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In a milestone year, gene therapy finds place in medicine

  • FILE - In this Monday, Nov. 6, 2017 file photo, Brian Madeux sits with his girlfriend Marcie Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Children's Hospital in Oakland, Calif. Madeux, who has Hunter syndrome, received the treatment on Monday, Nov. 13. Gene therapy aims to treat the root cause of a problem by deleting, adding or altering DNA, rather than just treating symptoms that result from the genetic flaw. (AP Photo/Eric Risberg) Eric Risberg

  • FILE - In this Oct. 4, 2017, file photo, Dr. Albert Maguire, right, checks the eyes of Misa Kaabali, 8, at the Children's Hospital of Philadelphia. Misa was 4-years-old when he received his gene therapy treatment. On Tuesday, Dec. 19, 2017, the Food and Drug Administration approved the therapy which improves the vision of patients with a rare form of inherited blindness, another major advance for the burgeoning field of genetic medicine. (AP Photo/Bill West, File) Bill West

  • Jay Konduros, left, and his brother, Bill, at Jay’s home in Cambridge, Ontario, Canada. They recently received genetic treatment for hemophilia. photo via ap



Associated Press
Thursday, December 28, 2017

After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.

Almost 20 years ago, a teen’s death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures.

Gene therapy aims to treat the root cause of a problem by deleting, adding or altering DNA, rather than just treating symptoms that result from the genetic flaw.

The advent of gene editing — a more precise and long-lasting way to do gene therapy — may expand the number and types of diseases that can be treated. Tests of another editing tool called CRISPR, to genetically alter human cells in the lab, may start next year.

“There are a few times in our lives when science astonishes us. This is one of those times,” Dr. Matthew Porteus, a Stanford University gene editing expert, told a Senate panel discussing this technology last month.

It’s a common path for trail-blazing science — success initially seems within reach, setbacks send researchers back to the lab, new understandings emerge over years, and studies ultimately reveal what is safe and effective.

Here’s a look at what’s been achieved and lies ahead.

A STRING OF FIRSTS

The year started with no gene therapies sold in the U.S. and only a couple elsewhere. Then the Food and Drug Administration approved the first CAR-T cell therapies, which alter a patient’s own blood cells to turn them into specialized cancer killers. They’re only for certain types of leukemia and lymphoma now, but more are in the works for other blood cancers.

Last week, the FDA approved Luxturna, the first gene therapy for an inherited disease, a form of blindness. The therapy injects a modified virus containing a corrective gene into the retina so the cells can make the protein.

Children who received the treatment told what it was like to gain vision.

“Oh yikes, colors. Colors are super fun,” said 13-year-old Caroline Carper of Little Rock, Arkansas. “And the sunshine is blinding.”

Gene therapies also showed some promise against a variety of diseases including hemophilia, a blood clotting problem; “bubble boy” disease, where a flawed immune system leaves patients vulnerable to fatal infections, and sickle cell disease, a serious and painful blood disorder common among black people.

A NEW FRONTIER: GENE EDITING

In mid-November, Brian Madeux, a 44-year-old Phoenix man with a metabolic disease called Hunter syndrome, had just become the first person to try an experimental gene editing treatment.

“I believe in science,” he texted The Associated Press after doctors sent viruses containing a corrective gene and an editing tool through an IV into his body. The hope is that the gene and the editing tool would enter some of his liver cells and insert the instructions needed to start making an enzyme he lacks.

WHAT’S NEXT

Top of one list: muscular dystrophy and sickle cell.

There’s been so much progress that the NIH has modified an oversight panel that just a few years ago reviewed every gene therapy experiment in the U.S. Most are considered safe enough to go ahead without the Recombinant DNA Advisory Committee’s review. The panel hasn’t even met for a year.