me too, Nic,   do   u  think we will   make  it?  I'll be 80 in 3   weeeks

-----Original Message----- 
From: Nic Marais
Sent: Tuesday, November 10, 2015 2:32 AM
To: [log in to unmask]
Subject: Re: Phenylbutyrate Abstract

After 20 plus years I'm getting an exciting feeling of anticipation...;-)

Things are starting to happen!!!

Nic 63/21




On 10 November 2015 at 03:04, Rayilyn Brown <[log in to unmask]> wrote:

>             another abstract from Joanie McGuire
>
>             Program#/Poster#:
>            12.02
>
>
>
>
>             Presentation Title:
>            Stopping progression of Parkinson's disease with the drug
> phenylbutyrate
>
>
>
>
>             Location:
>            N230
>
>
>
>
>             Presentation time:
>            Saturday, Oct 17, 2015, 1:15 PM - 1:30 PM
>
>
>
>
>             Topic:
>            ++C.03.a. Human studies
>
>
>
>
>             Authors:
>            *C. R. FREED1, M. WANG1, J. CUMMISKEY1, K. B. BJUGSTAD1, B. A.
> SYMMES1, C. A. JOHNSON2, R. C. MURPHY2, M. A. LEEHEY3, W. ZHOU1;
>             1Div. of Clin. Pharmacol., 2Dept. of Pharmacol., 3Dept. of
> Neurol., Univ. Colorado Sch. of Med., Aurora, CO
>
>
>
>
>             Abstract:
>            Parkinson's disease has excellent symptomatic treatment with
> drugs such as L-DOPA, but the underlying disease process causes a
> relentless downhill course.
>
>
>
>             Several drugs have been tested in an effort to alter disease
> progression, but all have failed. We have developed a new concept for
> stopping the progression of Parkinson’s disease by turning on a
> neuroprotective gene with an FDA-approved drug. In Parkinson’s disease, 
> the
> protein alpha-synuclein forms abnormal deposits called Lewy bodies as well
> as toxic fibrils which contribute to the death of dopamine neurons. We 
> have
> discovered that the drug phenylbutyrate can prevent aggregation of
> alpha-synuclein in transgenic mice which are genetically programmed to
> develop a form of Parkinson’s disease as they age.
>
>
>
>             The drug works by upregulating the neuroprotective gene DJ-1.
> Gene activation leads to an increase in lysosome and exosome activity,
> promoting transfer of alpha-synuclein from neurons into the bloodstream
> where the protein is eliminated. In mice, phenylbutyrate increased plasma
> alpha-synuclein by about 100 per cent compared to non-treated control
> animals.
>
>
>
>             To see if phenylbutyrate has the same effect in people, we
> have given the drug for three weeks to 20 people with newly diagnosed
> Parkinson’s disease and to 20 age-matched subjects without the disease. We
> found that the drug increased the level of alpha-synclein in plasma of all
> 40 subjects from 50 to 150 per cent of baseline values, just as it had 
> done
> in transgenic mice, strongly suggesting that the drug can mobilize
> alpha-synuclein from neurons into blood plasma. While baseline plasma
> alpha-synuclein varied between subjects, the average alpha-synuclein 
> plasma
> concentrations at baseline and during phenylbutyrate administration did 
> not
> differ between patients with Parkinson's disease and normal subjects.
> Results are compatible with a neuroprotective effect of phenylbutyrate by
> accelerating clearance of alpha-synuclein from brain into plasma.
>
>
>
>             A double-blind, placebo-controlled trial in newly diagnosed
> Parkinson patients will be needed to prove whether phenylbutyrate can stop
> the progression of Parkinson’s disease in humans.
>
>
>
>
>             Disclosures:
>              C.R. Freed: C. Other Research Support (receipt of drugs,
> supplies, equipment or other in-kind support); Hyperion Pharmaceuticals. 
> M.
> Wang: None. J. Cummiskey: None. K.B. Bjugstad: None. B.A. Symmes:None. 
> C.A.
> Johnson: None. R.C. Murphy: None. M.A. Leehey: None. W. Zhou: None.
>
>
>
>
>             Keyword (s):
>            PARKINSON'S DISEASE
>
>
>
>
>
>            NEUROPROTECTION
>
>
>
>
>
>            DOPAMINE
>
>
>
>
>             Support:
>            Walter S. and Lucienne Driskill Foundation
>
>
>
>
>
>            Michael J. Fox Foundation
>
>
>
>
>
>            Mr. Charles Ackerman
>
>
>
>
>
>            Mr. Ray Sidhom
>
>
>
>
>
>            Mr. John Anderson
>
>
>
>
>
>
>
>
> Ray
> Rayilyn Brown
> Past Director AZNPF
> Arizona Chapter National Parkinson Foundation
>
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