Over a series of four blogs — read the first blog here — we are tracing the path of Kymriah, a recent immunotherapy and gene therapy breakthrough for kids with high-risk leukemia, like Honored Kid Austin. This 9-year-old pioneer was one of the first patients to receive this revolutionary type of CAR T cell therapy, which was made possible because of the hard work of the St. Baldrick’s – Stand Up To Cancer Dream Team.
Continuing from Part One, Austin’s bone marrow transplant has failed and we find his parents at a dead end in terms of treatment options — until a ray of light appears.
During his treatment, Austin always just wanted to be a kid. After finishing a chemotherapy and radiation treatment, he’d often joyfully run out to his backyard to play on the swings.
The options were few and the stakes were huge, but the choice was clear for Austin’s parents. With their 4-year-old son months away from death, they had to choose hope – hope in the form of a clinical trial testing a promising gene therapy called Kymriah.
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.
1. Over 80% of children diagnosed with cancer will be cured, joining the growing population of long-term childhood cancer survivors.
Thanks to advances in chemotherapy, radiation and surgical techniques, more children and adolescents are being cured of cancer every year. There were ~380,000 survivors of childhood and adolescent cancer in the United States as of 2010, and that number is expected to exceed 500,000 by the year 2020.
Ever since Joe and Erin Martorano’s daughter Sara was diagnosed with childhood cancer, St. Baldrick’s has been part of the family. From shaving to organizing events and starting a St. Baldrick’s Hero Fund, they’ve done it all. Read on to find out why.
The Martorano family smiles together during Sara’s treatment for cancer. From left to right: Anna, Erin, Mary, Joe and Sara.
Joe Martorano first shaved his head with St. Baldrick’s in 2008, alongside his colleagues at the Chicago Police Department. He didn’t have a direct connection to childhood cancer at the time. Fundraising for kids’ cancer research just seemed like the right thing to do.
Later that year, that would all change.
Alyson Weissman is a dedicated St. Baldrick’s shavee℠, a member of the 46 Mommas and the founder of a St. Baldrick’s Hero Fund which raises crucial funds for lifesaving research. Why does she do so much? Because Alyson is also the parent of a cancer survivor. Read on for more about what being a survivor really means, how she conquers fear and why she works so hard to fund kids’ cancer research.
Alyson shaves her head with St. Baldrick’s as her son holds her hand.
My son Jared was diagnosed with Hodgkin lymphoma in 2007. This year, in July, he will be a nine-year cancer survivor.
The Furco family spent their holiday vacation traveling somewhere they’ve dreamed of going — to Italy with their child Ambassador Abby, who had cancer. How’d they do it? Read on for 11 travel tips from Abby’s mom, Patty, who is ready to share how you can successfully take a child with medical needs on a trip of a lifetime.
Ambassador Abby pretends to hold up the Leaning Tower of Pisa while on vacation in Italy.
Traveling with family is tough. But vacationing with a child in treatment or with ongoing medical needs brings it to another level. It takes a lot of forethought, from the what ifs, to making sure you have the right supplies, to planning for proper medication storage during travel – whew, it’s exhausting just thinking about it. But all the hard work is so worth it.
When a child survives cancer, the journey isn’t over. Childhood cancer survivors face the effects of their treatment for the rest of their lives — even when they decide to have children of their own. Meet 2012 St. Baldrick’s Ambassador Sarah, a 28-year-old survivor who’s ready to be a mom, and Dr. Jill Ginsberg, a St. Baldrick’s researcher who’s determined to preserve the fertility of people like Sarah.
Sarah and Patrick have been together for 10 years, through Sarah’s relapse and beyond. Last year, they got married. Now they want to start a family and they are ready to face the challenges together.
Cancer survivorship issues are close to Dr. Brandon McNew’s heart. It’s not just because he treats kids with cancer as a pediatric oncologist — the St. Baldrick’s researcher was diagnosed with leukemia when he was 10 years old. Read on for more about his childhood cancer journey, why he was drawn to pediatric oncology and what he’s doing (with a little help from St. Baldrick’s) to help fellow cancer survivors live long, healthy lives.
Dr. McNew is both a St. Baldrick’s researcher and a shavee. He rocked the bald at a Cedar Rapids, Iowa event in 2015.
For Dr. Brandon McNew, treating kids with cancer isn’t just a professional calling. It’s personal.
Honored Kid Brooke was diagnosed with PH+ acute myeloid leukemia in 2015. She is now a survivor, but that doesn’t mean life is easy, ‘normal’ or back to a fraction of what it was like before cancer. Brooke explains…
(Left) Brooke during treatment after her 2015 diagnosis. (Right) Brooke poses for a photo during her first day back at school this year.
Cancer survivorship isn’t pretty. When I was diagnosed, I imagined that if I survived, my life after cancer would somehow be sweeter. Maybe I would appreciate the little things more or unlock some secret wisdom that would render me happier, more peaceful. This was believable through my first few rounds of chemo.
Then, I had a bone marrow transplant and became so ill that I spent five months inpatient post-transplant.
Spunky, determined, and positive, 12-year-old Lily is a childhood cancer survivor in a family continually facing the disease. Read on for more of her inspirational story and see how this two-time shavee℠ is facing cancer survivorship head on and helping other kids like her.
Lily with her mom, Jennifer, during Lily’s 2014 shave.
For the Mallory family, hope is a curly-haired, 11-year-old girl named Lily.
In 2008 at the age of 3, Lily was diagnosed with two cancers — an adrenal cortical carcinoma and a sarcoma in her leg. Years later, her mom was diagnosed with two cancers too — breast cancer and sarcoma in her arm. The breast cancer has since metastasized to her bones, lungs and brain.
“There’s only so much you can do, but you could always be that one. You could be the Lily that defies all the odds,” said her mother, Jennifer.
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