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Local surgical excision remains the preferred therapy for periocular and facial basal cell carcinoma. However, interest is growing in nonsurgical drug therapy for locally advanced or metastatic basal cell carcinoma.

There have been promising results in a Phase II trial showing that gene transfer treatment of patients with heart failure and reduced ejection fraction was safe and successful in over 90% of patients.

Chimeric antigen receptor-modified T-cell therapies have demonstrated durable complete responses for patients with relapsed/refractory B-cell acute lymphoblastic leukemia; however, several questions remain regarding their optimal use and applicability outside of this disease.

The FDA has approved Captisol-enabled melphalan (Evomela) as a high-dose conditioning treatment for use in patients with multiple myeloma prior to autologous stem cell transplantation, as well as for the palliative treatment of patients with myeloma for whom oral therapy is not appropriate.

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to the investigational human IgG1 monoclonal antibody durvalumab (MEDI4736) for patients with inoperable or metastatic urothelial bladder cancer that is programmed-cell-death-ligand 1 (PD-L1)-positive and refractory to platinum-based treatment.

In a phase IIa study of patients with neovascular age-related macular degeneration, a single subretinal injection of rAAV.sFlt-1 gene therapy demonstrated acceptable safety, but not a complete or durable anti-VEGF response. Additional preclinical research is under way.

The second interim analysis of the phase III METEOR trial has revealed a statistically significant improvement in overall survival with cabozantinib (Cometriq) versus everolimus as a treatment for patients with advanced renal cell carcinoma following progression on one prior therapy.

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for BI 1482694, which is an investigational third-generation, epidermal growth factor receptor (EGFR), mutant-specific tyrosine kinase inhibitor (TKI) for patients with non-small cell lung cancer (NSCLC).