USFDA advisory panel unanimously approves first ever leukemia gene treatment


The advisory panel determined, unanimously, 10-0, that the benefit of the therapy outweighed the risks.

The one-time leukemia treatment, which will cost in the hundreds of thousands of dollars, would be for children and young adults with the most common form of childhood cancer, B-cell acute lymphoblastic leukemia, and who are facing death because other treatments have failed. "This is truly a turning point in the management of this disease". "It's an entirely new way of treating cancer". Many of these patients required treatment in the intensive care unit (ICU), but all of them survived.

ALL affects about 5,000 Americans each year, about 60 percent of whom are children or young adults.

Read: A Cure For Cancer?

In 2014, USA regulators designated CTL019 as a "breakthrough therapy" and put the experimental immunotherapy agent on the fast track to market approval, after 89 percent of leukemia patients in early trials saw their cancers disappear.

Speaking to the Times, Don McMahon described 12 hard years his son Connor spent in various treatments after he developed the illness at age 3. McMahon displayed painful photographs of Connor, bald and intubated during treatment.

Emily Whitehead, the first child to have been saved by the living drug treatment, at the age of six, also attended the panel discussion with her parents.

FDA reviewers said ahead of the meeting they were concerned about the potential for these viruses to infect patients and cause secondary cancers. The treatment nearly killed her, but she recovered and today is cancer free.

About 6,000 people are diagnosed with ALL in the United States each year and about 60 percent are children, according to the American Cancer Society.

Timothy Cripe, a panel member who is an oncologist with Nationwide Children's Hospital in Columbus, Ohio, called the treatment the "most exciting thing I've seen in my lifetime".

Here's how the approach works.

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If the FDA clears the treatment, it will be the first medical procedure of its kind to reach the masses.

In 2016, a Novartis exec told MM&M that the company plans to use a medical device-like sales model to market its CAR-T cell technology, which would rely more heavily on education for physicians than on detailing.

That tinkering instructs them to target and kill the ALL cells, the Times explained.

As Novartis edges closer to FDA approval for tisagenlecleucel-T, its engineered immune-cell therapy for leukemia, scientists continue to search for ways to improve upon this new mode of immuno-oncology treatments.

This super charges the T-cells to attack B-cells, which are immune cells that turn malignant in leukemia.

The recommendation of ODAC was based on the evaluation of the CTL019 r/r B-cell ALL development program that included the firm-lead ELIANA study, which is claimed to be the first pediatric global CAR-T cell therapy registration trial.

The treatments involve extracting white blood cells or T-cells which defend the immune system, from cancer patients. The treatment beat back her daughter's cancer and brought back "the sparkle" in her eyes.

A single one can destroy up to 100,000 cancer cells, the New York Times reported. The panel recommended patients who receive the treatment be monitored for 15 years.

The treatment will not be cheap, however.

How does CAR-T therapy work?

A novel cell treatment that saved the life of 9-year-old Austin Schuetz was given the green light by USA regulatory advisers on Wednesday and doctors hope it can save the lives of more children with the most common type of childhood cancer.