Making Progress in Rare Malignancies

| Comments (0)

We often hear about progress in common tumors such as breast and colon cancer, but are making slower progress in many rare malignancies. There are many reasons for this discrepancy, including lower levels of research funding, less public awareness and, importantly, difficulties in finding enough patients with a given rare tumor to perform rigorous scientific studies.  

Adenocarcinomas of the small bowel are an excellent example of this, as there are only an estimated 2,000 cases a year compared to the 150,000 cases of colon cancer diagnosed per year. However, concerted efforts by Dr. Michael Overman in the Department of Gastrointestinal Medical Oncology and Dr. George Chang in the Department of Surgical Oncology at M. D. Anderson have resulted in a better understanding of the disease.

They reported (#4596) a better method to judge the risk of recurrence for patients who have their small bowel cancer resected. By analyzing outcomes of 1,991 patients with adenocarcinoma of the small bowel from a national database, they were able to distinguish patients at moderate risk (42%) and high risk (63%) of the cancer returning in five years. Similarly, if enough lymph nodes were taken at the time of surgery and analyzed, those patients with no cancer in the lymph nodes had excellent outcomes (only a 12% chance of recurrence). These results will be very informative in discussing the role of chemotherapy after surgical resection of localized small bowel adenocarcinoma.

Dr. Overman and colleagues recently reported one of the only modern trials of small bowel adenocarcinoma in the Journal of Clinical Oncology (J Clin Oncol. 2009 Jun 1;27(16):2598-603). Compared to older chemotherapies, this regimen resulted in the best reported outcome to date by using a combination of capecitabine, oxaliplatin and the anti-angiogenic agent bevacizumab. Future studies in this disease plan to evaluate antibodies to the epidermal growth factor receptor.  

An article written by Amy Marcus, a Pulitzer Prize winning reporter at The Wall Street Journal, which accompanies Dr. Overman's study gives one perspective on how to move forward (Journal of Clinical Oncology, Vol 27, No 16 (June 1), 2009: pp. 2575-2577). She suggests that the key component to moving research forward in rare malignancies is the existence of active patient advocacy for the disease. Indeed, she offers examples of how individual patients advocated for (and participated in) research of their rare tumors and the progress they are able to make.   

How, then, can we make progress in these rare diseases? To paraphrase Margaret Mead: Never doubt that a small group of patients, scientists and advocates can change the world of rare malignancies. Indeed, that is the only thing that ever has.

Scott Kopetz, M.D., Assistant Professor, Department of Gastrointestinal Medical Oncology, from ASCO 2009

Leave a comment


Connect on social media

Sign In