1. Eastman, Peggy

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A new report from ASCO has named progress in the treatment of rare cancers as its major advance for 2019. "Clinical Cancer Advances 2019" is the 14th edition of ASCO's annual report on the most noteworthy progress in clinical oncology.

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For the first time, ASCO has also cited nine specific research priorities that need special focus in the future to make continuing progress in clinical oncology. ASCO chose to include these nine priorities in the report because they represent areas of vital unmet need or gaps in knowledge.


As it has done in previous reports on clinical cancer advances, ASCO stressed the importance of strong federal support for cancer research. Nearly one-third of the advances in the 2019 report received funding from the NIH and other federal agencies. According to ASCO's 2018 National Cancer Opinion Survey, 67 percent of Americans say the U.S. government should spend more money on finding effective treatments and cures for cancer, even if that means higher taxes or adding to the nation's deficit. The new report notes that after 10 years of "stagnant funding," Congress has passed four consecutive annual increases for the NIH and NCI.


But the report also states that "we're only now nearing pre-recession NCI funding levels." Indeed, there was a troubling 75 percent drop in the number of studies primarily funded by NIH presented at ASCO's annual meeting during the years 2008-2017.


Rare cancers are those that account for less than six of 100,000 diagnosed cancers; they account for 20 percent of all U.S. cancer cases each year. As previously reported in Oncology Times, the U.S. cancer mortality rate has continued to drop over the last 25 years, especially for the top four cancers: lung, breast, prostate, and colorectal. Progress has been slower against rarer cancers, in part because the small number of diagnoses makes clinical trials difficult and because such patients have fewer treatment options. But in the past year, according to the ASCO report, research and regulatory achievements against five rare cancers were especially noteworthy-which is why they were collectively chosen as the top advance for 2019.


The new ASCO report "reflects the impressive gains we've made in understanding these so-called 'orphan diseases' and in tailoring treatments to target their unique characteristics," said ASCO President Monica Bertagnolli, MD, FACS, FASCO, in her introductory message to the report. Progress against the five rare cancers highlighted in the new report represents achievements "that hold real promise for patients," added Bertagnolli, Chief of the Division of Surgical Oncology at Brigham and Women's Hospital and Professor of Surgery at Harvard Medical School.


The new ASCO report specifically cites research funded by the federal NIH as playing a significant role in progress against rare cancers. Three of the advances that collectively make up the advance of the year received federal funding. Among federal research initiatives that benefit patients with rare cancers are the following.


* The Cancer Genome Atlas (TCGA) is a federally funded project that has led to the characterization of a broad range of tumors, which in turn has led to effective targeting of activated pathways. The TCGA has catalogued key genomic changes in 33 types of cancer, of which 10 were rare cancers.


* The federally funded Molecular Analysis for Therapy Choice (MATCH) trial of precision medicine treats patients based on genomic changes in their tumors. More than 60 percent of enrolled subjects in MATCH have cancers other than the four most common types.


* The federally funded Dual Anti-CTLA-4 and Anti-PD-1 Blockade in Rare Tumors (DART) immunotherapy trial is investigating nivolumab and ipilimumab in patients with rare types of cancer who are usually ineligible for current immunotherapy trials. ASCO has closely followed progress in immunotherapy. Its "Clinical Cancer Advances 2018" report named adoptive cell immunotherapy as the 2018 advance of the year, specifically citing CAR T-cell therapy for its efficacy in young patients with acute lymphoblastic leukemia and in adults with lymphoma and multiple myeloma.



Research Priorities

Looking to the future, the new ASCO report on clinical cancer advances named the following research priorities as especially important in continuing to make strides against cancer.


1. Identify strategies that better predict patient response to immunotherapies.


2. Better define the patient populations that benefit from postoperative adjuvant therapy.


3. Translate innovations in cellular therapies to solid tumors.


4. Increase precision medicine research and treatment approaches in pediatric cancers.


5. Seek to achieve optimal care for older adults with cancer.


6. Increase equitable access to cancer clinical trials.


7. Reduce the long-term consequences of cancer treatment.


8. Reduce obesity and its impact on cancer incidence and outcomes. In a statement issued after release of the new ASCO report, Bertagnolli noted: "If current trends continue, it is estimated that obesity will lead to more than 500,000 additional cases of cancer each year in the U.S. by 2030." Unfortunately, she said, public awareness of the link between obesity and cancer risk is low. ASCO's 2018 National Cancer Opinion Survey found that, while 80 percent of Americans know that cigarette smoking is a risk for cancer, only 35 percent are aware of the link between obesity and cancer risk.


9. Identify strategies to detect and treat premalignant lesions.



"These priorities represent our vision for finding the next generation of cancer cures and reducing cancer's impact on patients' lives," said ASCO Senior Vice President and Chief Medical Officer Richard L. Schilsky, MD, FACP, FSCT, FASCO, who served as one of the report's executive editors. "From prevention through survivorship, these priorities are intended to identify areas where progress is most needed and most promising."


ASCO has been especially vocal about achieving equitable access to participation in clinical trials. As previously reported in Oncology Times, Bertagnolli recently commented on ASCO's "longstanding concerns about the underrepresentation of individuals from lower socioeconomic populations in cancer clinical trials and the access to treatment advances these trials may provide." Her comments came in reaction to a new American Cancer Society study showing a widening socioeconomic divide in U.S. cancer mortality.

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More Treatment Advances

In addition to naming its advance of the year and describing its nine priority research areas, the 2019 ASCO report highlights other advances in cancer treatment, diagnostics, and patient care. In the field of immunotherapy, for example, the report highlights research showing that checkpoint inhibitors have a valuable role as initial therapy for advanced non-small cell lung cancer (NSCLC); a combination regimen of nivolumab and ipilimumab boosts survival for patients with renal cell cancer, and has been established as a new standard; the treatment combination of nivolumab and ipilimumab can reduce brain metastases in patients with melanoma; and a new anti-PD-1 checkpoint inhibitor, cemiplimab, shows promise in treating metastatic or locally advanced cutaneous squamous cell carcinoma, a skin cancer with few treatment options.


Advances in targeted therapy highlighted in the new report include use of the EGFR inhibitor osimertinib as initial treatment of choice to delay progression in patients with NSCLC who have certain EGFR mutations, including those not responsive to older EGFR-directed therapies; and use of the protein-targeted therapy abemaciclib to delay progression in patients with previously treated metastatic hormone-receptor positive, HER2-negative breast cancer.

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Even as political volatility in Washington threatens sustained funding for cancer research, Bertagnolli is calling for strong, continued federal support.


"With U.S. cancer cases set to rise by roughly a third over the next decade, we must continue to advance research that saves lives," she commented in a statement on the new report. "Federal investment plays a key role in continuing progress-in rare and common cancers alike. We need to prioritize federal funding of cancer research in the years to come. Americans are counting on it."


Peggy Eastman is a contributing writer.


Five Rare Cancers in ASCO Report


1. Anaplastic thyroid carcinoma (ATC). The FDA approved the first treatment for this rare form of thyroid cancer in nearly 50 years. The new treatment is a targeted therapy combination of dabrafenib plus trametinib for patients with BRAF-mutated ATC. The combination treatment produced tumor shrinkage in more than two-thirds of patients in clinical trials.


2. Desmoid tumors. Sorafenib became the first treatment to improve progression-free survival in patients with this rare form of sarcoma.


3. Midgut neuroendocrine tumors. The FDA approved lutetium Lu 177 dotatate, a treatment that delivers targeted radiation to tumor cells. The new therapy, which binds to somatostatin receptors to deliver radiation directly into tumor cells, is based on research showing that it lowers the risk of disease progression or death by 79 percent for patients with this kind of rare cancer who have advanced disease.


4. Uterine serous carcinoma. The drug trastuzumab was shown to slow the progression of HER2-positive uterine serous carcinoma, one of the most aggressive forms of endometrial cancer.


5. Tenosynovial giant cell tumor. Research identified the first promising therapy, pexidartinib, for this rare cancer of the joints. This promising treatment, a colony-stimulating factor 1R inhibitor, produced responses in nearly 40 percent (39.3%) of patients who received it.