At just three months old, Ryan Frazier was diagnosed with acute myeloid leukemia (AML), a rare and aggressive blood cancer. For his parents, Steven and Carmen Frazier, this was the unthinkable.
“I had no idea what neutrophils were. I didn’t even really know what leukemia was,” recalls Steven. “It was a completely foreign world—shocking, terrifying. Nothing prepares you for that when you have a baby.”
After six months and five rounds of inpatient chemotherapy Ryan finally achieved remission. But by the time he was two, the leukemia had returned. This time, it had spread to his central nervous system, with tumor infiltration around the optic nerves—threatening not only his vision but his life. With all standard treatment options exhausted, Ryan’s parents were told his only hope lay in a Syndax clinical trial located hours away from home.
Upon arriving for the trial, the Fraziers were informed that there would be a delay in receiving the study drug. While they waited, Ryan began emergency radiation therapy necessary to slow the cancer’s progression. The treatment bought him precious time—but by then, the damage was already well underway. Caught in a no-win situation, the combination of aggressive AML and radiation-induced inflammation ultimately robbed Ryan of his sight. As the delay continued, the cancer rapidly progressed. Ryan’s life expectancy had now dwindled to mere days.
“We were watching our son die in front of us,” the Fraziers remember.
Determined not to give up, Steven sent out countless emails to anyone who might help. His persistence ultimately paid off when he connected with Syndax Pharmaceuticals. It was then that Steven learned the delay was due to the lack of regulatory approvals needed to begin Ryan’s treatment. The Syndax representative urged the Fraziers to transfer Ryan to Stanford, where the required approvals for the pediatric expanded access study were already in place, and assured them that Ryan could begin treatment within 24 hours. Expanded access studies allow patients with serious or life-threatening conditions to access investigational treatments outside of standard clinical trials when no other treatment options are available.
With no time to waste, the plan was clear—get Ryan to Stanford, where his treatment could finally begin.
A path forward
On a quiet Thursday morning in February 2024, Nancy Sweeters, RN, PNP, associate director pediatric hematology oncology research group and clinical operations at the Stanford Cancer Institute Clinical Trials Office, was heading to her office from the parking garage when she received an urgent text: “We hear you have the pediatric Syndax study open. Do you have drug available? We need to transfer a kid to Stanford today.”
Sweeters heard that Syndax had assured the family that Stanford would have Ryan on treatment within 24 hours of arrival. At the time, she thought, That’s a bold promise coming from someone all the way in New Jersey.
Under normal circumstances, transferring a patient like Ryan would take over a week, but time was something Ryan didn’t have. Sweeters knew it wouldn’t be easy, but she also knew she and her team would do whatever it took to make it happen.
"If there is a child that needs to go on study, we’ll move mountains to get it done," she said with clear resolve.
Sweeters contacted Stanford Cancer Institute associate director of pediatric cancer, Tanja Gruber, MD, PhD, division chief of pediatric hematology at Stanford Medicine Children’s Health, who was the doctor on call that day. Together, Gruber and Sweeters’ team worked tirelessly behind the scenes to expedite the transfer, including gathering all necessary clinical information, coordinating insurance coverage, and securing Ryan an inpatient bed.
Twelve hours after the initial text, Ryan arrived at Stanford Children’s by ambulance at 10 p.m.
The next day, Stanford Cancer Institute member and physician investigator of the expanded access study Norman Lacayo, MD, associate professor of pediatrics, enrolled Ryan onto the study. Before Ryan could receive the study drug, he had to undergo a battery of tests—blood draws, electrocardiograms, and a host of other screenings. The real challenge was piecing together the puzzle: identifying which tests had already been completed, getting the results transferred to Stanford, and finishing the remaining ones—all within the same day.
There was an immense amount of coordination. The team worked tirelessly behind the scenes and into the night to ensure Ryan received treatment by Friday. They entered data into the electronic health care record system, the pharmacist prepared the drug, and the electrocardiogram (EKG) team arrived at 8:30 p.m. to perform triplicate EKGs before and after administering the drug, as required per the research protocol. Physicians were on standby to answer any questions, and the nursing staff was fully committed to doing whatever it took.
At long last
On a rainy Friday night, Ryan finally received the study drug.
“We arrived at Stanford at 10 p.m. Thursday, and by 9:00 p.m. the next day, Syndax was flowing through his body,” Steven recalls.
Around 20-30% of pediatric AML patients respond to the Syndax menin inhibitor (revumenib). Ryan was one of the lucky ones. Initial imaging revealed that Ryan had tumors riddled across both his spinal cord and brain, which posed a real risk of paralysis.
“He responded quickly and beautifully,” Lacayo recalls. “The treatment prevented him from becoming paraplegic, but unfortunately, it was unable to reverse the damage to his optic nerve and restore his vision.”
One of the first signs of progress came when his bladder function began to return. Before treatment, he had trouble voiding, likely due to tumors pressing on his spinal cord.
“Before we arrived at Stanford, he would just lie on our chest, completely still—nothing at all for months. But after those 10 days of treatment, he sat up, and that’s when we knew he was starting to feel better,” Steven said.
