一道本不卡免费高清Lara Stuart and her husband, David Lodge, first noticed the peculiar rash on their son Quincy’s face when he was just shy of 4 months old.
Trips to the pediatrician and a dermatologist didn’t offer up any concrete answers – and within weeks, Quincy’s abdomen started to swell.
Stuart and Lodge drove Quincy to the nearby emergency department, and the infant was admitted to the hospital, where doctors ordered a battery of tests.
Later that day, the family received the news that would change their lives forever: Quincy had a rare and aggressive childhood blood cancer called juvenile myelomonocytic leukemia, or JMML.
Through the next few months, Quincy’s treatment would take him and his family across the Bay to San Francisco and to the frontier of using precision medicine to unearth a drug that would save the infant’s life.
While Quincy recovered from his splenectomy surgery, Stieglitz approached his parents about trying an oral medication called sorafenib, an FLT3 inhibitor that has been effective in treating many adult liver and kidney cancer patients whose cancers are driven by changes in FLT3.
一道本不卡免费高清The drug had never been tested in clinical trials for infants, so even the proper dosage was unknown. In fact, an earlier study even recommended against sorafenib for JMML because it had found no FLT3 alterations in a group of patients with the disease.
一道本不卡免费高清It was, as everyone understood it, a last-ditch effort.
“I was scared but felt comfortable knowing this was the shot we needed to take, and really felt like everyone was doing their due diligence,” Stuart said. “It was a risky but promising approach to his treatment, and we needed to pray, cross our fingers, and believe in the best. They asked us to trust them, and we did.”
Like Witnessing a Miracle
一道本不卡免费高清A mere three days after Quincy started taking sorafenib, his white blood cell count plummeted from an abnormally high 70,000 to a normal level of 10,000. Two weeks later, the normal levels held steady, and Quincy finally was able to move back home after more than four months in the hospital.
About two months after beginning the medication, Quincy was healthy enough to undergo a bone marrow stem cell transplant, but he presented a challenging case for the team in many ways. Because Quincy is biracial, there were no matches for him in the National Marrow Donor Program’s 20-million person registry. Nor did he have any siblings who could be potential donors. A “half-matched” transplant from his father was the only option, but Quincy’s illness had required many platelet transfusions, which caused his immune system to develop antibodies against a wide range of proteins, meaning he would likely reject his father’s stem cells. The transplant team, led by , MD, devised a novel combination of medications and procedures to eradicate these antibodies.
一道本不卡免费高清As an additional precaution, stem cells from Quincy’s father were filtered through an experimental procedure, called T-cell receptor alpha-beta depletion, to remove most of the cells that could potentially cause an attack on the recipient’s body. This state-of-the-art procedure by the UCSF Pediatric Cellular Therapy Laboratory has made stem cell transplant a possibility for all patients, regardless of racial background.
一道本不卡免费高清With these combined efforts, Quincy’s stem cell transplant was a success and he bounced back beautifully. In July 2017, Quincy celebrated his first birthday at home with his family.
Now more than two years after a a bone marrow stem cell transplant to treat JMML, Quincy is a thriving toddler. Photo by Barbara Ries
After a relatively new genetic test called the UCSF500 helped doctors find a drug to treat his JMML, Quincy (center) was able to undergo a bone marrow transplant with his father, David Lodge (right), as the donor. Photo by Barbara Ries
Quincy’s family has slowly reintegrated into a more normal day-to-day life after his treatment for JMML at UCSF Benioff Children’s Hospitals. Photo by Barbara Ries
Quincy’s mother, Lara Stuart, reads to him at their family home. Photo by Barbara Ries
Quincy plays in the backyard of his family backyard. Just years before, his parents noticed a rash on his face, and weeks later he was diagnosed with a rare and aggressive childhood blood cancer. Photo by Barbara Ries
“It was a risky but promising approach to his treatment, and we needed to pray, cross our fingers, and believe in the best,” said Quincy’s mother, Lara Stuart, of his treatment for JMML. “They asked us to trust them, and we did.”
“Sometimes children are trying to tell us that standard chemotherapy is not the right choice for their specific type of cancer and that they require personalized treatments,” Stieglitz said. “We read the letters of his DNA, saw what mutations were in his tumor, and gave him medication specific to that mutation that we never would have thought of otherwise. When you use precision medicine, it not only works better, it’s also safer because there are fewer side effects.”
Now more than two years after his transplant, Quincy no longer needs to take sorafenib and is in complete molecular remission. His family has slowly reintegrated into a more normal day-to-day life, happily accepting birthday party invitations and even taking the talkative toddler on his first plane ride to visit extended family.
“This whole experience was just the most beautiful symphony of collaboration and people putting together their knowledge and expertise to save a little boy,” Stuart said. “He’s a miracle, and there’s no way powerful enough to say thank you.”