StemCell Therapy

In 1990, geneticist William French Anderson injectedcells with altered genes into a four-year-old girl with severe immunodeficiency disorder. This was the first sanctioned test of gene therapy, in which genetic material is used to treat or prevent disease.

If were lucky, Anderson told The Chicago Tribune, with this little girl weve opened the door for genetic engineering to attack major killers and cripplers, particularly AIDS, cancer and heart disease.

Gene therapy has never fulfilled these grand hopes. In the decades since Andersons experiment, thousands of clinical trials of gene therapies have been carried out. But the first gene therapy was only approved for sale in the U.S. this week. The Food and Drug Administration announced its approval of Kymriah, a gene therapy produced by Novartis for a form of childhood leukemia. A few gene therapies have previously become available in Europe and China.

An FDA press release emphasizes the historic nature of the approval. Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb says.

As I have noted previously, for gene-therapy proponents have long predicted that it will eliminate diseases such as cystic fibrosis and early-onset breast cancer, which are traceable to a defective gene. Enthusiasts also envisioned genetically engineered "designer babies" who would grow up to be smarter than Nobel laureates and more athletic than Olympians.

Gene therapy turned out to be extremely difficult, because it can trigger unpredictable, fatal responses from the body's immune system.The National Institutes of Health warnsthat gene therapy can have very serious health risks, such as toxicity, inflammation, and cancer.

Kymriah is a case in point. The FDA press release warns that Kymriah can cause life-threatening immune reactions and neurological events, as well as serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). According to The New York Times, the FDA is requiring that hospitals and doctors be specially trained and certified to administer [Kymriah], and that they stock a certain drug needed to quell severe reactions.

Kymriah illustrates another problem with gene therapy: high cost. Novartis estimates the cost of its treatment at $475,000 per patient. As a recent Reuters article notes, over the past five years two gene therapies have been approved for sale in Europe, one for a rare blood disease and the other for the bubble-boy immunodeficiency disorder. The therapies cost $1 million and $700,000, respectively. So far, the companies that make the therapies have achieved a total of three sales.

As journalist Horace Freeland Judson points out in this excellent 2006 overview, The Glimmering Promise of Gene Therapy, most individual diseases caused by single-gene defectsthe kind that seem most likely to be cured by gene therapyare rare. (Sickle-cell anemia and some other hemoglobin disorders are among the few exceptions.)

Judson adds that because different diseases have different genetic mechanisms and affect different types of tissue, each presents a new set of research problems to be solved almost from scratch. As the millions burned away, it became clear that even with success, the cost per patient cured would continue to be enormous. And success had shown itself to be always glimmering and shifting just beyond reach.

The advent of CRISPR, a powerful gene-editing technique, has aroused hopes that gene therapy might finally fulfillexpectations. Researchers recently reported that they employed CRISPR to counteract a mutation that causes heart disease. Potentially, The New York Times reported last month, the method could apply to any of more than 10,000 conditions caused by specific inherited mutations.

CRISPR has also renewed concerns about the ethics of producing designer babies with enhanced physical and mental traits. But as Science noted recently, CRISPR poses some of the same risks as gene therapies. CRISPR still has a long way to go before it can be used safely and effectively to repairnot just disruptgenes in people.

So to answer the question posed in the headline: No, the gene-therapy era has still not arrived.

See the original post here:
Has the Era of Gene Therapy Finally Arrived? - Scientific American (blog)

This entry was posted on September 1, 2017 at 12:42 pm and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |