Novartis and Gene Therapy: Hope for Patients with Stage 4 Cancer

Mia Cappuccitti
Nov 5 · 3 min read

Novartis, a multinational pharmaceutical company, is using gene therapy to revolutionize the way cancer is being treated. The technology surrounding it is on the frontier of medical innovation. It is essentially a therapy for diseases that so far do not have any other cure (it is specifically geared to certain types of cancer). It’s purpose is to replace faulty genes or introduce new ones into the human body to prevent or fight deathly diseases.

Who is Novartis?

Novartis’s aim is to discover, develop and perfect breakthrough medicines and technologies that will impact millions of lives for the better.

“Our purpose is to re-imagine medicine to improve and extend people’s lives. We use innovative science and technology to address some of society’s most challenging healthcare issues.”

Their goal is to be a leader in changing the practice of medicine. And they are doing just that. In 2017, the FDA approved Novartis’s gene therapy treatment, naming it the “first gene therapy in the U.S.”

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA commissioner Scott Gottlieb in a statement.

A Closer Look at Gene Therapy.

The technology behind gene therapy is very complex, but let me give you a crash course in how it works and how Novartis is implementing it:

Okay, so, what actually is a gene? Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body. Genetic diseases happen when genetic mutations occur (a crucial part of the DNA is duplicated, deleted, etc.). Through gene therapy, we can go directly to the source of the problem and change a faulty gene for a clean one or introduce a new gene to the body to help rid the body of a disease or help the body fight the disease.

What are They Doing with Gene Therapy?

Novartis has started a clinical trial for patients with, “an often lethal type of blood and bone marrow cancer that greatly affects children and young adults.” The therapy is essentially a customized treatment that uses a patients own T-cells.

“Though the Novartis therapy has shown extraordinary results in patients, questions remain about how the company will be able to manufacture personalized therapies quickly enough to get them to patients across the country.”

Novartis has said that it takes a average 22 days to create the therapy from the time a patient’s cells are extracted to when they are infused back in.

A patient’s T cells are taken out and cryogenically frozen and are then transported to Novartis’s manufacturing center which is in New Jersey.

“ The cells are genetically altered to have a new gene that codes for a protein — called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient,” explains the associate editor for biomedicine at the MIT Technology Review.

The treatment will be sold by Novartis for $450,000.

Novartis says eventually a total of 32 hospitals/sites will offer this therapy. The therapy is known as Kymriah. Kymriah will start by available at 20 U.S. hospitals within a month.

Mia Cappuccitti

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