Kymriah. For kids with high-risk leukemia, those seven letters spell hope. But what is this lifesaving ‘living drug’? And how did this exciting new therapy come to be? Buckle your seat belts, because today we are starting an epic journey – a journey made possible by St. Baldrick’s supporters like you. Over four blogs, we’ll follow the path of this immunotherapy breakthrough — from the Phase 1 clinical trial to its recent FDA approval — and see the process through the eyes of the kids, families and St. Baldrick’s researchers who made this revolutionary research happen.
In Part One of our blog series, meet St. Baldrick’s Honored Kid Austin, a now 9-year-old cancer survivor whose last chance at life was the first human trial for Kymriah.
Honored Kid Austin was diagnosed with a high-risk form of acute lymphoblastic leukemia when he was 2 years old.
Kim Schuetz can’t forget the moment she saw the symptoms of her son’s childhood cancer. It was May 2011 and Austin was nearing his third birthday. Austin and his grandpa were playing together when the man noticed something odd. He called Kim over and together they crouched by the living room couch to look at large bumps on the sides of Austin’s neck.
St. Baldrick’s Scholar Dr. David Barrett gives us an inside look at immunotherapy, and we meet the first pediatric patient to ever receive this new cancer treatment.
Emily Whitehead, the first pediatric patient to be treated with immunotherapy, 18 months post-treatment and starting third grade.
For most of us, our immune systems fight off disease every day. A healthy immune system can even fight off life-threatening diseases like cancer. And when we do get sick, a healthy immune system attacks sickness and help us get back to feeling like ourselves again.
It is a different story for kids with cancer. A child with cancer has a suppressed immune system. It’s as if a blindfold has been pulled down, and the body is unable to target invasive cells. You might say the immune system is asleep.
What if doctors could wake up a sleeping immune system?
St. Baldrick’s supporters, this is a day to celebrate! You have helped make history. Today, the FDA approved the first gene therapy available in the United States. Called Kymriah, it’s an entirely new way of treating cancer and it’s saving lives.
This “living drug” is for patients with a type of acute lymphoblastic leukemia (ALL), a cancer of the blood and bone marrow and the most common form of childhood cancer. Scientists genetically modify a patient’s own immune cells in the lab, then infuse these new cells back into the patient’s body. These modified cells – called CAR T cells or chimeric antigen receptor T cells – then prompt the child’s own immune system to attack and kill leukemia cells.
What do childhood cancer researchers do when they see a problem? They band together to solve it. Read on to learn why institutions across Texas and California are joining forces and what they’re doing to help kids with cancer.
Dr. Philip Lupo is the principal investigator of the REDIAL Consortium, as well as an Associate Professor of Pediatrics at Baylor College of Medicine and Co-Director of the Epidemiology Program at Texas Children’s Cancer and Hematology Centers.
Years ago, St. Baldrick’s researcher Dr. Philip Lupo and his colleagues at Houston’s Baylor College of Medicine noticed a problem.
According to studies they read and stories Dr. Lupo heard from clinicians, kids who are Hispanic don’t do as well on treatment as other kids with acute leukemias. They are more likely to experience complications and are at higher risk of relapse than kids of other backgrounds.
But scientists don’t know exactly why that is.
We have big news. Yesterday, the the FDA Reauthorization Act passed the Senate which includes critical components of the Research to Accelerate Cures and Equity (RACE) for Children Act. Read on for more about the RACE Act and what this news means for Rob Lenfestey, the dad to Honored Kid Scott, and for families everywhere.
Honored Kid Scott ‘speaks up’ for kids’ cancer research on Capitol Hill during this year’s Childhood Cancer Action Days.
When our son, Scott, was diagnosed with acute lymphoblastic leukemia at age 3, my mind was swimming with questions about his treatment options. During the last 20 years, the FDA has approved approximately 190 new cancer therapies for adults; only three new treatments have been approved specifically for treating kids with cancer. Our hopes hinged on the existing treatments available, and the prospect of drugs that may be waiting in the pipeline if conventional treatments weren’t effective at killing his leukemia.
Last week, we shared Honored Kid Zach’s amazing story and the news that the three-time cancer fighter was going to get a bone marrow transplant — a procedure that could put the 15-year-old into remission for good. Today, about a week after the transplant, Zach’s mom has an update.
The family gathers before Zach’s bone marrow transplant. From left to right: Nancy, Ben, Tom and Zach, with Gabe on Facetime.
My son, Zach, is fighting cancer for the third time, which means this is the third time going through tests, treatments and all the uncertainties that follow. Zach had three months of intense chemo therapy to try to get him into remission before his bone marrow transplant or BMT. At the end of his treatments, through numerous hospitalizations, we learned that Zach was not in remission.
Honored Kid Zach has faced leukemia three times since he was 6 years old. On Wednesday, after 10 years of fighting, he hopes that this is it — that a bone marrow transplant will put him in remission for good. Learn about his tremendous journey and why children’s cancer research is important to him.
Zach clowns around during treatment.
Wednesday is a big day for 15-year-old Zach. After two relapses and years of childhood lost to cancer, it’s a new start.
“Our son would not be with us today if it weren’t for St. Baldrick’s,” says Phineas’ dad, Carlos. Read on to see how research saved the little boy’s life.
VIDEO: Phineas’ Story >
On a mountain bike ride with a friend, 9-year-old Phineas was sailing along when he decided to take a risk and pedal over a bridge not meant for bicycle traffic. He wiped out in a big way.
But without so much as a single tear, he picked himself up, dusted himself off, and got back on the bike.
Compared to what this boy had been through two years before, that was nothing.
Katie Pierantozzi always wanted to be a mom. But she never imagined she’d be a cancer mom. In celebration of Mother’s Day, read on for a message and a Mother’s Day wish from Katie for all those moms out there who have a child with cancer.
The Pierantozzi family from left to right: Nicky, Justin, Michael and their mom, Katie.
Her name was Junie. I loved to rock my favorite baby doll in a little wooden crib, painted pale pink. I liked real babies, too. I looked forward to being a mom. I would be good at it.
Eventually, God gave me three beautiful baby boys. It didn’t take long to realize that motherhood is no Pampers commercial, where babies crawl around on spotless ﬂoors and moms look perfect.
Honored Kid Ryan C. faces childhood cancer with a contagiously positive attitude — and our social media manager, Alison Sutton, got to experience this firsthand when she hung out with him last month! Read on to see what life is like for Ryan and bask in the glow of this amazing kid, his awesome family, and their St. Baldrick’s fundraising team — Team Rally for Ryan.
St Patrick’s Day is a special day at the St. Baldrick’s Foundation because 17 years ago, our founders hosted their first head-shaving event!
This St. Patrick’s Day I spent the day at Arnold Palmer Children’s Hospital in Orlando, Fla. with Honored Kid Ryan C. to see what a day in his life is like.
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