MOON SHOTS PROGRAM
Stem cell transplantation offers promise to multiple myeloma patients. But those with the high-risk form of the disease face a dismal 2-3 year median survival rate. MD Anderson physicians are intervening during the disease’s earlier stages and finding new reasons for optimism.
Multiple myeloma is the second most common blood cancer, with 27,000 new diagnoses in the U.S. annually. Prevalence is steadily increasing. The disease is caused by rogue plasma cells that accumulate in the bone marrow and interfere with the normal production of other blood cells. Symptoms include bone lesions, anemia and kidney failure.
Myeloma is unique for having two well-defined precursor states: monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). Intervening successfully at these earlier stages would improve patient outcomes, but predicting the disease’s progression is tricky. Even today, patients with high-risk precursors are rarely treated. This wait-and-see approach cannot continue.
MD Anderson’s High-Risk Multiple Myeloma Moon Shot™ is consolidating resources across MD Anderson and aiming to delay and ultimately prevent progression of the disease — especially for high-risk patients who have had few answers until now.
Listen to the podcast below by Robert Orlowski, M.D., Ph.D., professor of Lymphoma/Myeloma, as he talks about the nature of high-risk multiple myeloma, its precursor states and the goals of this Moon Shot™.
Donald Berry, Ph.D.
Professor, Biostatistics
Eric Davis, M.D.
Associate Professor, Lymphoma/Myeloma
Robert Orlowski, M.D., Ph.D.
Chair Ad Interim, Lymphoma/Myeloma
Perry Rupp, of Roanoke, Texas, received a successful stem cell transplant at MD Anderson after his multiple myeloma diagnosis. He’s now giving back to the nurses who took care of him — and doing it with an eye on innovation.
The risk of death and a lack of curative therapy for patients with high-risk multiple myeloma have driven the cost of care to among the highest for any cancer, leaving myeloma as an important public health challenge nationally and abroad.
An underlying goal of this Moon Shot is to better understand the molecular changes in mature myeloma cells and myeloma stem cells that trigger the cancer’s aggressive behavior. Moreover, researchers need to learn why a patient’s immune system allows the process to continue unchecked.
Findings from these studies can help identify and engage vulnerable targets using new drugs and immune-based therapies. Resulting treatments will be validated first in patients with high-risk disease, then in all myeloma patients.
The results are just out: a first-in-human Phase I study of multiple myeloma patients in which expanded cord blood-derived natural killer cells were combined with stem cell transplantation and high-dose chemotherapy had little or none of the side effects seen with current treatments.
Learn more about the clinical study led by Elizabeth J. Shpall, M.D., professor of Stem Cell Transplantation and Cellular Therapy.
Immunotherapy — a treatment approach designed to boost the body’s natural defenses — will play a major role in the High-Risk Multiple Myeloma Moon Shot.
One project is identifying relevant peptides, which are short amino acid chains, for their ability to stimulate the immune system. The PR1 chain has already been identified as a promising target for flipping the immune switch against invading myeloma cells. Researchers are also learning how antigen byproducts from myeloma cell replication can suppress the immune system. Another project is determining the efficacy of natural killer cells that are collected from umbilical cord blood.
More than 11,000 patients die from multiple myeloma in the U.S. annually. When clinical trials related to these projects run their course, our teams expect to finally have the tools needed to lower that number dramatically. Ideally, these clinical answers will also have applications for the treatment of other blood-related cancers, like leukemia and lymphoma.
