Rare cancers contribute to 30 percent of deaths from the disease. Large randomized trials are impractical because of the low incidence of rare cancers.

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Small clinical trials or case series can be used to provide proof and help identify targets for new treatments.
He wrote that ovarian cancer was a good example of how approaches to rare cancer treatment needed to evolve.
“One feature of common cancers like breast cancer or bowel cancer, where survival has been significantly altered over recent years, is that there is a screening test for early detection. The heterogeneity of ovarian cancer suggests that a population screening test based on a panel of biomarkers will be difficult to achieve.”
“Also, the symptoms of ovarian cancer are non-specific — such as bloating; abdominal, pelvic or back pain; bowel menstrual irregularities; and fatigue — so that diagnosis is often late when the disease has spread beyond the ovaries.”
However, he noted that there are similarities between ovarian subtypes and other cancers. “These will be helpful in identifying targets for new treatments. For example, high-grade serous carcinoma of the ovary shares similarities with triple-negative breast cancers.” Drugs used for metastatic breast cancer are now being trialed in patients with ovarian cancer. Professor Olver also suggested using small efficacy trials or case series to provide proof of principle for targeted therapies.
“Reassessing research into cancers or cancer subtypes classified as histologically rare may involve finding molecular and genetic similarities across a range of cancers, which suggest that a targeted therapy in one may be successfully trialed in another,” Professor Olver concluded.
Source-Medindia
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