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.
Usually, we pick one international winner of the Robert J. Arceci Innovation Award, but what happens when there are two equally deserving researchers with big ideas and big hearts for kids with cancer? Read on to find out!
After being nominated for the International Robert J. Arceci Innovation Award, (left) Dr. Franck Bourdeaut and (right) Dr. Jan-Henning Klusmann were both selected by a committee of experts and are being presented with the award today at the annual conference for the International Society of Paediatric Oncology.
Dr. Robert Arceci was a passionate innovator who dreamed big. He was a pioneer who knew that kids with cancer deserve better than what doctors can offer them and that breakthroughs are born from taking risks.
That’s why the international winner of the award established in his memory – the Robert J. Arceci Innovation Award – is given the resources and the freedom to follow their curiosity, pioneering spirit, and their passion for kids’ cancer research, wherever it leads.
Except this year, it’s winners of the Robert J. Arceci Innovation Award!
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.
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?
There’s nothing sweeter than a reunion with someone you love. Nancy Swart knows that better than anyone. Earlier this month, she had the happiest of reunions with her son Zach, who has been away from home to get treatment for leukemia. Read on for more from Nancy about the time they spent together, how Zach is doing since his bone marrow transplant and the news that made him smile.
Nancy and Zach have a special moment together.
Zach’s dad, Tom, has been living with Zach in an apartment we have been renting in New York City, so that we can be close to the hospital for follow-up visits. It has been great for Tom to be able to spend time with Zach and I know Zach has needed some quality time with dad, so it has been a nice change to get some male bonding time.
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.
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.
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.
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.
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.
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