Individually, sarcomas, neuroendocrine tumors, and uveal melanoma are rare cancers. But taken together, rare cancers account for 50% of all cancer diagnoses. At the Herbert Irving Comprehensive Cancer Center at NewYork-Presbyterian/Columbia University Irving Medical Center, researchers are at the forefront of investigations and treatment of rare cancers are leading initiatives in rare cancers for cooperative study groups.
Collectively, rare cancers account for half of all cancer diagnoses.
Clinical trials are critical to gaining a broader understanding of and making headway against these challenging tumors. At NewYork-Presbyterian/
Uterine Leiomyosarcoma
With funding from the National Cancer Institute, researchers are assessing olaparib plus temozolomide in women who have uterine leiomyosarcoma with BRCA-like alterations. Preclinical studies have shown that these two drugs work synergistically to inhibit cancer growth.
Advanced Sarcomas
Investigators are evaluating the immunotherapy drug APX005M, a CD40 agonistic monoclonal antibody, in combination with doxorubicin to treat patients with advanced sarcomas. They believe that doxorubicin, the current standard of care for most advanced sarcomas, could be more effective when combined with APX005M.
Alveolar Soft Part Sarcoma
In a phase II clinical trial, researchers are assessing the use of atezolizumab immunotherapy to treat patients with inoperable metastatic alveolar soft part sarcoma. Atezolizumab is FDA-approved for lung, breast, and bladder cancers; its use in this study is investigational.
Neuroendocrine Tumors
In a phase II clinical trial, investigators are assessing lanreotide for pheochromocytomas and paragangliomas. Lanreotide is FDA-approved to treat neuroendocrine tumors, but patients with pheochromocytomas and paragangliomas—rare and very special types of neuroendocrine tumors—were not enrolled in the registration trials.
Ocular Melanoma
Columbia University investigators are evaluating the use of ipilimumab and nivolumab immunotherapy after treatment of primary uveal melanoma—a disease with a 50% risk of metastasis—to reduce the chance of disease recurrence and increase the likelihood of a cure. In addition, investigators are now evaluating the use of PLX2853, a novel bromodomain inhibitor, for the treatment of metastatic uveal melanoma—a disease with no known effective systemic therapy.