Combinations of small molecule as potential therapeutic agents for APBD
Awardee: Or Kakhlon
Institution: Research Fund of the Hadassah Medical Organization
Grant Amount: $50,000.00
Funding Period: February 1, 2024 - January 31, 2025
Summary:
This proposal intends to significantly improve already very promising compounds for obtaining a cure for the adult neurodegenerative disorder Adult Polyglucosan Body Disease which inflicts paralysis, loss of sensation, and lack of urination control on its victims. These compounds will be applied in special formulations which can stabilize them and increase their tissue penetration. Using this methodology and also compound combinations we anticipate that a cure for this devastating disorder will be within reach as a syrup or a pill. APBD, or Adult Polyglucosan Body Disease, is a neurological disorder inflicting paralysis, loss of sensation, and lack of urination control on its victims. The purposes of this proposal are (1) to further improve already promising compounds for the treatment of APBD and (2) to establish new markers for the disease. The compounds will be improved by applying them in combinations and in special formulations which can stabilize them and increase their tissue penetration. The new blood markers will be: (a) neurofilaments, which are proteins derived from fragmented dead neurons and thus should decrease if treatment is successful; (b) a set of molecules in the blood, called metabolites, which can recognize the severity of the disease and how effectively it can be improved by interventions, such as the mentioned compounds.