20α-hydroxycholesterol

20α-hydroxycholesterol (also known as 20S-hydroxycholesterol or TT-20) is a naturally occurring, breast milk-derived oxysterol being developed by Tellus Therapeutics for the treatment of neonatal white matter injury (WMI) and diffuse white matter injury (DWMI) in preterm infants. It acts as an allosteric activator of the Hedgehog signaling pathway by binding directly to the Smoothened (SMO) receptor, which promotes the differentiation of oligodendrocyte progenitor cells (OPCs) and subsequent myelination. Additionally, 20α-hydroxycholesterol exhibits anti-inflammatory properties by inhibiting the NF-κB pathway, thereby suppressing the expression of pro-inflammatory cytokines such as IL-1β and IL-6. This dual mechanism of action makes it a promising therapeutic candidate for neuro-regenerative and anti-inflammatory indications, including neonatal brain injury and potentially adult demyelinating diseases like multiple sclerosis.

Other names

20(S)-hydroxycholesterol20(S)-OHC20HC20S-hydroxycholesterol20α-hydroxycholesterol5-cholestene-3β,20α-diolcholest-5-ene-3β,20-diol