USA clears first 'living drug' for childhood leukaemia

Safety and efficacy of tisagenlecleucel were demonstrated in a multicenter clinical trial comprising 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. Tisagenlecleucel (Kymriah) is a personalized genetically-modified autologous T cell immunotherapy created to use a patient's own T cells to target and kill leukemia cells with CD19 antigens on the surface, according to a press release.

Novartis' CAR-T, which will be marketed as Kymriah, leads an emerging field of cell therapies created to engineer the body's immune cells to seek out and destroy cancers.

This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia, which strikes more than 3,000 children and young adults in the United States each year.

The treatment is not a pill or a form of chemotherapy.

The disease is the most common type of childhood cancer in the US, making up 25 percent of cancer diagnoses for children under 15 years old, according the National Cancer Institute.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", stated FDA Commissioner Scott Gottlieb. It's these patients for whom Kymriah is intended. Some 83 percent of those given the therapy were in remission within three months of treatment, the agency said. Once modified, the cells are infused back into the patient to kill the cancer cells.

While promising, CAR-T treatments won't be like other drugs that win FDA approval, which can quickly wind up on pharmacy shelves and hospitals.

Kymriah treatment also has the possibility of serious side effects, including cytokine release syndrome (CRS), which consists of high fever and flu-like symptoms and can be life-threatening, and neurological events, which are also life-threatening. In addition, clinicians involved in the prescribing, dispensing, and administering of the therapy will need training to recognize these side effects. Kymriah's maker, Novartis, hasn't provided a price for the drug.

"Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need", Novartis CEO Joseph Jimenez said in a statement.

The others still are being tracked to see how they fare long-term.

For more on childhood acute lymphoblastic leukemia, visit the U.S.