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

> 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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