With its recent commitment of $500,000 for the Target Pediatric AML initiative, the St. Baldrick’s Foundation adds another chapter to its long story of support for innovative and impactful research in childhood acute myeloid leukemia (AML).
While great progress has been made over many decades to help children survive the most common childhood cancer – acute lymphoblastic leukemia (ALL) – the same has not held true for children with AML.
St. Baldrick’s is helping change that.
A child’s eyes see the love in their parents’ faces and the joy of their siblings. They see the vibrant colors in a box of crayons and the sparkle of rain on a flower. Sight helps kids navigate their classroom, their playground and their world. But what happens when pediatric cancer attacks that precious sense?
With retinoblastoma – an eye cancer in children that is usually diagnosed before the age of 3 years old – a kid can lose their vision to the cancer and their long-term health to the harsh treatment. They can even lose their lives.
That’s what tumor immunologist Dr. Vanessa Morales-Tirado and her St. Baldrick’s Summer Fellow, Zachary Goldsmith, are working to change.
Dr. Vanessa Morales-Tirado, with the University of Tennessee, works with Zachary Goldsmith, a St. Baldrick’s Summer Fellow and PhD candidate, in the lab.
What happens when you give a researcher funding and freedom to follow the science?
Just ask the Robert J. Arceci Innovation Award winner Dr. Kimberly Stegmaier. For her, that powerful combination of funding and freedom led to the discovery of a promising combination of molecules that could change the lives of kids with Ewing sarcoma, the second most common bone cancer found in children.
Jason Yustein, M.D., Ph.D., a St. Baldrick’s Scholar at Baylor College of Medicine in Houston, Texas, explains Ewing sarcoma symptoms, treatment options, and research opportunities.
What is Ewing sarcoma?
Ewing sarcoma is a type of childhood cancer that is most frequently found in children and adolescents between the ages of 10 and 20 years old.
Ewing sarcoma — the second most common bone cancer after osteosarcoma — often originates in the long, large bones of the body, including the hip, thigh, shin, chest, and arm bones.
This month is the official start of spring. Flowers are beginning to bloom, birds are chirping and a shiny new flock of doctors and medical students are about to blossom into childhood cancer researchers. Yep, it’s that time – it’s time to award $2 million in Summer Fellow and Fellowship grants!
These grants encourage and support the newest generation of doctors and students in pursuing childhood cancer research, so they can move the field forward and save the lives of the next generation of kids.
Childhood cancer knows no borders – but neither does research. In fact, funding kids’ cancer research saves the lives of kids across the globe. This International Childhood Cancer Day, meet Honored Kid Shauntelle, a 19-year-old from Ireland who left everything familiar behind so she could have her best chance at life.
Thanks to a successful fundraising campaign, Shauntelle and her family were able to move from their home in Ireland to Houston, Texas for cancer treatment.
Honored Kid Shauntelle lives about an hour south of the city of Dublin in Ireland. Four years ago, when she was 15 years old, she noticed rashes popping up on her body. She thought she was scrubbing too hard in the shower or was allergic to her body wash.
“For a while, I just didn’t think to speak up. It was a big pain and a big part of my life, but it was a bit embarrassing,” Shauntelle said. “I wasn’t sure if I was doing something wrong.”
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.
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.
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.
McKenna Claire was 7 years old when she was diagnosed with a rare brain tumor called DIPG. The McKenna Claire Foundation was established in her memory and in 2013, St. Baldrick’s partnered with the McKenna Claire Foundation to fund DIPG research, like the work done by Dr. Rameen Beroukhim at the Dana Farber Cancer Institute.
Honored Kid McKenna Claire was bright, spirited, and loved soccer and gymnastics. McKenna was full of grace, joy and grit through it all, even as her childhood cancer progressed and she could no longer run across a soccer field, jump on a trampoline, talk or swallow. She died just six months after her diagnosis with a rare, fatal type of brain tumor called DIPG – weeks before her birthday. She would have been 8 years old.
Stories like this are why St. Baldrick’s researcher Dr. Rameen Beroukhim studies DIPG, otherwise known as diffuse intrinsic pontine glioma. In fact, McKenna’s photograph hangs in his lab.
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