FDA Panel Recommends Approval for Gene-Altering Leukemia Treatment

Emily Whitehead a now 12-year-old girl was close to dying from leukemia but survived thanks to a cutting edge cancer therapy being pioneered when she was seven

Advisers to the FDA recommended approval of a new experimental cancer treatment called CAR-T therapy for kids with leukemia.

"This is a major advance", said panel member Dr. Malcolm A. Smith of the National Cancer Institute.

"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. "It represents a paradigm shift in treating cancers", said Maloney, who is extensively involved in auto T-cell research but not in the Novartis product.

"We believe that when this treatment is approved it will save thousands of children's lives around the world", Whitehead told the meeting, his voice cracking as he looked at his daughter beside him.

It's not without risks - some children who received the treatment spiked fevers of as high as 107 degrees and not all of them were cured. The T-cells hone in on a protein called CD-19 that is found on the surface of most B-cells. Earlier this year, Kite Pharma, which also has a CAR-T therapy product being reviewed by the FDA, reported the death of a patient from brain swelling, and Juno Therapeutics placed its vehicle T program on hold when its scientists weren't able to overcome the toxicity.

Novartis seeks approval of CTL019 for treatment of pediatric and young-adult patients with relapsed or refractory B-cell ALL. That happens to more than 600 patients in the US each year. One possibility is that the agency is anticipating that CAR-T products will be developed for patients who aren't at the end of the line. ALL accounts for a quarter of all cancers in children under age 15.

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In a test conducted, gene therapy results exceeded chemotherapy, and even newer types of cancer medicines.

There are potentially lethal side-effects associated with cytokine release syndrome (CRS), where the body comes under attack from its own over-stimulated immune system. Last month, updated results from the phase 2 ELIANA trial showed that 83% of pediatric patients achieved complete remission (CR) or CR with incomplete blood count recovery within the first 3 months of treatment.

The recommended therapy could be the first of a stream of treatments custom-made to target a patient's cancer, Fox News noted.

Whitehead's treatment process took about 16 weeks, which the FDA advisors said can be too long for some desperately ill patients. For one thing, the manufacturing process is extremely intensive since each individual batch of the drug needs to be tailored to each individual patient.

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. The advisory panel determined, unanimously, 10-0, that the benefit of the therapy outweighed the risks. Its treatment was for a type of leukemia, and it was working on similar types of treatments in hundreds of patients for another form of the disease, as also multiple myeloma and an aggressive brain tumour. The dramatic effect of the treatment, known for years as CTL-019, was never questioned at the meeting. The prognosis for these patients is very poor, especially in patients who have relapsed more than two times or who relapse following allogenic stem-cell transplants.

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