A small Boston biotech is at the heart of a groundbreaking approach to tackling cancer

Lydia Ramsey/Business Insider

• Agios Pharmaceuticals, a company that got its start less than a decade ago, already has one drug in front of the FDA.
• The company makes drugs that essentially try to repair cancer cells so they can grow old.
• If the drug's approved, it could be the first to target the metabolism of cancer cells, a relatively new area of research.

The idea is a simple one: Starve out cancer cells.

But finding the right way to do that isn't nearly as simple. Cancer cell metabolisms - the process of converting food into energy - worked differently than normal cells.

Cancer cells take in more glucose than normal cells, a phenomenon known as the Warburg effect. It's named after the German scientist Otto Warburg, who first observed in the early 1900s that cancer cells didn't need as much oxygen to metabolize sugar as normal cells. The Warburg effect is estimated to occur in roughly 80% of cancers.

That different metabolic process opens up the opportunity to target just cancer cells, leaving healthy cells untouched. While the Warburg effect has been around for close to 100 years, it's only been in the past decade that researchers have figured out ways to use it in treatments, Dr. Anne Le, a professor at Johns Hopkins who researches the cancer metabolism, told Business Insider.

It's why Le and companies like Agios Pharmaceuticals are working on drugs that target the metabolism of cancer cells. Agios submitted its first cancer drug to the FDA, which has until August 30 to decide whether it will approve the drug.

"Agios led the way - and others are now doing it as well - to build the tech to interrogate cellular metabolism in ways that just haven't been done before," Agios CEO David Schenkein told Business Insider.

Here's how it works

While a normal cell goes through a whole life cycle (gets born, ages, makes new babies, then dies), cancer cells get stuck at the baby stage. While there, they just make more and more of themselves.

Lydia Ramsey

A troll keeps watch over one of Agios's mass spectrometers, the one that discovered the oncometabolite 2HG that's key to Agios's IDH program.

"What our drugs do is allow those babies to grow up," Schenkein said. "They'll then get old and die off."

The drugs do that by pinpointing enzymes that are key to the cancer cell's metabolism, and acting on those. In order to take the treatment, a person first has to have that specific enzyme in their system.

"There are 3,000 different metabolic enzymes in our cells, and cancers have found a way to screw up a lot of them," Schenkein said.

What these treatments look like so far

Agios's first drug, enasidenib, is a pill that treats acute myelogenous leukemia, a cancer of the blood and bone marrow. That one's in front of the FDA right now, and could be approved by August 30. Agios also has a treatment for bile duct cancer, as well as a treatment for a rare genetic disease. Beyond that, there are still thousands of metabolic enzymes that still haven't been targeted.

On Wednesday, Agios came out with new data on its phase 1/2 trial, which will be presented at the American Society of Clinical Oncology annual conference. Out of 176 patients in the trial, 40% had an overall response rate (meaning the cancer responded and shrank by a certain amount), with 19% going into remission. The patients had a median response rate of 5.8 months.

Even with the progress in the last decade the field of cancer metabolism, Le wants everyone to know that it's not simple. As new treatments go through development in the coming years, cancers may be able to adapt and work around these drugs.

"That's an opportunity for us," Schenkein said. "We are at the beginning of this field,"