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FDA Approves First Gene Therapy

4 September 2017
Giulio Prisco

FDA, Food and Drug Administration, US Department of Health and Human Services

"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, referring to the "historic" approval of the first gene therapy in the US (see below), adding that new technologies such as gene and cell therapies could transform medicine. "With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care," said Novartis CEO Joseph Jimenez.

Kymriah is based on Chimeric Antigen Receptor T (CAR-T) cells, immune T cells modified with engineered receptors, also featured in other spectacular research news released last week.

There is, however, a catch: Novartis priced the drug at $475,000 for one treatment, Bloomberg reports, “well below even higher expectations of up to $700,000.”

This raises ethical concerns. Of course, drug development costs money, and a private company like Novartis is entitled to recover costs and make a profit. But, in the US, the FDA approval process itself adds billions of dollars to drug development costs. Therefore, it could be argued that suffering US patients are ultimately required to pay hundreds of thousands of dollars each to support inefficient regulation.

In related news, Peter Thiel and other libertarian investors are funding an offshore human clinical trial of a herpes vaccine, “skirting FDA regulations and sparking a heated debate over US safety rules,” as reported by TechCrunch. As usual, facts play a very minor role in the heated and heavily politicized debate, but a Reason article tries to at least get the facts right.

Futurism reports that scientists at the Albert Einstein College of Medicine claim that they have discovered the maximum lifespan of human beings, which is about 125 years. But the “cut-and-paste” CRISPR gene editing technique “is making it possible to modify DNA, the source code of life itself,” which may allow us to extend that maximum lifespan.

See also Pulse 22 for a review of the recently published book, A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution, co-authored by Jennifer Doudna, one of the inventors of CRISPR, and the recent AMA (Ask Me Anything) session hosted by CRISPR researcher Paul Knoepfler, Professor at UC Davis, on Reddit.

First gene therapy approved by the FDA, officially available in the US. The US Food and Drug Administration (FDA) has approved Kymriah, a Chimeric Antigen Receptor T cell (CAR-T) therapy, which becomes the first gene therapy officially available in the United States. Kymriah (tisagenlecleucel), from Novartis, has been approved for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

First response of central nervous system tumor to CAR T-cell therapy. Scientists at Massachusetts General Hospital (MGH) have found a remarkable treatment response in a patient participating in a clinical trial of an investigational CAR T-cell therapy. The research results, published in New England Journal of Medicine, indicate that the treatment induced complete remission of a brain metastasis of the difficult-to-treat tumor diffuse large-B-cell lymphoma (DLBCL), which had become resistant to chemotherapy. This is the first report of a response to CAR T-cells in a central nervous system lymphoma.

Single-molecule nanomachines penetrate cell membranes to heal or kill cells. Researchers at Rice University, North Carolina State University and Durham University have developed motorized single-molecule nanomachines, driven by light, able to penetrate the membranes of individual cells, either bringing therapeutic agents into the cells or directly inducing the cells to die. The study, published in Nature, describes how the scientists created several different light-activated motorized molecules designed to home in on specific cells, drill through cellular membranes using their molecular-scale nanomechanical action, induce necrosis, and introduce chemical species into live cells. The researchers expect that molecular machines could eventually also be used in live subjects.

Nanoparticles deliver mRNA to cells for ‘hit-and-run’ gene therapy. Scientists at Fred Hutchinson Cancer Research Center have used nanoparticles to deliver transient gene changes to targeted cells. A study published in Nature Communications describes a nanoparticle delivery system to extend the therapeutic potential of messenger RNA (mRNA), which delivers molecular instructions from DNA to cells in the body, directing them to make proteins to prevent or fight disease. According to the scientists, their method could someday make “hit-and-run” gene therapy feasible around the world, even in areas with little or none of the specialized technology now needed to genetically engineer cells.

DNA nanoparticles permit analyzing how neurons work. Researchers at the University of Chicago have developed a way to use nanopackages made out of DNA to deliver a payload of tiny molecules directly into a cell. DNA is a suitable material because it can dissolve harmlessly once its purpose is achieved and, like LEGO, it has standard interlocking pieces that make it easy to build into configurations. The research results, published in Nature Nanotechnology, describe how the technique permits analyzing the fast interaction between neurons and neurosteroids, chemical messengers that play a key role in the brain.

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