The Grapevine

Cancer Treatment: New Gene Therapy Uses Patients' Immune Cells

After being diagnosed with cancer in 2012, patient Mary Beth Zolik-Smith underwent successful chemotherapy for the non-Hodgkin lymphoma. But a few years later, her cancer returned and she had to receive treatments once again.

“My perception of clinical trials at that time was they were highly experimental and not much of a guarantee," she said, speaking to MedicalDaily. 

Zolik-Smith explains that standard-of-care treatments were the best options in her eyes since they were tried and true. It was only after both the chemotherapy and stem cell transfer failed, that she looked into the option of a clinical trial.

“I think patients sometimes think that clinical trials are for people on their death bed. I know I did. But if I knew then, what I know now, I would have opted for the CAR T-cell therapy instead of the stem cell transfer. Hindsight is 20/20,” she said.

After showing remarkable results in clinical trials, CAR T-cell therapy treatment became the second gene therapy approved by the FDA in 2017.

As a part of the treatment, T-cells are first separated from the blood drawn from patients. A virus is then used to genetically engineer these cells and produce chimeric antigen receptors (CARs). Thus, the CAR T-cell is developed and ready to be inserted back into the body in order to act like a “living drug.”

After undergoing this treatment in a trial at the Cleveland Clinic, Zolik-Smith is now in complete remission. “Today, it seems to me, clinical trials are happening fast and furiously with the new technology and medical advances in immunotherapy, gene therapy and hormone therapy,” she said, adding that she no longer views clinical trials as a last resort.

Dr. Brian Hill from Cleveland Clinic Cancer Center was Zolik-Smith’s medical oncologist. He explains that expanding the breakthrough therapy towards treating solid tumors has been challenging and would likely take many years to become a reality.

He identifies two main challenges: “The first would be identifying the tumor target to attack because many times that these targets are also present in normal tissue. The second challenge is the delivery of the therapy to the tumor, which is a hostile environment where CAR T-cells may not be able to become activated.”

Dr. Hill states the CAR T-cell treatment is not a first option for patients, but rather an option for patients who have relapsed after standard-of-care treatments for blood cancer. Additionally, he acknowledges that there may be side effects involved in opting for the procedure.

“Patients are typically hospitalized 1-2 weeks, and often experience toxicities related to the rapid proliferation/activation of the new immune cells. These toxicities can include fever, low blood pressure, low oxygen levels as well as potential confusion and sometimes severe somnolence and even seizure,” he said.

“The hope in the field is that is as CAR T-cell therapy evolves, the side effects will become more manageable.”