This week, the German biotech company, MODAG, announced that their first drug candidate for neurodegenerative conditions has started Phase I safety testing in humans.
The drug, Anle138b, is being developed for Parkinson’s and it has previously shown to rescue neurons in pre-clinical models of alpha synuclein-associated Parkinson’s.
Anle138b targets a protein called alpha-synuclein which is abundant in the healthy human brain, concentrated near the tips of nerve cells. In the Parkinsonian brain, however, alpha-synuclein clumps together forming toxic masses called ‘Lewy bodies’ and these clumps are associated with neuron death. Furthermore, alpha-synuclein appears to exert deleterious effects on neighboring neurons by seeding the aggregation and possibly contributing to disease progression.
Pre-clinical studies of Anle138b have shown that it specifically binds to the toxic clumps of alpha-synuclein and prevents new clumps from forming thus potentially stopping the aggregation process advancing.
Anle138b is administered orally and efficiently passes the blood-brain-barrier directly acting on the toxic protein aggregates. If the results of this Phase I safety study are positive it would be interesting to see this drug evaluated in a cohort of people with Parkinson’s.
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