No Action Without Reaction

"[Kymriah represents] a transformative therapy ... It represents an entirely new class of cancer therapies that holds promise for all cancer patients."
Dr. Crystal L. Mackall, associate director, Stanford University Cancer Institute

"Those patients [children and young adults who do not respond to chemotherapy or have their leukemia return] don't make it [survive] -- none of them do."
"Certainly for blood cancers, this is a game-changer."
"[Adapting this therapy for patients with solid tumours will be] the work of the next five years."
Dr. Stephan A. Grupp, director, cancer immunotherapy program, Children's Hospital of Philadelphia

Emily Whitehead, shown with her parents, Thomas and Kari Whitehead, was the first child treated with a new type of cancer therapy that uses the patient's own genetically modified cells to fight cancer. The Food and Drug Administration approved the therapy on Wednesday. (Sean Simmers for The Washington Post)

Dr. Grupp, in his specialty and in his exposure to children and young adults, estimated to be around 600 in each year whose lives are threatened -- knows the reality of the situation first-hand. And it was he who pioneered the use of Kymriah five years earlier while it was still an experimental treatment called CTL019. Now 12 years old, Emily Whitehead of Philipsburg, Pennsylvania experienced the miracle of her leukemia undergoing a complete remission within three weeks of the first administration of the treatment.

The process she underwent began when her T cells -- fighters of the immune system -- were harvested, delivered to a specialized laboratory where the DNA underwent alteration by scientists to reprogram them to specifically target cancer cells aggressively. The re-engineered cells are called CAR-T cells: chimeric antigen receptor T cells. Infused into the bloodstream, the CAR-T cells that have been engineered to relentlessly hunt down cancer cells and kill them do their work.

In the near future, this new gene therapy which is now out of the experimental stage will see patients up to age 25 attending one of the 32 certified treatment centres that Novartis, the pharmaceutical company that developed Kymriah plans to build, to have their T cells harvested, processed and reintroduced in their modified form. The Swiss company is preparing to provide Kymriah to up to 600 patients yearly. At a charge of $475,000 for each treatment.

Acute lymphoblasic leukemia, the most common type of pediatric cancer, afflicts around three thousand children and young adults in the United States annually. For most patients the condition is thought of as highly curable through conventional treatment. For a sizeable number of patients, however, about 600 yearly, their exposure to that treatment fails to work, and they die.

The U.S. Food and Drug Administration has now approved this type of highly effective gene therapy specifically geared at that aggressive form of leukemia in young patients with no other options left to them. This a new personalized medical concept whose function is that of a "living drug" where patients' own T cells have been fortified and multiplied then reinfused back into the patients' blood stream to help fight their cancer.

Clinical trials involving 88 patients with relapsing or treatment-resistant forms of acute lymphoblastic leukemia resulted in 73 of those trial subjects going into remission. Oddly enough Scott Gottlieb, FDA Commissioner, is personally involved in the sense that he is himself a survivor of blood cancer, so he took particular pleasure in predicting the new approach would have the effect of "chang[ing] the face of modern medicine".

On a more sobering note, the FDA has also called for continuing studies of the new therapy with respect to improving its safety. For in approving Kymriah the agency also warned of its potential to cause severe side effects which can themselves be life-threatening. Kymriah has the potential to cause cytokine release syndrome as an overreaction to the activation and proliferation of immune cells, to cause fever and flu-like symptoms and possibly neurological events.

Carrying a $475,000 price-tag

AP