* Amylase-resistant fluorescence of Periodic Acid Schiff’s reagent (PAS) staining.
The selected compounds were further investigated in vitro and in vivo.
In an APBD mouse model (Gbeys/ys), our lead compound, GHF-201 (Compound A), was effective in lowering polyglucosan bodies, improving motility and , carbohydrate burn and lifespan extension.
Furthermore, our extensive cell and in vitro analyses show that GHF-201 targets the lysosomal protein LAMP1 and enhances autophagic flux as well as respiratory and glycolytic ATP production, transforming the adversely excessive glycogen into a useful fuel source.
First In Human – GHF-201 was approved for an Urgent Compassionate Use program in Israel limited to 3 APBD patients.
GHF partnered with LDS to formulate GHF-201 in a highly-bioavailable oral nanodroplet solution, to be administered daily.
Currently, there are 2 APBD patients in this program, under the care of Prof. Alexander Lossos and his team at Hadassah University Hospital-Ein Kerem, Jerusalem Israel. Data from clinical/laboratory tests and patient feedback is being collected by the medical team.
The growing knowledge and evidence about GHF-201 suggest that it may offers a new, safe, and efficacious approach to treat APBD and additional glycogen and lysosomal storage disorders, as well as diseases with greater prevalence.