FDA panel backs new cancer treatment

FDA panel backs new cancer treatment

FDA panel backs new cancer treatment

In the meantime, the panel recommended that the FDA approve the drug for patients ages 3 to 25 with relapsed B-cell acute lymphoblastic leukemia, the most common form of childhood cancer in the US.

Loncar said approval of tisagenlecleucel would represent an inflection point for investors.

CAR-T is a novel therapy and could lead to changing the treatment standards for cancer. Those prior treatments involved multiple spinal taps and painful bone marrow testing, all of which was tough to tolerate and left his son infertile, McMahon explained. The trial lacked a control group, so investigators can not yet say with certainty how much of an effect the treatment had.

At the staring of this year, Novartis filed a BLA for CTL019 to the FDA.

One of those patients, Emily Whitehead, now 12, was at the panel's meeting with her parents, who advocated for approval of the drug. Of 68 young people receiving it, 52 of them had an excellent response nearly immediately, with their cancer disappearing within the first three months. But many of those participants have remained cancer free for months or years.

The missing men are identified as 19-year-old Dean Finocchiaro, 19-year-old Jimi Tar Patrick, 21-year-old Tom Meo and 22-year-old Mark Sturgis.

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.

Here's how the approach works.

Close on Novartis' heels is a team from Kite, a CAR-T-focused biotech which had hoped to be the first to the market. The cells would be frozen and sent to Novartis's facility, where they would be engineered to hunt for a protein marker called CD19 found in some blood cancers.

That tinkering instructs them to target and kill the ALL cells, the Times explained. Then sensor cells (antigen receptors) that can seek out certain cancers and destroy them are added to the T-cells. Past trials of the cells in patients have shown that they don't cause graft vs. host disease-a risky effect of unmatched T cells, which can attack normal tissues. Gene therapies, this camp might argue, require more editing, and CAR-T is simply a cell therapy. It involves removing a patient's blood and essentially replacing it with a population of blood cells stacked with cancer-fighting immune cells known as T cells.

Approval of tisagenlecleucel would have implications not only for Novartis, but for companies developing similar treatments, including Kite Pharma Inc, Juno Therapeutics Inc and bluebird bio Inc. Doctors call it a "living drug" - permanently altered cells that continue to multiply in the body to fight the disease.

Treatment with a single dose of the resulting product had produced long remissions, and possibly cures, in scores of patients in studies who were facing death due to the failure of every other treatment.

The treatment will not be cheap, however. Long-term management of leukemia can cost tens of thousands of dollars per year, and can even run to more than one hundred thousand dollars.

Novartis will limit the use of CTL019 initially because the treatment process is complex and managing side effects demands expert care. According to researchers, only time will tell for sure if the latter fear is unfounded or not.

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