A Peek Behind the Scenes: The St. Baldrick’s Grant and Scientific Review Process
Top: St. Baldrick’s researcher Akiko Shimamura (left) in her lab with a colleague at Fred Hutchinson Cancer Research Center in Seattle. During her St. Baldrick’s-supported study, Dr. Shimamura studied a rare group of inherited bone marrow disorders that are associated with heightened risk of cancer. Bottom: Based at the University of Medicine and Dentistry of New Jersey, St. Baldrick’s researcher Scott Kachlany tested a new therapeutic agent for kids with ALL during his St. Baldrick’s-supported study.
The St. Baldrick’s Foundation makes a bold statement: We support the most promising childhood cancer research, wherever it takes place. And because we grant more childhood cancer research than any non-government funder, it’s crucial that those dollars go to the right projects. But how do we pick the right projects?
Rising to that challenge is a widely respected grant application and scientific review process created by the St. Baldrick’s Foundation.
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
Formerly known as the St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team, this team is now the St. Baldrick’s EPICC Team (Empowering Pediatric Immunotherapies for Childhood Cancer).
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.
Immunotherapy Q&A with St. Baldrick’s Researcher, Dr. Grupp
We sat down virtually with St. Baldrick’s researcher, Stephan A. Grupp, MD, PhD, for a Q&A on immunotherapy. Dr. Grupp, located at Children’s Hospital of Philadelphia, is a key member of the Stand Up to Cancer – St. Baldrick’s Pediatric Dream Team*. He treated the first child with CAR T cell therapy, which was recently approved by the FDA for acute lymphoblastic leukemia (ALL). Check out our Twitter chat with Dr. Grupp and see what he thinks the future is for immunotherapy and childhood cancers.
What Is Immunotherapy?
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?
If Only There Had Been a Cure in Time for David
Honored Kid David was bright, charming and totally dedicated to embracing life. He also had childhood cancer. David asked his family to fight for a cure — a cure that didn’t come soon enough for him. Read on to learn more about this spectacular 10-year-old, his childhood cancer journey and why his mom, Susan, is passionate about research.
David hugs his mom, Susan, at a fundraising event.
Who would David be if he had grown up?
His mom can’t help but wonder. If he’d lived, he’d be a teenager now.
Would he still have that obsession with colorful skinny jeans? Would that cello he insisted upon getting still be gathering dust? Would he still be that bubbly, charismatic kid she remembers?
What if childhood cancer research hadn’t failed him?
That’s what Susan Heard wonders.
BIG NEWS: FDA Approves Groundbreaking CAR T Cell Therapy for Kids With Leukemia
Formerly known as the St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team, this team is now the St. Baldrick’s EPICC Team (Empowering Pediatric Immunotherapies for Childhood Cancer).
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.
CAR T Cell Therapy and a Turning Point in Treatment: News From the Dream Team
Formerly known as the St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team, this team is now the St. Baldrick’s EPICC Team (Empowering Pediatric Immunotherapies for Childhood Cancer).
As one of the most exciting projects supported by St. Baldrick’s – the SU2C-St. Baldrick’s Pediatric Cancer Dream Team – nears the end of its funding, a group of expert reviewers met at Stanford University on July 14th to evaluate its impact to date. To say they were impressed would be an understatement.
Members of the Dream Team gather at a meeting in July to discuss the impact of their research efforts.
The day began with a report from Dr. Stephan Grupp, director of the Cancer Immunotherapy Program at Children’s Hospital of Philadelphia.
He had flown to the Dream Team meeting straight from the Maryland headquarters of the Food and Drug Administration (FDA), where hundreds had packed a hearing room the day before. An FDA advisory panel was considering what could soon be the first gene therapy to be marketed in the United States, pioneered by Dr. Grupp and his colleagues.
Stop, Collaborate and Listen: How Teamwork Makes the Dream Team Work
Formerly known as the St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team, this team is now the St. Baldrick’s EPICC Team (Empowering Pediatric Immunotherapies for Childhood Cancer).
When people come together for a cause, incredible things happen. That’s true for St. Baldrick’s head-shaving events AND for the lifesaving work done by St. Baldrick’s researchers, especially the Stand Up To Cancer – St. Baldrick’s Pediatric Dream Team. As they say, teamwork makes the dream work! Read on to learn more about how cooperation and sharing between these researchers means big advances for kids with cancer.
They say two heads are better than one. But what about 149? That is how many brilliant brains are working together to conquer childhood cancers as part of the SU2C – St. Baldrick’s Pediatric Cancer Dream Team.
And it’s that cooperation that’s accelerating progress for kids and laying the foundation for better treatments and for cures, said Dr. Crystal Mackall and Dr. John Maris, co-leaders of the Dream Team.
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