Blood cancers make up a large portion of cancer types—these can include lymphomas, myelomas, leukemias, and myelodysplastic syndromes (MDS). There are over 100 different types of blood cancers, highlighting the importance of getting a proper diagnosis and individualized treatment plan into place.
Because of the large number of blood cancer types and the increasing complexity of diseases, finding breakthrough therapies has proven to be increasingly difficult, yet increasingly important. There are a variety of existing treatment options available for blood cancers such as chemotherapy, radiology, surgery, stem cell transplants, and CAR-T (chimeric antigen receptor T-cell) therapy. These therapies, or a combination, are used by hematology-oncology (hem-onc) professionals to treat patients based on their varying diseases and individual needs. While some of these treatments, like chemotherapy, have been around for a while, others, like CAR-T, are relatively newer to the hem-onc landscape.
CAR-T, while being slightly more recent than treatments such as chemo, has proven to be successful in treating blood cancers in over 30,000 patients to date. At a high level, CAR-T works by collecting T-cells from the patient, genetically modifying them, and then infusing them back into the patient to bind to and attack the existing cancer cells. While this therapy is typically used in blood cancers, it’s being studied to potentially be used to treat other forms of cancer.
Hem-onc patients are generally known to be at a greater risk for developing a secondary primary malignancy (or a second cancer that develops in a person already diagnosed with and treated for cancer), which is partially attributable to their previous exposure to cytotoxic chemotherapy. Recently, the FDA had become aware of and further investigated 22 patients who received CAR-T therapy and subsequently developed T-cell lymphomas1.
In partnership with Eddie Cliff, MBBS, MPH (Peter MacCallum Cancer Centre), David Russler-Germain, MD, PhD (Siteman Cancer Center, WUSTL), Kai Rejeski, MD (LMU University Hospital, Munich), and Aaron Kesselheim, MD, JD, MPH (Harvard Medical School), Medidata and collaborators sought to further quantify this incidence of developing secondary primary malignancies. Medidata leveraged aggregated data from trials run on the Medidata platform and found that of the >1500 patients analyzed, the rate at which patients developed secondary primary malignancies was 2.45% per patient-year. This is comparable to previous data on lymphoma patients treated with other cell therapies, like autologous or allogeneic stem cell transplants.
While it’s important for the FDA, physicians, and patients to understand the risk of any cancer treatment, it’s equally important that any risk is contextualized and weighed against the potential positive outcomes and alternative treatment options. CAR-T has proven to be a life-saving therapy for many individuals who have been diagnosed with blood cancer. While the risk of developing a secondary malignancy exists, our collaborative research demonstrates that it puts patients at no greater risk than other cellular therapies.
The findings of this research are incredibly important in understanding the risk vs benefit of CAR-T therapy in comparison to other therapies used to treat blood cancers. The fight against these unfortunately common cancers is not over, and having a variety of life-saving treatment options is crucial.
“The Medidata Research Alliance’s core mission is to advance medical breakthroughs for patients through the power of data and collaborations with academic physician-scientists. It’s through these partnerships that we can bridge insights from these aggregated data into clinical practice. This research demonstrates the importance of understanding the risks and benefits of therapies within the appropriate context.”
– Sheila Diamond, MS, CGC, Director, Scientific Engagement, Medidata
Medidata is proud to co-present this collaborative research at the 66th American Hematology Society Annual Meeting in December this year. ASH will take place in San Diego, CA from December 7-10. If you would like to set up a meeting, please contact us here.
References
-
-
- Center for Biologics Evaluation and Research. “FDA Investigating Serious Risk of T-Cell Malignancy.” U.S. Food and Drug Administration, FDA, www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-serious-risk-t-cell-malignancy-following-bcma-directed-or-cd19-directed-autologous. Accessed 29 Oct. 2024.
-