Researchers have published data from laboratory studies demonstrating the neuroprotective effects of a potential new drug for Parkinson’s called IkT-148009. Interestingly, the treatment worked even when the researchers delayed giving the drug until Parkinson’s-like pathology had appeared in the lab models of the condition.

In 2016, a drug called nilotinib, already available and widely used in the treatment of cancer, was reported to have shown encouraging results when used in a small clinical study treating people with Parkinson’s.

Two much larger and better controlled studies were then conducted (one trial was supported by Cure Parkinson’s and Van Andel Institute) to determine if these preliminary findings could be replicated. However, both studies failed to repeat the results of the small study, with one conclusion being that nilotinib was not able to fully access the brain to achieve the intended effect.

This led biotech companies developing similar drugs to nilotinib, to design their drugs with better abilities to access the brain, and in sufficient concentration to achieve a therapeutic effect. One such drug is IkT-148009 designed by biotech company Inhibikase Therapeutics.

Like nilotinib, IkT-148009 targets a protein called c-ABL which becomes over-active when cells are stressed. In the Parkinson’s brain, c-ABL levels are elevated in the populations of cells that are vulnerable to the disease; c-ABL targeting drugs like nilotinib and IkT-148009 stabilise the over-active protein, slowing its activity. Laboratory (preclinical) studies investigating c-ABL drugs have demonstrated their neuroprotective properties.

Now researchers at Johns Hopkins University School of Medicine, working in collaboration with Inhibikase Therapeutics, have published new data demonstrating IkT-148009’s neuroprotective properties in laboratory models of Parkinson’s, even when they delayed starting the treatment for 4 weeks.

Phase 1 clinical trials investigating the safety of IkT-148009 have been conducted in humans and Inhibikase Therapeutics is now looking to conduct a much larger and longer clinical trial in people with Parkinson’s to determine if the drug can slow the progression of the disease.