MDACC 2018: What can we Learn From Basket Studies About Treating Rare Tumors?

Houston, Texas ( Clinical trials typically include patients with a specific cancer, likely as a result of different genetic mutations and using this population to evaluate the efficacy of different drugs. This type of design is known as an umbrella trial.

A new way to develop clinical trials is doing a basket trial, which includes a variety of patients of different types of cancers in different locations as a result of the same, single, specific genetic mutation and using this group to test a single drug targeted against the mechanism of that single mutation. The Rare Cancers Oncology Agnostic Research, (NCT02034110) or ROAR trial, is an international, multi-center, open-labeled phase 2 study of Dabrafenib plus Trametinib used to target 9 tumors with the BRAF V600E mutation. This trial has resulted in the FDA approval of drugs for Erdheim-Chester1 disease and anaplastic thyroid cancer2.

Dr. Subbiah then discussed the MD Anderson phase II trial investigating Pembrolizumab in treating several rare tumors, including medullary renal cell carcinoma, penile carcinoma and germ cell tumors (NCT02721732). There has been an accrual of evaluable patients in all three GU tumor categories with preliminary data suggestive of a response to treatment. The estimated study completion date is August 20, 2020. What do we need for the success of basket studies? More publicly available data and increased molecular characterization and sequencing of rare tumors. Basket trials are an exciting, promising approach to finding better treatment options for rare tumors and I anticipate an increased number of basket trials as sequencing and molecular characterization of tumors becomes more commonplace in the care of our cancer patients.

Presented by: Dr. Vivik Subbiah, MD. Assistant Professor of Investigational Cancer Therapeutics MD Anderson Cancer Center Houston, Texas

1. Diamond EL, Subbiah V, Lockhart AC, Blay JY, Puzanov I, Chau I, et al. Vemurafenib for BRAF V600-Mutant Erdheim-Chester Disease and Langerhans Cell Histiocytosis: Analysis of Data From the Histology-Independent, Phase 2, Open-label VE-BASKET Study. JAMA Oncol. 2018;4(3):384-8.
2. Subbiah V, Kreitman RJ, Wainberg ZA, Cho JY, Schellens JHM, Soria JC, et al. Dabrafenib and Trametinib Treatment in Patients With Locally Advanced or Metastatic BRAF V600-Mutant Anaplastic Thyroid Cancer. J Clin Oncol. 2018;36(1):7-13.

Written by Dr. Amy H. Lim, MD, PhD, Urologic Oncology Fellow with Dr. Ashish M. Kamat, MD, (@UroDocAsh), Professor of Urologic Oncology & Cancer Research, MD Anderson Cancer Center, Houston, TX at the 13th Update on the Management of Genitourinary Malignancies, The University of Texas (MDACC - MD Anderson Cancer Center) November 9-10, 2018, Dan L. Duncan Building, Houston, TX