Pediatric Cancer Dream Team Works Toward More Breakthroughs
When the St. Baldrick’s Foundation — Stand Up 2 Cancer Pediatric Cancer Dream Team was created in 2013, the idea was to pursue breakthroughs, specifically in the area of immunotherapy enabled by the application of modern genomic technologies. Now, with St. Baldrick’s as the primary funder and each member institution also investing financially — the Dream Team looks to build on the tremendous momentum in immunotherapy for childhood cancers.
When Research Offers Hope, Parents Continue to Advance the Mission
I was recently invited to serve as a patient family advocate for the St. Baldrick’s / Stand Up to Cancer Pediatric Cancer Dream Team representing the National Cancer Institute. If you’re not familiar with the Dream Team and what they’re doing, it’s worth taking a few minutes to read up on the project.
In short, it’s a multi-institutional effort to accelerate cures for childhood cancer by sharing the skill, knowledge and unique resources of 8 top-notch research institutions.
Carlos Sandi with his son, Honored Kid and Ambassador Phineas.
New Immunotherapy Breakthrough Could Bring Hope to Kids With DIPG
Honored Kid Madelyn was diagnosed with DIPG in 2010. A fashionista with a big spirit and an equally big heart, Maddy was an inspiration to those around her. She passed away in November 2011 after an 18-month battle with DIPG.
No child has ever survived a diffuse intrinsic pontine glioma brain tumor, commonly called DIPG.
This aggressive pediatric brain cancer multiplies in the brainstem, which controls some of our most basic functions for living – our breathing, heart rate, blood pressure and ability to swallow and speak. And because the cancer is so intertwined with its delicate surroundings, DIPG is often inoperable.
A DIPG diagnosis and a zero percent survival rate has remained the reality for kids and families for decades.
But what if there might be hope? What if that hope came from within the child’s own body?
The Dream Team is Back — Thanks to You!
I have something exciting to tell you, but first, here’s something I heard and quickly scribbled down during a meeting about research priorities: “It’s like we caught lightning in a bottle – and you just don’t know when that will happen again.”
That was one of our expert advisers talking about the amazing, incredibly fast and impactful work of the Stand Up to Cancer – St. Baldrick’s Pediatric Cancer Dream Team, funded jointly by the two organizations from 2013 through 2017. It was such a perfect description, it has stuck with me for more than a year.
This team of 150 researchers from eight institutions across North America has been working on new immunotherapy treatments for childhood cancers. In less than four years, their work is already saving lives.
Honored Kid Austin was diagnosed with a high-risk form of acute lymphoblastic leukemia when he was 2 years old. Now a survivor, he was one of the first kids treated on a trial of the immunotherapy Kymriah, which was developed with help from the Dream Team.
Every one of the experts who had looked closely at the team’s work agreed that this was work that must continue. To me, it was only natural for St. Baldrick’s to make that happen. And by St. Baldrick’s, I mean you – our donors and volunteers who make the funding possible.
So, I’m thrilled to announce that the Dream Team is back – with a new name and a continuing determination to find cures for childhood cancers.
The St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team will continue from 2018 through 2022, with primary support from St. Baldrick’s.
Kymriah’s Journey From Lab to Lifesaver: FDA Approval and Beyond [Part Four]
From its inaugural Phase 1 clinical trial through its successful Phase 2 trial, we have traced the path of Kymriah, a recent immunotherapy and gene therapy breakthrough for kids with high-risk leukemia and few options, like Honored Kids Austin and Ori. (Read the whole series here.) Now, with a historic decision made in August, we are at the end of our story, but not at the end of the story of Kymriah. Instead, Kymriah’s is just beginning – and starting a new chapter of hope for kids with cancer.
Austin (left) and Ori (right) are both alive today thanks to Kymriah and their participation in the Phase 1 and Phase 2 trials.
On August 30, 2017, big news rippled through the childhood cancer community. On that late summer day – before the start of Childhood Cancer Awareness Month – the FDA made a historic move that changed the landscape of childhood cancer research forever.
Kymriah’s Journey From Lab to Lifesaver: The Phase 2 Trial [Part Three]
Over a series of four blogs — catch up with parts one and two about the Phase 1 trial — we are tracing the path of Kymriah, a recent immunotherapy and gene therapy breakthrough for kids with high-risk leukemia, like Honored Kid Ori.
After relapsing for the second time and with his cancer spreading to his nervous system, Ori’s best chance at life was a Phase 2 trial of this experimental CAR T cell therapy. With a sunny attitude and staggering strength of spirit, Ori gave this new treatment a shot – with astonishing results.
Ori was in cancer treatment for much of his young life and throughout the journey, his strength and positive attitude have been remarkable. “He has been through so much, but has done it all with a great attitude and a smile on his face,” said his mom, Kaye.
When a child with cancer relapses the first time, their treatment options shrink. But when a child with cancer relapses again, their options and chances at survival don’t just shrink – they’re nearly extinguished. That is what happened to Ori.
St. Baldrick’s Top 10 Highlights of 2017
2017 was a big year, full of breakthroughs, incredible stories and some pretty amazing achievements in the childhood cancer world. Join us as we reflect on St. Baldrick’s top 10 highlights of the past year — and make sure you give yourself a pat on the back, because much of this was possible because of YOU!
Are you ready to take a trip down memory lane? Here we go …
1) Passage of the RACE Act
Kymriah’s Journey From Lab to Lifesaver: The Phase 1 Trial [Part Two]
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’s Journey From Lab to Lifesaver: The Phase 1 Trial [Part One]
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.
Dream Team Discovers Potential New Weapon Against Neuroblastoma
Last week, in the pages of the medical journal Cancer Cell, St. Baldrick’s researchers announced a discovery that could radically transform treatment for kids with neuroblastoma – a new immunotherapy drug candidate that harnesses the immune system to fight cancer.
Neuroblastoma is a cancer that begins in the nerve tissue outside the brain, usually in a child’s abdomen. It strikes very young children, up to about age 7, and is the most common cancer diagnosed in infants. Only about 50% of patients survive the high-risk form of neuroblastoma.
All of this makes this new targeted immunotherapy for neuroblastoma especially good news, but it gets even better.
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