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.