T gene therapy for leukemia clears FDA hurdle

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

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

The FDA is expected to decide whether to approve the Novartis treatment in the next few months. Novartis is also vying to have CTL-019 approved to treat adults with the most common forms of non-Hodgkin lymphoma. Ten committee members voted in favor, and one left early without voting.

At UNC, 60-year-old Ian Dale is one of those in remission after having his immune cells removed and returned, engineered to fight his anaplastic large cell lymphoma.

"It's a pretty awesome new treatment", Dr. David Agus, director of the USC Norris Westside Cancer Center and CBS News medical contributor, said on "CBS This Morning". Diefenbach, who was not involved in researching the drug and has no ties to its manufacturer, Novartis, described its results as "astounding".

Although the outside panel of experts raised questions about concerns with the safety and manufacturing of the Novartis treatment, known as CTL019 (tisagenlecleucel-T), the panel did not question the efficacy of the CAR-T therapy in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). Dr. Megan Polanin of the National Center for Health Research, a think-tank, said she was anxious that most of the patients had only been followed for a few months. Treatments for an aggressive type of brain tumor, as well as myeloma and other varieties of leukemia, are also in development. The company that's able to do that quickly and safely could have the advantage in the competitive vehicle T space. But when the cancer comes back, the prognosis can be dire.

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.

Now Emily's survival has stretched to five years, cancer-free. Scientists use a virus to make the genetic changes in the T cells, raising fears about possible long-term side effects. This could limit the drug's availability to those hospitals that are specially equipped to deal with this complication, she added.

The vote came after lengthy discussion and impassioned pleas from the fathers of two young patients whose lives were saved by the therapy.

Slightly fewer patients experienced neurological side effects, such as seizures and hallucinations, according to the committee's briefing document.

The cells are grown in culture before they are reintroduced into the patient. At least three patients died with various infections - including viral, bacterial and fungal - more than a month after the drug's one-time infusion, according to the brief. There are about 3,100 new cases of ALL each year, but roughly 70 percent can be pushed into remission by standard therapy.

Novartis officials said they would follow patients for at least 15 years and investigate any cases of secondary cancers. Brody has helped design trials for similar drugs but not for Novartis. He told the Times that just one such cell could destroy 100,000 cancer cells.

By modifying immune cell DNA, this method could, in theory, lead to other cancers - a longtime concern for gene therapy.

The cost of CAR-T therapy is likely to be hundreds of thousands of dollars, but it's only given once. Brody said it could take decades to conclusively say this does not happen. "I think you will see a rush of investment in the cellular therapy space, both from investors but also companies". "This is straightforward numbers". Once infused back into the patient, the cells reproduce at a fierce pace to attack the cancer.

"You can put someone else's red blood cells into you", he said.

Nevertheless, several family members of children who benefited from the treatment in its experimental phase have made emotional appeals to the committee to recommend approval during a public comment period.

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