Overcoming the Challenges of Rare Cancers

People with rare cancers need information about their disease and treatment options, frank and transparent descriptions of the limitations to those options, and help understanding and navigating how their care will be organized, step by step. They also need better access to clinical experts, including travel and housing support, as well as referrals to psychosocial care services, advocacy groups such as the Pancreatic Cancer Action Network (PANCAN), and research networks, Houlihan said. Second opinions and specialized clinical centers of excellence such as Penn Medicine’s Neuroendocrine Cancer Program are crucially important, she added.

Clinicians, in turn, need better knowledge about these cancers, clinical networks for rare cancer specialists, and more research into how best to address the needs of these patients. Multidisciplinary professional education in rare cancers and patient support services will be key in improving patients’ quality of life and other outcomes.

New approaches to research are also key. “Basket” trials are a type of clinical trial that tests a new drug or treatment on patients with different types of cancer, but whose diseases share a common gene mutation or molecular target.

Coordinated multicenter research efforts are underway that will advance our understanding and treatment of these cancers. National and international research collaborations increase the number of patients participating in clinical trials and improve researchers’ access to data, Houlihan pointed out. The US National Institutes of Health is funding numerous research initiatives for rare cancers, and the National Cancer Institute is building a national Rare Tumor Patient Engagement Network for coordinated clinical trials and is implementing President Biden’s Moonshot initiative to cure rare malignancies.

Sequencing and characterization of a broad range of tumors’ genomes by The Cancer Genome Atlas (TCGA) project has yielded targeted treatment options for neglected cancer types. The National Comprehensive Cancer Network (NCCN) is expanding its clinical practice guidelines to include rare cancers, as well.

In 1 year alone, 2019, combined treatment with dabrafenib (Tafinlar) plus trametinib (Mekinist) for patients with BRAF-mutated anaplastic thyroid carcinoma was shown to shrink tumors in 66% of patients, sorafenib (Nexavar) offered improved progression-free survival to patients with rare desmoid sarcomas, trastuzumab (Herceptin) was found to slow HER2-positive uterine serous carcinoma tumor progression, and targeted ¹⁷⁷Lu-Dotatate radiation reduced disease progression and mortality risk by 79% for patients with somatostatin receptor-positive midgut tumors, Houlihan said.

“Positive outcomes have been demonstrated with basic oncology strategies of standardizing care, creating networks to improve access to expertise, establishing research initiatives, and providing support for individuals within their unique disease and social [situations],” Houlihan said. “Resources exist for patients and health care providers to overcome the challenges of a rare cancer diagnosis and nurses play a key role in assuring quality of care for these patients.

“Use your influence to support access to clinical trials for all patients and to advocate for greater funding for cancer clinical trials,” she concluded.

Disclosures: The presenter had no financial relationships to disclose.

Reference

Houlihan N. Caring for patients with uncommon diagnoses. Presented at: ONS Bridge 2023; September 12-14, 2023.