Naturally Occurring Enzyme Improves ALS Symptoms in Mice, Study Shows

Naturally Occurring Enzyme Improves ALS Symptoms in Mice, Study Shows

A naturally occurring enzyme improves ALS symptoms in mice and could lead to effective therapies in humans, a study suggests.

The research, “Deletion of NAMPT in Projection Neurons of Adult Mice Leads to Motor Dysfunction, Neurodegeneration, and Death,” appeared in the journal Cell Reports.

Data from the ALS Registry indicated that the prevalence of ALS in the United States was about five cases per 100,000 persons in 2013. This would translate into 14,000 to 15,000 Americans with the disease.

ALS is a progressive movement disability condition characterized by death of nerve cells. Patients initially exhibit weakness in legs, feet, and hands. This spreads to other parts of the body and leads to muscle atrophy, difficulty walking and swallowing, and slurred speech.

“Our previous studies indicated that an enzyme known as NAMPT (nicotinamide phosphoribosyltransferase) is primarily expressed in the neurons [nerve cells] in mouse models, and overexpression of NAMPT can protect against further brain injury following a stroke. For these reasons, NAMPT became a good target of study,” Shinghua Ding, the study’s senior author, said in a press release. He is with the University of Missouri’s Dalton Cardiovascular Research Center.

The Nampt gene codes for the NAMPT enzyme. Researchers discovered that deleting the gene in the central nervous system of mice engineered to have ALS led to the animals developing symptoms similar to humans with the disease — loss of weight, drop in body temperature, degeneration of cells that control movement, and loss of movement capability.

At the cell level, deleting the Nampt gene impaired the functioning of mitochondria, the cells’ power plants. It also led to muscle atrophy and defective communication at neuromuscular junctions, which as the name implies is where nerve cells and muscles come together. Lack of the NAMPT enzyme ultimately led to paralysis and death, researchers said.

The team then treated mice with nicotinamide mononucleotide, a naturally occurring molecule that regulates NAMPT activity. It served as a substitute for NAMPT,  reducing loss of movement capability and increasing the lifespan of NAMPT-deficient mice.

In ALS patients, levels of NAMPT are significantly lower in spinal cord cells, supporting the notion of a link between ALS and NAMPT.

“Our data from human ALS subjects potentially link NAMPT to ALS,” the researchers wrote.

“What we’ve shown is that NAMPT is essential to neuronal function and viability,” Ding said. “Based on our findings, it is an ideal candidate for further study, and the possible development of drugs in the diagnosis and treatment of ALS and stroke victims.”


  1. Dave Shuey says:

    My God … Please, please, please somebody sponsor a clinical trial for the NAD+ precursor nicotinomide riboside (NR). Elysium has already conducted what was essentially phase I for NR. Now just couple this dosage in a double blind using ALSFRS-R as the metric. It’s so damned easy!

  2. Charlie says:

    Well now… a ‘naturally occurring enzyme…’
    Big Pharmas will steer clear of this in their droves.

    There’s no profit for them in something ‘naturally occurring.’
    As this looks like a very interesting potential line of research, we might hope that benefactor/charitable donations make possible further investigation.
    To me it looks like a similar possibility to Dr.Bedlach’s Lunasin project, which hopefully reports something good, and soon.

    • Mike Leist says:

      I was on a webinar that Dr. Bedlack hosted a couple of weeks or so ago. He didn’t find anything promising about Lunasin from his studies, and said he is moving on from that. On the call he said he wanted to look at curcumin next, which is found in turmeric.

      • Charlie says:

        Oh dear, if this is true then another possibility bites the dust. It simultaneously casts a shadow over the makers of Lunasin who were quite happy to take $400 a month off desperately ill patients whilst doing nothing to quash the rumour of some efficacy.
        I imagine stockholders of Lunasin were happy to ‘buy on the rumour, sell on the facts.’
        Many pALS ie Desperate Innocents, probably bought bucket-fulls of Lunasin even though they were not in Dr.Bedlack’s trial.

    • Mary B says:

      Charlie…Big Pharma will take advantage of nutrients and the like to increase this enzyme. Big Pharma has often taken advantage of nutrients/supplements to produce Rx products. Deplin is just the active form of folic acid. There are Rx fish oil products. And a lot of other Rx products start with something natural and then change one molecule so they can patent it. And, of course, these products are more expensive than equivalent high quality supplements. But, most people are happy to go to a doctor and get an Rx product for whatever symptoms they are having and never ask if there are alternatives. By the way, resveratrol has been shown to increase this enzyme:

      • Charlie says:

        I would caution you to be extremely wary of a Pharma which takes a ‘naturally-occurring’ substance and seeks to patent it by a superficial means such as a small technical tweak.

        Getting a patent on an everyday substance and somehow implementing an Rx pricing strategy would seem to be something Al Capone would endorse with great enthusiasm.

        “But, most people are happy to go to a doctor and get an Rx product for whatever symptoms they are having and never ask if there are alternatives.”
        Indeed. Fools and money are soon separated.

        Sadly, ‘Desperate Innocents’ and money are also soon separated. Just take a look at the ALS Today article on ‘Radicava and Ethics.’

  3. Colorado 18yr ALS Research has stopped ALS progression in Denver man. Eight weeks into protocol. 25 pounds gained. Facial muscle use returning. Muscle gain in arms. Initiating breathes on his own from the ventilator. (Unprecedented). Full recovery expected. No enzyme was needed. Solve how mammals “trigger” the biochemical physics of ALS. Show a recovery methodology. Period.

    • Charlie says:

      Very interesting. Is this report of success been peer-reviewed, gone through a Phase 1,2,3 trial ?
      You can understand why we might ask…there are so many BS propositions out there for money-making purposes. We all want trialling to be quickened up.

    • Charlie says:

      Testing and trialling are probably seen as a waste of time for this -maybe it is thought of as having ,say, no substance whatsoever….

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