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Society leaders: 2017 likely to be time of ‘unprecedented’ advances in hematology, oncology
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This calendar year could be a pivotal one for oncology and hematology research, drug approvals and clinical trial results.
In 2016, several new immunotherapies, chemotherapies and targeted therapies were approved for cancer indications. This includes atezolizumab (Tecentriq, Genentech), the first drug approved for bladder cancer in 3 decades; venetoclax (Venclexta; AbbVie, Genentech), a targeted therapy for patients with chronic lymphocytic leukemia and 17p deletion; and eribulin (Halaven, Eisai), the first drug approved for liposarcoma, among others.
This year, regulatory approval is sought for immunotherapy candidates from Kite Pharma and Novartis. Kite Pharma has filed KTE-C19 therapy for patients with diffuse large B-cell lymphoma for FDA evaluation, and Novartis plans on submitting CTL019 therapy for acute lymphoblastic leukemia in the coming months.
HemOnc Today asked some of the nation’s leaders in cancer research to weigh in on where they see the greatest breakthroughs in 2017. Several areas of promise include genomic profiling, immunotherapy, gene editing, and personalized cancer screening and prevention. Experts also discussed the need for continued federal funding, greater enrollment of patients onto clinical trials, and efforts to reduce the costs of potentially lifesaving therapies.
Kenneth C. Anderson, MD, president of ASH, director of the Lebow Institute for Myeloma Therapeutics and the Jerome Lipper Myeloma Center at Dana-Farber Cancer Institute
It is an unprecedented time in our field and medical practice is being transformed as we speak. The most exciting areas of research that have been identified in our ASH agenda are multiple. One area is genomic profiling and the ability to precisely understand the genomic characterization of disease so that we can further refine pathogenesis in the laboratory, better understand the heterogeneity of disease, better define prognosis and, ultimately, deliver on the promise of precision medicine in the clinic.
Another exciting area of research is in immunotherapy. There has been tremendous progress in checkpoint inhibitors. In hematology, we also have immunomodulatory drugs, monoclonal antibodies, vaccine initiatives and cellular therapies. Not only single-agent immune therapies, but a combination of immunotherapies used earlier in the disease course in blood cancers have tremendous potential to transform practice.
A third area that is very exciting is genome editing and gene therapy, which has been a long time coming, but there is still great excitement. Sickle cell anemia is a disease caused by a single abnormality in the hemoglobin gene. Wouldn't it be wonderful if we could edit out the abnormal gene and repair the deficit that causes the disease? In the past, this has been “Star Wars”–type thinking, but that is no longer the case. With the availability of CRISPR technology, it appears possible to edit out an abnormal gene in stem cells and repair the defect in sickle cell disease.
Another area of exciting research includes stem cell biology, not only for stem cell transplantation for hematologic malignancies, but also for other diseases including cardiovascular, neurologic and others. There is excitement around epigenetics, and in terms of practice, we are starting to better understand new methods of dealing with complications in our field, such as thromboembolism, so that some of the precision medicine ideas extend not only to diseases such as sickle cell anemia or hematologic malignancies, but also to understand even how we can avoid complications, such as venous thromboembolism. These are the areas among the most exciting in hematology today.
Nancy E. Davidson, MD, president of American Association for Cancer Research and director of University of Pittsburgh Cancer Institute and UPMC Cancer Center
This year will be exciting for my organization, the American Association for Cancer Research — which is celebrating its 110th anniversary — and for the cancer research community, because there are unprecedented opportunities to make breakthroughs in so many areas of cancer research. In the past few years, we have harnessed our understanding of the immune system to develop immunotherapeutics that are being used to successfully treat patients with a variety of cancers, but we have more work to do. This year, I expect advances in our understanding of why only a fraction of patients respond to these groundbreaking treatments and why many patients who respond develop resistance later. I also expect new immunotherapeutics to emerge that utilize the immune system in new ways. We will see a continuing refinement of the way we use precision medicine to select targeted therapies, improve the way that we apply immunotherapy, and refine surgical and radiotherapeutic techniques.
I believe that we are also going to see the impact of precision medicine in improving early detection and cancer prevention strategies, and that we will make significant progress in realizing the concept of personalized screening and cancer prevention. Studies on epigenetic changes that influence cancer development are being embraced by many groups, and we will, hopefully, see this line of research bearing new fruit in terms of clinical applicability.
Given the number of clinical trials in progress, we are likely to see practice-changing data that will improve patient outcomes and quality of life. However, continued progress is going to require sustained federal commitment to the research agenda, and it will be key for the entire community to come together this year to ensure that policymakers and the administration make lifesaving cancer research a national priority.
Jeff Vacirca, MD, FACP, president of Community Oncology Alliance, chief executive officer of New York Cancer Specialists
I expect there to be a continued increase in utilization of immuno-oncology in multiple other tumor types. However, I also expect a realization that, although immuno-oncology has been a tremendous step forward in our ability to treat multiple cancers, it is, unfortunately, not the cure. For those of us in clinical practice, I hope to see the barriers broken down between the newer tests that determine potential for targeted therapies looking at tumor DNA mutations, amplifications, and so on, and a model for which we can pay for these treatments that could be groundbreaking for some patients. We have great precision testing abilities with next-generation sequencing that show the potential for all these drugs to work, but no way to pay for it. It is on everyone — insurers, Medicare, and the FDA, just to name a few — to fix that. Finally, if we truly want research to move the needle forward on fighting cancer, the bottom line is that we need to enroll more patients in clinical trials. We have an unbelievable amount of trials that never get populated because a system has not been set up to get patients into them. Every practice has patients that qualify for clinical trials, but we don’t have a system that makes that happen.
Daniel F. Hayes, MD, FASCO, president of ASCO, professor of internal medicine, Stuart B. Padnos professor in breast cancer, and clinical director of the Breast Oncology Program at University of Michigan Comprehensive Cancer Center
Immunotherapy, ASCO’s “Cancer Advance of the Year,” continues to be an incredibly promising area of cancer research. However, immunotherapy is still in its infancy. We expect that this year will continue to yield insights into patient and cancer characteristics — ie, biomarkers — that might predict whether immunotherapy will work in one patient but not in another. In addition, research into immunotherapy combination treatments will continue to expand, hitting the two known immune checkpoints; there is some early evidence that this may be a promising strategy. Further, we look forward to translation of ongoing basic immunologic science that has identified other checkpoints that may serve as future therapeutic targets. Also, the use of chimeric antigen receptor T cells in selected malignancies — especially those of hematologic origin — is a particularly exciting area of research that is just beginning to pay off.
We continue to be enthusiastic about advances in precision medicine as applied to oncology. However, this strategy is not applicable to all patients. We anticipate increasing knowledge about which biomarker changes — whether at the genetic, transcriptional or protein levels — might help us select the right drug for the right patient. In this regard, we are excited by the first clinical trial conducted by ASCO: The Targeted Agent and Profiling Utilization Registry (TAPUR) study. TAPUR was initiated to address whether targeted drugs approved for one type of cancer should be prescribed off-label for another. This issue raises significant challenges in precision medicine, including a lack of data regarding the safety and efficacy of such treatments and poor understanding of the optimal tests that might be used to match a targeted drug with a tumor’s biology. TAPUR is addressing issues by making targeted drugs available to participants at no cost and by creating a registry to monitor which tests, and the associated results, were used to “match” the patients who are entered and to record and share key clinical outcomes. We’ve already enrolled more than 140 patients, and will continue to expand our research this year. – by Chuck Gormley