Small cell carcinoma of the ovary, hypercalcemic type (SCCOHT) is a rare but very aggressive type of ovarian cancer affecting young women. Conventional chemotherapy is seldom effective in curing SCCOHT and its rarity has hampered the development of novel effective treatments. In 2014, our team discovered that SCCOHT is almost always caused by mutations in a gene called SMARCA4. Our follow-up studies have uncovered several clinical drugs that may be used to suppress the growth of SCCOHT cancer cells. While these drugs may provide options for treating SCCOHT patients, drug resistance is inevitable when they are used alone. Moreover, the rarity of this disease limits the feasibility of clinical trials testing all possible options. We are currently working to identify the most robust approaches, such as optimal treatment combinations, to be prioritized for clinical testing. This will improve the success rate of translating research findings into effective treatments for SCCOHT patients. It may also benefit patients affected by more common cancers that are caused by SMARCA4 mutations in various organs, such as the lung, brain, pancreas, and skin.