By Peter J. O’Dwyer, MD (left)
and Mitchell D. Schnall, MD, PhD

It is a time of change in our clinical cancer research environment. Recent leadership changes at NCI and NIH involve researchers with strong track records of support for clinical and translational research; they are looking at our landscape and reimagining how we might increase the efficiency and reach of current studies, as well as develop new studies in structures designed to accelerate progress.

Though short on specifics at this point, the reassessment of how we get new treatments to patients faster and more effectively is a healthy development for a system that for many years has seemed mired in process and regulatory complexity. The various participants in this research, ranging from those of us who design and implement the trials, to NCI, NIH, FDA, pharma companies, and—most important—patients and advocates will all have an impact in what novel systems may emerge. Not surprisingly, we are embracing this challenge with enthusiasm.

As examples of the government’s commitment to these goals, the White House’s Office of Science and Technology Policy (OSTP) has convened a forum to identify barriers, and NCI, FDA, and cooperative group leaders have been brought together in the NCI Clinical Trials Innovation Unit (CTIU). The CTIU is a vehicle to explore novel ideas and technologies, and to accelerate progress in the field. In addition, NCI Director Dr. Kim Rathmell has appointed Dr. M. Shaalan Beg as Special Assistant to the Director to bring his expertise in clinical trial methodologies to bear nationally. Weaving these and other threads together into a more dynamic system will take time, but early opportunities among all the groups should start to emerge in months—not years.

The OSTP forum met on June 26 in Washington, DC to address advancing clinical trials as a component of cancer care to which patients should have access. Two introductory sessions addressed aspects of trial promotion. A set of brief talks on People-Centered Clinical Trial Innovation highlighted recent progress in assuring the interoperability of electronic health record programs (a requirement for data analysis across health systems), ARPA-H funding priorities in this area, and the patient view that prioritization of clinical trials provides therapy options not otherwise available.

Improving Clinical Trial Access and Participation was a session that NIH Director Dr. Monica Bertagnolli introduced by emphasizing the importance of trial access for patients in underserved areas, such as American Indian reservations in the Northern Plains. Her remarks were followed by those of FDA Commissioner Dr. Robert Califf, who emphasized that to fully understand drug activity and variability, efforts must be made toward comprehensive study across diverse populations. A patient advocate and clinical trial participant, Ms. Chaundra Bishop, shared both the challenges and the opportunities that trial participation brought her. Each one of these positions has implications and opportunities for ECOG-ACRIN research, and as additional information emerges, we will continue to update you in this newsletter.

The remarkable results of E1910, presented by Dr. Mark Litzow (Mayo Clinic) at ASH 2023, were published in the New England Journal of Medicine on July 25. These results led to the FDA approval of blinatumomab for adults with newly diagnosed BCR-ABL1-negative B-lineage acute lymphoblastic leukemia. Eligible patients had achieved complete remission with standard therapy and were MRD-negative. At 3 years of follow-up, 85% of the patients who went on to receive additional standard consolidation chemotherapy plus experimental blinatumomab were alive, compared to 68% of those who received chemotherapy only. The survival curves suggest a durable effect in the patients who received blinatumomab. The contribution of all of the adult cooperative groups is recognized in this practice-changing advance. Blinatumomab is a BiTE, a bispecific T-cell engager, that approximates T-cells to CD28-expressing leukemia cells. BiTEs have striking activity in other hematological malignancies, including myeloma, and show great therapeutic promise.

Read the August 2024 issue here.