Health Rounds: Editas gene editing treatment restores sight in inherited blindness

Editas Medicine

07/05/2024 20:23

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    By Nancy Lapid
       May 7 (Reuters) - Hello Health Rounds Readers! Today we
have another potential win for the field of gene editing -
restoring eyesight to the blind - in this case, in people with
an inherited retinal disease. We also report on a tiny
implantable pumping device that may allow small children to live
more normal lives while awaiting a heart transplant, and data on
a drug that may help people quit nicotine vaping.   

    Gene editing restores vision in inherited blindness
    A gene editing treatment from Editas Medicine  EDIT.O 
showed promise for treating an inherited form of blindness in a
small early trial, researchers reported on Tuesday at the
Association for Research in Vision and Ophthalmology meeting in
Seattle and in The New England Journal of Medicine.  
    The 12 adults and two children in the trial were born with a
form of the retina disease called Leber Congenital Amaurosis 
caused by mutations in the centrosomal protein 290 (CEP290)
gene. They each received a single injection of the Editas gene
editing treatment - EDIT-101 - in one eye. 
    The main goal of the study was to assess the safety of the
treatment. No serious adverse events occurred, the researchers
said. 
    Eleven of the 14 patients experienced “measurable
improvements” in visual acuity, use of vision for navigation,
vision-related quality of life, and other outcomes, the
researchers found. 
    “To hear from several participants how thrilled they were
that they could finally see the food on their plates, that is a
big deal,” study leader Dr. Eric Pierce of Mass Eye and Ear and
Harvard Medical School said in a statement.
    “These were individuals who could not read any lines on an
eye chart and who had no treatment options, which is the
unfortunate reality for most people with inherited retinal
disorders."
        
  
        Children may be able to wait at home for new hearts
  
    Small children who now spend months in the hospital waiting
for heart transplants may someday be able to wait at home
instead with the help of an implanted pumping device,
preliminary trial data suggest.
    In seven small children who were waiting for new hearts,
researchers tested the Jarvik 2015 ventricular assist device,
which is slightly larger than an AA battery and can be implanted
in children weighing as little as 18 pounds (8.16 kilograms). 
    With the pump implanted, children can take part in many
normal activities while they wait for a heart transplant, the
researchers said.
    Children in the study ranged in age from 8 months to 7
years. They used the implanted pump for an average of 149 days.
    Six of the seven eventually underwent heart transplants,
although one child had to be switched to a larger external
pumping device. One child’s heart recovered, rendering a
transplant unnecessary, researchers reported on Wednesday in the
Journal of Heart and Lung Transplantation. 
    For decades, implantable ventricular assist devices have
allowed adults on heart transplant waiting lists to live at
home, go to work or school, take walks and ride bicycles, the
researchers said. 
    But the only devices available to support small children
with failing hearts weigh up to 200 pounds (90.7 kg) and are
attached to the child with tubes almost as large as garden
hoses. Children using these older devices typically remain
hospitalized for months.
    “Ventricular assist devices for adults have been improving
every decade, but in pediatrics we're using technology from the
1960s,” study leader Dr. Christopher Almond of Stanford Medicine
in Palo Alto, California said in a statement.
    The U.S. National Institutes of Health has awarded funding
for a trial involving 22 children in the United States and
Europe.
    Because far fewer children than adults need heart
transplants, manufacturers have little incentive to develop a
miniaturized pump for them, the researchers said.
    “Whether this device will survive to commercialization
remains unclear because of the financial challenges,” they said.
    
    Drug therapy may help vapers quit
    An experimental drug that has been shown to help people quit
smoking traditional cigarettes can also help them quit vaping
nicotine, a small randomized trial suggests.
    In the trial, 160 daily nicotine vapers who had already
attempted to quit at least once and were not currently smoking
cigarettes were randomly assigned to receive either oral
cytisinicline from Achieve Life Sciences  ACHV.O  or a placebo
three times daily for 12 weeks. 
    Everyone also received weekly behavioral support. 
    During weeks 9 to 12 of treatment, 32% of participants who
received cytisinicline were successfully abstaining from vaping,
compared to about 15% of those who received the placebo.
    The U.S. Food and Drug Administration has not yet approved
cytisinicline, or any medication, for helping people to stop
vaping nicotine, the researchers said on Monday in JAMA Internal
Medicine. 
    An accompanying editorial points out that in the only other
randomized trial of a drug to help people quit vaping,
varenicline, originally sold by Pfizer  PFE.N  under the brand
name Chantix, was superior to placebo. 
    Cytisinicline, however, has fewer side effects than
varenicline, the editorial notes. 
    The drug has been used for decades as a smoking cessation
aid by millions in Central and Eastern Europe with a good safety
record, the research team noted. 
    The editorial called the findings “a positive step toward
developing targeted interventions to address e-cigarette use,”
but said new tools and biomarkers are needed to measure
e-cigarette use and determine dosing of treatments. 
    Nicotine replacement therapies are now dosed on the basis of
cigarettes smoked per day, the editorial notes. “Unlike
combustible cigarettes... e-cigarettes lack easy visual and
quantifiable units” to track nicotine use and concentrations, it
said.
    Achieve Life Sciences in February said it had agreed with
the FDA on requirements for applying for approval of
cytisinicline for smoking cessation and nicotine dependence and
plans to do so in the first half of the year.


 (Reporting by Nancy Lapid; Editing by Bill Berkrot)
 ((nancy.lapid@thomsonreuters.com))