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Briana Ayala, with cancerous tumors in the abdomen and elsewhere, has been treated with an experimental drug called "precision medicine" that has just been approved by the Food and Drug Administration. (Courtesy of Children's Health / Juan Pulido)
The Food and Drug Administration on Monday approved a drug for a wide range of cancers based on a shared mutation rather than on the location of tumors – a breakthrough for the sometimes controversial field of "precision medicine."
The drug, called Vitrakvi, is the second treatment to receive FDA clearance based on a common biomarker found in a range of cancers. The drug, also called larotrectinib, has been approved simultaneously for adults and children. Generally, oncology medications are considered much later for children.
The drug approved Monday is for patients with advanced solid tumors containing what is called a fusion of NTRK genes, a hybrid of two genes that can promote uncontrolled cell growth. Thyroid, lung, head and neck cancers, among others, can be caused by the defect. The drug is intended for patients whose cancer has spread or who would suffer serious complications during a surgical procedure and who have no satisfactory alternative.
The drug's manufacturer, Loxo Oncology, Inc., faces a huge challenge: finding patients who could benefit. In the United States, it is estimated that only 2,000 to 3,000 people a year develop NTRK-related cancers.
"The million dollar question is: how do you know the drug is dealing with the merger?" Said Josh Bilenker, general manager of Loxo, who has a commercial partnership with Bayer. The mutation occurs in less than 1% of most types of solid tumors, but is common in malignancies such as salivary cancer in adults and infantile fibrosarcoma.
The only way to detect the mutation is to perform much larger tumor genetic tests. However, while patients at university medical centers with advanced stage cancer are generally tested, many people do not undergo genetic tumor testing in community settings where 80% of cancer patients receive cancer. care. And while Medicare covers some genetic testing, private insurers are far less likely to pay for it.
Precision medicine has generated tremendous enthusiasm in recent years. But this has also fueled skepticism, in part because drugs and tests tend to be expensive.
"Patient affordability is one of the major barriers to precision medicine at the present time," said Carolyn Presley, a geriatric oncologist at the University Cancer Treatment Center. from the Ohio State. "Show me the money – how are you going to pay for this?"
But Elizabeth Jaffee, an oncologist at Johns Hopkins, said that the cost of sequencing has decreased and that precision medicine "is going to be the way to treat cancer in the future." And David Hyman, oncologist at Memorial Sloan Kettering Cancer Center who led the pivotal studies on larotrectinib, said, "There is no way we are taking care of cancer patients and not trying to understand their situation. genetic."
When Briana Ayala, from El Paso, began having severe back pain at age 11, she was diagnosed with sarcoma – a soft tissue tumor – that was partially wrapped around her aorta. The growth was suppressed two years ago in a high-risk operation, but it returned to his spine, prompting doctors to test his tumors. When the NTRK changes were detected, she started taking larotrectinib as part of a clinical trial and her tumors disappeared. Ayala is now 14 years old. She studies in high school and dreams of becoming a fashion designer.
"It saved her life," said Theodore Laestch, pediatric oncologist at UT Southwestern and Children's Health in Dallas, who was involved in her treatment.
The FDA said the drug's effectiveness had been studied in three clinical trials involving 55 children and adults. Patients had an overall response rate of 75% on different types of solid tumors, with almost all responses lasting six months and 39% lasting for a year or more. Fatigue, nausea and dizziness are common side effects.
For Acra Samuels, the drug had a dramatic effect but did not last. In 2016, she had tumors throughout the colon, liver and lungs. "I was really in trouble," said the resident of Big Sky, at Mt. When sequencing tumors at the Dana Farber Cancer Institute in Boston showed that NTRK fusions were causing malignancies, she enrolled in a clinical trial.
The Loxo treatment has been running for seven months, said George Demetri, a Dana-Farber oncologist. But like many other targeted therapies, the cancer has mutated and in June 2017, tests showed that tumors in her abdomen and pelvis were developing rapidly. The pharmaceutical company, worried about this resistance, was already working on a second-generation drug for new mutations. "We knew that if it did not work, I had one month left," Samuels said.
Tumors have decreased sharply in a few weeks. She then underwent surgery to remove a tumor on the outside of her liver.
Today, "I'm fine for the moment," said the 47-year-old in a recent e-mail. "My last three exams showed that my illness was stable. I enjoy life and I am grateful for each day. "
Read more:
"It's not the end": using immunotherapy and a genetic problem to give hope to cancer patients
First approved treatment for breast cancer with BRCA genetic mutation
FDA Approves First Gene Therapy for Hereditary Disease
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