Young Emily Whitehead, who turned 7 in May, was saved from near death from leukemia after relapsing twice after chemotherapy – and with all viable options running out. In desperation, her parents sought experimental treatment at the Children’s Hospital of Philadelphia, using a disabled form of the virus that causes AIDS to reprogram her immune system genetically to kill cancer cells.
“She is the first child and one of the first humans ever in whom new techniques have achieved a long-sought goal — giving a patient’s own immune system the lasting ability to fight cancer,” reports The New York Times.
The treatment, developed at the University of Pennsylvania, the Times noted in a separate story, “may signify a turning point in the long struggle to develop effective gene therapies against cancer. And not just for leukemia patients: other cancers may also be vulnerable to this novel approach — which employs a disabled form of H.I.V.-1, the virus that causes AIDS, to carry cancer-fighting genes into the patients’ T-cells. In essence, the team is using gene therapy to accomplish something that researchers have hoped to do for decades: train a person’s own immune system to kill cancer cells.”
“Our goal is to have a cure, but we can’t say that word,” said Dr. Carl June, lead of the U Penn research team, echoed by his colleague, Dr. John Wagner, director of pediatric blood and marrow transplantation at the University of Minnesota, who said the Pennsylvania results were “phenomenal” and “what we’ve all been working and hoping for but not seeing to this extent. I think this is a major breakthrough.”
Cue Novartis, which has committed $20 million to building a research center on the university’s campus to ready the treatment for public consumption. In August 2012, Novartis acquired exclusive rights from Penn to CART–19, the therapy now known as CTL019. Unlike trials for commercial development of drugs like Viagra or cholesterol meds where millions consume the same drugs, Emma’s treatment requires a new batch of T-cells for each patient.
Hervé Hoppenot, president of Novartis Oncology, called the research “fantastic” and likely “to revolutionize the treatment of leukemia and related blood cancers,” reports the Times. Future applications could include reprogramming a patient’s immune system against tumors like breast and prostate cancer. Hoppenot also said Novartis is looking for cancer treatments with big impact for small numbers, “home-run” drugs that require smaller studies and gain quicker approval. “The economic model is totally acceptable,” he said.
Novartis International AG is a Swiss multinational pharmaceutical company with US revenue of $58.566 billion (2011) from the manufacture of clozapine (Clozaril), diclofenac (Voltaren), carbamazepine (Tegretol), valsartan (Diovan), imatinib mesylate and (Gleevec / Glivec). Additional agents include cyclosporin (Neoral / Sandimmun), letrozole (Femara), methylphenidate (Ritalin), terbinafine (Lamisil), among others.
Engineered T-cells cost about $20,000 per patient, much less than a bone-marrow transplant and scale will likely reduce that price tag. “We are excited to see that the CTL019 approach may be effective in untreatable cases of pediatric ALL as well,” stated pediatric oncologist Stephan A. Grupp, M.D., Ph.D., of The Children’s Hospital of Philadelphia. “Our hope is that these results will lead to widely available treatments for high–risk B cell leukemia and lymphoma, and perhaps other cancers in the future.”
In Emily’s case, the treatment nearly killed her, but after hours of “shake and bake” reaction, raging fevers and chills, placed on a ventilator, unconscious and severely swollen, it resulted in stabilization and recovery and Emily woke after one week on May 2nd, the day she turned 7.
Collaboration between major drug companies and cutting-edge medical research such as that being carried out at the Children's Hospital of Philadelphia is the best possible partnership, leveraging resources, profit and humanity in an eco-system that merges what makes us human: our hope, hearts and minds. And the gift that keeps on giving: kids like Emily.