Novartis AG's Leukemia Drug CTL01 Clears Cancer In 90 Percent Of Patients

Published: Oct 20, 2014

Novartis's Leukemia Drug Clears Cancer In 90% Of Patients

October 16, 2014

By Jessica Wilson, BioSpace.com Breaking News Staff

Novartis AG and the University of Pennsylvania's Perelman School of Medicine (Penn) this week published in The New England Journal of Medicine (NEJM) preliminary results showing a 90 percent remission rate in patients treated for relapsed/refractory acute lymphoblastic leukemia (r/r ALL) with experimental drug CTL019.

The data from a nearly two year study showed 27 of 30 patients experienced complete remission after receiving CTL019, an investigational chimeric antigen receptor (CAR) therapy.

“These interim results, which supported the recent FDA Breakthrough Therapy designation, reinforce the potential CTL019 has as a life-saving therapy for patients with relapsed/refractory ALL,” Usman Azam, the global head of the Cell & Gene Therapies Unit at Basel, Switzerland-based pharma Novartis, in a statement.

The study ran from April 2012 to February 2014 and involved 25 pediatric patients and five adult patients. The patients were infused with autologous T cells, which are T cells derived from the same individual who receives the infusion. The T cells, prior to the infusion, were transduced with a CD19-directed CAR (CTL019) lentiviral vector. Transduction is a process that involves introducing DNA into another cell via a viral vector, or virus.

Novartis describes the process as follows, “CTL019 uses CAR technology to reprogram a patient's own T cells to ‘hunt’ cancer cells that express specific proteins, called CD19. After they have been reprogrammed, the T cells (now called CTL019) are re-introduced into the patient's blood; they proliferate and bind to the targeted CD19+ cancer cells and destroy them.”

Because the treatment uses the body’s own defenses to fight cancer cells, CTL019 is classified as immunotherapy, an exciting emerging field of cancer research. According to Bloomberg, immunotherapies as a whole could end up yielding $35 billion in annual sales for the pharmaceutical industry.

“We are excited by these results, which indicated how effective CTL019 may be in fighting ALL, a leading cause of childhood cancer deaths,” lead investigator Stephan Grupp, the Yetta Deitch Novotny Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania, said in a statement.

Grupp, who is also the director of Translational Research in the Center for Childhood Cancer Research at the Children's Hospital of Philadelphia (CHOP), where 25 pediatric patients were treated in the study cohort, continued, “This represents the largest experience to date of CD19-CAR T cells and demonstrates the ability of this approach to achieve sustained complete responses in a patient population with few other treatment options. We are especially hopeful for those patients who remain in remission for 1-2 years without further therapy.”

Novartis has worldwide rights to commercialize any CAR treatments for cancer developed in collaboration with Penn.

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