The American Society of Clinical Oncology (ASCO) has named “Progress in Treating Rare Cancers” as the “Advance of the Year” in its annual Clinical Cancer Advances report released on Thursday.

According to the report, rare cancers account for about 20% of all cancers diagnosed annually in the United States. But progress in their treatment has not kept pace with the more common types of the cancer.

The report highlights treatment advances for five rare cancers. Many of these new treatments are targeted therapies.

1. Anaplastic thyroid carcinoma:  FDA approved the first treatment for this form of thyroid cancer in nearly 50 years, a targeted therapy combination of dabrafenib (Tafinlar) + trametinib (Mekinist) for patients with BRAF-mutated ATC. This approach produced tumor shrinkage in over two-thirds of study participants.
2. Desmoid tumors: Sorafenib (Nexavar) became the first treatment to improve progression-free survival for patients with this rare form of sarcoma.
3. Midgut neuroendocrine tumors: FDA approved 177Lu-Dotatate (Lutathera), which delivers targeted radiation to tumor cells, based on research showing it lowers the risk of disease progression or death by 79% for patients with advanced disease.
4. Uterine serous carcinoma: Trastuzumab (Herceptin) was shown to slow progression of HER2-positive uterine serous carcinoma, one of the most aggressive forms of endometrial cancer.
5. Tenosynovial giant cell tumor: Research identified the first promising therapy, pexidartinib, for this rare cancer of the joints, producing responses in nearly 40% of patients.

(Source: ASCO Press release)

Dr KK Aggarwal

Padma Shri Awardee

President Elect Confederation of Medical Associations in Asia and Oceania   (CMAAO)

Group Editor-in-Chief IJCP Publications

President Heart Care Foundation of India

Past National President IMA