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.
Meet the St. Baldrick’s Innovation Award Winners
What do researchers Dr. Alex Huang and Dr. Carl Allen have in common? Passion, curiosity, drive, brilliant ideas, a desire to help kids — the list goes on! And now there’s something else. They are both recipients of the first St. Baldrick’s Innovation Award. What do they want to do with this unique grant? Read on to find out.
Dr. Carl Allen (left) is an associate professor at Texas Children’s Cancer Center and one of the investigators involved in the North American Consortium for Histiocytosis (NACHO), which received a St. Baldrick’s Consortium Grant. St. Baldrick’s researcher Dr. Alex Huang (right) is a professor of pediatrics at Case Western Reserve University School of Medicine and a 10-time shavee with St. Baldrick’s.
St. Baldrick’s researchers Dr. Alex Huang and Dr. Carl Allen work on different projects, in different labs about 1,300 miles away from each other.
Dr. Huang primarily studies how immunotherapy can help kids with cancer, while Dr. Allen studies Langerhans Cell Histiocytosis or LCH, which is caused by out-of-control immature white blood cells. The disorder can cause inflammatory tumors, damage organs and even cause brain degeneration in some patients.
The two researchers may work in different areas on different projects, but since the start of their careers in medicine, they’ve shared a goal — to help sick kids get better. And now they have something else in common.
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.
Teaching Good Cells to ‘See’ Cancer: Researcher Uses CAR T Cells to Help Kids Like Zach [VIDEO]
From CAR T cell therapy in May to a bone marrow transplant in June, over the past few weeks we’ve been following the tremendous journey of Honored Kid Zach Swart. Now we bring you another angle — a closer look at the St. Baldrick’s-funded research that has changed Zach’s life.
St. Baldrick’s researcher Dr. Kevin Curran meets with a patient at Memorial Sloan Kettering Cancer Center.
A couple months ago, Zach Swart went through yet another medical procedure; one more to add to an already substantial history of biopsies and blood draws.
But this procedure was different than a typical needle poke.
A Life Saved in Six Minutes: An Update on Zach [VIDEO]
Last week, we shared Honored Kid Zach’s amazing story and the news that the three-time cancer fighter was going to get a bone marrow transplant — a procedure that could put the 15-year-old into remission for good. Today, about a week after the transplant, Zach’s mom has an update.
The family gathers before Zach’s bone marrow transplant. From left to right: Nancy, Ben, Tom and Zach, with Gabe on Facetime.
My son, Zach, is fighting cancer for the third time, which means this is the third time going through tests, treatments and all the uncertainties that follow. Zach had three months of intense chemo therapy to try to get him into remission before his bone marrow transplant or BMT. At the end of his treatments, through numerous hospitalizations, we learned that Zach was not in remission.
How Immunotherapy Saved Phineas [VIDEO]
“Our son would not be with us today if it weren’t for St. Baldrick’s,” says Phineas’ dad, Carlos. Read on to see how research saved the little boy’s life.
VIDEO: Phineas’ Story >
On a mountain bike ride with a friend, 9-year-old Phineas was sailing along when he decided to take a risk and pedal over a bridge not meant for bicycle traffic. He wiped out in a big way.
But without so much as a single tear, he picked himself up, dusted himself off, and got back on the bike.
Compared to what this boy had been through two years before, that was nothing.
Childhood Cancer Stories: My Day With Ryan [PHOTO ESSAY]
Honored Kid Ryan C. faces childhood cancer with a contagiously positive attitude — and our social media manager, Alison Sutton, got to experience this firsthand when she hung out with him last month! Read on to see what life is like for Ryan and bask in the glow of this amazing kid, his awesome family, and their St. Baldrick’s fundraising team — Team Rally for Ryan.
St Patrick’s Day is a special day at the St. Baldrick’s Foundation because 17 years ago, our founders hosted their first head-shaving event!
This St. Patrick’s Day I spent the day at Arnold Palmer Children’s Hospital in Orlando, Fla. with Honored Kid Ryan C. to see what a day in his life is like.
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.
The Kid With Superhero Cells: Leon’s Immunotherapy Story
Honored Kid Leon is one tough cookie. He’s fought childhood cancer not just once, but twice, and this time, Leon and his family hope it’s gone for good — thanks to an immunotherapy trial run by Leon’s buddy Dr. Daniel Lee, an investigator with the Stand Up to Cancer – St. Baldrick’s Dream Team*.
Leon and St. Baldrick’s researcher Dr. Daniel Lee share some smiles.
Everything changed one hot summer day in Colorado. Leon was spraying his cousins with a water gun and playing with the hose. Then the 9-year-old slipped on the slick deck and fell hard.
It was a badly bruised hip, said the doctors in the emergency room. It will heal. But it didn’t. Leon’s grandmother, Lisa, watched her normally active grandson walk gingerly and even resort to crutches.
Then she watched Leon get tired more quickly than a kid should. And then she watched him sleep. He slept and slept.
“And I knew then,” Lisa said. “I was like, ‘I think he’s sick again.’”
Destroying the Defenses: Researchers Fight Brain Tumors With Immunotherapy
St. Baldrick’s Scholars Dr. Alex Huang and Dr. Agne Petrosiute are studying how switching off a protein could lead to new treatments and cures for kids with brain tumors. Read on for more about their unexpected discovery, its implications for immunotherapy, and why Dr. Huang compares himself to those fuzzy little bears in Star Wars.
Dr. Agne Petrosiute (left) and Dr. Alex Huang study how the immune system can be harnessed to fight pediatric brain cancer.
Dr. Alex Huang likens himself and his colleague, Dr. Agne Petrosiute, to Ewoks battling the Death Star.
“We are the Ewoks that found the controller on this planet, and all of a sudden the Death Star cannot put up the shield anymore,” he said. “And so now, Luke Skywalker can go in there and blow it up.”
It may seem like a curious explanation, but it fits.
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