Ataxia UK
2,494 members2,314 posts

Oral Ingestion of Trehalose. A human clinical trial!!!

Ladies and Gentelman, I am sorry that this study has not been published on every ataxia, huntington, parkinson, etc forum, but this morning I found what I consider the Holy Grail of trehalose studies. This was a double blind human clinical trial of oral ingestion of trehalose. The study proved trehalose ingested orally improved blood flow in elderly patients. This means, ladies and gentlemen, that eating trehalose gets some of the molecule into the blood. I realize this does not go so far as prove that the trehalose molecule can then continue on to the brain, but it is a HUGE step forward and should give every neurologist who is treating ataxia patients a giant moment of reflection.

Oral trehalose supplementation improves resistance artery

endothelial function in healthy middle‐aged and older adults

For those who don't want to read the details I will say this: in the study patients ate 100g daily and there were two reported problems 1) intestinal upset and 2) some patients gained weight. Otherwise 100g daily of trehalose was tolerated and efficacious.

14 Replies

Thank you for sharing the link. This is so interesting/exciting/hopeful. Good to have you back! 👍😀

1 like

Very exciting : )


As Sue said in an earlier post, one should never embark on eating mass quantities of anything without consulting their doctor. I am providing information and an explanation of why I think it is important, but it is critical that anyone with ataxia take this information and discuss it with their own neurologist before trying anything! That being said. . . it has just come to my attention that there may be a few people who don't fully understand the implications of the study I posted above. For those who get it please forgive this lengthy explanation, but if by chance you are scratching your head let me just say I am excited because I believe this study is one piece in a puzzle that provides more evidence that trehalose ingested orally may be beneficial to some ataxic patients, especially those who do not yet have symptoms but have a genetic diagnosis, and here is why . . .

1) BioBlast Pharma has evidence that trehalose crosses the blood brain barrier and can halt the progression of symptoms in patients with OPMD (a form of ataxia) and SCA3 but BioBlast was using injections of trehalose not oral ingestion so the question remained as to whether or not trehalose could get into the blood stream just from eating it.

2) Numerous people around the world who have genetic diagnoses of Huntingtons or SCA have been taking trehalose orally for nearly a decade and have gone well past the time when symptoms should have been present. Until now there was no evidence that trehalose was making it into their blood stream and was potentially the cause of their delayed onset.

3) There are more than 2 dozen studies in mouse models demonstrating trehalose efficacy in halting the progression of various ataxias and other similar neurodegenerative diseases.

4) Prior to the human study I linked above there was a mouse study that showed trehalose improved blood flow in mice. The clinical trial showed trehalose acted the same way in humans even when simply ingested with water.

5) Before BioBlast proved efficacy in humans, mouse studies showed trehalose could cure mice with OPMD and SCA3. BioBlast now has evidence that trehalose acted the same in humans as it did in mice.

So now we put it all together. The BioBlast research plus the anecdotal evidence of people who are symptom free plus this latest research demonstrating the trehalose molecule reaches serum levels high enough that it impacts human cellular activity leads to one epic conclusion.

In some cases, trehalose acts in humans just like it does in mouse models even when simply ingested orally.

Doctors thought ingesting trehalose would be ineffective because they believed the body could not absorb any of the whole trehalose molecule as it passed through the intestines. That is why the patients ate 100g a day in the study and why BioBlast is using IV administration.

The research from the last decade showing trehalose can prevent symptoms from ever appearing in mice by them simply drinking trehalose saturated water now has clinical evidence it may work in humans as well. Tragically proving this in humans would take literally decades because the ideal study would be a double blind trial of people with a genetic diagnosis who then ingest trehalose or a placebo over a multiple decade period starting before any symptoms had appeared. If it works the people on trehalose would never get ataxia and the people on a placebo would. On top of that trehalose is a sugar that costs $10 a lb so I'm pretty sure no pharmaceutical company in the world will be interested.


Here is my question: Does Trehalose help all ataxias? And why have the traditional ataxia orgs, shied away from this?? Thanks and more power 2u. N

1 like

This one is not going away is it! Most positive prospect of a treatment I've heard of.......could be the answer to Ataxia UK 2020 pledge to find a cure....doesn't seem to have been any other major break throughs! So how do we get this usefully trialed for those who need it sooner rather than (much...much) later!?


Today's find . . .

Turns out a Taiwanese University has applied for a patent to use trehalose and trehalose derivatives to treat numerous SCAs:

In the present invention, the type of the abnormal polyglutamine-mediated disease is not particularly limited. Preferably, the abnormal polyglutamine-mediated disease is spinocerebellar ataxia. In spinocerebellar ataxia, the expansions of CAG trinucleotide repeats encoding a polyglutamine stretch have been shown to cause dominantly inherited SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, SCA17 and dentatorubropallidoluysian atrophy (DRPLA) . . .

To implement the method according to the present invention, the above pharmaceutical composition can be administered via oral administering, parenteral administering, inhalation spray administering, topical administering, rectal administering, nasal administering, sublingual administering, vaginal administering, or implanted reservoir, and so on.

More hope!

1 like

Dear All, we don't want any potential treatments or cures for the ataxias to go away! They just need to be substantiated with scientific evidence before we can promote them!

Up to now we have safety information from various studies and a small six-month open label Phase 2a study with promising results, but which didn't have a placebo control group. We now need a much larger blind trial - the problem with this is the cost: squillions of £ (or $) which Ataxia UK sadly doesn't have! There is no doubt that we could get a lot more done much more quickly if we had more money! We are always grateful to our committed fundraisers - but there's always more to be done.

But, at the moment, I would argue we don't have the evidence for Joe's 'epic' conclusion. What we have at best is an epic hypothesis, based on anecdotal evidence from ataxia and other conditions, safety trials, the study mentioned above, and a small study which didn't have a control group - which laid it open to the placebo effect. All this needs to be tested in a larger trial to prove the hypothesis.

On the other hand, I'm not as pessimistic as Joe regarding the possibility of a pharma (presumably Bioblast) continuing to work on trehalose. Yes it's just a sugar, but Bioblast knew that when they started their work and they were still prepared to invest in it

Ataxia UK has maintained a dialogue with Bioblast and suggested to them that they could use somewhere in the UK as one of their trial centres when they get to the point of the large scale trial.

Trehalose does present a possibility, which Ataxia UK and the other ataxia charities are definitely not ignoring - with only a few possibilities existing, we can't afford to ignore i! And indeed, we have reported on it on our website and in the magazine.




Thank you for providing a balanced view for all to consider, but I hope I may be permitted a small counter point.

First the trial I provided above was a double blind placebo trial. The placebo was simply identified as maltose in the paper, but the patients were blind to which they received. In the trial 15 older adults between the ages of 50 and 77 ate 100g of trehalose per day and realized significant vascular improvement in a p=0.02 study result. That means the study was a smashing success in what it was trying to do. That also means the fact that the cohort (number of patients) was small is not as relevant because the results were so overwhelmingly positive. At a minimum one now has evidence that eating trehalose is good for vascular health and may help reduce cardiovascular risk, and that alone may be reason to speak with your doctor.


Hi Joe,

I do not suffer from ataxia but interested in trehalose due to another disease. I do have a question for you as I saw you have got quite extensive knowledge on this subject.

What would stop us to just proceed and inject endotoxin free trehalose?

Many thanks,



Is this something we can ask our GPS to prescribe?

1 like

Did the trial include participants with Ataxia? If not will there be such trials', Also is it available in UK and is it recognised by 'NICE' or perhaps the research still in its infancy stage.


What exactly is 'Trehalose' and is it to be found in food products?


Could be a coincidence but my husband was getting worse....very wobbly....and during a chat I said how upsetting it was for me that he never even shows interest in any of the things I research to help him! Fast forward a month and he has been using Trehalose in his coffee regularly (2 heaped spoonfuls per cup, 3-5 cups a day) and he seems steadier on his feet. Don't think he could balance on one leg but certainly he doesn't stumble half as much as he was?!?! He's not a placebo effect kind of a person either. ... he certainly wouldn't of expected it to help! Live in hope........x


How much Trehalose should I take each day? I am diabetic but it's supposed to be safe


You may also like...