Groundbreaking New Leukemia Treatment Will Save Lives and $Billions

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

A pioneering gene therapy created to treat leukemia has moved one step closer to becoming the first of its kind marketed in the United States, thanks to a crucial recommendation.

"The panel's unanimous recommendation in favour of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need", said Bruno Strigini, chief executive of Novartis Oncology.

This treatment has given patients, who were facing death as other treatments failed, long remission periods and possibly even cured them.

The gene therapy, also known as CTL019, uses a new technology called chimeric antigen receptor T-cell therapy, or CAR-T for short. Cripe was a member of the FDA advisory panel that voted Wednesday to support the drug's approval.

A group of cancer experts advising the FDA has asked the agency to approve a Novartis AG (ADR) (NYSE:NVS) leukemia drug now going by the name CTL019. The drugmaker is seeking approval to use the one-time treatment for children and young adults with advanced leukemia.

This revolutionary new method of treating blood cancer is customized to target a specific type of cancer, that is, it is adjusted according to the DNA information of a particular patient.

CAR-T involves extracting special immune system cells from the patient and then re-programming them.

"Although this therapy is technologically somewhat complicated and is associated with certain serious side effects, it, indeed, has been proven to be amazingly effective", said Kanti Rai, chief of the chronic lymphocytic leukemia research and treatment program at Northwell Health Cancer Institute in NY.




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.

In a Novartis' sponsored ELIANA study, which formed the basis of Novartis' application of approval, treatment with Tisagenlecleucel resulted in a best overall response rate of 83%, with 63% of successfully infused patients experiencing a complete response. "When other organizations, including the NIH, considered gene therapy too risky, ACGT believed in the science and funded us when no one else would", said Dr. June.

Patients with ALL who fail chemotherapy typically have only a 16 percent to 30 percent chance of survival.

Assuming this treatment is approved, it will be a first for the FDA.

"It is not uncommon for the companies who make these products to manage supply closely", said Elizabeth Kalina, a spokeswoman for Shire, which is building a new manufacturing facility that's slated to open in Georgia in 2018. Her father said his family was more afraid of the side effects of the full-body radiation that's used ahead of a transplant than of the experimental immunotherapy.

"Now that people see there is a path to approval at the FDA, that usually gets people off the sidelines", explained Brad Loncar, founder of a cancer immunotherapy exchange-traded fund. Seven dropped due to an insufficient batch of CAR-T formulation, five did not reach a 3-month followup, and four were still in the process of infusion at the data cutoff time point.

Treatments involving live cells, known as "biologics" are generally far more hard to manufacture than standard drugs, and the panelists also expressed concerns about whether Novartis would be able to produce consistent treatments and maintain quality control as it scaled up its operation.

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