Breakthrough- Treat the Disease, Not Just the Symptoms!

No Gravatar
Schemat parkinson
Image via Wikipedia

Under the direction of Drs. W. Zhou and C. Freed (University of Colorado, School of Medicine), a new treatment for Parkinson’s disease is being tested.  After proving its worth in mice, by activating a protective gene in mice, phenylbutyrate (which protects dopamine neurons) is being tested in humans.

These researchers have been studying the DJ-1 gene since 2003, when Dr. Bonifati et. al. (Erasmus Medical Center, Rotterdam, Netherlands) found it to be in the critical path of decreasing the accumulation of certain proteins and antioxidant production (oxidative stress response) and was associated with Parkinson’s disease. (There are six genes are linked to Parkinson’s disease- Parkin, UCHL1, DJ-1, PINK1, LRRK2, and %-synuclein; DJ-1 is important to the oxidative stress response and the elimination of the abnormal protein aggregates. ) The lowered oxygen and protein levels preserve the life of the dopamine producing neurons.  (Dopamine is a critical need for Parkinson’s patients; it’s why L-Dopa has been used to treat the symptoms of the disease for more than 40 years.  Its administration, however, does not prevent the continued deterioration of brain cells.  Dr. Freed’s group has been transplanting dopamine cells into the brain- they provide dopamine, but don’t stop the brain deterioration that affords the progression of the disease )  The Colorado research group knew that overexpression of the DJ-1 gene protected against the oxidative stress, by regulating the rate-limiting step in glutathione synthesis.  It has been searching to find a drug to turn on the DJ-1 gene.  Phenylbutyrate was chosen as the candidate, since it inhibits histone deacetylase (and is so currently employed for children with a rare neurodegenerative disease).  In 2009, the researchers tested the safety of the drug (“no harm” testing).

The required dose of phenylbutyrate is huge- 16 g per day (32 tablets throughout the day, each at 500 mg).  As such, even though testing with this drug is ongoing, other drugs are being investigated to see if they, too,  can activate the DJ-1 gene more effectively.

Enhanced by Zemanta
Share this:
Share this page via Email Share this page via Stumble Upon Share this page via Digg this Share this page via Facebook Share this page via Twitter
Share

7 thoughts on “Breakthrough- Treat the Disease, Not Just the Symptoms!”

  1. Hi Roy! Thanks for this information. As someone with Multiple Sclerosis, I know that some of these same therapies/ research could work for MS patients. Unfortunately, it doesn’t seem like the two fields talk to each other and share information. It’s so frustrating!
    Kelly Green recently posted..A Special Gift Just For You!

    1. Hello, Kelley:
      The fields of medicine rarely talk to one another. They rely on their drug reps to pass the information around :-).
      I am not sure that this one would work for MS, since it is specific to the dopamine cells. But, there are others that could certainly be “cross-fertilized”.
      Thanks for your comment.

    1. Janette:
      The drug will come down in price (it is a simple chemical), since it is only used for a very small audience now. My guess is the price will be on the order of $ 600- 1000 per year or LOWER. (LDopa is cheap, too.) Insurance will pick it up, as well.

  2. Thanks for sharing this research with us Roy. In my years of nursing I have seen many people and their spouses hugely affected by the impact of parkinsons on their life.
    A whopping 32 tablets per day. Thats some hard pills to swallow for many people. Now that they have identified the DJ-1 gene, hopefully they can find a more palatable drug/dosage formulation to activate it.
    Maureen Hunter recently posted..Men and Grief

  3. Pingback: go learn web.

Comments are closed.