Phase 1 trial of VRG50635 - a novel therapeutic discovered & "delivered into the clinic in just 4 years" by an AI-powered platform

I know it is for ALS but it gives me hope that AI can deliver so fast.

vergegenomics.com/news-blog...

[park_bear]

This is just a phase I safety study. As far as I can tell there is no animal data - apparently they do not use animal models at all, so there is no evidence of efficacy other than computer modeling. I wish them luck but this is a longshot. They are also looking at Parkinson's: vergegenomics.com/pipeline

So this is only "delivered to the clinic" for the purpose of testing.

[Hidden in reply to park_bear]

I understood that the whole point is to skip the animal models and doze humans.. If that is the case we might be loking at a whole new fast track of tsting new drugs.

[park_bear in reply to Hidden]

Yes that is the whole point. Whether it works or not is another question. We shall see.

[Bolt_Upright]

SilentEchoes

[PDWarrior1900]

Thanks! Great post! VERY VERY interesting study.... My niece just gave birth to a daughter.... I believe within 30 to 40 years all disease will be wiped out and she BILLIONS of others will live long and healthy lives....

[SilentEchoes]

Thanks for the heads up. Seems like this is more about new technology than the actual treatment for ALS. "Verge discovered the relevance of PIKfyve in ALS using their computational platform, which incorporates large genomic and transcriptomic data sets from patients with the disease."

There's an ALS Registry at the CDC - some of these ALS groups are collecting blood samples and data for research.

Autophagy-related gene (TRIM68, PIKFYVE, and DYNLL2) research is a hot topic. I've said this before - viruses, bacteria, toxins and cancer share the same biological pathways. This small molecule is also being studied for the Vid and cancer.

The endolysosomal pathway is a critical cell process that clears the “garbage” within a cell. PIKFYVE is an enzyme that regulates endolysosomal function within a variety of cells, including neurons. In ALS, this “garbage” pathway is dysregulated, which leads to neuronal death and the progression of the disease.

In addition, viruses are able to co-opt PIKFYVE in order to gain entry into host cells and replicate, including SARS-CoV-2. Inhibiting this target prevents COVID-19 infection in cells as well as blocking Ebola, MERS, SARS and Marburg virus entry. This technology is applicable to Parkinson's disease too.

'Glucose starvation induces autophagy via ULK1-mediated activation of PIKfyve in an AMPK-dependent manner'

sciencedirect.com/science/a...

I might just have a high school education, but isn't this also achieved with fasting and a ketogenic diet??? 🤔

SE

vergegenomics.com/news-blog...