Despite many successes, modern medicine still struggles to find effective treatments for serious pathologies such as cancer and neurodegenerative and genetic diseases. For Alzheimer's disease, for example, only 30% of patients respond to therapy but for cancers the figure is only 25%.
Iawn The lack of an effective drug is not the real drug but a difficulty in reaching the therapeutic target, says Francesco Nicotra, the EU-funded NABBA project coordinator. O Many therapeutic targets are protected behind biological barriers, limiting access to conventional drugs or diagnostic tests.
The body's defense system has several mechanisms for eliminating non-self-substance substances. This is even more problematic for most innovative biological pharmacies, such as recombinant proteins, antibodies and gene therapeutics.
Although the project has just finished, the results are promising and the commercialization of one discovery has already started.
Neglect of the body's defenses
The challenge faced by NABBA researchers, working in a consortium of six research institutions and five companies, was how to protect drugs until they reach their destination in the body. Their answer is to summarize the executive agent in a 'nano-vehicle' vehicle that can travel through the body without hindering it until it releases its wage of drugs.
The NABBA project has developed nano-vehicles based on different auto-assembling molecules of natural and synthetic origin, such as liposomes and some specific polysaccharides. Researchers designed the vehicles to be invisible to the immune system and prepare them to overcome the biological barriers providing a useful amount of drug to the right place in a controlled manner.
Getting to the right place itself is a big challenge. For some cancers, the nano-vehicles are small enough to slip through crevices occurring in lymphatic shipping walls in the tumor site, a phenomenon that has already been seen with nano-drugs. others. In other cases, more active targeting methods are needed.
‘The most challenging goal was to promote specific active targeting – in other words, the ability to selectively release the drug where it is needed, explains Nicotra.
NABBA scientists have been working on two methods. In the first, different targeting agents – peptides, carbohydrates or antibodies – are attached to the surface of the nano-vehicles to operate as er keys to access the target cells. In the second strategy, an ultrasound beam collapses the nano-vehicle when it reaches its destination; the drug is released only in that place and diffuses into the surrounding tissue.
Hope for Alzheimer's patients?
‘We have had very interesting and promising results, 'said Nicotra, particularly targeting different tissues and the pathology of the brain – the most difficult blood-brain barrier is to prevent it.
When could we see these results as effective treatments? Nicotra warns that it can take 10 to 15 years and about EUR 2 billion for a newly discovered drug to come to the market, with a high chance of failure. Yet a class of nanoparticles developed in NABBA is already being promoted by a new company, Amypopharma. It is intended to develop it as a treatment for some nerve pathologies and, in the longer term, as a possible therapeutic against Alzheimer's disease.
NABBA was a four-year project funded through the EU's Marie Skłodowska-Curie action program, where 12 early stage researchers worked with experienced scientists not only to improve their own knowledge and skills but also to contribute to a new field of work. increasing nanomedicine.