The annual cost of treating brain diseases is in Europe €798 billion, globally it amounts to €3 trillion. In addition, over 90% of novel drugs being developed for brain diseases fail during the development process. One of the main reasons for this is that there are no good preclinical models available, since animal models do not correspond well to humans.
Researchers at the University of Helsinki have investigated a new kind of technical solution to make it possible to test drugs in models that are a better match to humans. The new Living Human Brain (LHB) solution enables the culturing of human-derived brain organoids, also known as mini-brains, from the cells of any individual. In practice, this technical solution makes it possible to study drugs, before moving on to expensive clinical trials, on a platform that models the human brain. Furthermore, the solution is expected to offer significant help in the medicinal treatment of brain diseases. According to Professor Jari Koistinaho, who heads the research group, hospitals could utilise the technique in personalised therapies, as it enables the creation of patient-specific mini-brains.
For the first time, the solution incorporates all key cell types found in the human brain, including nerve cells, glial cells, cells of the immune system and cells that form blood vessels.
“LHB is brain tissue derived entirely from humans, which can be grown in a culture dish in a laboratory and which can be used to model the physiology of anyone’s brain. As raw material, we use human cells, which can be collected, for example, from a blood sample or skin. To begin with, we turn the cells into stem cells, after which we use them to grow mini-brains,” Koistinaho says. “The mini-brain can be also transplanted into living rodent brain, where it maturates further and allow high resolutions research on human brain as part of a living mammalian organism,” Koistinaho adds.
In recent years, the competence base associated with stem cell-based technical solutions has been enhanced at the University of Helsinki and the University of Eastern Finland for the prevention of brain diseases. LHB too is a technical solution based on stem cells. The new solution helps to improve the therapeutic process of brain diseases, while reducing costs.
"The LHB solution can accelerate the identification of suitable personalised drug therapies for individual patients as much as tenfold,” says Koistinaho.
Alleviating the burden of costs in drug development is another great opportunity. With the help of the new LHB solution, costs could be significantly reduced by testing drug candidates in human-derived brain tissue already at the preclinical stage alongside animal testing.
“Among other things, our solution can reduce the number of unnecessary clinical trials by roughly 30%”, Koistinaho notes.
Business Finland has awarded more than €600,000 to the project for finalising and commercialising the LHB solution. The overall budget of the project, which will officially launch in August 2021 and run for two years, is €860,000.
Over the course of the project, the research group will survey various opportunities to commercialise LHB.
“According to our current estimate, the market potential of LHB is over €600 million through automated production, licensing the solution, and business partnerships”, says Koistinaho.
The costs incurred by brain diseases are very high: it is estimated that the annual costs in Europe are almost €800 billion and globally up to €3 trillion. In addition, over 90% of new drug candidates being developed for brain diseases fail during the development process. One of the main reasons for this is the poor translatability of the efficacy of drugs from animals to humans.
Living Human Brain (LHB) technology enables growing human-derived brain tissue, so-called mini-brains, from the cells such as blood or skin cells of any individual. The mini-brains created using LHB technology contain all of the most important cell types of the human brain and thus can be used for studying and developing drugs in a human brain model before moving on to costly human trials. In addition, hospitals could potentially utilise the technology in personalised medicine, as it enables the creation of patient-specific mini-brains.
Patenting options are currently being explored. In addition, various business models are being considered for commercialising the technology – one option is a spinout company that produces mini-brains for a fee or licenses the right to manufacture them.
We are looking for partners in the pharmaceutical industry for validating our mini-brain technology. We are also looking for partners who provide equipment and methods for stem cell or mini-brain work. In addition, we are looking for investors interested in our technology to finance development efforts and the establishment of the spinout company after the two-year project funding finishes.
Gen Tamjar, Senior Commercialisation Officer, Helsinki Innovation Services, firstname.lastname@example.org