The Dawn of a New Age in Cancer Treatment

One of the most important events last year was the U.S. Food and Drug Administration (FDA) approval of a drug based on a tumor’s genetic profile as opposed to the tissue where it originated. “This marks another step in an important shift toward treating cancers based on their tumor genetics rather than their site of origin in the body. This new site-agnostic oncology therapy isn’t specific to a cancer arising in a particular body organ, such as breast or colon cancer” – FDA Commissioner Scott Gottlieb, M.D.

We’re at the dawn of a new era in medicine. Cancer is not just anatomic — it’s also genetic. Two decades ago, doctors could offer only “one-size-fits-all” treatments until they failed.

The FDA granted accelerated approval to Vitrakvi (larotrectinib), a treatment for adult and pediatric patients whose cancers have a specific genetic feature (biomarker). This is the second time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated. The approval marks a new paradigm in the development of cancer drugs that are “tissue agnostic.”

Today, thanks to research on the molecular landscape of prostate cancer, we know that more than 30% of patients with advanced prostate cancer harbor genetic mutations that are clinically actionable. And this same research is now also benefiting 67 other forms of adult and childhood cancers.

Genetically driven re-targeting of drugs is significant. As an example, discovering a BRCA mutation (best known from breast cancer) in a patient’s prostate cancer opened new therapeutic options. The key element is recognition; that an understanding of tumors beyond their cellular appearance allows us to link them with the appropriate molecular therapeutic. Molecular therapeutics target diseases on an early molecular level and thus modulate the disease at its roots. This is why there is so much promise in the FDA’s approval of a drug that ignores tissue of origin and focuses on genetic characteristics of a tumor. Immunotherapy (immune checkpoint inhibitors) hasn’t scored the quick wins in prostate that it has in melanoma or non-small cell lung cancer and other areas; those were the leap. Now we are in a phase of more fully exploring its potential in other cancers and understanding more about how to leverage it in combination with other therapies.

“Precision” medicine has potential that we have only begun to glimpse.

This is not the end. It is not even the beginning of the end. But it is, perhaps, the end of the beginning.” – Winston Churchill (November 10, 1942)

About the blogger @TomHulsey

After Tom beat cancer and achieved his post-surgery goals, he turned his focus to education and advocacy. He is a Board Member at ZERO The End of Prostate Cancer, volunteers at the Baylor Scott & White Cancer Health & Wellness Center, and is a Reviewer and Mentor for the DoD’s (Department of Defense) Congressionally Directed Medical Research Programs. Tom was a full voting member, (along with prominent scientists) to help determine how the $100 million appropriated by Congress would be spent on the peer reviewed Prostate Cancer Research Program (PCRP). “The advocacy work we (ZERO Cancer) do and the PCRP peer review go hand in hand. It is vital to protect and grow federal funding in order to ensure researchers have resources to develop new treatments and early detection methods.”

Tom is the author of THE WINNING MINDSET THAT SAVED MY LIFE ( 100% of the net proceeds from book sales will be donated to cancer research and education.

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