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Thread: Gene therapy research & treatments

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    waltky's Avatar Senior Member
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    Gene therapy research & treatments

    Drug to fix DNA gets go-ahead...

    Drug to fix faulty DNA gets go-ahead in landmark move that may alter medicine forever
    2 November 2012 | Gene therapy drug given green light for rare disease; Glybera to cost £1m ($1.6m) for each patient; Drug is first to get approval in Western world; EU go-ahead paves way for many more treatments
    Regulators yesterday approved the first therapy in the western world that can correct errors in a person’s genetic code. Europe has approved Glybera to be used against a rare inherited disorder which disrupts fat production in the body. The treatment uses a virus to counteract LPLD, lipoprotein lipase deficiency, which can led to acute inflammation of the pancreas.

    The illness affects around one or two people per million and sufferers have damaged copies of a gene which is essential for breaking down fats. The virus infects muscle cells with a copy of a healthy gene and a one-off treatment is effective. The treatment was backed by an advisory panel to the European Commission in July and full approval was granted yesterday. The medicine should be available next year.

    Professor John Kastelein, of the University of Amsterdam, said the therapy would have a ‘dramatic impact’ on patients. Only two other gene therapies have previously been approved for sale, both in China. It will cost around £1 million ($1.6 million) per patient, a new record for pricey modern medicines.

    Joern Aldag, chief executive of Amsterdam-based uniQure, said more such treatments would follow and argued a high price was justified because gene therapy restored natural body function and was not just a short-term fix. 'This provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated,' he said in a telephone interview on Friday

    Read more: http://www.dailymail.co.uk/news/arti...#ixzz2BMLPkXIl

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    Conley (11-05-2012)

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    Calypso Jones's Avatar Banned
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    I am assuming you have a particular agenda in posting this, Walt? Is this therapy derived from embryonic stem cells. I rather doubt it.

    Perhaps you assume that conservatives and christians have a problem with adult stem cell research? They don't. It's the growing of the unborn for harvesting their stem cells which in every case have been proven to be not just unsuccessful but catastrophic. Terrifyingly so. They don't call 'em terratomas for nuthin.

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    roadmaster's Avatar Senior Member
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    Perhaps you assume that conservatives and christians have a problem with adult stem cell research? They don't.
    No we don't in fact it's better than the other.

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    Calypso Jones (11-05-2012)

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    exactly Roadmaster. Do you have any idea as to Walt's intent. Is he just delighted that 1 or 2 people out of 1million of the population of the world? US? now have mAYBE the potential to MAYBE have their genes manipulated. maybe.

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    Quote Originally Posted by Calypso Jones View Post
    I am assuming you have a particular agenda in posting this, Walt? Is this therapy derived from embryonic stem cells. I rather doubt it.

    Perhaps you assume that conservatives and christians have a problem with adult stem cell research? They don't. It's the growing of the unborn for harvesting their stem cells which in every case have been proven to be not just unsuccessful but catastrophic. Terrifyingly so. They don't call 'em terratomas for nuthin.
    Why are you making all these assumptions?

    It's a very interesting article and the potential to repair genes is huge...this is just the first example on a rare disease, if it works it could theoretically be applied to many more

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    Cool

    Calypso...

    ... no agenda...

    ... most of my posts are of an informational nature such as this one...

    ... a thread about gene therapy in a health & medicine forum...

    ... also started threads for stem cell posts and a thread for heart related posts...

    ... health & medicine forum...

    ... get it?
    Last edited by waltky; 11-05-2012 at 03:45 PM.

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    Captain Obvious (11-05-2012)

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    Quote Originally Posted by waltky View Post
    Calypso...

    ... no agenda...

    ... most of my posts are of an informational nature such as this one...

    ... a thread about gene therapy in a health & medicine forum...

    ... also started threads for stem cell posts and a thread for heart related posts...

    ... health & medicine forum...

    ... get it?
    You tell 'em, Walt - don't take no $#@!.
    my junk is ugly

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    Lightbulb

    Gene therapy for ultra-rare genetic disorder approved in Europe...

    Europe gives green light to first gene therapy for children
    April 1, 2016 - The world's first life-saving gene therapy for children, developed by Italian scientists and GlaxoSmithKline, has been recommended for approval in Europe, boosting the pioneering technology to fix faulty genes.
    The European Medicines Agency (EMA) said on Friday it had endorsed the therapy, called Strimvelis, for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no matching bone marrow donor is available. Around 15 children a year are born in Europe with the ultra-rare genetic disorder, which leaves them unable to make a type of white blood cell. They rarely survive beyond two years unless their immune function is restored with a suitable bone marrow transplant. SCID is sometimes known as “bubble baby” disease, since children born with it have immune systems so weak they must live in germ-free environments.


    A vector, which is used to transport the healthy gene into the patient's cells

    Strimvelis is expected to secure formal marketing authorization from the European Commission in a couple of months, making it the second gene therapy to be approved in Europe, after UniQure's Glybera, which treats a rare adult blood disorder. The U.S. Food and Drug Administration has yet to approve any gene therapies but a growing number of U.S. biotech companies, such as Bluebird Bio, have products in development. Other large pharmaceutical companies are also eyeing the field, including Bristol-Myers Squibb, which has a tie-up with UniQure.

    MANY SETBACKS

    Research into gene therapy goes back a quarter of a century but the field has experienced many setbacks, including the high-profile death of an American patient in 1999 and some disastrous clinical trial results in the late 1990s and early 2000s. Now, though, optimism is building, helped by the discovery of better ways to carry replacement genes into cells. Martin Andrews, head of GlaxoSmithKline's rare diseases unit, believes the technology is proving itself, although it remains at an early stage of development. "We're on page one of chapter one of a new medicine text book," he told Reuters. A host of challenges still need to be overcome, including the complexity of delivering a product like GSK's new treatment, which requires bone marrow cells to be taken from the patient, processed and injected back.

    Trickiest of all may be pricing, given the tiny market for a therapy like Strimvelis. UniQure's Glybera made history in 2014 as the first drug to carry a $1 million price tag. GSK is not putting a price on its product but a source close to the company said that, if approved, Strimvelis would cost “very significantly less than $1 million”. GSK has several other gene therapies under development with researchers at Fondazione Telethon and Ospedale San Raffaele in Italy, including treatments for metachromatic leukodystrophy and Wiskott-Aldrich syndrome that could be submitted for regulatory approval in the next couple of years. Its Strimvelis treatment for ADA-SCID is also being lined up for submission to U.S. regulators, although Andrews said this would not happen before the end of next year.

    https://www.yahoo.com/news/europe-gi...--finance.html

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