A new study shows the cerebroprotective effects of ApTOLL on mice with acute ischemic stroke

6 June, 2024

ApTOLL cerebroprotective - Pharmaceutics
The results of a preclinical study demonstrate that ApTOLL, a single-stranded DNA aptamer modulating the Toll-like Receptor 4 (TLR4) developed by aptaTargets, has cerebroprotective effects on thrombectomy models in rats with acute ischemic stroke (AIS) as it reduces infarct/edema volume, neurofunctional deterioration and the risk of hemorrhagic transformation, in addition to inflammatory responses at a peripheral and local level. 

The study, recently published in the Pharmaceutics journal , was conducted by researchers at aptaTargets (Dr Macarena Hernández) and at the Health Research Institute Hospital La Fe (Dr Juan B. Salom).

The paper discloses information on the ApTOLL dose-response, the therapeutic time window, the target population, sex differences and the mechanism of action.

“We carried out our research based on the recommendations of the Stroke Treatment Academic Industry Roundtable (STAIR) to further characterize the effects and mechanisms of ApTOLL after transient ischemic stroke in rats. The obtained results will help us optimize the design of future clinical trials with ApTOLL,” explains Dr Macarena Hernández-Jiménez, Chief Scientific Officer of aptaTargets and co-director of the study together with Dr Juan B. Salom (IIS La Fe).

It was also noted that the cerebroprotective effects of ApTOLL administered prior to thrombectomy support the design of new clinical trials to assess its effect even before starting reperfusion treatment.

Potential sex differences in the effects of the drug and inflammation-related biomarkers could be considered when designing and analyzing larger clinical trials related to personalized medicine.

At present, ApTOLL has completed a Phase 1b/2a clinical trial (APRIL) in patients with acute ischemic stroke, and it will start Phase 2b in the coming months.

Reference article:

Cerebroprotective Effects of the TLR4-Binding DNA Aptamer ApTOLL in a Rat Model of Ischemic Stroke and Thrombectomy Recanalization

Aliena-Valero A, Hernández-Jiménez M, López-Morales M, Tamayo-Torres E, Castelló-Ruiz M, Piñeiro D, Ribó M, Salom J. Pharmaceutics (2024). DOI: https://doi.org/10.3390/pharmaceutics16060741