Some good medical news: a big breakthrough in fighting Lou Gehrig’s Disease

A fresh delivery of hope arrives from the medical community, as the Chicago Tribune reports a major advance toward understanding how amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s Disease, erodes the ability of victims’ brains to control motor functions and respiration.  It could have implications for treating other neurological disorders, including Alzheimer’s and Parkinson’s, as well. 

The breakthrough in a paper from Drs. Teepu Siddique, Han-Xiang Deng, Wenjie Chen, and twenty other contributing scientists hinges on isolating a protein called “ubiquilin2”:

Amelie Gubitz, a research program director at the National Institute of Neurological Disorders and Stroke, said the Northwestern research is a big step forward in efforts worldwide to conquer ALS.

“You need to understand at the cellular level what is going wrong,” said Gubitz. “Then you can begin to design drugs.

“ALS is a complicated problem, and Dr. Siddique’s research adds a big piece to the puzzle that gives us important new insights.”

A variety of proteins perform different functions within cells, and Deng and Chen led research that discovered a key protein, ubiquilin2, in the ALS mystery.

Ubiquilin2 in spinal and brain system cells is supposed to repair or dispose of other proteins as they become damaged. The researchers discovered a breakdown of this function in ALS patients.

When Ubiquilin2 is unable to remove or repair damaged proteins, the damaged proteins begin to pile up in the cells, eventually blocking normal transmission of brain signals in the spinal cord and brain, leading to paralysis.

Dr. Siddique previously helped isolate the gene that makes some people susceptible to hereditary ALS.  Nailing down which protein malfunctions with the onset of the disease should be a big step toward either preventing this cascade failure of body chemistry, or dealing with its effects.  This is really great news; I hope it doesn’t get buried in all the bad news.  Hearty applause to all involved!


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