USA Approves Historic Treatment Against Childhood Leukemia

USA Approves Historic Treatment Against Childhood Leukemia: Genetic Revolution Against Cancer Has Just Begun

The US FDA has just approved commercial gene therapy for the first time. Novartis' treatment, aimed at cases of leukemia with a very poor prognosis, has shown a brutal effectiveness in patients who until now have been evicted.
"We are crossing a new frontier in medical innovation thanks to the ability to reprogram a patient's cells to attack cancer," said Scott Gottlieb, an FDA commissioner. And he did not fall short.
The "precision medicine" is here
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The CAR-T treatment that will be called Kymriah uses (and reprograms) patients' white blood cells to attack the tumor. That is, unlike other treatments with pills, radiation or injections, Kymriah requires a personalized, individual and non-transferable manufacturing. It is the " precision medicine " live and lives.
With spectacular results, Kymriah is only the first of a long series of treatments (such as Kite Pharma or Juno Therapeutics ) that await FDA approval and promise to change forever (and for the better!)Everything we knew about the treatment of cancer.
The approval of this treatment to treat children and young adults with relapsed acute lymphoblastic leukemia is therefore a historical landmark. And cause for joy: in clinical trials Kymriah managed to eradicate cancer in 83% of participants in only three months. It is, according to the experts, is a very important advance for patients who still have few a very difficult future.
All this with relatively controlled side effects. The so-called cytokine storms are very strong immune reactions that, although they may become deadly, are controllable with immunosuppressants.
A high price, but lower than expected
The good news does not end there, it seems: analysts expected the treatment to be around $ 750,000 per cycle and the European regulator established that $ 700,000 would be an acceptable figure. However, Novartis (which has suffered significant media pressure on this issue ) has announced that the price will be at $ 475,000.
Beyond price and negotiations with administrations, a new era begins in the development of cancer treatments. With the advent of new gene therapies to hospitals, everything we have talked about in recent years suddenly becomes real. Now it remains to work to continue advancing both therapeutically and socially.

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