The Erling-Persson Foundation is donating SEK 8 million to KI researcher Johan Ericson to fund the verification of his ATMP (Advanced Therapy Medicinal Products) concept with the aim of producing a highly effective restorative Parkinson's therapy.
Parkinson's disease is a neurodegenerative motor disorder affecting over 10 million people around the world. There is currently no cure and the effects of symptom treatments wanes over time.
Parkinson's disease occurs when neurons, particularly those that produce dopamine, die. Research has shown that when these particular neurons are replaced, motor function can be restored.
Transplantation a challenge
However, the problem is that adult nerve cells die on transplantation, which means that doctors must transplant immature progenitor cells, which are subsequently expected to grow into functioning dopamine-producing cells.
Preclinical studies suggest that this cellular exchange is not as efficacious as hoped.
"Other studies have shown that only an estimated five per cent of the transplanted progenitor cells develop into dopamine-producing neurons," explains Johan Ericson , professor at the Department of Cell and Molecular Biology at Karolinska Institutet. "This provides a limited therapeutic effect and leaves a great many unwanted, non-curative and potentially harmful cell types inside the patient."
Johan Ericson has been researching dopamine-producing nerve cells and the stem-cell treatment of Parkinson's disease for over 15 years.
Validating method using unique signal substance
Professor Ericson's research group has recently developed a method that has shown very promising results in preclinical studies. The hypothesis is that a unique signal substance can be used to promote the intended development of the progenitor cells.
"We see a level of dopamine cell replacement that's ten times higher than that achieved by alternative methods," he says. "We can also see that motor functions in an animal model of Parkinson's disease are restored very quickly within three months, which is half the time of other studies. The results are incredibly exciting."
The grant from the Erling-Persson Foundation will now finance the validation of the method with the aim of demonstrating whether it can be taken to the industrial production of clinically viable cells that can be used in the treatment of people with Parkinson's.
Potential one-time treatment
Although the ATMP therapy is costly, since dopamine cells live for decades, it has the potential to be a one-time treatment that restores motor function while ideally obviating the need for symptomatic medication.
"We'll now be ensuring that this cell-production process is robust and upscalable so that it will be able to provide a stable, high-quality drug," says Professor Ericson. "The grant from the Erling-Persson Foundation makes it possible for us to keep the momentum going and hopefully fulfil our goal to start clinical studies within three years."
