Published: Fri, September 01, 2017
Medical | By Rita Mcdonald

Cancer drug a major breakthrough - but it has a hefty price tag

Cancer drug a major breakthrough - but it has a hefty price tag

Pharma giant Novartis on Wednesday won highly anticipated U.S. approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, an exhorbitantly priced treatment that nevertheless marks the start of a potential new treatment approach for some cancers.

US regulators approved a revolutionary new treatment from Novartis that modifies the body's own immune-system cells and reinjects them to treat, and in many cases cure, patients with deadly blood cancers who have run out of other options.

Stephan Grupp, MD, PhD, a researcher at Children's Hospital of Philadelphia who helped test Kymriah in early studies, said he hopes the therapy could eventually replace bone-marrow transplants for these young patients. Novartis CEO Joseph Jimenez, however, says developing the treatment cost more than $1 billion.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA commissioner Scott Gottlieb.

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The one-time treatment isn't cheap: Novartis said on Wednesday that the price would be $475,000, which is actually lower price than many expected.

Kymriah, which is made by Novartis, isn't your run-of the-mill pill, or even a biologic drug, like insulin, that can be mass-produced.

"Not only does Kymriah provide these patients with a new treatment option were very limited options exist, but a treatment option that has shown promising remission and survival rates in clinical trials", he added. "There are also over 800 cell therapy clinical trials now underway, and a considerable pool of research and pre-clinical work right across the cell therapy sector", said Bruce Levine, PhD, ISCT Commercialization and Immuno & Gene Therapy Committees, Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania Perelman School of Medicine.

"The efforts involved in being able to provide this novel therapy to patients is unlike any other therapy now offered", says Colleen Dansereau, MSN, RN and CPN, who is director of clinical research nursing and gene therapy program manager at Dana-Farber/Boston Children's.

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Swiss pharmaceuticals group Novartis has said it will charge $475,000 (CHF458,000) a patient for its new cancer therapy, putting it among the most expensive drugs of all time. It was identified in previous reports as CAR-T cell therapy, CTL019 or tisagenlecleucel. "The therapies that we're using to treat children 20 years from now will emerge from the investment in science that we make today".

Epstein said he envisioned cell therapies having much shorter life cycles than traditional drugs. Eventually, the therapy will be offered at 32 sites, the company said.

Health officials in the United States have approved a revolutionary treatment that uses genetic engineering to combat childhood leukemia. Instead of getting an updated therapy that works better on a disease every decade or so, we might begin to see second-generation cell therapies in just a few years.

Doctors don't yet know what the full range of long-term side effects will be. And previous year, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.

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