The combination of trametinib (Mekinist) and dabrafenib (Tafinlar) shows a strong and durable response in a single-arm phase II study of patients with anaplastic thyroid cancer (ATC) with BRAF V600E mutation and no satisfactory locoregional treatment options. Based on the results, the therapy is approved for the treatment of patients with locally advanced or metastatic ATC. The combination is the first therapy approved for ATC in nearly 50 years. Combination treatment with trametinib and dabrafenib become the standard of care for patients with ATC with tumors that have a BRAF mutation.

Two clinical trials produce positive data on a new medicine called larotrectinib (Viktravi), which selectively targets a genomic abnormality present in a variety of solid tumor cancers, called tropomyosin receptor kinase (TRK) gene fusion. This abnormality occurs in approximately 0.5% to 1% of many common cancers, but it is present in as many as 90% of certain rare, hard-to-treat cancers, such as salivary gland cancer, pediatric breast cancer, and infantile fibrosarcoma. In one early clinical trial, larotrectinib yields strong, long-lasting tumor shrinkage across multiple types of adult and pediatric cancers with TRK fusions, regardless of the tumor histology or location. A second trial focuses only on pediatric cancers and found that the treatment causes tumor shrinkage in nearly all patients with TRK fusions – becoming one of the earliest studies to demonstrate how precision medicine treatments can benefit patients with pediatric cancer. Collectively, these findings lead to the accelerated FDA approval of larotrectinib for adult and pediatric patients with advanced or inoperable solid tumor cancers with TRK fusions.

The 2019 FDA approval of the immunotherapy atezolizumab in combination with chemotherapy represents a major advance in the treatment of extensive small cell lung cancer. The approval was based on the results of the IMpower 133 study, which showed significantly longer survival with the addition of atezolizumab to carboplatin and etoposide chemotherapy. The combination therapy has become the standard of care for extensive-stage small cell lung cancer in the United States. 

Abemaciclib (Verzenio) is a type of targeted therapy that inhibits cyclin-dependent kinases (CDK) 4 and 6, which play roles in breast carcinogenesis and endocrine therapy resistance. In two studies, treatment with abemaciclib — either alone versus endocrine therapy or in addition to nonsteroidal endocrine therapy — results in improved progression-free survival for patients with metastatic hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer. These findings lead to the approval of abemaciclib as the first CDK 4/6 inhibitor for use as monotherapy in patients previously treated with endocrine therapy and chemotherapy in the metastatic setting.

A major study of women with early-stage HER2-positive breast cancer shows that adjuvant treatment with two drugs that target the HER2 protein – pertuzumab (Perjeta) and trastuzumab (Herceptin) – together with standard chemotherapy substantially reduces the risk of local or regional invasive breast cancer recurrence, distant recurrence, invasive breast cancer in the opposite breast, or death from any cause. in women with breast cancer that overproduces the HER2 protein. Importantly, the dual-targeted treatment does not increase in the rate of heart problems, a major concern with HER2-targeted therapy. The FDA approves pertuzumab for use in combination with trastuzumab and chemotherapy as adjuvant treatment of patients with HER2-positive early breast cancer at high risk of recurrence.

FDA grants accelerated approval for combined modality therapy with trametinib (Mekinist) and dabrafenib (Tafinlar) in patients with advanced or inoperable melanoma that harbor certain BRAF gene mutations. The approval is based on studies demonstrating that the new regimen leads to long-lasting responses in most patients. The two drugs target different molecules in a pathway involved in melanoma growth.