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Centre de recherche
Tuesday, May 21 2024

A “Formula 1” for administration of drugs and gene therapy

A team perfects even higher-performance lipid nanoparticles with a new technology

Montréal, May 21, 2024 - To ensure that drugs and other therapeutic substances get to the right place in the body and act efficiently, they need a vehicle - and the most effective to date is called lipid nanoparticles (LNP). A research team at CHU Sainte-Justine, directed by Dr. Pierre Hardy, Victor Passos and Dr. Houda Tahiri, in collaboration with Professor Xavier Banquy of the Faculty of Pharmacy of the Université de Montréal, has perfected a new layer-by-layer technology that improves the efficiency and versatility of LNP.  Having proved the benefits of this discovery for the treatment of glioblastoma, a very aggressive brain cancer, the researchers now wish to apply this therapeutic “Formula 1” to other pediatric diseases with the aim of accelerating the transfer from bench to young patients’ bedside.

LNP: the vehicle of choice for gene or combination therapy

Gene therapy, including reliance on messenger RNA vaccines, has emerged as one of the most promising pharmacological approaches to date. RNA is very fragile, so it is quickly destroyed when introduced in the body. It must therefore be conveyed in a high-performance vehicle, which protects it while taking it to the place where it must act. 

This is precisely what LNPs do. Once injected, they ensure the completely safe transmission of RNA to the right cells to help fight a virus or a cancer. However, several limitations hinder the full clinical potential of LNPs, primarily because they are quickly captured by the liver.  “Our layer-by-layer method allows us to bypass several LNP barriers,” Dr. Houda Tahiri explains. “For example, we can ensure that LNPs circulate longer in the body without being destroyed by the liver, or that they can cross the barriers of certain organs, which was impossible before. Moreover, the possibility of having up to five layers allows us to combine several therapeutic approaches in the same vehicle."

A versatile strategy available for clinical validation

Up to now, this improved LNP vehicle has proved itself for administration of a treatment against glioblastoma. The team has succeeded in orienting LNPs to the cancer cells and the immune cells, for a targeted gene therapy.  This makes it possible both to hinder the proliferation of cancer cells and ultimately reorganize the immune cells so that they attack the cancer cells, thus offering a twofold strategy against pediatric cancer.

Nonetheless, the possibilities offered by these five-layer LNPs are far from being limited to glioblastomas or even cancer. “We have perfected a very open and versatile technology, which can be useful for all types of pathologies, notes Dr. Pierre Hardy, also a professor at the Université de Montréal. “For example, we could use them for certain cardiovascular or brain diseases, or in immunology. It is also very promising for rare genetic diseases, because it supports gene therapy. It could even be used to administer drugs, which then could be conveyed in lower concentrations while remaining effective.” The team wishes to make this highly versatile technology available to other researchers to demonstrate its full potential.

Would you like to use the technology in your research?

Contact Dr. Pierre Hardy at pierre.hardy.med@ssss.gouv.qc.ca.

For information

Catherine Goulet-Cloutier
Conseillère en communication
Centre de recherche Azrieli du CHU Sainte-Justine

Personne-ressource auprès des médias :

Justine Mondoux-Turcotte
Conseillère - Relations médias et relations externes
CHU Sainte-Justine
514 345-7707

Persons mentioned in the text

Cette recherche a bénéficié d'un financement du Fonds d’innovation thérapeutique en hématologie-oncologie du CHU Sainte-Justine, ainsi que de l'initiative pilote pour validation clinique lancée par Axelys, le CHU Sainte-Justine et la Fondation CHU Sainte-Justine. 

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Updated on 5/16/2024
Created on 5/16/2024
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