Preclinical studies validate the potential of ApTOLL to reduce inflammation and regenerate myelin in models of multiple sclerosis

20 May, 2024

ApTOLL multiple sclerosis
In preclinical studies on animals with multiple sclerosis (MS), ApTOLL, a first-in-class aptamer developed by aptaTargets to antagonize Toll-like receptor 4 (TLR4), has been shown to have a therapeutic effect to slow the progression of the disease. The results of the research have been published in the British Journal of Pharmacology.

The aptaTargets research team, in collaboration with scientists at the Cajal Institute of the Spanish National Research Council (CSIC), evaluated the efficacy and safety of the innovative treatment on different animal models of MS.

The results demonstrated that ApTOLL reduces inflammatory response and myelin loss and, furthermore, has a remyelinating effect, in other words, it helps to regenerate myelin. Myelin is a layer that protects the neuronal axons responsible for transporting nerve impulses. In the case of MS, the immune system attacks the myelin as well as the cells that produce it, destroying it and thus affecting its production and regeneration.

The therapeutic solutions that are currently available to treat MS are made up of immunomodulators; however, but there are still no approved remyelinating drugs.

The results of this study are very promising and reveal a new therapeutic approach for treating inflammatory and demyelinating diseases such as MS. ApTOLL’s molecular nature generates not only an anti-inflammatory effect, but also a cytoprotective and remyelinating effect,” explains Dr. Macarena Hernández-Jiménez, Chief Scientific Officer of aptaTargets and co-director of the study together with Dr. Fernando de Castro (Cajal Institute).

“ApTOLL treatment positively impacted the clinical symptoms of mice in the different models of multiple sclerosis, which was also associated with better preservation and restoration of myelin and oligodendrocytes in the lesions of animals. Restoration was corroborated on purified cultures of oligodendrocyte precursor cells,” indicates Dr. Macarena Hernández-Jiménez.

The excellent safety profile that ApTOLL is proven to have in animals and humans (in clinical trials in healthy volunteers and patients who have other diseases such as acute ischemic stroke), together with the efficacy results disclosed in this work, show the potential for future clinical trials in patients with multiple sclerosis.

It is estimated that over 1.8 million people have multiple sclerosis worldwide. It is a chronic degenerative disease that affects cognitive, emotional, motor, sensory and visual functions. As stated by the World Health Organization (WHO), the goals of MS treatment are to reduce the frequency and severity of relapses, slow disease progression, manage symptoms, and improve quality of life.

Reference article:

ApTOLL: A new therapeutic aptamer for cytoprotection and (re)myelination after multiple sclerosis.

Fernández-Gómez B, Marchena M, Piñeiro D, Gómez-Martín P, Sánchez E, Laó Y, Valencia G, Nocera S, Benítez-Fernández R, Castaño-León A, Lagares A, Hernández-Jiménez M, de Castro F. British Journal of Pharmacology (2024). DOI: