Menin inhibitor clinical trial puts pediatric leukemia patient in remission

When David Stan was diagnosed with acute myeloid leukemia in the summer of 2023, his parents, Emil and Maria, were stunned. Cancer doesn’t run in either of their families. And until then, David had been a perfectly healthy 13-year-old. 

“In my line of work, I see some pretty awful things happening to children,” says Emil, a peace officer in southern California. “That can really mess with your head. But when it’s your kid, it’s an entirely different feeling. None of those situations affected me the same way David’s diagnosis did.”

Initial treatment’s failure exhausts family’s local options — and hope

Initially, the couple sought leukemia treatment locally for their son. Despite an encouraging start, the cancer returned after only three rounds of chemotherapy. And this time, it was in David’s brain and spinal fluid. 

“It was heartbreaking,” recalls Maria. “The doctors near our home said there was nothing more they could do. The only thing that might save David was a stem cell transplant, but he had to be in remission first. And, they’d already given him the strongest chemotherapy they could, yet he was worse off than before.” 

At that point, David was having such bad headaches and persistent fevers that he could neither eat nor sleep comfortably. 

“He was miserable,” adds Maria. “I couldn’t stop crying. We contacted several pediatric cancer hospitals. They had nothing to offer. They told us David had maybe a year left to live.”

Clinical trial at MD Anderson renews family’s sense of hope 

The Stans kept searching for new therapies and specialists. Finally, someone mentioned Branko Cuglievan, M.D., a pediatric oncologist at MD Anderson specializing in treating childhood leukemias. He gave the couple the first stirrings of hope.

“I loved Dr. Cuglievan’s confidence,” recalls Emil. “He was the only doctor who used the word ‘cure’ when talking about David. I thought, ‘Either this guy is nuts, or he knows something we don’t.’”

As it turns out, Cuglievan did know something.

He told Emil and Maria about an early-phase clinical trial that he thought might help David. It involved a new type of targeted therapy called a menin inhibitor and was available only at MD Anderson. It was showing great promise against a particular genetic mutation called KMT2A, which David had. If Emil and Maria could get David to Houston, he might be able to join the trial. 

Experimental drug combination results in full remission

The couple wasted no time. Within a few days, David received his first dose of revumenib (SNDX-5613) through the clinical trial at MD Anderson. He also received a targeted therapy drug called venetoclax and a chemotherapy drug called decitabine/cedazuridine. 

“Patient response to revumenib has been good in other clinical trials,” explains Cuglievan. “But it’s also been short-lived when used alone. We thought we could do better. So, we combined it with two other drugs. And we’ve gotten such good results, this may well become the new standard of care.” 

Just two weeks later, David’s bloodwork demonstrated why: he had no evidence of disease. Even his spinal fluid was totally clear. That meant David was eligible for a stem cell transplant. He finally received one from an unrelated donor on April 25, 2024. 

“Going to MD Anderson changed everything for us,” Emil says. “Thanks to that clinical trial, our son went from having 70% leukemia cells to having 0% in just 14 days. It was incredible.”

Second clinical trial resolves alarming side effect

Today, David remains in full remission. But his road to recovery has not been entirely smooth. About five days after the stem cell transplant, he developed severe mucositis, or inflammation of the digestive tract. 

For David, that meant a sore throat so painful he couldn’t swallow anything — not even his saliva. His care team managed that side effect with a combination of pain relievers and IV fluids. 

“It finally cleared up on its own a couple of days after the cells engrafted,” recalls Emil. “That was about Day 16 after the transplant. His marrow just needed time to generate enough white blood cells to counteract it.”

David also began urinating blood. This was due to the BK virus, an opportunistic infection that crops up frequently among immunocompromised populations. 

“That’s when Dr. Cuglievan told us about another clinical trial involving CTLs — or cytotoxic T lymphocytes — that are specifically reactive to the BK virus,” says Emil. “We agreed to try it. After just two infusions, the problem cleared up completely.” 

Family now reclaiming a joyful future

David’s blood counts continue to improve daily, though he must remain cautious about what he eats and how he spends his time until his immune system recovers completely. But he is back to playing video games and anticipating family camping trips. He’s even considering a career in medicine.

“We are beyond grateful,” says Emil. “MD Anderson gave us our son back. Now, he wants to be a pediatrician and work with other cancer patients. Maybe someday he can be the next Dr. Cuglievan!” 

Clinical trials like the one that saved David are funded in part by generous donations from corporate partners like Energy Transfer, whose employees have collectively raised more than $17 million over the past 10 years to support pediatric cancer research at MD Anderson. 

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