First lab-made cancer-hunting blood cells approved by the FDA

It is the first gene therapy of its kind to hit the USA market.

Novartis licensed the therapy from Penn in 2016. "We worked on it and worked on it and finally to have such a discrete, palpable event like this is such an wonderful thing". Baldrick's Pediatric Cancer Dream Team (PCDT), which has been instrumental in the development of this new approach by committing millions of dollars to the research of vehicle T cell therapy.

Scott Gottlieb, the FDA commissioner, said of the treatment: "We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer". "We are actively preparing to offer this novel treatment at our center to patients at Dana-Farber/Boston Children's".

What is the therapy and how does it work?

"Patients are involved in making their own drug".

Like all CAR-T therapies, Kymriah involves reprograming body-guard T cells to contain a gene that codes for a protein called chimeric antigen receptor or vehicle. When the body's immune system kicks into high gear to fight the cancer, extremely high fevers can occur as well as the production of proteins that produce inflammation. They are then reintroduced back into the patient to do battle against the tumor. "To do that definitely shifts everything". And while Novartis' success was widely expected, the FDA's decision to rapidly clear the drug underscores the excitement that doctors and the life sciences community feel about such therapies, which have been shown to eliminate all signs of aggressive blood cancers in some patients after just one treatment in clinical trials. When that deal was finalized, June said in an interview with Bloomberg that he had never imagined that pharma companies would move into "ultra personalized therapy" and explained why they decided against developing a new company.

Despite its great promise, the FDA warns that Kymriah does have the potential for severe side effects.

Novartis plans to file for FDA approval of Kymriah for a type of non-Hodgkin lymphoma that affects adults later this year. Research is therefore focusing for now on refining the therapy for patients of this disease. The treatment is for those who have not responded to initial treatment, which occurs in an about 15-20 percent of patients, or those who have relapsed. The modification provides a chimeric antigen receptor (CAR) which directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. The disease progresses quickly and is the most common childhood cancer in the US. Mitchell said Novartis should not "get credit" for bringing an expensive drug to market "and claiming they could have charged people a lot more". "There will be shortages". Medications can control the extent of the reaction, but some early patients died of the syndrome.

And then there's the issue of cost.

Shares of Juno Therapeutics Inc, which past year reported a handful of patient deaths during trials of its CAR-T therapy, were down 8.9 percent at $39.92.

About The Leukemia & Lymphoma Society The Leukemia & Lymphoma Society® (LLS) is the world's largest voluntary health agency dedicated to blood cancer. Several recent patient deaths in CAR-T clinical trials have raised serious questions over the safety of the treatment.

  • Joanne Flowers