New Drug Stops Parkinson Disease in Mice by mikesanye


									                                            [ Research and Findings ]

New Drug Stops Parkinson’s Disease in Mice

    n a major breakthrough in the          convert their findings into a practical
    battle against Parkinson's disease,    treatment for Parkinson's disease,
    researchers at the University          they needed to find a drug to turn
of Colorado School of Medicine             on the DJ-1 gene.
have discovered a drug that stops               "We know some drugs can turn
the progression of the degenerative        on genes. For example, steroids like
illness in mice and is now being           testosterone act on genes in muscle
tested in humans.                          cells to create muscle bulk," said
     "Drugs currently used to              Freed.
treat Parkinson's disease just treat            After testing many drugs, the
symptoms; they do not stop the             team found that phenylbutyrate
disease from getting worse," said          could activate DJ-1 and keep
senior author Curt Freed, MD,              dopamine neurons from dying.               and 60s. Patients have a decline in
who heads the division of Clinical         Next, they put the drug in the             their ability to walk, talk, and write
Pharmacology and Toxicology at the         drinking water of mice genetically         because of slow movement and rigid
CU School of Medicine. "We've now          programmed to get Parkinson's              muscles. They develop tremors and
discovered that we can prevent the         disease as they aged.                      reflexes slow down. The current
progression of the disease by turning           Aging mice receiving the drug         treatment of Parkinson's is based
on a protective gene in the brain."        were able to move normally, had            on drugs that increase dopamine
     The results have been published       no decline in mental function, and         production in the brain.
on line in the  Journal of Biological      their brains did not accumulate the             Freed is a national leader in
Chemistry.                                 protein that causes Parkinson's. By        transplanting dopamine cells into the
     Lead author Wenbo Zhou, PhD,          contrast, older animals that did not       human brain to relieve symptoms.
Assistant Professor of Medicine,           get the drug saw a steady decline in       He and his neurosurgical colleague
and Freed, a national pioneer in           their ability to move as their brains      Robert Breeze, MD, have done the
Parkinson's research, have found           were damaged by abnormal proteins.         operation in 61 patients, more than
that the drug phenylbutyrate turns              The researchers began giving          any other group in the world. The
on a gene that can protect dopamine        phenylbutyrate to people in 2009,          procedure can replace the need for
neurons in Parkinson's disease. The        to test the safety of the drug in          drugs but even cell transplants do
gene, called DJ-1, can increase            Parkinson patients.                        not prevent the progression of the
production of antioxidants like                 Zhou and Freed will publish the       disease.
glutathione to reduce the debilitating     human results in the coming months.             Freed and Zhou are now looking
effects of excess oxygen in brain cells.        "We look forward to a future          for other drugs that might turn
In addition, activating DJ-1 helps         when Parkinson patients will be able       on the DJ-1 gene. One drawback
cells eliminate abnormal proteins          to take a pill that will turn on the       of phenylbutyrate is that patients
that otherwise accumulate and              DJ-1 gene and stop the progressive         must take very large doses, 16 grams
kill brain cells. Dopamine neurons         disability associated with the illness,"   per day or 32 large tablets taken at
are particularly susceptible to too        Freed said. "Right now, when you           frequent intervals. While the drug is
much oxygen and abnormal protein           get the diagnosis of Parkinson's, you      approved by the FDA for treating
deposits. Parkinson's disease is caused    can expect to have a steady decline        a rare genetic disease in infants,
by dying midbrain dopamine neurons.        in the ability to move. While drugs        whether it can stop Parkinson's in
     Zhou and Freed have studied           like L-DOPA are very important for         people remains to be seen.
the DJ-1 gene since 2003 when a            generating dopamine in the brain                But Zhou and Freed believe the
European group discovered that             and making movement possible,              discovery offers new hope for those
mutations in DJ-1 could cause              these drugs have little impact on the      suffering from Parkinson's disease.
Parkinson's disease. The Colorado          ongoing deterioration of the patients'          "If we can say to someone that
scientists immediately started work        own brain cells."                          as of today we can stop your disease
to see why the gene was so important            Over one million people in the        from getting worse, that would be a
and have published a series of papers      United State have the disease which        truly significant achievement," Freed
on the subject since 2005. But to          usually strikes those in their 50s         said. ■                                                                            Volume 15 > Number 3 > 2011   ■ 85

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