The STEMM research programme explores metabolic diseases with the help of stem cells and gene editing

At the turn of the millennium, stem cell research was expected to quickly yield revolutionary therapies. Even though this did not come to pass, research in the field has been successful, asserts Professor Timo Otonkoski.

According to Professor Timo Otonkoski, director of the Translational Stem Cell Biology and Metabolism research programme (STEMM), what links the research groups working under the programme is research focused on genetic metabolic diseases as well as their modelling with the aid of stem cell research and gene editing.

“Our programme is method-based, but offers synergies to all participants. All of the groups have challenging and novel perspectives, and it is productive to explore them together. Each group has specific research topics of its own, but the basic methods utilised in the research are the same.”

The STEMM programme is founded on the research programme for molecular neurology, active in the previous term, but the current programme has renewed itself in terms of both content and focus areas.

“Junior researchers, such as Satu Kuure who returned from the United States and Sara Wicktsröm who came back from Germany, have brought with them important new expertise associated with stem cells and genome editing, while Taneli Raivio’s group offers solid expertise in disease modelling.”

From dabbling to professional and top-level research

In the last 20 years, medical research at Meilahti Campus has taken a considerable leap forward.

“Many research groups got their start from a kind of amateur basis. It’s been great to see how high-quality and professional the research has become over the years,” Otonkoski says.

A less positive trend has been the reduction in the number of researchers trained as physicians. There are not many of them in the STEMM programme either.

“The majority of our junior researchers need not be physicians, but some are needed when conducting research focused on patient care. Personally, I feel – and this is pure conjecture, not based on any scientific figures – that the nadir is now behind us and that younger physicians are once more becoming increasingly interested in research.”

No quick wins in science – fruit takes time to ripen

In 1998, embryonic human stem cells were a hot topic in the media, which, according to Otonkoski, was a starting shot for ‘pretty incredible hype’. Stem cell research was subject to huge expectations; there was talk about revolutionary new treatments.

“Even many of those you would have expected to be more critical fell for the hype,” Otonkoski states with some surprise.

Fantastic dreams of a quick breakthrough in stem cell therapies remained just that, which made the media’s and public’s interest in the research field wane to a certain degree. However, researchers retained their enthusiasm and carried on with their work.

“In the end, stem cell research has been an enormous success in terms of basic research. We are now getting to the point where we can start expecting clinical applications as well.”

Still, Otonkoski does not believe that the central findings in stem cell research will be revolutionary cellular therapies; rather, the biggest benefit for medicine will be gained from disease modelling.

“I’m sure there will be new therapies, but what is most important is that stem cells and gene editing help create disease models, which can be used to study in detail the onset and progress of diseases. Such information will, in turn, help find effective methods of treatment and prevention,” he says.

“That’s what medical research is about. We break down diseases into their constituent parts to see what is central to them and how we can influence them.”

For the first time, researchers now have the opportunity to investigate diseases directly with the patient’s own cells. Both a disease model and a healthy version can be constructed from these cells in a laboratory, with comparisons providing accurate and reliable data.

“Our everyday work entails fixing genetic mutations found in patients’ cells, or creating mutations in healthy cells and then investigating and comparing them. This helps us understand the most important mechanisms of diseases.”

A link is needed between the laboratory and clinical trials

Otonkoski estimates that the researchers will be much more adept at controlling cellular reprogramming when the term of the STEMM research programme ends in five years . His research group is currently developing the utilisation of CRISPR tools to increase the accuracy of regulating cellular differentiation.

“For the time being, cells differentiated from stem cells don’t reach maturity, nor do they function as efficiently as they should. In five years, the results will certainly be much better. What benefits that will bring about is difficult to predict. Who knows, we might even have therapeutic applications in five years.

“And I can’t even imagine where we will be in 20 or 30 years!”

According to Otonkoski, the current focus should be on how basic research and clinical research could be better integrated so that laboratory work would smoothly lead to trials involving patients.

“It’s nice to investigate interesting things in the laboratory, but enormously laborious to start processing the results in clinical trials. And yet, that’s what we’re here for, to bring benefits to patients. Now that stem cell research has advanced and is offering increasing opportunities when combined with gene editing, it's vital to try to get clinical trials started. I hope relevant support measures will be actively developed on our campus.”


The Research Programs Unit of the Faculty of Medicine

The Research Programs Unit (RPU) of the Faculty of Medicine is a high-level research community.  An international scientific advisory board evaluates and selects the research programmes and groups through a competitive application procedure every four to six years. RPU director is Associate Professor Henna Tyynismaa.

The current Research Programs are the following (until the end of 2025):