There’s a new drug in town.
Tertomotide, or GV-1001, is a small polypeptide, which means that it’s a string of amino acids, like a protein. But with only 16 amino acids, it’s much smaller than any protein. In fact, it’s a critical fragment of a protein, in this case an enzyme called telomerase reverse transcriptase. The drug was originally developed as an anti-cancer drug. Initial results there were unfavorable, but trials continue. In the lab, tertomotide protects brain cells growing in a dish from various insults including free radicals and inflammatory attack.
As many of the effects of tertomotide might be relevant to neurodegenerative diseases, it has also been tested in Alzheimer’s disease. A study published in 2021 found the drug to slow the rate of decline by upwards of two-thirds on a standard, 40-item test for AD called the “Severe Impairment Battery.” In one patient sub-group, the rate was slowed from 3.7 points to 0.1 point, or 97%.
Normally, a drug trial attempting to slow the rate of progression of a neurodegenerative disease would be happy with a 25 or 30% slowing and overjoyed to see 40%. So, this extravagantly positive tertomotide result in AD might be too good to be true, and in fact none of the other five tests of various aspects of AD gave a favorable result to any statistically significant degree. So, more study is needed. At least there were no side effects among the 55 patients receiving the active drug.
These results have encouraged not only further trials in AD, but also a trial in PSP! It’s not yet recruiting and it’s taking place only in South Korea. (Sorry — but glad for the South Koreans.) The study will randomly assign 75 patients to high dose, low dose and placebo groups. As in the AD trial, the drug is injected subcutaneously once a week for 4 weeks and then every 2 weeks for a total of about 6 months. The outcome will be measured by the rate of progression on the PSP Rating Scale.
The drug company’s website is here. http://www.gemvax.com/bio_en We wish them well. I’ll report back when I know more.
PSP Blog 
On June 20, the first patient recruitment for clinical trials began.
I hope the result will be the same as the preclinical result.
In addition, the changes in Alzheimer’s patients were more surprising than the data.
Thank you, Woo, for that information. Good news.
I wish trial sponsors would keep their clinicaltrials.gov listings updated properly, especially if they’re trying to recruit their new trials! LG
I am just a family member of an Alzheimer’s patient.
I was interested, so I gathered as much information as possible and found your blog.
Please understand that I am not familiar with English, so I use a translator to write ^^
That shows you how much of a techno-ignorant Boomer I am.
A paper has been published on what you mentioned.
https://dnd.or.kr/DOIx.php?id=10.12779/dnd.2023.22.e16
Dear woo: Thanks for sending that link, which reports a cognitive benefit of tertomotide in Alzheimer’s disease. The benefit was only in language function, not overall cognition or memory. The placebo group showed no change over the study’s 24 weeks, but the tertomotide group showed improvement. This was probably a symptomatic, not a neuroprotective, effect, and its magnitude was modest, but it’s still good news.
Thank you very much. I wanted to know the objective meaning of the above paper.
While watching the patient of an acquaintance who participated in the previous clinical trial, I got an expectation.
But I think I’ll have to wait a little longer.
I look forward to hearing good things from you.
woo: There are so many new drugs being developed for PSP that your optimism is more than justified. Dr. Golbe
Dear Dr. L. Golbe
I’m sorry to ask you a lot of annoying questions.
https://www.mdpi.com/1422-0067/24/16/12566
I have a question because a new paper came out this time.
I’ve seen a paper in the past that periodontitis is associated with Alzheimer’s and cranial nerve-related diseases.
Can the content in the above paper play a positive role?
Thanks for this question, Woo. The drug you refer to, GV-1001 (also called tertomotide), is given by subcutaneous injection, and has a number of different actions. One small, double-blind clinical trial in South Korea in moderate-to-advanced Alzheimer’s disease gave modestly favorable results. It is now being pursued in a larger trial of moderate-to-advanced AD patients, which has not started recruiting, and a small trial of early-to-moderate-stage AD patients, which is under way. But I’ve saved the best news for last: GV-1001 will be tested in PSP as well. This was the topic of my recent post that prompted your comment, “Tertomotide to the rescue?” GV-1001 is the same thing. That trial is listed as not yet recruiting, but its completion date is given as May 2024. All of these trials are only in South Korea.
Hi Dr. Golbe, Checking in on this now about a year later —
Do you think that we might expect to hear any preliminary results from the GV-1001 trial any time soon?
Thank you as always.
Maura & Pedro
Hi, M:
My latest info from clinicaltrials.gov is that as of December 2023, that trial was still recruiting. (Note that it’s only in South Korea.) It’s a six-month study, so even if the recruitment was completed a couple of months after that last update (let’s say February), the last patient would finish in August and then it would take about three or four months to clean and analyze the data before announcing the results.
I’ll make inquiries next chance I have, though I don’t consult for that company.
LG