Currently, there are no treatment options that could cure the diseases, or even stop or slow the progression. Therefore, our research is especially important for those who suffer from ALS or PD. A promising novel neurotrophic factor, cerebral dopamine neurotrophic factor (CDNF), moved on to clinical trials for PD in Finland and Sweden in 2017. It has the potential to be the basis of a treatment strategy which could stop disease progression.
ALS is a rapidly progressing fatal motoneuron (MN) disease characterized by progressive degeneration of MNs. There is no treatment for ALS that could halt the progression or cure the disease, thus there is a crucial medical need to find drug therapies that would slow disease progression, treat the disease and extend survival of the patients. The clinical hallmark of ALS is the combination of upper and lower MN signs and symptoms, causing muscle weakness with a wide range of disabilities. Most patients with ALS die from respiratory failure, usually within 1 to 3 years from the onset of symptoms. Our aim is to, using a wide array of preclinical methods, find therapies that could protect from motoneuron loss and improve the survival from the disease.
Parkinson’s disease (PD) is a progressive neurodegenerative disorder characterized by the cardinal motor symptoms of tremor, rigidity, postural stability and bradykinesia. In PD, dopaminergic cells die most prominently in the area of substantia nigra. Current therapies of PD do not prevent the progression of the disease, and the efficacy of these treatments wanes over time. We aim to find a treatment that stops the neurodegeneration and restores dopamine function above the level of clinical symptoms.
The aim of the neurorestoration therapy is to reverse the intracellular environment towards restoration of the dopamine phenotype. In developing restoration therapy we have focused on neurotrophic factors and small molecules. We are using the 6-OHDA model in mice and rats and the MPTP-mouse model of Parkinson’s disease (Airavaara et al., 2013; Voutilainen et al., 2011). We are looking at the mechanisms of neurodegeneration as well as neuroprotection and neurorestoration with a wide array of methods. We study these phenomena starting at the molecular and cellular level, and up to the neuronal networks. Furthermore, we are using electrophysiological and electrochemical methods to look at the function of nigrastriatal dopaminergic pathway, ie. activity of the neurons in the substantia nigra and dopamine kinetics (release and reuptake) at their terminals in the striatum. We have several novel mouse models in which we can study GDNF, CDNF and MANF biology, and master a wide array of behavioral tests routinely used to study dopamine-regulated behaviors.