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SCIENCEFriday 29 December 2017
In a milestone year, gene therapy finds a place in medicine
By MARILYNN MARCHIONE In this Monday, Nov. 6, 2017 file photo, Brian Madeux sits with his girlfriend Marcie Caroline Carper of Little
AP Chief Medical Writer Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Rock, Arkansas. “And the
After decades of hope Children’s Hospital in Oakland, Calif. sunshine is blinding.”
and high promise, this was Gene therapies also
the year scientists really Associated Press showed some promise
showed they could doctor against a variety of diseas-
DNA to successfully treat nucleases for a man with a back to the lab, new un- works for other blood can- es including hemophilia ,
diseases. Gene therapies metabolic disease. It’s like derstandings emerge over cers. a blood clotting problem;
to treat cancer and even a cut-and-paste opera- years, and studies ultimate- Last week, the FDA ap- “bubble boy” disease,
pull off the biblical-sound- tion to place a new gene ly reveal what is safe and proved Luxturna, the first where a flawed immune
ing feat of helping the blind in a specific spot. Tests of effective. gene therapy for an inherit- system leaves patients vul-
to see were approved by another editing tool called Here is a look at what’s ed disease, a form of blind- nerable to fatal infections,
U.S. regulators, establish- CRISPR, to genetically al- been achieved and what ness. People with it can’t and sickle cell disease, a
ing gene manipulation as ter human cells in the lab, lies ahead. make a protein needed serious and painful blood
a new mode of medicine. may start next year. A STRING OF FIRSTS by the retina, tissue at the disorder common among
Almost 20 years ago, a “There are a few times in The year started with no back of the eye that con- black people.
teen’s death in a gene our lives when science as- gene therapies sold in the verts light into signals to the It’s not all good news,
experiment put a chill on tonishes us. This is one of U.S. and only a couple brain, enabling sight. The though. The therapies
what had been a field full those times,” Dr. Matthew elsewhere. Then the Food therapy injects a modified don’t work for everyone.
of outsized expectations. Porteus, a Stanford Univer- and Drug Administration virus containing a correc- They’re shockingly expen-
Now, a series of jaw-drop- sity gene editing expert, approved the first CAR-T tive gene into the retina sive. And no one knows
ping successes have re- told a Senate panel dis- cell therapies, which alter so the cells can make the how long some results will
newed hopes that some cussing this technology last a patient’s own blood cells protein. last, though scientists say
one-time fixes of DNA, the month. to turn them into special- Children who received the the aim is a one-time repair
chemical code that gov- It’s a common path for trail- ized cancer killers . They’re treatment told what it was that gets at the root cause.
erns life, might turn out to blazing science — success only for certain types of like to gain vision. “The whole promise ... is to
be cures. initially seems within reach, leukemia and lymphoma “Oh yikes, colors. Colors are cure diseases. It’s based
“I am totally willing to use setbacks send researchers now, but more are in the super fun,” said 13-year-old on the rationale of fixing
the ‘C’ word,” said the Na- the problem,” not just im-
tional Institutes of Health’s proving treatment, said Dr.
director, Dr. Francis Collins. Carl June, a University of
Gene therapy aims to treat Pennsylvania scientist who
the root cause of a prob- pioneered CAR-T therapy.
lem by deleting, adding or A NEW FRONTIER:
altering DNA, rather than GENE EDITING
just treating symptoms that In mid-November, Brian
result from the genetic Madeux, a 44-year-old
flaw. Phoenix man with a meta-
The advent of gene editing bolic disease called Hunter
— a more precise and long- syndrome, had just be-
lasting way to do gene come the first person to try
therapy — may expand an experimental gene ed-
the number and types of iting treatment. “I believe
diseases that can be treat- in science,” he texted The
ed. In November, Califor- Associated Press after doc-
nia scientists tried editing tors sent viruses containing
a gene inside someone’s a corrective gene and an
body for the first time, us- editing tool through an IV
ing a tool called zinc finger into his body. q
