CB-103
In human trialsMeasured in people, not heightBy mouthSmall molecule
A tablet that blocks the Notch signal lower down than the approved Notch drug does, at the point where the freed piece meets its DNA partner. No regulator has approved it anywhere. The company that owns it has staff among the authors of both papers on it.
An orally active small molecule that disrupts the Notch transcription activation complex, blocking the CSL-NICD interaction rather than the γ-secretase cut above it, which is why it also works in tumour lines resistant to γ-secretase inhibitors. It produced Notch loss-of-function phenotypes in flies and mice. No regulator anywhere has approved it for any indication. Cellestia Biotech holds it, and the company's people are among the authors of both papers behind it: cofounders and employees on the discovery paper, seven staff on the trial.
Risks. No regulator has approved it, so it has no label, and nobody knows what it does to a growing skeleton. In its only human study, in adults with advanced cancer, 19% had a severe side effect caused by the drug, most often raised liver tests, anaemia or changes in vision.
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What has been measured, in whom, with every source, and what it does to the rest of you. And every article in the library.
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Educational content, not medical advice. Nothing here is a recommendation to take or stop taking anything — talk to a doctor about your own situation.