Blood & Marrow Transplantation: Innovative Treatments

Pictured: Susan Prockop & Nicole Zakak Pediatric Bone Marrow Transplant specialist Susan Prockop (left) discusses a patient with nurse practitioner Nicole Zakak.

During the first twenty years of our program's existence, we achieved many milestones in the field of transplantation. Our priorities for the next five years are to build on developments in both the laboratory and the clinic so that every patient who comes to Memorial Sloan Kettering Cancer Center for a blood stem cell transplantation procedure leaves a success story.

Memorial Sloan Kettering's doctors are working to improve the use of donor-derived T cells after a transplant to fight infections, and to prevent or reverse cancer relapse after a transplant. Research in gene therapy is also under way, with the goal of one day being able to transfer therapeutic genes into blood cells and immune cells to correct diseases of genetic origin. Clinical trials are vital to bone marrow transplantation. Read about the importance of clinical trials and learn about our newest clinical trials in our clinical trials section.

Another blood stem cell source is showing promise: studies have shown that infusions using cord blood stem cells, collected from the umbilical cord and placenta of newborns, are successful between matched siblings in the treatment of both genetic and acquired blood disorders and can also be used for unrelated adult patients.

Memorial Sloan Kettering Cancer Center investigators are also helping to develop two additional transplant techniques. Reduced-intensity transplants (RIT) use lower doses of chemotherapy and radiation in the preparative regimen, suppressing a patient's immune system just enough so that it cannot attack and reject the donor's blood stem cells. RITs rely on the patient's new immune system — growing from the donor's cells — to destroy disease. Memorial Sloan Kettering Cancer Center physicians use this approach for older patients, those with chronic leukemias and lymphomas and multiple myeloma, or patients who cannot tolerate standard transplants due to poor overall health.

In a tandem stem cell transplantation — sometimes referred to as a “double” transplantation — an autologous transplant is followed six months later by a second transplantation, which is either autologous or an allogeneic reduced-intensity transplantation. Although the benefits of this procedure are still not fully understood, early results have shown that tandem transplants may improve the survival rate of some cancer patients.

Memorial Sloan Kettering Cancer Center laboratories are also making progress in blood stem cell transplantation research, with mouse models proving to be particularly helpful. Using mouse models allows us to mimic the transplantation process in humans. This helps us understand some of the major complications, like GvHD, that occur after a transplant. We can use this knowledge to develop therapies to treat these problems and to prevent them in the future.

Our investigators have advanced the use of adoptive cell therapies with T or natural killer cells after transplantation to treat or prevent infections, post-transplant lymphoproliferative disorder, and disease relapse.

Memorial Sloan Kettering investigators have developed and used new strategies (including Interleukin-7 and Keratinocyte Growth Factor) to reduce the complications of immune deficiency after transplantation.