Big Ideas

Anthony Letai, M.D., Ph.D.

Sometimes, a little discouragement can go a long way.

Anthony Letai, M.D., Ph.D., of the Dana-Farber Cancer Institute, is a distinguished cancer researcher whose work has led to a new FDA-approved drug. It’s a scary thought to imagine the cancer research world without the bright mind of someone like Letai. Luckily, a teacher, who surely had the interest of the general public in mind, steered Letai back to the direction he was meant to go.

“I always knew I’d likely be a scientist and never seriously considered anything else, except being a violinist,” said Letai. “A kind teacher gently disabused me of that notion.”

While the music world may have missed out on its next great violinist, the world of cancer research gained a star.

Letai’s research focuses on apoptosis, a process the body uses to get rid of cells that have gone bad. Normally, cells undergo apoptosis – essentially, they commit suicide – if something goes wrong with the cell’s growth or functioning. But this doesn’t happen with cancer cells, which means they can grow out of control. Letai and his team discovered one reason for this is BCL-2, a particular family of proteins that interfere with apoptosis in cancer cells.

Their work has led to FDA approvals for venetoclax, a drug that causes apoptosis in two types of leukemia cancer cells.

“When BCL-2 family proteins are targeted by the right drugs, cancer cells are convinced to commit suicide,” said Letai. “We have developed some useful tools to identify what drugs work best on an individual’s cancer, which helps in getting the right one of these drugs to the right patient. I expect that, in this decade, doctors will be making use of this class of drugs for nearly all blood cancers.”

Building on venetoclax’s success, many clinical trials for similar drugs designed to trigger apoptosis in other types of cancer are currently being conducted internationally. In addition to blood cancers, Letai expects the approach will work against solid tumors as well, especially when combined with other cancer-killing drugs.

Back in 2005, Letai received a V Scholar grant from the V Foundation, and then in 2015, he received a Designated grant. That early support from the V Foundation was instrumental in Letai’s future success.

“I was a V Scholar early in my career, and that boost allowed me to be a little more ambitious and take a little more risk than I otherwise might have taken,” said Letai. “You have to take these risks to do something new and make a true jump in understanding.”

Thankfully for all of us, Letai decided to be a risk-taker in the lab and not in a concert hall. Because of that, we are closer than ever to Victory Over Cancer®.

“The relentless, step-by-step conquest of cancer will not happen via one idea or advance,” said Letai. “We will just get better and better with one cancer after another. It’s the type of progress we are witnessing right now, right in front of us.”