59-year-old Judy Wilkins, a mother of two, had been battling lymphoma for years. Thanks to CAR T-cell therapy, an emerging new treatment for some types of blood cancers, Wilkins is cancer-free.
59-year-old Judy Wilkins, a mother of two and owner of a hair salon, had been battling lymphoma for years. The cancer became so aggressive that she tried four different chemotherapy regimens over a course of 18 months, with an aim to put it into remission. Nothing worked until CAR T-cell therapy. Today, Wilkins is cancer-free.
The CAR T-cell therapy ‘poster child’
CAR (Chimeric Antigen Receptor) T-cell therapy is an emerging treatment option for some types of blood cancers such as leukemia, lymphoma, and others. CAR T-cell is a type of immunotherapy utilizing the immune system of the patient to target cancer. A procedure call apheresis is used to remove T-cells from the patient’s blood. T-cells are white blood cells whose job is detect and kill off cancer cells; they don’t always recognize the cancer cells, though. That said, the cells taken from the patient are re-engineered in a laboratory to provide special structures (CARs) that attach to their surface. With the newly re-engineered T-cells infused back into the patient’s bloodstream, the cells go on to multiply in the body and they can become particularly skillful at targeting and killing off cancer cells. Their effectiveness may even grow over time, helping to guard against recurrence and resulting in long-term remissions.
In a nationwide clinical trial, it was found that more than 80% of patients with diffuse large B-cell lymphoma who underwent CAR T-cell therapy achieved positive outcomes and 50% -- like Wilkins – experienced complete remission. “These are phenomenal numbers, and are consistent with similar trials for this and other lymphoma types going on around the country. Standard chemotherapy will be effective for many patients, but not all of them. If we can identify those patients who do not respond well, and may be strong candidates for CAR T-cell therapy, we might spare them months of ineffective chemotherapy” said Dr. Caron Jacobson, Head Investigator for the clinical trial at Dana-Farber Cancer Institute and Wilkin’s oncologist.
Dr. Jacobson the one who recommended that Wilkins undergo CAR T-cell therapy. “I never really thought at that point it would be making choices on my life. But, then, the more I realized how sick I really was-- because I was in total denial about it-- and that’s when Caron said we are doing this” said Wilkins.
Three weeks later, the Dana-Farber team drew out Wilkins’s blood, sending the blood sample to a laboratory in California. Once there, the technicians re-engineered her T-cells to they would target a protein on her B cells. She didn’t feel well after the initial infusion of re-engineered cells, but in a matter of just six weeks, she went back to work. “Every three months I go back for PET scans and bloodwork, but I feel terrific. I really lucked out that I was under the care of Dr. Jacobson, who had done the work needed to start this trial and had a spot for me right when I needed it” she said. Now, more than a year later, she remains cancer-free and feels like her old self again.
The 2018 Advance of the Year
As of May 2018, the United States Food and Drug Administration (FDA) has approved CAR T-cell as a standard therapy for patients that are aged 25 and under, as well as various adult patients with relapsed or refractory aggressive non-Hodgkin’s lymphoma. “In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer” said Dr. Scott Gottlieb, FDA Commissioner. CAR T-cell therapy could also prevent some patients from undergoing donor-based stem cell transplant. This type of transplant requires extended hospitalization stay, months of isolation at home, as well as one-year time away from work.
However, there are post-operative side effects, including cytokine release syndrome, that need to be considered and addressed. Cytokine release syndrome can cause a high fever, low blood pressure, as well as respiratory and other organ-related health issues – but these symptoms tend to disappear after a couple of days. Another challenge is its cost. The two newly approved CAR T-cell therapies, Kymriah and Yescarta, are priced at $475,000 and $373,000, respectively.
At present, a clinical study is ongoing at Dana-Farber Cancer Institute for patients diagnosed with sarcomas. Other solid-tumor cancers, including prostate cancer, ovarian cancer, and brain tumors, are also being researched across the nation.
In her own research study, Marcela Maus, Director of Cellular Immunotherapy at Massachusetts General Hospital, is analyzing the ways in which CAR T-cell therapy works in glioblastoma patients. Glioblastom is the deadliest form of brain cancer. It will take some time, but she is driven by the speed with which CARs therapy has been discovered, tested and then approved.
Both Dana-Farber Cancer Institute and Massachusetts General Hospital will be among the first 16 sites across the nation to offer Yescarta. This means that more patients will have access to the therapy. However, now that it is no longer in the experimental stages and it is FDA-approved, the patients and their insurance companies will have to cover the expenses. Wilkins and other patients enrolled in the clinical trials had a majority of their expenses covered by the pharmaceutical companies.
Transforming the outlook for incurable cancers
CAR T-cell therapy is still in its early stages and whether this approach will be a cure for patients, like Wilkins, remains to be determined. Nevertheless, this form of therapy is showing encouraging results and its future is looking quite promising. “This is revolutionary,” said Dr. Jacobson.