- In a project headed by Saarma, the companies GeneCode and Argobio are developing small drug molecules that could support the functioning of the dopamine system of the brain.
- The goal of the collaboration is to provide patients with a ground-breaking treatment for Parkinson’s disease.
- In the best case scenario, the first clinical trials could begin in 2025.
Parkinson’s disease, a neurodegenerative disorder, affects roughly 10 million people globally, and the number of patients is expected to only increase in the future. For the time being, no drugs are available that would slow the progression of the disease. Over the years, Research Director Mart Saarma of the Helsinki Institute of Life Science (HiLIFE), University of Helsinki, has joined forces with several companies to tackle this problem.
“Whenever I have the opportunity to apply my research findings, I definitely try to do so,” Saarma notes.
He is now involved in an ambitious project carried out by two companies: GeneCode, based in Estonia and the United States, and Argobio, a French startup, are pursuing a breakthrough in the treatment of Parkinson’s disease. Under Saarma’s direction, the companies are developing small molecules that could protect dopamine-producing neurons in the brain. Their destruction causes symptoms typical of Parkinson’s disease, such as resting tremors.
If all goes well, the new therapy can soon be tested on humans.
“We hope to initiate phase 1 trials next year,” Saarma says.
The benefit of small molecules is that they function similarly to the glial cell line-derived neurotrophic factor (GDNF), a growth factor discovered in the 1990s. In spite of opening promising therapeutic avenues for Parkinson’s disease, GDNF has one big problem: administering the growth factor to humans requires penetrating the blood–brain barrier.
“This means that patients must undergo brain surgery,” Saarma says.
Small molecules are not associated with the same problem. GeneCode recently received €16 million in funding from the European Innovation Council. In the best case, molecules that mimic GDNF could also be helpful in other diseases, such as ALS or irritable bowel syndrome.
Saarma’s collaboration with GeneCode began in 2012. Today, he serves as the company’s lead scientist and is one of its shareholders. Saarma and his grouphave long been conducting groundbreaking research on neurodegenerative diseases at the Institute of Biotechnology under HiLIFE: he has identified GDNF receptors and the cerebral dopamine neurotrophic factor (CDNF) associated with the maintenance of dopaminergic neurons. This has also made businesses interested.
“Companies have contacted me on the basis of conference presentations or publications.”
There are 18 patent families under Saarma’s name, and he has licensed a number of technical solutions related to Parkinson’s disease. Even today, businesses have therapies in the drug development pipeline dating back to his GDNF receptor discovery in the 1990s. Saarma explains that, for example, MedGenesis Therapeutix has successfully tested the GDNF growth factor in Bristol in patients with Parkinson’s disease.
“For the first time in history, the progression of the disease was at least slowed in roughly half of the patients.”
Among the general public, the most well-known businesses in the field are probably those established by Saarma with his colleagues. The diagnostics company Mobidiag was sold to the United States for a whopping €668 million, of which the University of Helsinki received nearly €50 million. Herantis Pharma is developing a drug based on the CDNF growth factor for Parkinson’s disease, which may soon progress to phase 2 trials and, in the best case, revolutionise the treatment of neurodegenerative diseases. Of course, competition in the field is fierce.
“So far, Herantis Pharma’s results look very promising.”
Saarma, who was also among the leadership of the European Research Council (ERC) in the 2010s, thinks that the commercialisation of research benefits society as a whole. Academic startups can create jobs, generate tax revenues for the state and attract international investors. For researchers, joining forces with businesses brings, among other things, valuable research tools – and produces influential scholarly publications.
“I’ve got a great deal out of business collaboration,” Saarma sums up.
Saarma sees a lot of potential for successes in the research conducted at the Helsinki Institute of Life Science. Researchers at the Institute of Biotechnology, Saarma’s own unit, have already secured 16 competitive ERC grants, and there are also a number of junior group leaders at the institution who are developing interesting applications.
“There are several groups conducting excellent, high-quality research,” Saarma points out.
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