Revolutionary Gene Therapy Treatment For Cancer Gets Approval In The USA

Phillip Cunningham
September 1, 2017

Despite these negatives, FDA approval of gene therapy is a big step for the treatment both in the U.S. and around the globe. The treatment is called a living drug because involves using genetically modified immune cells from patients to attack their own cancer. The first gene therapy to gain FDA approval for an inherited disease could be a treatment for a form of blindness.

The drug was one of several CAR-T therapies rushing to be the first to market.

But the new therapy doesn't come cheap: Developed by Novartis Pharmaceuticals and the University of Pennsylvania, the one-time infusion is priced at a reported $475,000. The new cells would then continue to multiply to fight disease for months or years. The FDA approval of this therapy is based on the results of the Phase II ELIANA trial, which was sponsored by Novartis and included 25 centres in the US, EU, Canada, Australia and Japan.

Novartis plans additional filings for Kymriah in the U.S. and EU later this year, including applications with the FDA and European Medicines Agency (EMA), for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL). "Not only does [tisagenlecleucel] provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials".

There are other severe side effects that can pop up as well that can require hospitalization. After being infused back into patients' bodies, these newly built "hunter" cells both multiply and attack, targeting cells that express a protein called CD19. "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. Kymriah is approved for use in pediatric and young adult patients with B-cell ALL and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 percent of patients.

Until recently, the use of vehicle T-cell therapy has been restricted to small clinical trials, largely in patients with advanced blood cancers.


"Patients who get this therapy basically have no chance to survive", Satwani said.

"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. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of vehicle T-cells causing high fever and flu-like symptoms, and for neurological events.

"The only potentially curative option for these pediatric and young adult patients is allogeneic stem cell transplant, which costs $500,000 to $800,000 for the first year", Cooper said.

For one, the treatments would have to treat more types of cancer than the one Kymriah was approved for on Wednesday. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years. CAR-T's side effects can be deadly.

Over the last few years, CAR-T cell research has exploded, and there are now nearly 300 clinical trials underway experimenting with the treatment.

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