The emerging field of modifying and deploying a patient’s own cells to fight cancer just got some big legs under it with the late August approval of a new treatment for pediatric and young adult leukemia. The therapy, known as chimeric antigen receptor T cell (CAR-T), was awarded to Novartis for Kymriah, “a novel immunocellular therapy.”

David Lebwohl, M.D., head of the CAR-T Global Program at Novartis, told CenterWatch, “This is a significant step forward in the treatment landscape and for this patient population, which has limited treatment options and historically poor outcomes.”

According to Dr. Lebwohl, 83% of patients who received treatment with Kymriah achieved complete remission or complete remission with incomplete blood count recovery within three months of infusion. This compares to traditional multiple treatments, including chemotherapy, radiation, targeted therapy or stem cell transplant, which resulted in less than 10% of patients surviving five years.

CAR-T is powered by personalized medicine. Each dose, noted Lebwohl, is tailored individually to, and manufactured for, each patient using the patient’s own blood cells. The therapy, which is not a pill or traditional chemotherapy, is produced via pioneering technology and a sophisticated manufacturing process.

“Our manufacturing process uses cryopreserved leukapheresis, which enables patients to be leukapheresed at a time that is in their best interest, including, at times, months in advance of manufacturing,” he said. “Our cryopreserved leukapheresis process allows for manufacturing and treatment of patients from around the world.”

While Novartis gets credit for the first FDA approval, several other companies in the CAR-T research sector are targeting other cancers.

“We at the Fred Hutchinson Cancer Research Center believe this is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers,” David Maloney, M.D., Ph.D., medical director of Cellular Immunotherapy at Fred Hutch and medical director of the Bezos Family Immunotherapy Clinic at the Seattle Cancer Care Alliance, said in a statement.  “Approvals are an important step, but they’re just the beginning. The safety, availability and affordability of these new immunotherapies can be improved, but to achieve these goals we need an increase in industry partnerships, more corporate investments and continued government funding.”

According to the statement, Fred Hutch is testing new experimental T-cell therapies for breast cancer, leukemia, lymphoma, lung cancer, Merkel cell carcinoma and mesothelioma. Other companies told CenterWatch that they, too, are excited about work in the CAR-T field.

“Novartis’ therapy raises the standard and expectations for cancer therapy everywhere, and brings a new technology to the forefront of oncology,” Cheng Liu, Ph.D., CEO and founder of Eureka Therapeutics, told CenterWatch.

However, Dr. Liu noted that, to date, CAR-T therapy is applicable only to “liquid cancers.” He said that Eureka has engineered CARs with TCR-like antibodies that target peptides from intracellular antigens that are processed and presented by class/MHCs on the cell surface. This approach, he added, will enable the use of CAR-T therapy against solid tumors that were previously considered “undruggable.”

“Approval of the first CAR-T for treatment of cancer brings cell therapy as a viable and an orthogonal therapy to the traditional chemotherapy regimens,” Maria Fardis, Ph.D., MBA, CEO of Iovance Biotherapeutics, told CenterWatch. “It demonstrates that cell therapy for oncology indications are real, commercially suitable, and a viable therapy option.”

According to Dr. Fardis, Iovance is focusing on tumor infiltrating lymphocytes therapy (TIL). These, she explained, are host T-cells that come to the site of the tumor and try to launch an attack against the tumor. Iovance has licensed this technology from the National Cancer Institute. The concept is to extract the TIL cells from the site of the tumor, isolate the TIL cells, expand them to multiple billions and then provide the cells back to the patient for treatment.

As part of the FDA approval announcement, Novartis also announced a “groundbreaking” collaboration with the Centers for Medicare and Medicaid (CMS). According to a company statement, the effort will focus on improving efficiencies in current regulatory requirements related to value-based care and access for specific patient populations. This is intended to translate to “indication-based” pricing for such medicines as Kymriah.

“Novartis has been at the forefront of outcomes-based pricing and is very pleased to work with CMS on this first-of-its-kind collaboration with a technology that has the potential to transform cancer care,” said Joseph Jimenez, CEO of Novartis. “We look forward to continuing to work with CMS to potentially expand this approach to other products and disease states.”

There can be side effects from Kymriah. These include Cytokine Release Syndrome and neurological toxicities. Because of this risk, the treatment is only available through a REMS restricted program.

“Although the treatment was shown to be very effective in clinical trials for types of blood cancer, there’s no doubt that this approach can be toxic. It’s not without risks and, in the study on which this approval is based, almost half of the patients had grade 3 or 4 side effects,” Alan Melcher, professor of Cancer Research, London at The Institute of Cancer Research, told CenterWatch.”  The $475,000 price tag is also a concern. It remains to be seen whether health services around the world will be able to foot the bill.”

However, it’s fair to say the entire immunocellular therapy community is enthused by the FDA approval. Mark W. Sawicki, Ph.D. and chief commercial officer for Cryoport, an approved vendor to ship CAR-T materials, believes the therapies open the door to treat diseases that have been resistant to treatment by conventional means.

“CAR-T therapies ... are the next generation of medicines, using cells from an individual’s body to fight diseases that have been intractable in the recent past,” said Dr. Sawicki. “Therapies of this type create an entire new paradigm of possibilities and new approaches to treating disease and illnesses.”

This article was reprinted from Volume 21, Issue 36, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »