losangelesnewstoday.com May 23, 2018

T gene therapy for leukemia clears FDA hurdle

14 July 2017, 12:37 | Violet Powell

FDA advisers back Novartis' game-changing personalized CAR-T for cancer, approval in sight

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

Indeed, the FDA panel raised concerns about the potential for the kind of severe side effects that Emily Whitehead experienced, or whether gene therapy might raise the odds for secondary cancers many years later.

Data from the United States multicenter trial and single site trial assessing the safety and efficacy of CTL019 among pediatric and young adult patients with r/r B-cell ALL also backed the recommendation and the biologics license application (BLA). This process gives the patient's immune system a tumor-attack roadmap for the treatment of leukemia and other cancers, including those of the ovaries and myeloma.

The FDA is not required to follow the panel's recommendation but often does.

The treatment was developed by researchers at the University of Pennsylvania and licensed to Novartis.

"This therapy will no doubt save the lives of many children and young adults who have had no other effective therapy", said Dr. John Maris, a pediatric oncologist at The Children's Hospital of Philadelphia and leader of the SU2C-St.

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.

"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.

Patients who had received CTL019 and their families descended upon the panel meeting en masse Wednesday to urge an approval recommendation. Millions of copies of the new T cells are grown in the lab and then injected into the patient's bloodstream where they can seek out and destroy cancer cells. An approval decision for the Kite CAR-T is expected on November 29.

Old meningitis B vaccine 'may also protect against gonorrhoea'
Kramer said that if a vaccine against multiple strains of gonorrhea could be developed, it would be very beneficial. Though it does have many similar components, we don't know if these are useful in protecting against gonorrhoea.

And because the treatment kills one type of immune cell, patients are more likely to come down with certain infections.

"It's a pretty fantastic new treatment", Dr. David Agus, director of the USC Norris Westside Cancer Center and CBS News medical contributor, said on "CBS This Morning".

In the case the FDA approves the launch of the drug, called Tisagenlecleucel (CTL019), it will be the first ever leukemia drug introduced on market.

Other panelists called the treatment a "major advance" and that novel therapy meets a "dire, unmet need". To generate a batch of tisagenlecleucel, white blood cells are purified from a sample of a patient's blood and shipped to a central processing centre.

"It's a new world, an exciting therapy", said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment.

But the drug has side effects that can be fatal, such as cytokine release syndrome or CRS, which "looks like sepsis" and causes blood pressure to drop dangerously low, said Diefenbach.

"These engineered T-cells have proven to be active in B-cell leukemia in adults", Grupp said in a statement in 2012. She was the first child to receive the treatment. While patients on this engineered vehicle cell therapy have been repeatedly hit by potentially lethal cases of cytokine release syndrome and neurological toxicity with the threat of new malignancies, advisers were clearly willing to accept the risk in order to achieve the potential gains.

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