ALS Research May Benefit Alzheimer’s Patients

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The success of the ALS Ice Bucket Challenge has been the envy of many charities. Now it appears that ALS research, having been revitalized by the flow of $220 million from the Challenge, may lead to better understanding of other neurological diseases as well, including Alzheimer’s.

ALS, also known as Lou Gehrig’s disease, attacks neurons (nerve cells) in the brain and spinal cord that control muscles. As these neurons degenerate, ALS patients’ muscles atrophy, robbing them of the ability to walk, use their hands, speak and breathe. However, their intellect remains fully intact.

For the last decade, ALS researchers have known that a specific protein, TPD-43, seems to be implicated in ALS, although they were not sure of its function. This protein builds up in clumps outside the nuclei of neurons and seems to lead to cell death. Researchers describe these proteins as becoming “sticky”. Until now, researchers did not know if these sticky clumps were the cause or the result of the disease.

Last week, the journal Science published the research results of a team at Johns Hopkins. They determined that TPD -43 is essential to decode DNA, and when it fails to function properly, essential decoding ceases.

The researchers developed a synthetic protein designed to mimic normal TPD-43 and injected it into neurons that had stopped functioning due to “sticky” TPD-43. What they found was that the cells that had stopped functioning came back to life and started functioning normally again.

There is huge early promise in this result, although it is premature to call it a break through for ALS patients. The next step for the researchers is to work with live mice, not just mouse neurons, and see if mice who are engineered to manifest ALS can regain normal muscle function when given synthetic versions of TPD-43. If those trials go well, clinical trials with people will follow.

What does this mean for Alzheimer’s patients? In the short run, probably nothing. However, since Alzheimer’s is also a neurological disorder marked by abnormal clumping of protein outside of brain cells, what scientists learn about clumping protein, how it affects brain cell function, and how its impact might be reversed could be a huge leap forward for Alzheimer researchers.

Jonathan Ling, the graduate student who was one of the primary researchers on the Hopkins ALS study, said that the availability of large amounts of funding due to the Ice Bucket Challenge gave researchers the ability to try for “high risk, high reward” studies. It will be a wonderful dividend if this research advances our understanding of Alzheimer’s as well.

See below as Jonathan and his graduate professor Philip Wong take the Ice bucket Challenge themselves:

Video Credit: Johns Hopkins Hospital

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