Pioneering gene therapy to treat blood cancer in children set to get US approval
Developed by Swiss pharmaceutical major Novartis, the new therapy genetically alters the patient’s own cells to combat a form of leukaemia that mostly affects children and young adults. Norvartis plans to seek approvals in the European Union in 2018.health Updated: Jul 17, 2017 10:18 IST
A revolutionary new cancer therapy developed by Swiss pharmaceutical major Novartis moved closer to the market on Wednesday after getting unanimous approval from a panel of US experts.
The panel recommended that the Food and Drug Administration approve the drug, tisagenlecleucel, for patients ages 3 to 25 with relapsed B-cell acute lymphoblastic leukaemia (ALL), one of the most common form of childhood cancer.
The treatment, called CTL019, has been developed by Swiss pharmaceutical major Novartis. The therapy genetically alters the patient’s own cells to combat a form of leukaemia that mostly affects children and young adults.
The treatment involves drawing some blood from a patient, genetically modifying a type of immune cells call T-cells in it and re-infusing it into the patient. These re-programmed cells then find and kill all the cancerous cells within the body.
Novartis said that 52 of 63 cancer patients who received the treatment in a clinical trial were cancer-free after three months. These patients had mostly failed to respond to standard treatments or had relapsed.
When approved, the treatment will initially be available at 30 or 35 dedicated centres in the US. Some estimates put the cost at around US $300,000,
Norvartis plans to seek approvals in the European Union in 2018.
Indians will be able to access the treatment at US centres, which will also accepting international patients.
“This is a major advance and is ushering in a new era,” said committee member Malcolm A Smith, who is associate branch chief for paediatric oncology at the National Cancer Institute in Bethesda, Maryland.
The two significant concerns about the treatment are its severe side effects, which is some cases could be life-threatening, and the risk of secondary cancer from the re-infused cells in the long-term.