Community and the Care of Patients With Rare Cancers

Richard D. Carvajal, MD

Director, Experimental Therapeutics and Melanoma Service

New York-Presbyterian Hospital/ Columbia University Medical Center

New York, NY

At a recent patient symposium, I had the opportunity to speak with a number of individuals affected by uveal melanoma, a rare and aggressive form of the disease that is clinically and molecularly distinct from its more common cutaneous counterpart. Unlike for advanced cutaneous melanoma, a disease for which there have been dramatic advances in treatment options, there is no FDA approved effective treatment option for metastatic uveal melanoma. Without the time pressures and relative formality associated with clinic, the discussions I had with the attendees focused less on clinical management options, and rather on the experience of being a patient with uveal melanoma or the challenges of caring for an individual with this disease. Fear and a sense of isolation are common themes raised by those affected by cancer; however, these emotions seemed amplified and were echoed repeatedly by those present at the symposium.

Although these emotions were partially allayed by the presentations by the researchers who discussed recent advances in our understanding and treatment of uveal melanoma, as well as by the nurses and social workers who spoke about managing the impact of this disease upon the patient and family, it was the opportunity for patients and family members to share their own experiences with the other symposium participants that was most effective in alleviating the fear and feeling of isolation. It was the knowledge that others were going through similar challenges to what they themselves were facing and the sense of community that rapidly developed over that weekend that the participants most appreciated.

The acknowledgment of the importance of community by those affected by rare cancers is reflected in the number of patient-established rare cancer support groups. For uveal melanoma, there is the Melanoma Research Foundation CURE OM (Community United for Research and Education of Ocular Melanoma) initiative, the Ocular Melanoma Foundation, OcuMelUK, the Eye Cancer Network, and others. For gastrointestinal stromal tumors, there is the Life Raft Group, the GIST Cancer Awareness Foundation, GIST Support UK, and GIST Support International. For neuroendocrine tumors, there is the NET Patient Foundation, the Carcinoid Cancer Foundation, and the Caring for Carcinoid Foundation.

The groups listed here are just examples of some of the many support groups available for patients and family members facing these and other rare tumors. These groups allow individuals to learn from one another, help each other navigate the complexities of our health system, identify potential treatment options, and provide emotional support. While similar organizations exist for those affected by more common cancers, the importance of these groups for those with rare cancers is augmented by the limited awareness by others regarding their diseases, the frequently more limited treatment options, and their own knowledge of the relatively limited funding and focus by the research community on finding a cure for their diseases.

The creation of rare cancer consortiums within the medical community to foster research in and enhance awareness of these malignancies, in many cases, has lagged behind the development of these patient and caretaker communities. While groups such as SARC, the CanLiv International Hepatobiliary Cancers Research Consortium, and the Lustgarten Foundation Pancreatic Cancer Research Consortium have successfully led significant research efforts in the fields of sarcoma, hepatobiliary malignancies, and pancreatic cancer, respectively, similar efforts either do not exist or are in their infancy for other rare cancers, such as adrenocortical cancer, small bowel cancers, thymomas, and many others.

Over the past several years, the uveal melanoma research community has been brought together due to the efforts of groups such as CURE OM and the International Rare Cancer Initiative (IRCI). CURE OM is the Melanoma Research Foundation’s initiative to increase awareness, education, treatment and research funding for uveal melanoma, and aims to meet these goals by providing grants, sponsoring biannual international scientific meetings, and forging partnerships with leading academic and industry organizations. Thus far, CURE OM has provided over $800,000 in grants specifically for uveal melanoma research.

IRCI is a joint initiative between the National Institute for Health Research (NIHR) Cancer Research Network (NCRN) and Cancer Research UK (CR-UK) in the United Kingdom, the National Cancer Institute (NCI) in the United States, and European Organisation for Research and Treatment of Cancer (EORTC), and the Institut National Du Cancer (INCA) in France. The aim of this initiative is to facilitate the development of international clinical trials for patients with rare cancers in order to boost the progress of new treatments for these patients. Uveal melanoma is one of the nine cancers initially targeted for the IRCI efforts, and the IRCI uveal melanoma committee meets twice yearly to coordinate international research efforts in this disease.

As defined by the National Institutes of Health (NIH), each rare cancer affects fewer than 200,000 individuals in the United States at any one time; however, approximately half of all individuals diagnosed with malignancy are diagnosed with a rare cancer. Outcomes for patients with these rare cancers are generally inferior to those diagnosed with more common cancers. Thus, it is critical that research aimed specifically at understanding and improving outcomes for patients affected by each rare cancer is supported and promoted. It is equally critical that those of us whose research focuses on rare cancers learn from our patients who have long recognized the importance of community.

It is only through the development of effective collaborations between clinicians and researchers focused on rare cancers within the United States and internationally, industrial partners, and those affected by rare cancers that the progress toward our goal of cure for these diseases will be most rapidly achieved.