- The Washington Times - Wednesday, August 30, 2017

Federal regulators green-lighted Wednesday an “historic” gene therapy that uses the body’s own immune cells to attack an acute form of leukemia in children and young adults.

The Food and Drug Administration approved Kymriah for treating patients up to age 25 who suffer from a type of quick-acting cancer in the bone marrow and blood.

The pioneering treatment collects white blood cells from patients, isolates components known as T-cells and genetically engineers them to multiply. The T-cells then seek and destroy leukemia cells after they’re infused back into the patient.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

The pursuit of this breakthrough in immunotherapy spans more than a century, back to when New York-based surgeon William Coley used a toxin in 1890 to stimulate cancer patients’ immune responses, according to a blog post Wednesday by Francis Collins, director of the National Institutes of Health.

He said interest in the field waxed and waned over the decades and required many advances in bio-sciences to get to this point, though there is more work to be done.

“Many questions must be addressed before we can herald immunotherapeutic approaches to cancer an unqualified success. There are still too many severe reactions, too many non-responses or relapses, and, potentially, a very high price tag for their widespread use, which will be truly challenging to scale up,” he wrote. “But we’re off to a promising start.”

Dr. Gottlieb praised the researchers, drug companies and patients who spent decades developing and trying out the new class of drugs, saying Wednesday’s announcement marked the first step in delivering on the “original promise” of gene therapy.

He said the form used in Kymriah, known in lab-speak as CAR-T, holds promise for treating genetic and autoimmune disorders, diabetes, HIV and heart disease, among other conditions.

Among the leading contenders are new treatments for cancer, including leukemia in adults.

Right now, the agency is reviewing 76 investigational drug applications related to CAR-T therapies, such as Kite Pharma’s request to approve its product “axi-cel” for aggressive non-Hodgkin lymphoma.

“Today is just the first approval in this promising new class of medical products,” Dr. Gottlieb said.

Kymriah is administered through a one-time, intravenous infusion. It was developed by Novartis Pharmaceuticals Corp. and the University of Pennsylvania to treat patients with acute lymphoblastic leukemia (ALL), which is the most common type of cancer among U.S. residents up to age 20.

Roughly 3,100 patients are diagnosed per year.

One trial found that 83 percent of 63 participants went into remission within three months of treatment.

“This therapy is a significant step forward in individualized cancer treatment that may have a tremendous impact on patients’ lives,” said Dr. Carl June, a professor of immunotherapy at UPenn’s Perelman School of Medicine and who helped to develop the treatment.

The FDA warned that Kymriah could have severe side effects related to an immune response known as “cytokine release syndrome,” which can cause high fever, flu-like symptoms and neurological problems. Other side effects include high blood pressure and kidney problems.

Because of the risks, the government says hospitals and clinics that dispense the drug must be specially certified. They also must be ready to treat cytokine release syndrome.

Novartis also will be required to study the effects of its drugs on patients treated with its product.

The ground-breaking treatment won’t be cheap. The one-time treatment will cost $475,000, although that is less that what some outside analysts anticipated.

Novartis said it would have been justified in charging $600,000 to $750,000, but set a lower price to ensure wider access.

“We believe this [price] will support sustainability of the healthcare system and patient access while allowing a return for Novartis on our investment,” the company said in a statement.

Novartis said it will work with patients to navigate their insurance coverage and offer financial assistance to the uninsured and under-insured.

It also struck an agreement with the U.S. government to seek payment only if a patient responds well to the drug within the first month after treatment.

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