The European Medicines Agency (EMA) has designated as an orphan drug a compound based on the bioactive lipid molecule Maresin-1 (MaR1), currently being investigated by a research team from the UAB for the treatment of spinal cord injuries.
The designation represents an important step forward towards the development of a potential new treatment, by recognising that MaR1 could provide relevant clinical benefit in an area with high unmet medical need.
Spinal cord injuries have an estimated incidence of between 20 and 45 cases per million inhabitants per year. There is currently no pharmacological treatment that improves neurological recovery after injury; the only drug approved in the acute phase is methylprednisolone, the use of which is discouraged in many countries due to its adverse effects and limited efficacy.
MaR1 is a lipid produced by macrophages (immune system cells) during the resolution phase of inflammation and acts to promote the termination of the inflammatory process and the protection of tissue. The treatment being investigated by the Neuroplasticity and Regeneration Group of the Institut de Neurociències (INc-UAB) and the Department of Cell Biology, Physiology and Immunology of the UAB, led by Rubèn López-Vales, has demonstrated in animal models an outstanding capacity to significantly reduce post-injury inflammation and promote processes that limit neurodegeneration. These actions translate into a substantial improvement in locomotion recovery.
"This designation is the recognition of many years of research in the biology of inflammation resolution and in spinal cord injury models and allows us to move forward towards the goal of one day being able to offer therapy to people affected by this serious condition", says Rubèn López-Vales.
The EMA's orphan drug designation means benefiting from regulatory advantages that speed up the development of the compound, such as priority scientific advice and accelerated evaluation pathways, and protect marketing exclusivity in the event that it reaches the market.
To advance towards a clinical trial with the treatment, it is now necessary to complete regulatory preclinical studies that support product safety and establish a fully validated manufacturing process, as well as await approval by the regulatory agency and ethics committee of the documentation presented by the research team. All of these steps require approximately 3 to 5 years of work before a trial in patients can begin.
The research carried out by the UAB research team is funded by the Spanish Ministry of Science, Innovation and Universities, the "la Caixa" Foundation, the Agency for the Management of University and Research Grants (AGAUR), Barcelona Activa, and the University's own funds.
