Penn State Cancer Institute Advances Clinical Research for AML Treatment Paradigm

For more than 20 years, Shin Mineishi, MD, director of the blood and marrow transplant program at Penn State Cancer Institute, had treated acute myelogenous leukemia (AML) according to the long-established paradigm focusing on allogeneic stem cell transplant. In the past five years, however, he has begun to see a shift in that paradigm with transplantation being the center piece of a larger treatment sequence. The new approach emphasizes pre-transplant therapy and post-transplant maintenance together with the transplant itself to improve the transplant outcome. Over the next several years, Dr. Mineishi and his colleagues at the Cancer Institute will be conducting numerous trials to test aspects of this new paradigm.

In the past, only chemotherapy was available for induction and transplant conditioning. Transplantation, particularly using extremely toxic myeloablative regimens, was associated with numerous serious side-effects. One of Dr. Mineishi’s early efforts is to improve transplant conditioning by using new drugs. The standard of care has been fludarabine, but clofarabine has far better anti-leukemic activity, with little increase in toxicity, bringing disease into remission. In this new paradigm, clofarabine enhances the opportunity for successful posttransplant therapy, which cannot occur unless a patient’s disease is in remission. Now, many options exist including molecular targeting agents, monoclonal antibodies, immunotherapy and gene therapy. All options have the potential to be combined with transplantation. As Dr. Mineishi states, “This is a very exciting time for AML therapy, as we are seeing so many new developments.”

Research is a top priority for Dr. Mineishi. He is spearheading the initiative to open new Cancer Institute studies for AML posttransplant therapy and is seeking to develop trials of immune therapy and gene therapy. While these studies are still in the earliest planning stages, Dr. Mineishi has developed relationships with a number of other institutions engaging in multi-center trials and hopes to collaborate with them moving forward. These studies, started with a few centers, can be proposed to the Bone Marrow Transplant Clinical Trail Network (BMT-CTN) or Eastern Cooperative Oncology Group (ECOG), which performs multi-center clinical trials for translational and clinical cancer research. This coordination could lead to engaging more patients in a national study, and advancing the clinical knowledge base.¹

He states, “In the future, I want for us to develop studies here at Penn State Cancer Institute or at one of the other sites in the consortium that grow into national studies and ultimately change the standard of treatment for AML. I think we have the potential to do that.”

Reference

1. http://ecog-acrin.org/. Accessed September 7, 2016.