Parkinson's Disease Tulip


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Old 05-23-2011, 06:08 PM #1
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Default Single brain injection 'can ease symptoms' of Parkinson's

http://www.dailymail.co.uk/health/ar...-symptoms.html
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Born in 1953, 1st symptoms and misdiagnosed as essential tremor in 1992. Dx with PD in 2000.
Currently (2011) taking 200/50 Sinemet CR 8 times a day + 10/100 Sinemet 3 times a day. Functional 90% of waking day but fragile. Failure at exercise but still trying. Constantly experimenting. Beta blocker and ACE inhibitor at present. Currently (01/2013) taking ldopa/carbadopa 200/50 CR six times a day + 10/100 form 3 times daily. Functional 90% of day. Update 04/2013: L/C 200/50 8x; Beta Blocker; ACE Inhib; Ginger; Turmeric; Creatine; Magnesium; Potassium. Doing well.
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Old 05-24-2011, 09:39 AM #2
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I hardly dared believe when I read this in my Dail Mail yesterday Rick - let's hope it comes to something in the future!

All good wishes.
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Old 05-24-2011, 09:40 AM #3
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Ooops - just read the article you posted and your response thereunder Rick - spoke too soon - sorry!
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Old 05-24-2011, 01:41 PM #4
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Quote:
Originally Posted by reverett123 View Post
More information on this, from Oxford Biomedica's website

ProSavin®

ProSavin® is a gene-based treatment for Parkinson's disease, a progressive movement disorder caused by the degeneration of dopamine producing nerve cells in the brain. In Parkinson's disease, there is degeneration of the cells in the brain that produce dopamine. ProSavin® uses the Company's LentiVector® gene delivery technology to deliver the genes for three enzymes that are required for the synthesis of dopamine. The product is administered locally to the region of the brain called the striatum, converting cells into a replacement dopamine factory within the brain, thus replacing the patient's own lost source of the neurotransmitter.
Encouraging Phase I/II results

ProSavin® is being evaluated in a Phase I/II trial in patients with mid-stage Parkinson's disease. The first stage of the study is an open-label dose escalation of ProSavin® in cohorts of three patients. The first two dose levels (1x and 2x) were safe, well-tolerated and showed promising evidence of efficacy with a maximum improvement in motor function of up to 56% at the 2x dose. If these results are confirmed in placebo-controlled studies, ProSavin® would represent a significant advancement to current treatment options.
Enhanced administration

Results from the third cohort of patients treated using a new infusion technique reduced the surgical delivery time by 50% at the 2x dose. The enhanced technique requires fewer needle tracks into the brain and is capable of delivering higher doses of ProSavin® compared to the previous administration procedure. Following approval from the Data Monitoring Committee in December 2010, Oxford BioMedica plans to proceed to a 5x dose level in early 2011, with the aim of advancing ProSavin® into a randomised Phase II study in 2012.

Market opportunity

Parkinson’s disease currently affects 4.1 million patients globally which is projected to rise to 8.7 million by 2030. A patient with Parkinson’s disease progressively loses the ability to make the neurotransmitter dopamine, the mediator of the control of movement. There is no cure for Parkinson’s disease, and no treatment can prevent the disease from progressing, however current treatments can usually ease symptoms. The current treatment market for Parkinson’s disease is approximately US$3.5 billion (source: Datamonitor). ProSavin® has the potential to address an unmet medical need in Parkinson’s disease, offering long-lasting benefit from a single administration with an excellent safety profile. The product could also reduce the social care burden that is associated with mid to late-stage disease.

Oxford BioMedica plc
Medawar Centre, Robert Robinson Avenue,
The Oxford Science Park, Oxford,
OX4 4GA United Kingdom



Telephone: +44 (0) 1865 783 000
Fax: +44 (0) 1865 783 001
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Old 05-25-2011, 06:19 AM #5
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Quote:
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I see a problem with this approach. It seems to me that SN cells are particularly sensitive to dying because of their dopamine production process and the dopamine rich environment within them. Wouldn't it be possible that these re-converted cells start to die after some time? Maybe I haven't understood well the process, but it seems risky, at least in the long term.
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Old 05-25-2011, 07:24 AM #6
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Quote:
Originally Posted by JoeM View Post
I see a problem with this approach. It seems to me that SN cells are particularly sensitive to dying because of their dopamine production process and the dopamine rich environment within them. Wouldn't it be possible that these re-converted cells start to die after some time? Maybe I haven't understood well the process, but it seems risky, at least in the long term.
for us, the main concern with this approach is that it takes existing cells and converts them to dopamine-producing cells....do we know that our body can handle the loss of the cellular function(s) those cells used to have before they were "instructed" to make dopamine? Also, what effects do changing a cell's function have on that cell and surrounding cells? We've never seen these questions addressed and until they are, unfortunately this therapy is not on our list....at least yet. I hope for everyone that this shows to be a long-term safe and effective choice, one of many PWP will have to choose from.
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Old 05-26-2011, 05:55 AM #7
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Even if this procedure would work, wouldn't the cells continue to die off because the original problem causing cells to die off has not been corrected?
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Old 05-26-2011, 10:15 AM #8
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Default Good points Greg and Lurking ....

Nathan Klein, an early PD gene therapy trialist, had a good recovery then his "new" cells started to die and he was back to square one.

However he got back a good few years and remember DBS doesn't halt the progression of PD and Prosavin is competing with DBS.

In terms of losing the functions of the converted brain cells, well I am no expert but :

It is estimated, the human brain consists of 100 billion brain cells (neurons) and around 1 trillion supporting cells (glia), which helps neurons. The brain weighs about 1.4 kg (3 pounds). But after the age of 20, naturally people will lose about 1 gram of brain mass per year.

If the human brain has a weight of 1.4 kg (1,400 grams) and there are about 100 billion neurons, then it can be calculated that there are about 70 million neurons per gram.

If people lose 1 gram of brain mass per year, this means that the brain loses 70 million neurons per year with about 190 thousand per day slowing down or dyeing.

Even if it is said that about 5 percent from 1 gram of neurons really die, then the human brain loses about 9,000 neurons per day. These numbers may seem high, but when compared with the remaining neurons, the number is small.

So there is still a lot of capacity to go round even allowing for the "loss" of the converted cells.

Take care,
Neil.
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