A new era in medicine was heralded yesterday with a Food and Drug Administration panel, unanimously recommending that the agency approve the first-ever treatment that genetically altered a patient's own cells to fight cancer, transforming them into what scientists called ''a living drug'' that powerfully bolstered the immune system to cure the condition.
If the FDA were to accept the recommendation, which was likely, the treatment would be the first gene therapy ever to reach the market, according to commentators.
Researchers and drug companies had engaged in intense competition for decades to reach this milestone and Novartis was now poised to be the first. 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 use of the technique would entail a separate treatment for each patient, removal of their cells at an approved medical centre, freezing them, shipping to a Novartis plant for thawing and processing. The cells would need to be frozen again and shipped back to the treatment centre.
Treatment with a single dose of the resulting product had produced long remissions, and possibly cures, in scores of patients in studies who were facing death due to the failure of every other treatment.
Timothy Cripe, a panel member, an oncologist with Nationwide Children's Hospital in Columbus, Ohio, called the treatment the "most exciting thing I've seen in my lifetime."
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval for the treatment in children and young adults whose leukemia was not responsive to traditional treatments.
The approach is also being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumours.