I maintain my positive disposition by reading a good number of upbeat stories. This one ought to make you feel good even though... geez... the author sure loves paragraph breaks!
January 8, 2025
13-Year-Old Leukemia Patient in Remission After Clinical Trial for Groundbreaking New Cancer Treatment
by Frank Bergman
SlayNews.com
A 13-year-old leukemia patient has been brought into full remission during a clinical trial for a groundbreaking new cancer treatment.
The patient, David Stan, is recovering from a devastating diagnosis of acute myeloid leukemia (AML) with a KMT2A genetic mutation.
Led by Dr. Branko Cuglievan, the trial tested a novel combination therapy using the menin inhibitor revumenib, venetoclax, and decitabine/cedazuridine.
Within just two weeks of treatment, David’s leukemia cells dropped from 70% to 0%, clearing him for a successful stem cell transplant.
Though post-transplant complications like severe mucositis and BK virus infection arose, these were effectively managed, including through another clinical trial involving cytotoxic T lymphocytes.
Dr. Cuglievan completed his Pediatric training at Miami Children’s Hospital.
He subsequently completed both Pediatric Hematology and Oncology training at MD Anderson, during which he was selected as the Chief Fellow.
He obtained board certification in Pediatrics and Pediatric Hematology and Oncology and stayed on faculty as Assistant Professor in the Department of Pediatrics.
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.
Until the diagnosis, 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.”
Initially, the couple sought leukemia treatment locally for their son.
Despite an encouraging start, the cancer returned after only three rounds of chemotherapy.
This time, however, 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.”
The Stans kept searching for new therapies and specialists.
Finally, someone mentioned Dr. Cuglievan, a pediatric oncologist 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.
The family wasted no time.
Within a few days, David received his first dose of revumenib (SNDX-5613) through the clinical trial.
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.”
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, an 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.”
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!”
http://slaynews.com/news/13-year-old-leukemia-patient-remission-clinical-trial-groundbreaking-new-cancer-treatment/
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