- The goal is to identify cellular mechanisms underlying hereditary mitochondrial diseases and to establish therapies for people with these diseases.
- Using self-built disease models, researchers are testing the effect of promising molecules developed by pharmaceutical companies on disease progress.
- These efforts will increase understanding of mitochondrial diseases and related therapeutic opportunities.
When pharmaceutical companies on the other side of the world are looking for a research partner for their projects, they may look to Finland and Academy Professor Anu Wartiovaara. A research group head at the University of Helsinki, Wartiovaara is a global leader in the study of mitochondrial diseases. In her profession, she enjoys investigating the progression of rare diseases and the causes underlying them.
“This really is extremely interesting work,” Wartiovaara sums up.
Mitochondrial diseases affect an estimated two million people worldwide. The diseases are caused by gene defects that lead to disruptions in cellular energy metabolism. They can manifest as, for example, heart, muscle and brain diseases or, in children, severe systemic diseases.
Internationally speaking, there are exceptionally competitive groups specialising in the study of molecular metabolism at the University of Helsinki.
Wartiovaara’s group investigates the mechanisms of mitochondrial diseases. Recently, they demonstrated that mitochondria also contribute to the body’s defences against viruses, the disruption of which can advance the onset of a certain neurodegenerative disease. Accumulating knowledge is making it easier to identify rare diseases.
“Our research has helped to provide diagnoses for thousands of patients around the world, and even treatments for some.”
So far, mitochondrial diseases cannot be cured, but new therapies are being investigated. Wartiovaara’s research has in fact provoked interest in international pharmaceutical companies, of which several have approached her over the years. At the moment, Wartiovaara’s team is collaborating with two California-based pharmaceutical companies.
“Businesses make it possible to develop therapies in a way that would not be possible for an academic group on its own.”
Pharmaceutical companies are interested in disease models designed by Wartiovaara’s group, which have identified potential drug targets. In the collaborative projects, Wartiovaara and her colleagues are testing promising molecules developed by the companies to determine whether they affect disease progression. The results are published in scholarly articles, promoting research on mitochondrial diseases more broadly.
Wartiovaara and her group have also demonstrated that, at times, cellular metabolism can be redirected by relying on the body’s own regulators.
“You don’t always have to develop drug molecules for years on end.”
Wartiovaara has also been involved in Finnish collaborative projects. In 2018, a working group she headed also received a substantial grant (link in Finnish only) from Business Finland for an ecosystem concept implemented in cooperation with the pharmaceutical company Orion Pharma. Together with their teams, Wartiovaara and Professor Anna-Elina Lehesjoki sought metabolic pathways common to diseases with similar symptoms.
“Project results are published in a number of articles.”
Recently, Wartiovaara and her group received two grants from international foundations that give a significant boost to their research activities: €1.2 million from the PolG Foundation of Prince and Princess de Luxembourg and pilot funding awarded by the Chan Zuckerberg Initiative, with Professor Pekka Katajisto and his team involved in the latter project. Carried out by several groups, the study of metabolic processes at the molecular level meets exceptionally high standards at the University of Helsinki.
“We are very well known around the world.”
A couple of years ago, Wartiovaara and her colleagues established NADMED, a business of their own. It all started with the discovery that the progression of a specific mitochondrial disease can be slowed by using vitamin B3 to increase nicotinamide adenine dinucleotide (NAD) levels. The NAD molecule regulates tissue metabolism. The NADMED startup has introduced a test to measure NAD levels, important to health, from blood.
“We already have customers on four continents.”
NADMED’s board includes Juha Tuominen, former CEO of HUS, and Ilkka Salonen, former CFO of the construction company YIT. Indeed, diving into the world of startups with the support of an expert group has been rewarding.
“For me, it’s been a tremendous learning experience,” Wartiovaara notes.
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