The PR team at the Emily Whitehead Foundation, established in 2015, has been busy of late.
The month of June saw the premiere of Of Medicine and Miracles, a documentary about Emily Whitehead and directed by Academy Award winning filmmaker Ross Kauffman, which played at the Tribeca Film Festival in New York City.
Last week, July 15 to be exact, the Foundation held its 8th annual golf tournament fundraiser – “Tee Off for T-Cells” – in the Foundation’s home of Philipsburg, PA. I have yet to learn how the event did, but last year’s fundraiser raised over US$53,000 – contributing to a grand total of US$350,000.
And this week People magazine ran a feature on Emily Whitehead. If you didn’t know who Emily Whitehead is, there’s a good chance you do now.
This year marks the 10-year anniversary of Emily’s first infusion of CAR T cells to treat the acute lymphoblastic leukemia (ALL) she was diagnosed with at the age of five. Oncology Times describes her as follows: “She is the first pediatric patient in the world to receive CAR T-cell therapy for relapsed/refractory acute lymphoblastic leukemia (ALL). She is a singular figure in the annals of medicine. She is a soldier on the front lines of the war on cancer. And like the “shot heard round the world,” her personal medical assault sparked a revolution in cancer care that continues to power forward.” (Read blogger Sara M. Nolte’s post to learn about CAR T-cell therapy.)
Emily remains in remission today, thanks to the tremendous efforts and perseverance of Dr. Carl June and colleagues at the University of Pennsylvania, Dr. Stephan Grupp at the Children’s Hospital of Philadelphia, and her parents who refused to give up.
Dr. June’s pioneering research with CAR T cells for HIV/AIDS and cancer has brought him much acclaim and resulted in a “breakthrough therapy” designation from the Food and Drug Administration for CART19, which you can read about here. (Written by Dr. June, it’s a fascinating overview of his life and work, beginning with being drafted in the early 1970s but avoiding the Vietnam War, and continuing through to his trailblazing work at the University of Pennsylvania. As he recounts his past, he shares words of advice and important lessons he learned along the way and describes the “happy accident” that launched his studies “toward adoptive transfer of T cells for patients with HIV/AIDS” in the 1990s.)
Emily’s clinical trial was not a walk in the park. There was a period when Dr. June was convinced the treatment had failed and he expected the trial would have to be halted. But Dr. Grupp wouldn’t give up. As told by Dr. June in Oncology Times, “We treated Emily with tocilizumab out of desperation. Steve [Grupp] has told me that when he went to the ICU with tocilizumab as a rescue attempt for CRS, the ICU docs called him ‘a cowboy.’ The ICU docs had given up hope for Emily. But she turned around—unbelievably rapidly. Today, tocilizumab is the standard of care for CRS, and the only drug approved by the FDA for that complication. Emily’s recovery was huge for the entire field.”
From Dr. Grupp: “If things had gone differently, if Emily had experienced fatal toxicity, it would have been devastating to her family and to the medical team. And it might have ended the whole research endeavor. It would have set us back years and years. The impact that Emily and her family had on the field is nothing short of transformational.”
However, all the success and attention for CAR Ts has unfortunately resulted in an overcrowded market. As reported in Fierce Pharma, “The pipeline of personalized medicines for blood cancers is fit to burst.” Data and analytics company Global Data indicates there are 800 cell therapy prospects for just five major blood cancers and Dr. Sakis Paliouras, with Global Data, told Fierce Pharma that “If all of the in-development drugs for blood cancer got the go-ahead from the FDA, competition would be much too fierce.”
While so many products may sound like good news to patients, it’s a problem that the industry will have to contend with, including insufficient manufacturing capacity to meet demand.
Not wanting to end on a negative note, it’s incredible the progress we have seen with CAR T-cell therapy in a decade. With Dr. June and his colleagues tackling acute myeloid leukemia and solid tumours, one expects we will hear of other breakthroughs soon.
From Oncology Times: “In addition to targeting different types of cancer, June said contemporary research is also exploring the use of different types of cells. ‘Our initial CAR T trial used T cells, and that is what all the FDA-approved CARs are. But we now have trials using different cell types, like natural killer cells, monocytes, and stem cells. An entirely new field has opened because of our initial success. This is going to continue for a long time, making more potent cells that cover all kinds of cancer, not just leukemia and lymphoma.’”
Even with everything I know about Emily, her family, Drs. June and Grupp, I am still curious to watch Kauffman’s documentary. Although live showings have ended, it could end up on a streaming service near you. You can watch a clip from the film below.
Stacey Johnson
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