UVA Lab Research Becomes FDA-Approved Leukemia Drug

After years of failed treatments, some leukemia patients finally have new hope thanks to research that began in a University of Virginia lab nearly two decades ago.

In November, the FDA approved ziftomenib for patients with treatment-resistant acute myeloid leukemia. The drug targets patients with a specific NPM1 gene mutation, offering a lifeline when other options have failed.

The breakthrough started with Tomasz Cierpicki and Jolanta Grembecka, a married couple from Poland who met in graduate school. Back then, developing cancer drugs was just a distant dream.

In 2008, working as assistant professors at UVA, they focused on menin inhibitors under mentor John Bushweller. The couple spent countless late nights in the lab, sometimes starting conversations about breakthrough ideas at midnight.

"To some degree, it helped that we were married and worked together to support each other," Cierpicki said. But he admits the work followed them home, making it hard to separate lab life from personal life.

Their dedication paid off. In 2009, they patented the menin inhibitors that would become ziftomenib through a collaboration between UVA and the University of Michigan, where they now teach.

Acute myeloid leukemia strikes more than 22,000 Americans each year, killing over 11,000. It primarily affects people over 68, making it one of the deadliest blood cancers.

Ziftomenib works by blocking a key protein that drives leukemia growth. Instead of becoming cancer cells, the cells develop into healthy white blood cells.

The Ripple Effect

The drug represents more than just one successful treatment. It proves that academic research labs can develop life-saving drugs for diseases that pharmaceutical companies might overlook due to smaller patient populations.

"I believe academic drug discovery plays a very important role, particularly in rare diseases where industry interest may not be a primary priority," Cierpicki said.

Grembecka has already heard stories of patients entering remission after just one cycle of ziftomenib, following several failed treatments. These results fuel the couple's continued research efforts.

"What Tomasz and Jolanta started in a UVA research lab two decades ago is now saving lives," said Richard Chylla, executive director of UVA Licensing & Ventures Group. The success story exemplifies the work expected at the new Paul and Diane Manning Institute of Biotechnology.

The couple's midnight conversations and years of persistence transformed an impossible dream into reality for patients who had lost hope.