Molecular ‘Tweezers’ Might Be The Answer to Degenerative Diseases
 

A research team at UCLA, along with about 20 collaborators around the world, believe they have found a drug that may help to prevent or cure over 30 devastating diseases including Alzheimer’s and Parkinson’s. The potentially life-saving experimental drug has already shown improvement in seven (7) different disease models with no known side effects.

This experimental ‘Molecular Tweezer” Treatment is the first of its kind and targets degenerative diseases. Pre-clinical research to date is very promising. This pre-clinical research has shown that the unique ‘molecular tweezer’ drug may benefit those with Alzheimer’s, Parkinson’s, Huntington’s, Lou Gehrig’s, type-2 diabetes, and many other diseases and conditions. The ‘molecular tweezers’ work in an entirely different way from any drug developed before. The California-based dedicated team of scientists behind the discovery is being led by Gal Bitan, Ph.D., an Associate professor at UCLA.

This promising experimental drug is poised to take the next step and move into move to clinical trials (and FDA approval); but first they need your help. The preliminary research has been funded by both foundation and government grants supporting Alzheimer’s, Parkinson’s, and ALS research. They need the public’s help to gather the significant infusion of funds necessary to initiate clinical trials. Without these trials the drug can never reach the market. That is why the new crowd funding campaign on Indiegogo is so critical for this research to be a success.

Read more: http://www.digitaljournal.com/pr/1836014#ixzz2yFUAjoqM


This is the link to the PD a-syn study abstract. The study itself was quite complex. However, the net result was that in pre-clinical research, the "tweezer" CLR01, was able to inhibit the aggregation of a-synuclein by making the protein more diffusive.

http://www.jbc.org/content/early/201...24520.abstract

Why crowd funding ?
 

Tupelo3 --

Why haven't the venture capitalists, large drug companies, MJF, PDF or NIH funded these next steps ?

Lab Rat, that is certainly the question of the day with regard to this study. Actually, when I first read the article I just passed over it after reading about the crowd funding. It seemed like just another group looking to take advantage of this new way to get some money. But when I came across a second article by the lead author, I looked into the group in more detail. They certainly seem to have the right credentials and have published a lot of legitimate research. So, I agree, seems like a strange way to go about getting funding for what appears to be some important research.

Hopeful but 10 years too late.
 

While encouraging, there appears to be many years of research and trials before a drug like this reaches the public. Also, it's not clear if these tweezers can remove established synuclein.

You may or may not be able to view the full pdf report below.

http://www.jbc.org/content/early/201....full.pdf+html

The model suggests that -synuclein behaves as
a random chain restrained by both repulsive and
attractive interactions (excluded volume repulsion,
hydrophobic attraction, and charge repulsion and
attraction). Disruption of this balance of interactions, in
this case by reversing the charge on certain residues,
substantially changes the ensemble properties and
therefore the dynamics. A more expanded chain is more
diffusive and therefore more likely to avoid bimolecular
association and subsequent oligomerization. The work
presented here shows how CLR01 binds to α-synuclein
and prevents its aggregation by perturbing key
hydrophobic and electrostatic interactions, resulting in
an increased reconfiguration rate of the protein. The
data provide important insight into the mechanism of
aggregation of α-synuclein and suggests that compounds
that increase the reconfiguration rate may lead to
efficient therapeutic drugs for Parkinson’s disease and
other synucleinopathies.

Yes, this is only pre-clinical research and remains many years away, if ever, from becoming an approved drug for human use. However, it's research that moves the science on misfolded and aggregated proteins forward. Every journey has to begin with a first step.