KXIP vs KKR Live Score

The U.S. Food and Drug Administration on Wednesday approved the first gene therapy in the United States, to treat children and young adults with a particularly tough type of leukemia. Patients will be followed for 15 years to evaluate the long-term safety of Kymriah. "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses". Kymriah extracts a patient's autoimmune T-cells and sends them to a facility, where they are modified to include a new gene that directs the T-cells to target particular leukemia cells.

Tisagenlecleucel T-suspension (Kymriah, Novartis) - the first chimeric antigen receptor (CAR) T-cell therapy approved in the United States - is indicated for patients aged up to 25 years with refractory B-cell precursor ALL, as well as those whose disease is in second or later relapse.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells.

"Until today, there has never been an FDA-approved treatment to manage severe cytokine release syndrome associated with auto T-cell therapy, which is marked by a rapid onset and can cause life-threatening complications", Sandra Horning, MD, chief medical officer and head of global product development at Genentech (Roche), the manufacturer of tocilizumab, said in a statement.

First developed by the University of Pennsylvania, Kymriah uses the 4-1BB costimulatory domain in its auto to enhance cellular responses as well as persistence of the treatment after it is infused into the patient, which may be associated with long-lasting remissions in patients.

Novartis' investigators registered a game-changing 83% remission rate in its pivotal study.

Tisagenlecleucel is the first auto T-cell therapy to demonstrate early, deep and durable remission in this patient population, and it likely will become the new standard of care, according to Stephan A. Grupp, MD, PhD, Yetta Deitch Novotny professor of pediatrics at Perelman School of Medicine at University of Pennsylvania and director of the Cancer Immunotherapy Frontier Program at Children's Hospital of Philadelphia.

But the promise of T-cell therapy is believed to be vast. "We started using tocilizumab in our patients about 5 years ago, and that has really saved the field of active cell therapy".

Novartis has not yet announced the cost for the therapy, but British health authorities have said a price of $649,000 for a one-time treatment would be justified given the significant benefits, according to a Kaiser Health News report last week. The modified cells are then transfused back. "Future potential indications would be reviewed for the most relevant outcomes-based approach", the drug company said in a statement.

"We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program", added Bruno Strigini, CEO of Novartis Oncology. The response, in a word, is heartening. And our promise to you is that we will always strive to provide indispensable journalism to our community.

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