Australian Researchers Found a Breakthrough Drug That Beats the Crap Out of Leukemia

A new drug could be a major breakthrough in the fight against cancer.

If leukemia is Popeye, BCL-2 is its spinach. It's a protein in the body that makes chronic lymphocytic leukemia, or CLL, grow. And now, researchers say, a new drug called Venetoclax, recently granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration, could effectively target BCL-2. 

So far, the medication looks effective. A study, published in the New England Journal of Medicine, observed 116 patients with advanced chronic lymphocytic leukemia and found cancer was reduced with Venetoclax in 80% of cases. Those patients had already undergone multiple treatment methods, including chemotherapy, with minor rates of success.

Finding a successful way to attack the BCL-2 protein could make Venetoclax a cancer-fighting game-changer. "If you have too much BCL-2, it prevents cells from dying appropriately," Dr. Michael Grossbard, director of the Hematologic Malignancies Clinical Program at the Laura & Isaac Perlmutter Cancer Center at NYU Langone Medical Center, told Mic. "In CLL, there's an overexpression of that protein so the cells don't die, and then they accumulate. That's what leads to the accumulation of CLL."

"There is no other drug or medicine previously available that has had the ability to inhibit this BCL-2 protein," said professor John Seymour, a lead researcher on the study, according to the Sydney Morning Herald. "That potential for broader application, and the fact that this is a completely new class of drugs, takes this beyond simply being a very good outcome for patients."

While the hype of a "miracle drug" that "melts away" cancer cells might be a little overblown, Grossbard told Mic, this is an exciting development in the cancer field, one that can be added to a few other up-and-comers. "It's a good drug, but I wouldn't neglect that you could've said the same for Zydelig and another called Imbruvica, both of which also showed high response rates in CLL patients," Grossbard said.

"Which of these will turn out to be most effective and have the largest side effect profile remains to be seen."