Childhood Cancer

Kymriah’s Journey From Lab to Lifesaver: FDA Approval and Beyond [Part Four]

by Erinn Jessop, St. Baldrick's Foundation
January 19, 2018

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.

A collage of Austin and Ori

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.

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Childhood Cancer

Kymriah’s Journey From Lab to Lifesaver: The Phase 2 Trial [Part Three]

by Erinn Jessop, St. Baldrick's Foundation
January 11, 2018

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 smiles in bed

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.

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Families

Kymriah’s Journey From Lab to Lifesaver: The Phase 1 Trial [Part Two]

by Erinn Jessop, St. Baldrick's Foundation
November 22, 2017

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.

Austin in his hospital bed

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.

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Families

Kymriah’s Journey From Lab to Lifesaver: The Phase 1 Trial [Part One]

by Erinn Jessop, St. Baldrick's Foundation
November 21, 2017

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.

Austin wears a face mask

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.

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