Drug Research

Discussion in 'Fibromyalgia Main Forum' started by Slayadragon, Jan 27, 2007.

  1. Slayadragon

    Slayadragon New Member

    A question to ponder.

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    Why Nonprofits Fund For-Profit Companies Doing Drug
    Research
    Wall Street Journal
    January 26, 2007
    SCIENCE JOURNAL By SHARON BEGLEY

    Science has made paralyzed rats walk, cured mice of
    cancer and eliminated Alzheimer’s in more lab rodents
    than you can count. Human patients? Not so much.

    “There’s frustration that developments from academic
    labs don’t get picked up by [drug and biotech]
    companies,” says Dayton Coles. As a board member of
    the Juvenile Diabetes Research Foundation, he has seen
    promising discovery after promising discovery emerge
    from the university labs that JDRF has funded, but
    none has turned into a cure for type-1 diabetes, which
    his daughter has.

    Fed up with breakthroughs that fill journals rather
    than medicine chests, private foundations and
    charities that have traditionally funded academic
    scientists have started doing the once-unthinkable:
    writing checks for millions of dollars to for-profit
    companies.

    It’s a sign of desperation. One reason there have been
    so few drug breakthroughs lately is that the profit
    motive actually works against the development of new
    pharmaceuticals. Drug companies suffer from
    blockbuster-itis, the belief that only billion-dollar
    almost-sure things need apply for development. As a
    result, even the most brilliant discovery may not be
    translated into a drug unless it has 10-figure sales
    potential. Also, short time horizons on the part of
    venture capitalists, who generally want to see their
    biotech bets pay off in three years, don’t mesh well
    with the lengthy drug-development process.

    Enter the charities. Earlier this month, JDRF
    announced that it was giving $2 million to MacroGenics
    Inc., a Rockville, Md., biotech, for a phase-2/3
    clinical trial of an antibody that might slow
    progression of type-1 diabetes. The antibody basically
    puts an immune-system cell called CD3 in a headlock,
    preventing it from orchestrating an immune attack on
    cells that produce insulin. Destruction of those cells
    causes type-1 diabetes.

    MacroGenics acquired the rights to the antibody in
    2005, after a major pharmaceutical company got it
    through safety tests with flying colors but then
    dropped it for financial reasons, says Scott Koenig,
    MacroGenics president and CEO. “Only” 1.7 million
    people in the U.S. have type-1 diabetes, so anti-CD3
    therapy will never ring up Lipitor- like profits.
    Blockbuster-itis had struck again.

    The macrogenics deal follows three others JDRF
    unveiled in 2006. In October, it announced that it
    would pay up to $3 million to Sangamo BioSciences
    Inc., Richmond, Calif., for a phase-2 trial of a
    protein drug that shows promise against diabetic
    neuropathy, in which nerve damage due to diabetes
    causes numbness, pain and, eventually, loss of motor
    function. It is also funding a phase-2 trial by
    Transition Therapeutics Inc., Toronto, of a drug that
    might make insulin- producing cells regenerate, and a
    phase-3 trial by TolerRx Inc., Cambridge, Mass., of an
    antibody that, like MacroGenics’, might protect
    insulin-making cells.

    Transition Therapeutics’s big-pharma partner for its
    clinical trial bowed out in 2006, so the trial “would
    have come to a halt,” says JDRF’s Richard Insel, vice
    president for research. “We wanted to see this go
    forward, which meant moving beyond our traditional
    support for academic research.”

    In every case, the companies are also sinking their
    own (or investors’) money into the trials. But they
    say the JDRF check makes a difference. “We were locked
    and loaded for our phase-2 trial, but the JDRF funding
    will let us look more closely” at how the drug works,
    says Sangamo CEO Edward Lamphier.

    Parents of kids with diabetes aren’t the only ones fed
    up with the slow pace of translational research. This
    week the Michael J. Fox Foundation announced that it
    had awarded Sangamo $950,000 to apply its
    gene-regulation research to slowing Parkinson’s
    disease. In March, Families of Spinal Muscular
    Atrophy, founded by parents of children with this rare
    disease, ponied up $402,500 to help Paratek
    Pharmaceuticals, Inc., Boston, develop a drug for the
    disease.

    “With 10,000 SMA patients in the U.S., the market is
    too small for companies to see this [disease] as a
    worthwhile bet unless we help them take a compound
    past the initial stages,” says Kenneth Hobby,
    executive director of the charity. “If that means
    funding a company, we have no problem with that.”

    At the Myelin Repair Foundation, “we decided we have
    to break the mold to bridge the translational research
    gap, which is getting bigger and bigger,” says the
    foundation’s chief operating officer, Russell Bromley.
    That means paying companies to conduct validation
    research, in which scientists test how a compound
    works in the body. Given how few bright ideas turn
    into FDA-approved drugs — fewer than one in 1,000,
    according to the pharmaceutical industry — companies
    want compounds “de-risked” for them. Translation: get
    someone else to do, or pay for, this step.

    Charities realize that writing checks to for-profits
    might not be what their donors had in mind. “We
    debated whether it was right for our money to go to a
    company that might make a profit,” says JDRF board
    member Michael White. “We’re not unconcerned about
    that. But we’ve invested so much in discovery, what we
    need now is to take these things to market. We’re
    taking on the role of ‘venture philanthropists.’ “
  2. Lichu3

    Lichu3 New Member

    Pharmaceutical companies are double-edged swords...they exist for a purpose but are mostly profit-generating machines. Look at executive compensation and the list of most profitable companies in the U.S.

    The biggest funder of research in the U.S. is NIH but pharma often comes in to steal ideas from the universities without much profit going back to the government/ universities.

    These companies have some of the largest lobbying groups in the capital and often rationalize that the reason drugs are so expensive in the U.S. is because of the costs of research and development. Nevermind the above point about funding, the fact that a much larger amount of money is spent on advertising than research, and much of the drugs out are "me,too" drugs and not new ways to really address problems.

    It would be great if the goverment had some way of getting these companies to develop drugs urgently needed (like for this disease) by witholding things like tax breaks for these companies. Lobbying groups make it difficult.

    Non-profit groups' hearts are in the right place but I don't know if they have the money to push forth on these matters and really, this is something which needs to be addressed by the goverment.