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Winter 2021 Human Interest Feature – Penn Medicine

Still Thriving Years After Groundbreaking Cancer Immunotherapy
Experts Gather this Spring to Share Progress and Chart Future Course

Their options had run out. Multiple rounds of chemotherapy had proven ineffective. The cancer could not be contained. This scenario is familiar to many oncologists and other medical specialists who treat diseases for which there is no cure. Patients face the news with emotions ranging from fear, sadness, hopelessness to determination and, in some cases, bravery.

Doug Olson, a leukemia survivor, rejects the notion of bravery. “When you’re told that you are out of options and you see an opportunity, that’s your focus. Hope is the most important thing for cancer patients – to have the opportunity to fight back. It’s a big part of the consideration when you make a decision to do something that has never been done before.”

Olson speaks from experience. In 2010, he was patient number two to receive an experimental therapy developed at Penn Medicine’s Abramson Cancer Center. The treatment, known as CAR T-cell therapy, was an entirely new form of immunotherapy.

For years, University of Pennsylvania scientist Carl June, MD, and colleagues had pursued the vision of a cancer therapy that would work by reengineering T cells (the immune system’s fighters). Unlike chemotherapy that attacks all fast-growing cells, immunotherapy works with the body’s best defense – the immune system. During CAR T-cell therapy, a patient’s immune cells, or T cells, are reengineered – they are removed, genetically modified so they can recognize and kill tumor cells, and returned to the body to find and destroy the cancer cells.

After a 1996 chronic lymphocytic leukemia diagnosis, Olson endured multiple rounds of chemotherapy with sporadic periods of remission until the cancer no longer responded. His doctor, David Porter, MD, at Penn’s Abramson Cancer Center, presented Olson with the option to join a new clinical trial for a treatment not yet tested in people. It would be a risk but June, along with Bruce Levine, PhD, Porter, and others had worked relentlessly towards this goal for years. It was time. Olson’s willingness to be person number two on this trial changed his life and the course of cancer treatment. One month after the T cell infusion, there was no evidence of leukemia by any test the doctors performed.

Then, in 2012, Emily Whitehead became the first pediatric patient to enroll in a pediatric clinical trial testing the same treatment. At age 5, Emily was diagnosed with acute lymphoblastic leukemia (ALL). Her parents brought her to Children’s Hospital of Philadelphia’s (CHOP’s) top-ranked cancer center where she received care from a team led by Stephan Grupp, MD, PhD, the oncologist leading the study at CHOP, and colleagues who specialized in hard-to-treat blood cancers. ALL is often treatable in children but, in Emily’s case, the disease failed to respond to any chemotherapy. A bone marrow transplant would be the next step toward a cure, but she needed to respond to treatment before that could happen, and she wasn’t responding. Transplant wasn’t possible.

At CHOP, Emily’s parents were introduced to the idea of treating their daughter with the innovative new therapy being tested in adults right next door at the Hospital of the University of Pennsylvania. With hospice as their only other option, they were desperate for their daughter to join the phase 1 trial of CAR T-cell therapy. The trial opened at the exact moment Emily needed it. Everyone involved rushed the paperwork and processes to collect her cells and enroll her in the trial.

Emily’s treatment went smoothly until she was admitted to the hospital with an extreme immune response beyond what had been seen in the handful of adults treated before her. The response, now known as cytokine release syndrome, ravaged Emily’s young body through the night. Desperate for solutions, June suggested a new arthritis drug designed to target a specific cytokine observed at high levels in CAR T patients. Miraculously, it worked.

Three weeks later, like Doug Olson, Emily Whitehead was cancer-free. This early trial led to the 2017 approval of the drug tisagenlecleucel (Kymriah®) to treat particularly tough ALL in children and young adults. Kymriah is unique in that it is a living drug. Years beyond treatment, many patients remain in remission and still retain powerful T cells in their bodies.

A scientist and a businessman, Doug Olson now lives a full life, an opportunity afforded him by the miracle of CAR T-cell therapy. Emily Whitehead is a teenager who is passionate about school (she’s a straight “A” student), art, healthy living goals, and her dog Luna. When asked what stands out, Olson reflects, “the fact that I’m alive and that what I participated in has literally changed the landscape of cancer treatment.”

Today, doctors study CAR T-cell therapy in cancers ranging from pancreas to melanoma to brain. Research also explores why the approach works better for some patients than others, and how to deliver the therapy out of the hospital and more safely. CAR T-cell therapy is just one kind of cellular therapy being studied at Penn and CHOP. Its approval has unleashed a floodgate of related research on other delivery mechanisms beyond CAR. Doctors and scientists on Penn/CHOP team have won many prestigious awards for this work, including June’s 2018 nomination for a Nobel prize.

In 2019, the Penn’s Abramson Cancer Center and CHOP hosted a large international gathering of doctors and scientists dedicated to cellular therapy. Nearly 1,000 medical and biotech professionals arrived in Philadelphia to hear from pioneers in the field who played pivotal roles in the work leading up to the approval of CAR T-cell therapy.

This May, Drs. June, Grupp, Levine and Porter will once again bring together experts from around the globe, this time virtually, to share findings and chart a future course for cellular gene therapies. Members of industry and advocates will join physicians, nurses, scientists and others for a two-day program. Highlights will include a keynote talk by Stanley Riddell, MD, of the Fred Hutchinson Cancer Research Center on “Engineering Smarter T Cells.” Dr. Riddell is a world leader in developing immunotherapies and the architect of groundbreaking work to develop longer-lasting T cells. General and breakout sessions will also cover critical topics such as current uses, globalization, gene delivery and editing, overcoming resistance, and new targets and technologies. An exhibit hall will showcase the state-of-the-art discoveries that are advancing the field.

Cellicon Valley ’21: The Future of Cell and Gene Therapies
Virtual Symposium
May 6 – May 7, 2021
Hosted by Penn Medicine’s Abramson Cancer Center and Children’s Hospital of Philadelphia
Register now – https://www.med.upenn.edu/cellicon2021/

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