Gene Therapy May Hold Promise for Advanced Parkinson's Disease
 

A new, preliminary treatment involving triple-gene therapy appears safe and effective in helping to control motor function in Parkinson's disease patients, according to new research.

The therapy, called ProSavin, works by reprogramming brain cells to produce dopamine, the chemical essential for controlling movement, the researchers said. Lack of dopamine causes the tremors, limb stiffness and loss of balance that patients with the neurodegenerative disease suffer.
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ProSavin also helps to smooth out the peaks and valleys often produced by the drug levodopa, the current standard treatment, Mitrophanous said.
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The treatment uses a harmless virus to deliver three dopamine-making genes directly to the area of the brain that controls movement, he explained. These genes are able to convert non-dopamine-producing nerve cells into dopamine-producing cells.

The researchers don't say new this therapy is a cure, because brain cells continue to die. "But the hope is that we would give patients an additional five years before the disease progresses further," Mitrophanous said.

"If you imagine you get five to 10 years of good control of symptoms with levodopa, we hope we would lengthen that. An extra five years, maybe longer, would be a real benefit to these patients," Mitrophanous said.

All patients showed significant improvements in motor scores in the 12 hours after they stopped taking their other medications and at six months and a year after surgery, the researchers found.

http://www.philly.com/philly/health/...s_Disease.html

Building on this ...
 

See

http://www.oxfordbiomedica.co.uk/pre...in-the-lancet/

For a recent publication re. Prosavin in the lancet.

Neil

sorry didn't see this
 

Wtf
 

Estimated Enrollment: 15
Study Start Date: May 2011
Estimated Study Completion Date: May 2022
Estimated Primary Completion Date: May 2021 (Final data collection date for primary outcome measure)

http://clinicaltrials.gov/show/NCT01856439

You've got something with encouraging results, something that could improve the lives of millions of people with Parkinson's. You would think that you'd try to get it to market as soon as possible. How naive of me.

Look at OxfordBioMedica's press release dated 10th January 2014

http://www.oxfordbiomedica.co.uk/pre...in-the-lancet/

Look at the "Next objectives" section:

"Oxford BioMedica is currently evaluating a more potent formulation of ProSavin®, called OXB-102, to ensure the greatest chance of success in future randomised studies. OXB-102 will also increase the commercial opportunity by offering extended patent protection and a relative reduction in cost of goods."

John

Unfortunately, the reality is that a drug company's primary objective is profits. Patient symptom control and disease cure is only a secondary objective.

I have been ...
 

Following prosavin for some time and it has a number of problems getting to market.

I) does it work ? What with the dreaded Placebo effect, the PD subtypes and our old friend "we all have our own blend of PD", this can be tough to prove.
Ii) is it better than the current best ? for prosavin this is DBS which has impressive stats for motor control at least. It takes no account of the risk and ordeal of fitting the DBS however DBS remains a tough act to beat (step forward neurologix to prove this point).
Iii) can I afford to take it to market ? Oxfordbiomedica have been trying to partner with big pharma for prosavin for years however the risk of gene therapy has proven a significant hurdle to date as well as the risk of the placebo effect, DBS, etc.

I have no doubt oxbio want to get prosavin to market, they get wealth unimagined if they can however their problems are not unique. Until we can get products to market at faster than geological time we will continue with a 40 year old drug, ldopa, or the trial of DBS.

As Mjf said a while ago, until we get a person in charge of curing PD then we are fighting with one arm only.

Take care,
Neil.

same
 

And they wonder why they can't get people to participate in trials:(

reasons for a long term study
 

Oxfordbiomedica have been trying to partner with big pharma for prosavin for years however the risk of gene therapy has proven a significant hurdle to date as well as the risk of the placebo effect, DBS, etc.

Yes, the risk of gene therapy is a major hurdle for Prosavin. It is a lentiviral based gene therapy. Lentiviral vectors integrate into host DNA and are expressed like any host gene. I donot think Prosavin is any different than the conventional lenti viral vectors. This means, once Prosavin is injected into a patient, genes of interest and the viral vector are a part of patients DNA in cells it gets into.

This is both good and bad news. If it works, you will have all the enzymes you need to make dopamine forever and can be considered a cure.

The uncertain part is that we dont know the long term effects of a viral DNA inserted into your own DNA in neuronal cells. There is no way to pull it out if something goes wrong. If I remember correctly, several years ago, a few patients treated with mouse version of an integrating virus (muLV) created some problems, (I cannot recall all the details, sorry.)>I am sure Oxford Biomedica is aware of this, and probably one of the reasons for a long term study.

girija

http://youtu.be/bK9M2uHt5gk

Trial participant talks of her experience