Panel Approves New Cancer Drug, Paving the Way for First FDA-Approved Gene Therapy

Pharma giant Novartis’s new cancer drug tisagenlecleucel passed a critical test by winning the support of a federal advisory panel – setting it up for final FDA approval in September.

If approved,this would mark the first U.S. gene therapy -and a significant medical breakthrough!

Tisagenlecleucel is idnicated for for patients ages 3 to 25 with relapsed B-cell acute lymphoblastic leukemia (ALL), the most common type of childhood cancer in the United States.

“This is a potentially paradigm-changing type of benefit,” said Dr. Brian Rini, a panelist and physician at Cleveland Clinic Taussig Cancer Institute.

The drug uses a new technology known as CAR-T (chimeric antigen receptor T-cell) therapy, which employs the use of the body’s own immune cells to target and attack cancerous cells. This powerful technique has been in development in one form or another for decades – and now is poised to have an effect on all forms of cell therapy.

Cell therapy is on the front lines of treating and curing countless diseases – especially cancers. By perfecting this CAR-T therapy, the door for researching, treating and ultimately curing these diseases is flung open. Not to mention the tremendous impact this could have on rare diseases.

And of course, this development offers a very tangible hope for those with ALL – many of whom are children.

Patient Worthy will keep a close eye on any developments – and we surely expect to get some good news in September!

To read more about the trials in Reuters, click here.

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