We have been hoping for and working toward this for five years. To not only have CAR-T cell therapy likely to be the first gene cell therapy approved by the FDA, but to also have it be approved first in pediatrics is very exciting. This is a step forward for pediatric cancer as new treatments are typically approved for adults first.

Our foundation’s mission is to advocate and provide research funding for treatments that are less toxic to children than the standard chemotherapy and radiation. CAR-T cell therapy is an example of this, and we hope such treatments will continue to be developed for other types of pediatric cancers.

After the final FDA approval, more children will be able to receive the CTL019 treatment outside of the clinical trial, opening the opportunity for more parents to take their children home healthy and cured of their cancer. We are so proud that our daughter Emily kept fighting and survived in order to give so many other patients hope and a new treatment so they too can live their lives cancer free.

Tom and Kari Whitehead, Co-founders

On Wednesday, July 12 the U.S. Food and Drug Administration's Oncologic Drugs Advisory Committee convened at a meeting to discuss the pending FDA approval of CAR-T cell therapy CTL019. Our co-founders, Tom, Kari and Emily Whitehead were present at the meeting, and Tom Whitehead spoke before the panel of experts during the afternoon's Open Public Hearing session.

The CTL019 treatment, known also as an experimental gene therapy or immunotherapy, is manufactured by Novartis. In May 2012, Emily Whitehead became the first child to receive the CAR-T cell treatment in the clinical trial at Children's Hospital of Philadelphia (CHOP).

In his speech, Tom shared Emily's story — from diagnosis, to being told that all remaining options had been exhausted, to calling CHOP as a last hope and entering the CTL019 trial. Tom focused also on Emily's fight following CAR-T cell infusion, referencing the cytokine release syndrome, or "storm", that made her very sick and the efforts of the team at CHOP to reverse this side effect.

Emily is now 5 years cancer free, the threshold for being considered "cured" of cancer. Since her pioneering treatment, many more kids have followed in the trial at CHOP and the doctors have been better prepared to handle the side effects of the storm. Dr. Stephan Grupp of Children's Hospital of Philadelphia also presented at the meeting, telling the advisory committee earlier in the afternoon that discovering the effective use of the drug tocilizumab to reverse the storm was a key finding and has since been used to treat more patients safely.

Tom said in his speech that, "Now when other parents enter their children into this treatment, they are more comfortable knowing that the doctors figured out a solution for Emily’s storm so quickly."

It has been a goal of mine for five years to some day speak at the FDA approval hearing and it was one of the most inspiring days of my life.

Such parents were also represented at the meeting. Amy Kappen spoke during the morning session’s Open Public Hearing, advocating on behalf of her daughter Sophia and the gift of a few extra months CAR-T cell therapy gave before Sophia passed away due to a mutation of her leukemia. She wanted the panel to know that their family is thankful to the doctors at CHOP, because while Sophia's cancer was too far progressed, the CTL019 treatment did give them more time to spend with their "spirited and strong daughter". Amy said in her speech: "CTL019 did what it was supposed to do for her bone marrow, but the mutation was too much," and also, "Our hope is to see this incredible treatment option available to children sooner. It is a remarkable therapy that spares children the toxic side effects of conventional chemotherapy."

Don McMahon of Connor's Hope spoke just before Tom in the afternoon about his son's journey to CAR-T cell therapy. Connor was diagnosed with Acute Lymphoblastic Leukemia (ALL) at age 3 and relapsed three times over 12 years before Don sought out the help of Tom Whitehead to get Connor into the CAR-T cell trial at CHOP. Connor was denied entry into the CHOP trial, but was accepted at Duke Children’s Hospital and received the same Novartis manufactured CTL019 treatment in October 2016. One of the most impactful slides Don presented was a side-by-side comparison on the effects that standard chemotherapy treatment vs. CAR-T cell therapy had for Connor. He pleaded with the advisory committee to adopt and approve this treatment, concluding that, “At the end of the day, that truly is Connor’s hope.”

Immediately following Don McMahon and Tom Whitehead's heartfelt testimonies, the advisory committee placed their votes. It was unanimous — 10 yes, 0 no.

The advisory committee's vote is not an official approval of the CTL019 treatment — that decision is to be expected of the FDA some time in September. You can read more about the decision in the Washington Post and The New York Times. [Read additional news coverage here]

Read more:

FDA Approves CAR-T Cell Treatment for Pediatric Patients with Acute Lymphoblastic Leukemia [August 2017]

Celebrating CAR-T Cell Therapy Advancements and Thirteen Years [May 2018]