Just this week, the Food and Drug Administration approved a drug that targets a wide range of cancers based on DNA, rather than where the tumor is in the body.
This is being hailed as an advance for the field of “precision medicine.”
The drug Vitrakvi (larotrectinib) is for patients with advanced solid tumors containing what’s called an NTRK gene fusion – a hybrid of two genes that can promote uncontrolled cell growth.
Cancers of the thyroid, lung, and head and neck, among others, can be caused by an NTRK gene fusion.
This breakthrough is not only important for treating the aforementioned cancers, but also for the ever-evolving field of precision medicine, which is the tailoring of medical treatment to a patient’s individual characteristics, including genetic makeup.
“[Precision medicine] is going to be the way to treat cancer in the future. There’s no way that in 5 to 10 years we will be taking care of patients with cancer and not trying to understand their genetic situation,” according to Elizabeth Jaffee, an oncologist at Johns Hopkins, and David Hyman, an oncologist at Memorial Sloan Kettering Cancer Center.
Obstacles still exist. Vitrakvi is expected to be very expensive, and finding patients who develop NTRK-related cancers
(who can benefit from Vitrakvi) is costly and difficult, because they are so rare.
But early signs are promising. Here is an anecdote, reported by the Washington Post:
When Briana Ayala of El Paso began having severe back pain at 11, she was diagnosed with a sarcoma — a soft-tissue tumor — that was wrapped partly around her aorta. The growth was removed two years ago in a high-risk operation, but it returned in her spine, prompting doctors to test her tumors. When NTRK alterations turned up, she began taking larotrectinib as part of a clinical trial, and her tumors disappeared. Now 14, Briana is a freshman in high school and dreaming of becoming a fashion designer.
According to the FDA, Vitrakvi’s efficacy was studied in three clinical trials involving 55 children and adults. The patients had a 75 percent overall response rate across different types of solid tumors, with almost all the responses lasting six months and 39 percent lasting a year or more.
So as this new, novel treatment climbs up the research and testing ladder, we are filled with hope that any new development involving genetic experimentation has a trickle down effect that can affect many rare diseases.
We’ll keep a close eye on it!
