with our colleagues in the leukemia, lymphoma and myeloma programs who are members of the Hematology/Oncology Division.
Transplant outcomes are to a large degree determined by prevention and treatment of infectious complications. We also benefit from close interactions with immucompromised infectious disease programs under the direction of Dr. Thomas Walsh. Thanks to our collaboration we will have access to important new medications and for example will collaborate on a pivotal study of zoster prophylaxis.