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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Clinical Trials: Sharing of Data and Living Up to Our End of the Bargain

Discussing clinical trials

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Today we took a huge step forward in our efforts to make sure that data from biomedical research is shared widely and rapidly. The NIH, in collaboration with our fine colleagues at the U.S. Food and Drug Administration (FDA), and with the valuable input from scientists, patients and other members of the public, has announced the HHS regulation and NIH policy to ensure that information about clinical trials is widely shared. In this blog I want to talk about what this will mean for patients, providers, and researchers. I also want to reflect a bit on how the new regulation and policy fit into our overall efforts to improve clinical trials and data sharing.

Clinical trials are essential for the translation of research advances to new approaches to prevention and treatment. Volunteers who take part in clinical trials often do so with no assurance of personal benefit, but with the expectation that their involvement will add to the growing body of knowledge about health and disease, and thus may help others someday. For that to be realized, all trial results information needs to be publicly reported in a timely fashion—and yet we know that doesn’t always happen. Today’s announcements aim to change that. The HHS regulation issued today, called a “final rule”, describes requirements for registering certain clinical trials and submitting summary results information from these trials to ClinicalTrials.gov, a database managed by NIH’s National Library of Medicine (NLM).

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Honoring Our Promise: Clinical Trial Data Sharing

Clinical Trials Data Sharing Word CloudWhen people enroll in clinical trials to test new drugs, devices, or other interventions, they’re often informed that such research may not benefit them directly. But they’re also told what’s learned in those clinical trials may help others, both now and in the future. To honor these participants’ selfless commitment to advancing biomedical science, researchers have an ethical obligation to share the results of clinical trials in a swift and transparent manner.

But that’s not the only reason why sharing data from clinical trials is so important. Prompt dissemination of clinical trial results is essential for guiding future research. Furthermore, resources can be wasted and people may even stand to be harmed if the results of clinical trials are not fully disclosed in a timely manner. Without access to complete information about previous clinical trials—including data that are negative or inconclusive, researchers may launch similar studies that put participants at needless risk or expose them to ineffective interventions. And, if conclusions are distorted by failure to report results, incomplete knowledge can eventually make its way into clinical guidelines and, thereby, affect the care of a great many patients [1].

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Meet Alex—Before and After NIH Clinical Trial

Photo of an infant with mottled skin adjacent to a photo of young man with clear skin being examined by a female doctor.

Caption: Alex, then and now, with Dr. Goldbach-Mansky
Credit: Kate Barton and Susan Bettendorf (NIH)

Alex Barton recently turned 17. That’s incredible because Alex was born with a rare, often fatal genetic disease and wasn’t expected to reach his teenage years.

When Alex was born, he looked like he’d been dipped in boiling water: his skin was bright red and blistered. He spent most of his time sleeping. When awake, he screamed in agony from headaches, joint pain, and rashes. After a torturous 14 months, a rheumatologist told his mother that Alex suffered from Neonatal-Onset Multisystem Inflammatory Disease (NOMID). The doctor showed her a brief and scary paragraph in a medical text. Kate Barton, Alex’s mother, admitted that it “knocked her over like a freight train.” Continue reading