The Food and Drug Administration on Wednesday approved the first-ever gene therapy in the US. The treatment, named Kymriah, was hailed by doctors and the life sciences community as a major advance in medicine and a boon to children and young adults with a certain form of leukemia (the group for whom the gene therapy is approved). In just the last few years, progress with vehicle T cells and other ACT approaches has greatly accelerated, with researchers developing a better understanding of how these therapies work in patients and translating that knowledge into improvements in how they are developed and tested.
The treatment involves altering the patient's own immune system by removing T-cells and genetically modifying them to recognize, attack and kill cancer cells. This is the first gene therapy of its kind to be approved for use in the United States, but the treatment is not without controversy as it has the potential for severe side effects as well as an extraordinarily prohibitive price tag. Meanwhile, this week Roche secured FDA approval to use its arthritis drug Actemra (tocilizumab) as a treatment for CAR-T-induced CRS. The new cells would then continue to multiply to fight disease for months or years.
Novartis will create a registry to follow patients for 15 years after being treated to monitor their progress and any potential, future side effects. "Emily's cancer remains in remission, and in larger trials, we're seeing overall remission rates over 80 percent, which is a remarkable improvement upon previous treatment success rates", said lead investigator of the CHOP and global trials of the therapy, Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at Penn and director of the Cancer Immunotherapy Frontier Program and chief of the section of Cell Therapy and Transplant at CHOP.
"We have to keep in mind that patients will get cured of leukemia", Satwani said.
Novartis estimates some 600 ALL patients a year would be eligible for Kymriah.
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"The efforts involved in being able to provide this novel therapy to patients is unlike any other therapy now offered", says Colleen Dansereau, MSN, RN and CPN, who is director of clinical research nursing and gene therapy program manager at Dana-Farber/Boston Children's.
The trial was successful, with 83% of the 63 clinical patients treated for relapsed or refractory B-cell undergoing successful remission within three months of treatment. Instead of getting an updated therapy that works better on a disease every decade or so, we might begin to see second-generation cell therapies in just a few years.
They'd also have to be less toxic so that more people could consider it, instead of only those who have very few other options. "The lessons learned from the clinical trials of auto T-cell operations will assist us in being able to provide to the larger population". In May, Kite disclosed that one person had died in a clinical trial for its late-stage CAR-T therapy from cerebral edema, a condition in which excessive fluid causes the brain to swell. The patient was a little girl nearly dying and is now five years cancer free.
And then, to impact more people, the cell therapies will need to go beyond the blood cancers.
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