Completed Heart, Stroke & Blood Brain & Nervous System

Development of UCCB01-144 as a new treatment for stroke.

In plain English

AI plain-English summary

A drug that protects brain cells during a stroke is about to be tested in humans for the first time. Stroke is a leading cause of death and long-term disability worldwide, yet treatment options remain limited. The only widely available drug, alteplase, must be given within a few hours and carries a risk of bleeding. UCCB01-144 works differently: it targets a protein called PSD-95 that sits inside the junctions between nerve cells. By blocking this protein, the drug appears to prevent a cascade of damage that follows a stroke, even when given hours later. Preclinical studies have shown strong results. If the Phase I trial confirms the drug is safe in humans, it could open the door to a new class of stroke treatments that are easier to administer and effective over a longer time window. That would change how emergency stroke care is delivered, potentially reducing the number of patients who die or are left permanently disabled. The research is not fundamental science—it is a direct push toward a new medicine, with a clear path to a Phase II trial if this first step succeeds.

View original technical description
UCCB01-144 has demonstrated great effect in preclinical studies of stroke, and combined with its unique mechanism of action (dimeric ligands) and a novel drug target (PSD-95), we believe that UCCB01-144 is a particularly promising candidate for the putative treatment of cerebral ischemia. The objective of this proposal is therefore to move UCCB01-144 forward through pre-clinical development and a human clinical phase 1 trial. This will be achieved by examining three key points: i) Show that UCC B01-144 has adequate exposure and safety profile in the pre-clinical program thereby enabling clinical development. ii) Obtain an increased understanding of the efficacy of UCCB01-144 in a broad range of in vivo stroke models in accordance with the STAIR (Stroke Treatment Academic Industry Roundtable) recommendations. iii) Examine UCCB01-144 in a first human dose Phase I clinical trial. If successful, UCCB01-144 will subsequently be employed in a Phase II clinical trial.

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Researchers

Kristian Stromgaard (EPMC Awardee)

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Original classification

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