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?
‘Pura Vida!’: Phineas’ Summer in Costa Rica
Ambassador Phineas’ dad, Carlos, shares what the family has been up to this past summer, and he looks back at where their family was 10 years ago — and where they might be now if it weren’t for the immunotherapy clinical trial that saved Phineas’ life.

Ten years ago I was nearing the end of the worst summer of my life.
‘My One Ticket to Live On’: How a Clinical Trial Saved Mitch Carbon’s Life
Like most college freshmen, Mitch Carbon is excited to be getting a fresh start. But unlike his peers, just two years ago, he didn’t think he’d live to see this day. Read on for more of Mitch’s story and the clinical trial that saved his life — all made possible by YOU.
When Mitch Carbon was a junior in high school, he was preparing to die.
The Dream Team’s First Year
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).

The review took place at The Children’s Hospital of Philadelphia (CHOP), and reporting for the Dream Team were co-leaders Dr. John M. Maris, director of the Center for Childhood Cancer Research at CHOP, and Dr. Crystal Mackall, chief of the Pediatric Oncology Branch of the National Cancer Institute (NCI).
The review panel included representatives from Stand Up to Cancer (SU2C), our partner in making this $14.5 million grant; the American Association for Cancer Research (AACR); and of course St. Baldrick’s.
I am excited to report that this Dream Team seems to be making more rapid progress than most. This is partly due to the fact that researchers in childhood cancer are more accustomed to working with colleagues from multiple institutions than are researchers in the adult oncology world.
The Dream Team’s Progress Explained by a Non-Scientist
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).
One year ago today, the St. Baldrick’s Foundation announced the Stand Up to Cancer – St. Baldrick’s Pediatric Cancer Dream Team, a group of the best and brightest pediatric oncology researchers focused on developing innovative therapies for childhood cancers. Becky Weaver, chief philanthropy officer of the St. Baldrick’s Foundation, shares this update on the Dream Team’s work.

Dr. Crystal Mackall, co-chair of the Stand Up to Cancer – St. Baldrick’s Pediatric Cancer Dream Team, speaks at the Stand Up to Cancer Scientific Summit.
To learn about the Dream Team’s first six months of progress, I went to the Stand Up to Cancer (SU2C) Scientific Summit in Pasadena, California. The St. Baldrick’s Foundation is the first pediatric cancer organization to partner with SU2C to fund a Dream Team. Representatives from other funding partners were there, too.
There we sat in a room full of people so smart they made my head spin. So if they can make me understand what’s going on, I can pass it along and you’ll be just fine.
A Six-Month Childhood Cancer Research Progress Report 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).
Dr. Crystal L. Mackall and Dr. John M. Maris, co-chairs of the Stand Up to Cancer – St. Baldrick’s Pediatric Cancer Dream Team, explain the Dream Team’s children’s cancer research goals and how they’ve been working to meet them.

Cure rates for childhood cancer haven’t improved for the last 20 years, and for some childhood cancers, less than 20% of patients survive. Current treatments for childhood cancer often cause lifelong side effects. We need new, more effective treatments for childhood cancers.
Cancer genomics is the field of research designed to define the genetic reasons that cancer arises in the first place, and behaves aggressively in some patients.
Immunotherapy is an approach to treating cancer that harnesses the power of the body’s immune system to kill cancer cells.
Pediatric cancer researchers in both fields have made exciting recent advances, but historically these have occurred in parallel with little cross-fertilization.
The St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team is made up of researchers from seven institutions in North America whose goal is to create new immune-based therapies for pediatric cancer based on the individual patient’s tumor genomics and what makes the cancer cells different from the rest of the body.
NCI Meeting Explores Barriers to Drug Development for Pediatric Cancers
Why are there so few drugs available to fight childhood cancers? Why have no new drugs been developed and approved specifically for pediatric cancers in decades?
These questions dominated the agenda on February 21 when the National Cancer Institute (NCI) convened the 64th meeting of its Director’s Consumer Liaison Group in Bethesda, Maryland. Entitled “Barriers to Drug Development in Pediatric Cancer Research,” the all-day meeting included presentations from oncologists, government scientists, physicians, and members of the childhood cancer advocacy community.
What Is Genomics?


Dr. Parsons, a genomics expert on the Stand Up To Cancer – St. Baldrick’s Pediatric Cancer Dream Team*.
For example, we know that childhood cancer is caused by genetic mutations. What we don’t know is how or why most of those mutations occur.
And we’re still trying to figure out what the mutations mean — in terms of the cancer and its ability to thrive, and in terms of our bodies and their ability to overcome disease.
That’s the focus of genomics, explains Dr. Donald Parsons, the principal investigator at Baylor College of Medicine for the Stand Up To Cancer – St. Baldrick’s Pediatric Cancer Dream Team*.
“Genomics is the study on a large scale of all the genetic changes that occur in a patient’s DNA,” Dr. Parsons says. “It really tries to look at — in a single patient or across a group of patients — all the different changes that occur and how they might interact with each other.”
New Grants Help Fight Childhood Cancers
St. Baldrick’s adds $2.2 million in grant funding for childhood cancer research.

You made it possible for St. Baldrick’s to fund $2.2 million in new grants at 39 institutions across the United States. Combined with grants awarded earlier this year, St. Baldrick’s has given over $24.5 million to childhood cancer research in 2013, including the Stand Up To Cancer-St. Baldrick’s Pediatric Cancer Dream Team**.
46 Mommas Then and Now: Curing Kids’ Cancer Together

The first 46 Mommas St. Baldrick’s event in 2010.

Tiffany’s daughter, Miranda, in 2007, shortly before she was diagnosed with childhood cancer; Miranda during treatment.
As the months of treatment passed, just getting through each day was challenging: Miranda’s body didn’t process all the antibiotics she got to help fight infection, her kidneys took a serious beating, she stopped eating, didn’t tolerate a feeding tube in her nose. I watched as my daughter’s little body shrank smaller and smaller as she lost one-third of her weight. I feared that the chemo fighting the cancer was going to kill her instead.
It was hard for to me rejoice when the doctor told me that, after three rounds of chemo, the tumor had shrunk by 75%, because I felt my baby was dying before my eyes.
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