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Sylvester Study Contributed to FDA Approval of Targeted Therapy for Lung Cancer

A patient was diagnosed with non-small cell lung cancer (NSCLC) five years ago and was told her tumor had a rare genetic rearrangement known as a RET fusion, which affects 2% of people with NSCLC and is also found in other types of malignancies. The patient responded well to chemotherapy for three years but then had a recurrence.

Gilberto Lopes
Gilberto de Lima Lopes Jr., M.D., M.B.A.

That’s when she discovered a clinical trial at Sylvester Comprehensive Cancer Center, part of the University of Miami Health System, offering targeted therapy for the first time to people with RET fusion cancers. The patient enrolled in the trial of pralsetinib at Sylvester. Leading the Sylvester site for the previous ARROW study that contributed to the FDA approval of pralsetinib in September was Gilberto de Lima Lopes Jr., M.D., M.B.A., professor of clinical medicine and co-leader of the Lung Cancer Site Disease Group at Sylvester.

“Until recently, patients with lung cancer and RET fusions, a type of molecular alteration, had no specific targeted options for treatment,” Dr. Lopes said. “Patients would usually receive chemotherapy. This trial showed that patients with RET fusions respond to pralsetinib.”

Pralsetinib (brand name Gavreto) is FDA approved for adults with metastatic RET fusion-positive NSCLC. The agency also approved the companion Oncomine Dx Target test to detect RET fusions. In a practical sense, Dr. Lopes said the approved RET fusion test is easy to use, and many platforms already test for RET fusions in common practice.

Dr. Lopes and colleagues conducted the ARROW clinical trial in 87 people with NSCLC and RET fusion gene alterations who were previously treated with standard chemotherapy. The results showed 57% of patients achieved the main outcome of overall response rate. Furthermore, among the patients who responded, 80% experienced effects lasting six months or longer.

The researchers in this multicenter trial also evaluated pralsetinib in 27 patients who had not received previous treatment. They found 70% achieved an overall response rate and 58% of people who responded had an effect lasting at least six months.

“This drug has really given hope for patients with this rare fusion mutation seen in lung cancer and thyroid cancers,” said Estelamari Rodriguez, M.D., M.P.H., associate director for community outreach-thoracic oncology at Sylvester.

“As an NCI-designated cancer center, we have access to early stage clinical trials and as such have more options for patients,” Dr. Lopes said. “We now have the first line trial open for patients with newly diagnosed non-small cell lung cancer and RET fusions.” That trial will evaluate if chemotherapy plus immunotherapy or pralsetinib is a better option for the initial treatment of patients with advanced lung cancer and RET fusions.