FDA Approves First CAR-T Cell Therapy for Pediatric Acute Lymphoblastic Leukemia

Emily Whitehead

Caption: Cancer survivor Emily Whitehead with her dog Lucy.
Credit: Emily Whitehead Foundation

Tremendous progress continues to be made against the Emperor of All Maladies, cancer. One of the most exciting areas of progress involves immunotherapy, a treatment strategy that harnesses the natural ability of the body’s own immune cells to attack and kill tumor cells. A lot of extremely hard work has gone into this research, so I was thrilled to learn that the Food and Drug Administration (FDA) just announced today its first approval of a promising type of immunotherapy called CAR-T cell therapy for kids and young adults with B-cell acute lymphoblastic leukemia (ALL)—the most common childhood cancer in the U.S.

ALL is a cancer of white blood cells called lymphocytes. Its treatment with chemotherapy drugs, developed with NIH support, has transformed ALL’s prognosis in kids from often fatal to largely treatable: about 90 percent of young patients now recover. But for those for whom the treatment fails, the prognosis is grim.

In the spring of 2012, Emily Whitehead of Philipsburg, PA was one such patient. The little girl was deathly ill, and her parents were worried they’d run out of options. That’s when doctors at Children’s Hospital of Philadelphia gave Emily and her parents new hope. Carl June and his team had successfully treated three adults with their version of CAR-T cell therapy, which is grounded in initial basic research supported by NIH [1,2]. Moving forward with additional clinical tests, they treated Emily—their first pediatric patient—that April. For a while, it was touch and go, and Emily almost died. But by May 2012, her cancer was in remission. Today, five years later, 12-year-old Emily remains cancer free and is thriving. And I’ve had the great privilege of getting to know Emily and her parents over the last few years.

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NIH Family Members Giving Back: Toben Nelson

Roseville Raiders

Caption: Toben Nelson (back row, far left) celebrates with his Roseville Raiders after winning Gopher State Tournament of Champions.
Caption: Heather Hammond Nelson

What was Toben Nelson, a University of Minnesota epidemiologist who studies the health risks of alcohol abuse and obesity, doing this summer lugging around a heavy equipment bag after work? Giving back to his community. Nelson volunteered as a coach for the Roseville Raiders, a 13-year-old-and-under traveling baseball team that just wrapped up its season by winning the prestigious Gopher State Tournament of Champions in their age group.

In the fall, Nelson will gear up for hoops as the volunteer president of the Roseville Youth Basketball Association, which provides an opportunity for kids in this Minneapolis-St. Paul suburb to take part in organized sports. Nelson says volunteering grounds him as a scientist. It reminds him every single day that his NIH-supported research back at the office affects real lives and benefits real communities like his own.

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NIH Family Members Giving Back: Rebecca Shlafer

Rebecca Shlafer

Rebecca Shlafer/Credit: Brady Willette

When Rebecca Shlafer clicks on her office lights each morning at the University of Minnesota Medical Center, Minneapolis, she usually has a good idea of what to expect from the day ahead as lead of a nine-person research team that studies the effects of incarceration on children and families. It’s her volunteer work that can be unpredictable.

For the past eight years, this developmental child psychologist has donated her free time to serve as a guardian ad litem for abused or neglected children who’ve been removed from their homes and placed under protective supervision of Minnesota’s Fourth Judicial District. In that volunteer capacity, Shlafer advocates in court for the well-being of the child, but doesn’t foster the youngster or provide any day-to-day care.

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NIH Family Members Giving Back: Kafui Dzirasa

Kafui Dzirasa at UMBC

Caption: Kafui Dzirasa (front center) with the current group of Meyerhoff Scholars at University of Maryland, Baltimore County.
Credit: Olubukola Abiona

Kafui Dzirasa keeps an open-door policy in his busy NIH-supported lab at Duke University, Durham, NC. If his trainees have a quick question or just need to discuss an upcoming experiment, they’re always welcome to pull up a chair. The donuts are on him.

But when trainees pop by his office and see he’s out for the day, they have a good idea of what it means. Dzirasa has most likely traveled up to his native Maryland to volunteer as a mentor for students in a college program that will be forever near and dear to him. It’s the Meyerhoff Scholars Program at the University of Maryland, Baltimore County (UMBC). Since its launch in 1988, this groundbreaking program has served as a needed pipeline to help increase diversity in the sciences—with more than 1,000 alumni, including Dzirasa, and 270 current students of all races.

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How Can You Take Part in Clinical Research? Looking Beyond “First in Human”


For a remarkable journey through the front lines of clinical research, I’d like to invite you to join me in viewing First in Human, which premieres tonight at 9 p.m. ET on the Discovery Channel. This three-part docuseries, to be aired August 10, 17, and 24, provides an unprecedented look inside the NIH Clinical Center here in Bethesda, MD, following four of the many brave patients who’ve volunteered to take part in the clinical trials that are so essential to medical breakthroughs.

You’ll learn about what it’s like to take part in an experimental trial of a new treatment, when all standard options have failed. You’ll see that the NIH Clinical Center and its staff are simply amazing. But keep in mind that you don’t have to travel all the way to Bethesda to be part of outstanding, NIH-funded clinical research. In fact, we support clinical trials all across the country, and it’s often possible to find one at a medical institution near your home. To search for a clinical trial that might be right for you or a loved one with a serious medical problem, try going to ClinicalTrials.gov, a web site run by NIH.

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