Tikomed's ILB® attenuates clinical symptoms and serum biomarkers of oxidative/nitrosative stress and mitochondrial dysfunction in ALS patients.
A myotrophic lateral sclerosis (ALS) is a lethal progressive disease that causes degeneration of motor neurons (nerve cells that control muscle cells) and muscle. As motor neurons are lost, the muscles they control become weak and non-functional, thus leading to muscle weakness, disability, and eventually death. The clinical signs of ALS are weakness and atrophy of voluntary muscles, increased muscular tone with increasing spasticity and decreased fine motor skills, as well as increasing difficulties of swallowing, speech and respiration. From the time of diagnosis, most patients die within three to five years. Numerous trials have so far been unable to identify any agent that reverses or even halts symptoms. Researchers now aim to slow disease progression by targeting known pathophysiological pathways or genetic defects.
Published in the Journal of Personalized Medicine, new study results for Tikomed's platform lead drug candidate ILB® in the treatment of patients with ALS revealed a significant normalisation of the serum levels of several key metabolites in addition to a significant improvement of patients' clinical conditions for the duration of the ILB® treatment period. The treatment response appears to be mediated by improvement of tissue bioenergetics, a decrease of oxidative/nitrosative stress and attenuation of (neuro)inflammatory processes.
The deranged neuronal function associated with the oxidative/nitrosative stress and mitochondrial dysfunction characterised by the pathophysiological progress of neurodegenerative conditions such as ALS is reflected by changes in related metabolites in the blood. When measured, these metabolites can be used as biomarkers of tissue function and, therefore, of disease progression and/or patient response to treatment. In this study of a cohort of patients with ALS who had participated in the clinical trial entitled “A single-centre, open single-arm study on the safety, tolerability and efficacy of subcutaneously administered ILB® in patients with amyotrophic lateral sclerosis”, repeated ILB® administration over four weeks led to a significant attenuation of the levels of key serum metabolites related to neural damage, oxidative/nitrosative stress and mitochondrial derangement.
"We are very pleased that these results strongly suggest that ILB® treatment produces metabolic benefits corresponding with the encouraging clinical improvements seen for the ALS patients participating in this study," said Anders Kristensson, CEO of Tikomed. "The adaptive, multimodal mechanism of action of ILB® and the growing scientific evidence supporting its ability to rebalance the body's own inflammatory response and enhance endogenous repair mechanisms gives us the limitless potential to pursue all disease areas driven by uncontrolled or dysfunctional inflammation, on our quest to provide safe and affordable medicines to as many patients as possible across the globe".