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<DIV>I got this from another list and thought you’d be interested.</DIV></DIV>
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<P class=MsoNormal><B>Doctors perform gene therapy treatment on boy with
inherited blindness<o:p></o:p></B></P>
<P class=MsoNormal>Article Link: <A
href="https://mandrillapp.com/track/click/30489975/mashable.com?p=eyJzIjoiYzM1SmNJc1hVUThFZDBDYnZpY1BiRUZVTVVBIiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL21hc2hhYmxlLmNvbVxcXC8yMDE4XFxcLzAzXFxcLzI0XFxcL2dlbmUtdGhlcmFweS1ibGluZG5lc3MtdHJlYXRtZW50XFxcLyNjMVhPZE5aMkFtcWZcIixcImlkXCI6XCI1YTc2OTkyZGI1ZmU0NDk3YmFmYWYzNjU4ZTg1M2JmNVwiLFwidXJsX2lkc1wiOltcImM5OWQxZGY4MTIwZjVkNDI0YWIzNmE3ZWNlZWQyMjcwOWJkODE2YTBcIl19In0">https://mashable.com/2018/03/24/gene-therapy-blindness-treatment/#c1XOdNZ2Amqf</A>
<o:p></o:p></P>
<P class=MsoNormal>On the morning of March 20, eye surgeon Jason Comander
injected viruses carrying lab-grown genes into the eyes of a boy whose vision
had been gradually disappearing.<o:p></o:p></P>
<P class=MsoNormal>If all goes as planned, the 13-year-old patient — who lives
with an inherited genetic defect that causes blindness — will experience an
improvement in eyesight in about a month.<o:p></o:p></P>
<P class=MsoNormal>After a series of tests, the U.S. Food and Drug
Administration (FDA) <A
href="https://mandrillapp.com/track/click/30489975/www.fda.gov?p=eyJzIjoiZ0dWVzBtQUpQOXpXYjB6eEZHOU1xNHdLVVc4IiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL3d3dy5mZGEuZ292XFxcL05ld3NFdmVudHNcXFwvTmV3c3Jvb21cXFwvUHJlc3NBbm5vdW5jZW1lbnRzXFxcL3VjbTU4OTQ2Ny5odG1cIixcImlkXCI6XCI1YTc2OTkyZGI1ZmU0NDk3YmFmYWYzNjU4ZTg1M2JmNVwiLFwidXJsX2lkc1wiOltcImNlYWE5ZmFhYjVkMDhkYWYyZGVkYWViYjUxY2YyNzQ4YjlmZTdmOTZcIl19In0"
target=_blank>approved this gene-therapy treatment</A>, called Luxturna, in
December 2017. <o:p></o:p></P>
<P class=MsoNormal>Three months later, the procedure Comander performed at
Massachusetts Eye and Ear served as the first time an FDA-approved gene therapy
was used on a person living with an inherited, and incurable, genetic disease.
There are no other effective treatments for this specific retinal disease.
<o:p></o:p></P>
<P class=MsoNormal>"It's a huge step in the right direction," said Comander,
associate director of the Inherited Retinal Disorders Service at the
Boston-based hospital, in an interview.<o:p></o:p></P>
<P class=MsoNormal>"Most of the patients are legally blind by their 20s or 30s,"
said Comander. "Suffice to say, these are really terrible
diseases."<o:p></o:p></P>
<P class=MsoNormal>Scientists have been working on this novel type of treatment
for decades, including as part of FDA trial programs. Comander notes the
treatment affects different patients in different ways. Sometimes the vision
improvement is dramatic. One previous patient, from an earlier FDA trial, no
longer needed to attend a specialized school for children with blindness,
Comander said. <o:p></o:p></P>
<P class=MsoNormal>"This has been a dream of the field for 30 years, and there
have been sobering setbacks," said Tim Cherry, who researches inherited
blindness and other visual disorders at Seattle Children’s Research Institute,
in an interview. Cherry disclosed he has collaborated with Comander on research
before, but played no part in the development of this treatment.<o:p></o:p></P>
<P class=MsoNormal>"It can improve people's lives in a way that might have been
considered science fiction years ago," said Cherry. It should be noted, however,
that while blind people face challenges due to inaccessibility, many live happy,
fulfilled lives.<o:p></o:p></P>
<P class=MsoNormal>The rare, mutated gene in afflicted patients, known as RPE65,
prevents the retina, a light-sensitive tissue behind the eye, from working
correctly. Specifically, the gene prevents retinal cells from properly producing
proteins, resulting in deteriorating eyesight. Many patients become legally
blind, and some lose all their eyesight.<o:p></o:p></P>
<P class=MsoNormal>This novel gene therapy, called gene-replacement therapy, is
designed to replace the mutated genes with genes that work, and accordingly,
improve a patient's eyesight. Synthetic genes are first grown in a lab and then
inserted into viruses commonly found in the human eye. The viruses — whose
mission is to invade eye cells and infuse genetic material into them — are then
carefully placed in the patient's retina. <o:p></o:p></P>
<P class=MsoNormal>These viruses, said Cherry, naturally live in our eyes and
are harmless. <o:p></o:p></P>
<P class=MsoNormal>Because there were no other meaningful treatments available
for this incurable disease, the FDA approved the treatments more rapidly than it
typically does, under programs called Breakthrough Therapy and Priority Review.
<o:p></o:p></P>
<P class=MsoNormal>These sorts of accelerated approvals require drug companies
and clinicians to continue monitoring the treatments after their initial FDA
approval, to ensure they're truly safe and effective enough for consumers to
use. The <A
href="https://mandrillapp.com/track/click/30489975/luxturna.com?p=eyJzIjoiWXZFZnNaVVktcjNSNDZ3TEVLT1YwUU1uVUFjIiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL2x1eHR1cm5hLmNvbVxcXC8_Z2NsaWQ9RUFJYUlRb2JDaE1JZ1lldDJPWDcyUUlWRUlacENoM0ZVZ3p5RUFBWUFTQUFFZ0liOVBEX0J3RVwiLFwiaWRcIjpcIjVhNzY5OTJkYjVmZTQ0OTdiYWZhZjM2NThlODUzYmY1XCIsXCJ1cmxfaWRzXCI6W1wiMDIzODY4YjU3NDIzMzY3NTk4YzE4YjFmNTk3YmQ0YmJlZjFjMmFmMVwiXX0ifQ"
target=_blank>safety warnings on the Luxturna website</A> underscore these
potential hazards, including potential eye infections and further declines in
vision. <o:p></o:p></P>
<P class=MsoNormal>Earlier in 2017, the FDA had approved two other gene-therapy
treatments for cancers, which aren't considered genetically-inherited diseases.
The FDA now plans for gene therapies to play a more prominent role in combating
previously untreatable diseases.<o:p></o:p></P>
<P class=MsoNormal>"I believe gene therapy will become a mainstay in treating,
and maybe curing, many of our most devastating and intractable illnesses,” FDA
Commissioner Scott Gottlieb <A
href="https://mandrillapp.com/track/click/30489975/www.fda.gov?p=eyJzIjoiZ0dWVzBtQUpQOXpXYjB6eEZHOU1xNHdLVVc4IiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL3d3dy5mZGEuZ292XFxcL05ld3NFdmVudHNcXFwvTmV3c3Jvb21cXFwvUHJlc3NBbm5vdW5jZW1lbnRzXFxcL3VjbTU4OTQ2Ny5odG1cIixcImlkXCI6XCI1YTc2OTkyZGI1ZmU0NDk3YmFmYWYzNjU4ZTg1M2JmNVwiLFwidXJsX2lkc1wiOltcImNlYWE5ZmFhYjVkMDhkYWYyZGVkYWViYjUxY2YyNzQ4YjlmZTdmOTZcIl19In0"
target=_blank>said</A> when announcing the approval of Luxturna. <o:p></o:p></P>
<P class=MsoNormal>Although gene therapies provide treatments for historically
unmanageable diseases, they can be markedly more expensive than conventional
treatments, especially if the disease — such as this retinal mutation — is
rare.<o:p></o:p></P>
<P class=MsoNormal>The price right now is prohibitively high for most Americans:
It costs <A
href="https://mandrillapp.com/track/click/30489975/www.cnn.com?p=eyJzIjoibV9oOEFVWWc5dm9LTHRudDQ2YjhVOFQ4dEFNIiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL3d3dy5jbm4uY29tXFxcLzIwMThcXFwvMDFcXFwvMDNcXFwvaGVhbHRoXFxcL2x1eHR1cm5hLXByaWNlLWJsaW5kbmVzcy1kcnVnLWJuXFxcL2luZGV4Lmh0bWxcIixcImlkXCI6XCI1YTc2OTkyZGI1ZmU0NDk3YmFmYWYzNjU4ZTg1M2JmNVwiLFwidXJsX2lkc1wiOltcImRhNTA0MTY3NWFlZDM3NjQ4NTNhMzhmYjRmYWMzZmUxMDdiZGM0MzFcIl19In0"
target=_blank>$425,000, <I>per eye</I></A>. The pharmaceutical company that
produces the treatment, Spark Therapeutics, notes on its <A
href="https://mandrillapp.com/track/click/30489975/mysparkgeneration.com?p=eyJzIjoicnhPTjdoVGZ3dF93Y3F0enNNdEo4c3JtTUwwIiwidiI6MSwicCI6IntcInVcIjozMDQ4OTk3NSxcInZcIjoxLFwidXJsXCI6XCJodHRwczpcXFwvXFxcL215c3BhcmtnZW5lcmF0aW9uLmNvbVxcXC9wYXRpZW50LXN1cHBvcnQuaHRtbFwiLFwiaWRcIjpcIjVhNzY5OTJkYjVmZTQ0OTdiYWZhZjM2NThlODUzYmY1XCIsXCJ1cmxfaWRzXCI6W1wiNDczYzI0MjM1M2M3MjhjNTYzNTcxOGY4MzYxYTAzZTMwZmE4NmE3MlwiXX0ifQ"
target=_blank>website</A> that it assists by directly contacting insurers and
offers payment plans. <o:p></o:p></P>
<P class=MsoNormal>But Cherry hopes this gene therapy can be expanded to other
similar diseases, driving down the cost of artificially producing the genes.
<o:p></o:p></P>
<P class=MsoNormal>"We’re all in it to try and improve people's lives. So making
the treatment more accessible is on everyone’s mind," said
Cherry.<o:p></o:p></P>
<P class=MsoNormal><o:p></o:p> </P>
<P class=MsoNormal> <o:p></o:p></P>
<P class=MsoNormal> <o:p></o:p></P>
<P class=MsoNormal><o:p></o:p> </P>
<P class=MsoNormal><o:p></o:p> </P>
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