Polish Dreams: Couple Saving Lives With Cancer Drug They Invented While at UVA

What must it be like to go through unsuccessful after unsuccessful cancer treatment. At a certain point, it might make even the most courageous and strongest-willed lose hope.

But former University of Virginia professors Tomasz Cierpicki and Jolanta Grembecka are restoring hope for people suffering from the deadliest form of blood cancer.

In November, their drug, ziftomenib, was approved by the Federal Drug Administration for patients with recurring or treatment-resistant acute myeloid leukemia who have mutation in the NPM1 gene.

Acute myeloid leukemia is a deadly blood cancer primarily seen in people over the age of 68. More than 22,000 Americans develop the condition each year, and more than 11,000 die, according to the American Cancer Society.

But now those numbers could be on the decline thanks to Cierpicki and Grembecka.

“What Tomasz and Jolanta started in a UVA research lab two decades ago is now saving lives,” said UVA Licensing & Ventures Group Executive Director Richard W. Chylla. “This is precisely the type of outcome we hope for with all of the work that comes through our office here at LVG, and an example of the work we expect to be accomplished at the Manning Institute.”

Natives of Poland, Cierpicki and Grembecka -- who are married and are now professors at the University of Michigan -- met while they were in graduate school at Wroclaw University of Technology in Poland. While there, they began thinking about research aimed at developing new drugs that could help cancer patients.

“It was a dream, but at the time, we didn’t realize it could actually become a reality,” Grembecka said.

“Nobody could foresee the future,” Cierpicki said.

It was while working as assistant professors in UVA’s Department of Molecular Physiology and Biological Physics in 2008 under mentor John Bushweller that the couple homed in on menin inhibitors, a class of targeted therapies used primarily to treat acute leukemias.

“We really value what John Bushweller did – he allowed us to have some independent work,” Cierpicki said. “And we were quite lucky that we got funding that could support what was needed to initiate and then continue the menin inhibitor project.”

There were many late nights.

“To some degree, it helped that we were married and worked together to support each other, but one of the issues is that when you work with your spouse, your work-related conversations continue even after you get home, so sometimes that is maybe too much,” Cierpicki said, smiling.

“We would sometimes start a conversation at midnight with an idea that couldn’t wait until the next day,” Grembecka said, with a laugh. “But on the positive side, we could closely collaborate with each other and use our complementary expertise while working on different aspects of the same project.”

Eventually, the hard work paid off. In 2009, the menin inhibitors that would eventually lead to the development of Ziftomenib were patented through a collaboration between UVA LVG and the University of Michigan Invention Partnership Office.

The drug works by disrupting interactions of a cellular protein, menin, that drives leukemia cell growth and survival. It allows cells to mature into white blood cells instead of becoming cancerous.

“Ziftomenib is a long-awaited and desperately needed new option for patients for whom other treatments have failed – for patients left with no hope. It’s a wonderful achievement by Drs. Grembecka and Cierpicki,” said Mark Esser, PhD, the head and chief scientific officer of UVA’s Paul and Diane Manning Institute of Biotechnology. “UVA has founded the Manning Institute specifically to advance exactly this type of important research. We are accelerating the development of new treatment and cures for the most complex and difficult diseases to benefit patients everywhere.”

Recently, Grembecka said she and her husband have learned of patients who have had several unsuccessful treatments for their leukemia , only for the disease to go into remission after just one cycle of ziftomenib.  

“The FDA approval of Ziftomenib is deeply rewarding, as the drug is now available to all patients who may need it,” Grembecka said. “It’s very inspiring as well. We feel even more motivated to continue our efforts.”

Cierpicki smiled when reflecting on his time at UVA.

“I believe academic drug discovery plays a very important role, particularly in rare diseases where industry interest may not be a primary priority,” he said. “Continuing these efforts is therefore essential.”