KAJO uses human stem cells to create a testbed for developing drugs for degenerative brain diseases

At present, no treatments slow down degenerative brain diseases such as Parkinson’s and Alzheimer’s. One of the reasons is that the cell models available for drug development do not correspond to human biology. For example, testing drugs in genetically modified mice or mice cells has proved profoundly disappointing in brain disease treatment.

“Using mice models, researchers have been unable to develop new drugs for Parkinson’s or Alzheimer’s disease,” says Mikko Airavaara, Professor of Pharmacology and Drug Development at the University of Helsinki.

Now KAJO team have taken a step towards solving the problem: they are developing a new and better disease model based on human stem cells, which can be used to test drugs intended for degenerative brain diseases before human trials.

The model is based on the differentiation of stem cells into the neurons central for each disease. They are then made to produce, for example, the protein plaques that characterise Parkinson’s disease. The innovation is particularly promising because it modifies cell function artificially only to a minimal extent.

A team of top experts refining the innovation

The innovation is based on stem cell technologies developed in the 21st century. Although researchers have used them in academic research, no Parkinson’s disease model based on human stem cells has been brought to the market and made available for pharmaceutical companies.

“This is precisely what we’re trying to do,” Airavaara states.

He has been examining the mechanisms underlying Parkinson’s disease for 20-odd years. In early 2023, Airavaara’s team received Business Finland’s Research to Business funding to commercialise their innovation. This team of experts is led by Vassileios Stratoulias, a developmental biologist with experience in extensive international collaboration who wrote his doctoral thesis on cell differentiation.

“It’s rewarding to work with skilled researchers seeking solutions to problems such as degenerative brain diseases. You have the chance to improve the wellbeing of many people,” Stratoulias notes.

Airavaara says that KAJO has a number of trump cards. He believes the team is capable of building a reliable cell model for measuring the effects of drugs accurately. Crucially, the same measures can also be used later when testing the drugs on patients. This is a typical feature of successful drug development projects.

“It’s important for us that the model can be replicated and has been validated in the same way as the current animal models,” he points out.

Promoting drug development for Alzheimer’s disease

If successful, KAJO will launch a new era in the treatment of degenerative brain diseases. The demand for the innovation is significant. As populations age, the number of people with Parkinson’s disease will increase. For example, about one million people in the US are living with Parkinson’s disease.

“If we can provide the only viable solution or one of a few such solutions for early-stage drug development, the commercial potential is considerable,” confirms Joel Noutere, the team’s commercialisation specialist.

The team’s two-year commercialisation funding boosts development work. As the scientific side of the project is currently ahead of schedule, the researchers can tackle new challenges too.

“We’ll explore during the project whether we can also build a model for Alzheimer’s disease.”

Towards a scalable business model

Noutere says that the team wishes to obtain a clear understanding of market needs by consulting with pharmaceutical companies. The goal is to establish a spinout when the project concludes at the end of 2024.

The project will also seek to determine the optimal business model. Noutere believes one option is to establish a commissioned research company, although this is not necessarily the best solution for commercialisation. Consequently, the team is keen to exchange ideas with private equity and angel investors.

“We’ve come up with potential business models that could be highly scalable and interesting to private equity investors funding startups.”