Gene therapy eases Parkinson’s symptoms b... - Cure Parkinson's

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Gene therapy eases Parkinson’s symptoms by rewiring parts of the brain

Farooqji profile image
8 Replies

In a trial published in 2011, researchers at the Feinstein Institute for Medical Research in New York found that a gene therapy designed to turn down the activity of the subthalamic nucleus improved motor control for people with Parkinson’s.

One year after treatment, the people in the gene therapy group were found to have new brain connections that weren’t seen in the placebo group. Shutting down the disease-causing pathways between the subthalamic nucleus and the brain’s motor regions appeared to encourage alternative pathways to develop instead, says David Eidelberg at the Feinstein Institute, who led the study.

These alternative pathways are not found in healthy people. This suggests that gene therapy lets people with Parkinson’s form novel, compensatory brain circuits for controlling movement, says Eidelberg.

The team is now planning a larger trial of gene therapy for Parkinson’s disease that is due to start at the end of 2019.

newscientist.com/article/21...

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Farooqji
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jimcaster profile image
jimcaster

This sounds encouraging, but the eight year gap between the initial trial and the next phase is discouraging.

Erniediaz1018 profile image
Erniediaz1018 in reply to jimcaster

Exactly my thought. The gap was immediately disappointing.

Farooqji profile image
Farooqji in reply to Erniediaz1018

see below in reply to jimcaster

Farooqji profile image
Farooqji in reply to jimcaster

I put the same question to Simon of scienceofParkinson.Com and his response was as under

"1.) Good question: This series of gene therapy clinical trials was actually started by Neurologix in 2003. Positive Phase I results (safety in 12 participants) were announced in late Sept 2005 ((link: eurekalert.org/pub_releases... eurekalert.org/pub_releases/2…), published in June 2007 ((link: ncbi.nlm.nih.gov/pubmed/175... ncbi.nlm.nih.gov/pubmed/1758630

2.) 'Modest clinical effect' Phase II results (45 subjects; 50:50 - treated:sham) announced in June 2010, published 2011 ((link: ncbi.nlm.nih.gov/pubmed/214... ncbi.nlm.nih.gov/pubmed/2141970…). It is not clear why this new result has taken so long to come out. Will do some exploring

Farooqji profile image
Farooqji

newsday.com/news/health/gen...

LAJ12345 profile image
LAJ12345 in reply to Farooqji

Interesting the placebo group also grew new circuits!

jeffreyn profile image
jeffreyn

SoPD blog post on this topic:

scienceofparkinsons.com/201...

Fighttolivenow profile image
Fighttolivenow

Wonder how the patients receiving the real therapy and thus have a new network developed in their brain are doing today....

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