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Possible breakthrough treatment for degenerative diseases

Date: 04/25/2014
Contact: Mark Wheeler ()
Phone: (310) 794-2265

UCLA neuroscientist turns to crowd funding for next stage of research

Crowdfunding is the idea that people will donate money, small amounts or large, to a project or a cause they want to support. It could be for an artist who wants to make a film, an entrepreneur who wants to market a new gizmo-or a scientist who may have a cure for a number of diseases that afflict human beings.

For nine years, UCLA neuroscientist Gal Bitan, and his colleagues have been working on an experimental drug that may prevent or cure over 30 currently cureless and devastating diseases. These include Alzheimer's, Parkinson's, Huntington's, Lou Gehrig's, type-2 diabetes, and many others that have a common underlying mechanism. They developed this drug first in cell cultures, then in animal models of these various diseases. They found that the drug worked without showing side effects.

This basic research has been funded by foundation and government grants supporting Alzheimer's, Parkinson's, and ALS research. But now Bitan needs a considerable amount of money for the critical next step, to begin human clinical trials. The amount needed exceeds by far the typical government or foundation grant. And possible private funding most likely from pharmaceutical companies who have shown interest, want the research to be a little farther along, i.e., already at the clinical trials phase, before they get involved. This, of course, is a catch-22 without adequate funding.

Given the downturn in the economy and its slow recovery, the emphasis on reducing the nation's debt, and the sheer numbers of researchers seeking money for their research, it is hard to find the funding they need to move their work forward.

In addition, said Bitan, "The challenge is that the drug we have developed works in a way that is entirely different from any drug developed before. And innovative ideas are often are met with resistance. But given the current results, we asked ourselves - with seven successful animal model tests and no side effects, can we afford to wait? Can the patients and families afford to wait?"

The drug in question is a molecular compound called CLR01 and nicknamed a "molecular tweezer" for the way it looks and functions. With Alzheimer's disease, for example, it's thought that an amino acid called lysine, part of the beta-amyloid protein believed to cause Alzheimer's, was the key to its transition from a regular component of every cell's normal biological function, to a toxin that kills brain cells and causes dementia. "The mechanism that causes this is the clumping together of these beta-amyloid proteins that cause the toxicity. It is therefore critical," Bitan said, "to find a way to stop this aggregation process."

Over the last two decades, researchers and pharmaceutical companies have attempted to develop drugs that would prevent this abnormal protein aggregation, but so far, with little success in translating laboratory findings to clinical results. "The problem is that there is very little understanding of how or even why these previous compounds worked." Bitan said. The molecular tweezers are the first case of using an out-of-the-box, rational approach to the problem, with a clear understanding of how it works. Shaped like the letter "C," the tweezers wrap around these chains of lysine using the same molecular forces that lead to the clumping and therefore they prevent the clumping itself. As they continued their research, Bitan and his colleagues realized that due to their unique mechanism of action, they also had the same effect on many other proteins, each of which causes a different disease. 

"We've shown that our drug safely crosses the blood-brain barrier, prevents and removes those aggregated proteins, and further, protects the existing healthy neurons and synapses in the brain," said Bitan.

With Alzheimer's, for example, more than 10,000 baby boomers are turning 65 each day according to the Alzheimer's Association. After 65, the risk of being struck by the disease doubles every five years, so almost half of the population suffers from this disease by age of 85. Based on these statistics, there is no time like the present to pursue FDA testing and clinical trials.

"It's crucial that we find a cure for these diseases," Bitan said. "If everyone who is dealing with the devastation or knows someone affected by these diseases contributes one dollar, we will have what we need to move forward as quickly as possible towards clinical trials!."

More information, and how to donate, can be found at
https://www.indiegogo.com/projects/breakthrough-treatment-for-degenerative-diseases

Bitan is also on Twitter and Facebook