PCMS_Philadelphia_Medicine_Spring2018 - 7

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"This therapy is a significant step forward in individualized cancer
treatment that may have a tremendous impact on patients' lives."
Carl June, MD, the Richard W. Vague Professor of Immunotherapy, Director of the Center for
Cellular Immunotherapies in Penn's Perelman School of Medicine

One of the things Dr. June
does for fun is ride his
bike, but definitely not the
way most of us do. When I
talked to him he was getting
in condition for the Dirty
Kanza 200 - a 200-mile race
through the Flint Hills of
Central Kansas.
the research that led to his fame. I learned about his unwavering
perseverance and determination through times when things were
going poorly, very poorly, when he no longer had funding, when
he had to lay off people, when he thought he would have to close
the lab. I learned how his diverse background and research training
in different areas of medicine were integral to him developing his
current research and finally putting it all together.

into a complete remission, and
most excitingly, nearly six years
later, she remains disease free.

Chimeric antigen receptor
T-cells or CAR T-cells work by
modifying the patient's own
immune system to fight cancer. CAR T-cells are created by first
I was amazed at how his knowledge in other areas of medicine removing T-cells from the cancer patient. These T-cells are then
allowed him to critically treat one of the major, potentially life-threat- changed by infecting them with a virus that contains the genetic
ening side effects of CAR T-cell therapy. I learned of the critical information for the T-cell to make a new type of receptor on its cell
importance of donor support at a time when the NIH (National surface that provides the information to identify the tumor cells,
Institute of Health) budget, the biggest source of most basic science in this case the CD19 receptor that was expressed on Emily's ALL
research in the United States, continues to be insufficient to support cells. The virus also contains information to activate the T-cells,
groundbreaking research. Dr. June also confirmed what I already programming them into their killing mode. These activated T-cells
knew - our academic institutions are vital in their support and are like super-charged killer cells on steroids. They are multiplied
development of this pioneering research. Without the support of in the lab and then infused back into the cancer patient. These
our academic institutions, this new lifesaving therapy would not cells then seek out and destroy the acute lymphoblastic leukemia
be available. "Disruptive advances," he said, "in biomedicine most B-cells, which are covered by the CD19 protein. According to Dr.
often occur in academia and not in the pharmaceutical industry." June, one of these cells and daughter progeny CAR T cells are
One of the great breakthroughs for his research came in 2012. capable of killing 100,000 cancer cells.
At that time, Emily Whitehead was six years old and her acute
I wanted background. I wanted to learn more about Carl.
lymphoblastic leukemia had relapsed. There was no further salvage
Dr. June grew up in Northern California, the eldest of five
chemotherapy to offer her. Her only chance to live was a new ex- children. His father was a chemical engineer. A quiet, supportive
perimental form of gene therapy that had only been tried in a few man who was what Dr. June describes as a mathematical genius.
adults and never in children. Emily became the first pediatric patient His mother, also from a family of engineers, was the nurturer and
to receive the then "experimental" CAR T-cell therapy. Her own co-conspirator to his active imagination. She helped him and his
immune system was now used to combat her cancer. Emily's tumor friend in sixth grade perform their first surgeries on rats. I also
burden melted away. She was Dr. June's first pediatric experience learned that he had a very creative streak. As a young adolescent,
with CAR T-cell therapy and it was a resounding success. She went he was interested in making hot air balloons. Unfortunately, he
Continued on page 8
Spring 2018 : Philadelphia Medicine

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