One of the most significant advancements made in treating disease in recent years is the development and commercialization of CAR-T therapy, which weaponizes a patient’s own white blood cells to attack certain types of blood cancer cells. For some cancer patients, CAR-T therapy hope holds the promise of a cure.
In fact, in early 2022, a 16-year-old who had participated in a CAR-T clinical trial celebrated her cure ten years after receiving treatment.1
Since the first CAR-T therapy was approved by the Food and Drug Administration (FDA) in 2017, five more therapies have gained FDA approval for treating specific types of leukemia, lymphoma and multiple myeloma in both children and adults. Research is underway to develop similar solutions for solid tumor cancers as well.
How CAR-T therapies are changing cancer treatment
FDA-approved CAR-T therapies can be used to treat patients who have already been through at least one unsuccessful standard treatment, such as chemotherapy or a stem cell transplant. With a success rate of about 30% to 40% for lasting remission2, CAR-T treatments are the only FDA-approved therapy to show significant benefit for those patients who, as few as five years ago, had no other course of treatment. That is why, despite potentially dangerous side effects, the adoption of CAR-T therapy as a treatment continues to grow.
A recent study published in Nature3 shows that CAR-T treatments may be more effective than other treatments as the first treatment for the relapsed/refractory patient, rather than second-line or later. If so, this would bring new hope for patients and reduce their total time of treatment: CAR-T treatments typically take about a month, compared to the six months often required for traditional chemotherapy or stem cell transplants.
Cardinal Health Chief Medical Officer, Bruce Feinberg, has been conducting observational research on the perceptions, obstacles and adoption of these therapies since 2015. “When the first CAR-T therapy was launched, there was significant concern among oncologists about the toxicity of these therapies: More than 43% of oncologists surveyed said that the toxicity profile was concerning enough to limit the number of patients they would refer for this treatment. However, over time and with improved understanding of toxicity and its treatment, as well as newer CAR-T therapies that have lower toxicity profiles and the benefits of real-world evidence insights, oncologists have become less concerned about toxicity and have found that the benefits outweigh the risks in many cases.”
Obstacles to patient access
Cardinal Health researchers monitor and explore other challenges associated with CAR-T therapies. Often cited concerns about CAR-T therapies are related to costs. Priced up to $1 million per person per treatment4, CAR-T therapies are some of the most expensive treatments on the market. However, researchers are finding that the high price tag is no longer deterring oncologists as much as it did when the first CAR-T therapy was approved as evidence of the benefits of these treatments stacks up.5
Beyond the sticker price, oncologists often cite the administrative burdens of using these therapies, including cumbersome logistics, long referral processes and payer approvals, as obstacles that need to be overcome.
Administrative delays often impact whether the patient will be able to receive the treatment at all. Because cancer can progress so rapidly, any delay may mean that a patient won’t get the therapy they need in time.
What’s next
“With more than one hundred companies around the globe now developing CAR-T technologies, we anticipate continued innovation in these therapies,” Feinberg said.
Something that could open more options for CAR-T treatments is a shift from autologous (unique to a single patient) to allogeneic CAR-T treatments, which are off-the-shelf options that can be used by many patients. Several pharmaceutical and biotech companies are in the process of developing these off-the-shelf products or even beginning clinical trials6 on them.
Additionally, many hope that the treatments will become available in the community oncologist setting, rather than only in accredited centers, often large research hospitals, where patients often must travel for treatment.
At Cardinal Health, our experts have been involved in the research, development and logistics of CAR-T therapies since 2015, partnering with the companies that are developing them to help get the treatments to patients faster. We provide solutions across the drug development cycle, from assisting with regulatory approvals and enhancing product launch strategies, to providing logistics solutions that help ensure patients are able to get the medication they need.
Learn more at www.cardinalhealth.com/cellandgene
1 “Emily Whitehead, First Pediatric Patient to Receive CAR T-Cell Therapy, Celebrates Cure 10 Years Later.” Children’s Hospital of Philadelphia. May. 11, 2022. https://www.prnewswire.com/news-releases/emily-whitehead-first-pediatric-patient-to-receive-car-t-cell-therapy-celebrates-cure-10-years-later-301545190.html
2 “Six years after CAR T-cell therapy for lymphoma, patient still cancer-free.” UChicago Medicine. December. 28, 2021. https://www.uchicagomedicine.org/forefront/cancer-articles/a-walking-miracle-car-t-cell-therapy
3 “First-line CAR T-cell therapy could help cure some lymphomas.” Nature Medicine. March. 22, 2022. https://www.mdedge.com/hematology-oncology/article/253020/aggressive-lymphomas/first-line-car-t-cell-therapy-could-help
4 “Study Finds Total Cost of Care for CAR-T, Post-Treatment Events Can Exceed $1 Million.” Pharmacy Times. April 13, 2021. https://www.pharmacytimes.com/view/study-finds-total-cost-of-care-for-car-t-post-treatment-events-can-exceed-1-million
5 “CAR-T provides value despite high price tag, oncologists say.” Healio. Dec. 13, 2021. https://www.healio.com/news/hematology-oncology/20211213/cart-provides-value-despite-high-price-tag-oncologists-say
6 “Allogene, slipping in ‘off-the-shelf’ CAR-T race, prepares for key trials.” Biopharma Dive. Feb. 24, 2022. https://www.biopharmadive.com/news/allogene-off-shelf-CAR-T-trial-plans/619338/