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New Answers About ALS May Yield Treatments

Posted on October 27, 2017


A new research study from the University of North Carolina School of Medicine and North Carolina State University may lead to breakthrough treatments for people with amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease.

ALS is a nervous system disease that weakens muscles. It is rare, with fewer than 20,000 people diagnosed each year. People with ALS suffer from muscle weakness, loss of balance, problems with motor function, muscle stiffness, spasms and the inability of control their reflexes.

ALS is a progressive disease and over time can impact the ability to eat, speak and breathe.

A Problem With a Protein

The North Carolina researchers worked with a specific protein called TDP-43, which is already known to contribute to the development of ALS because it causes clumping in neurons.

The clumping caused by TDP-43 eventually causes the death of neurons, which impedes nervous system function. The protein also induces a degeneration of muscle tissue in ALS patients who experience sporadic inclusion body myositis.

TDP-43 is found in the nucleus of cells and plays a critical role in how a cell functions. It also regulates gene expression. It becomes a problem when the protein moves from inside the nucleus to other parts of the cell and then clumps together. When this happens, the cell is unable to function or express genetic information.

ALS researchers believe that the inability of cells to function or regulate themselves after TDP-43 leaves the nucleus and clumps together causes premature cell death.

When this happens in ALS patients, it leads to the inability to control muscles, muscle degeneration and muscle weakness.

The study found that clumping occurs when a particular chemical modification known as acetylation goes awry. Acetylation is the process that turns body proteins on and off.

Researchers on the study believe that when the acetylation process malfunctions, it induces clumping of TDP-43, and using a natural anti-clumping agent can reverse clumping in muscle cells and stop muscular degeneration.

Getting ALS Answers

To test their theory, study authors produced sporadic inclusion body myositis in mice to cause TDP-43 clumping in the mice’s muscles.

Once the TDP-43 in the muscle cells showed signs of clumping, researchers observed that the cells were trying to get rid of the TDP-43 protein to survive.

To reverse the clumping and save the cell, they added another specialized protein known as heat shock factor 1 to neutralize the impact of the TDP-43 clumping. Once neutralized, the cells were able to survive.

Study authors hope their discovery could lead to anti-clumping therapy in a pill or injectable form.

New Treatment for ALS Now

Dr. Joel Singer is a New York physician who treats patients who have neurological diseases like ALS, Parkinson’s disease and muscular dystrophy using personal cell therapy.

This kind of regenerative medicine uses the patient’s own fat, eliminating the risk of rejection and other complications; there are also no harsh side effects that may occur with conventional treatments and medications.

Currently, there is no cure for ALS; mainstream medical treatments only work to slow the progression of the disease and keep patients comfortable.


University of North Carolina Health Care. “ALS: New clues to the cause and how future drugs might reverse disease: Researchers team up to reverse TDP-43 protein aggregation, a hallmark of degenerative diseases.” ScienceDaily. ScienceDaily, 19 July 2017.


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