IBM Researchers’ New Approach To Huntington’s Could Lead To Significant Breakthrough In Therapy


Huntington’s disease is a fatal genetic disorder that causes a progressive breakdown of the brain’s neurons and afflicts about five to seven people per 100,000. Despite millions of dollars spent over the past two decades to investigate the disease, there’s been little progress in developing a drug or therapy to stop its affects on patients.

Most scientists doing research in this field view genes as being responsible for the disease in a direct causative way that goes from the gene to the protein product to a toxic influence on a cell, let’s say a neuron.

Researchers at IBM IBM -1.95% are trying a new approach that lies at the intersection of medicine and information technology, using using a piece of software called the Neural Tissue Simulator, which mimics the way neurons communicate in the brain.

With this, they are trying to investigate Huntington’s effect on the brain’s electrical signaling system at its most basic level — how individual neurons “talk” and “listen” to one another.

“In our model, we consider the Striatum, a subcortical part of the brain which degenerates massively in Huntington’s Disease, and the system of nuclei it lives in, known as the Basal Ganglia, as a router of signals in the brain,” IBM’s neuroscientist and researcher James Kozloski, tells me.

The theory is that Huntington’s mutant protein subtly changes the way that neurons receive those signals, which causes them essentially to get the message wrong and then send an errant response that gradually cascades though the rest of its network.

The “router” sends the wrong messages around, or sends them in the wrong way, and over time, this causes the brain to break down,

“What we are saying is you can disturb the dynamics of a normal brain without causing it to basically breakdown completely, but instead cause it to have risks that over time build up and cause the system to fail. An analogy would be putting the wrong oil in your car. Over many years that will affect many parts of the engine in a way that isn’t indirectly caused by the oil, but it has to do with the overall dynamics of the engine not being quite the same given the error in your maintenance,” Kozloski says.

The goal, here, is not denying the validity of traditional approaches, but adding another area for investigation, one based on understanding the brain dynamics as a whole.

“We discovered the genetic basis of the disease 20 years ago,now, and we still have no therapeutics for Huntington’s,” Kozloski tells me, “It may be that hiding in those dynamics is the missing link between gene and neurodegeneration.”

The IBM research effort won’t translate directly into a cure, but it eventually could help medical professionals to understand exactly how Huntington’s damages the brain and eventually kills a person.


Source: forbes

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