'Astounding' second-chance cancer drug heading for FDA approval

Novartis dodged deaths from its CAR-T Therapy, but FDA still has safety concerns

It seems that the first gene therapy that will be available in the United States will be for the treatment of leukemia, one of the most common blood cancers.

During her research on the treatment, she discovered that the "memory" of the T-cells, introduced into cancer patients for whom bone marrow transplants had failed to work, ranged from two to 14 years. "We are hopeful about the promise of immunotherapy approaches for cancer patients, who deserve new and more effective treatments". The protocol involves removing a patient's white blood cells called T-cells, genetically modifying them, then infusing the newly transformed cells back into the patient's body following chemotherapy.

The FDA is not required to accept the recommendations of its advisory panel, but it usually does.

"This review by the FDA of CAR-T therapy is a major milestone in the successful treatment of cancer", noted John Walter, CEO and president of ACGT.

Novartis picked up rights to CTL019 under an agreement with the University of Pennsylvania in 2012, which also gives it worldwide rights to CAR-Ts developed in all cancer indications.

Novertis's CAR-T would be used in patients age 25 and under whose cancer comes back after chemotherapy - about 600 people a year in the US Acute lymphoblastic leukemia is the most common cancer in children. The drug was tried on patients whose cancer had relapsed or didn't respond to standard treatments. In light of the drug's side effects, the FDA advisory panel viewed this as a favorable risk-benefit balance.

The committee decision now gets passed to the FDA which is expected to render a final decision regarding CTL109 by Oct.3. The trial lacked a control group, so investigators can not yet say with certainty how much of an effect the treatment had. In one key trial of tisagenlecleucel, which started in 2015, 82.5% of 63 patients experienced overall remissions. It's risky: The cross-effect from revving up the immune system can sometimes cause a deadly internal storm of its own.

"The majority of calls we are getting are from patients looking for immunotherapies - particularly CAR-T therapies", said Dr. Gwen Nichols, chief medical officer at the Leukemia and Lymphoma Society. First, a process specific to each patient is conducted where millions of immune system T-cells are removed and frozen at an approved medical center. This modification of the cells causes them to attack the cancer cells.

"In the last five years, there have been a significant number of cell therapy companies that have gone public or gotten investment in hopes of moving this type of therapy forward", said Reni Benjamin, an analyst at Raymond James. In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancer cells. "As an investor I've never seen anything like it".

The global CAR-T market is estimated to hit $8.5 billion by 2028, according to a report released in February by Coherent Market Insights.

The FDA also discussed mandating a patient registry that would track patients for up to 15 years.

In addition to understanding that process, the staffers wrote, Novartis will need to understand critical product quality attributes unique to autologous vehicle T-cell products-as well as to understand and address sources of variability seen in the products.

Novartis has confirmed it will file for approval of the cell therapy later this year in the European Union, where it became one of the therapies to enter the EMA's new PRIME expedited approval pathway.



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