Treating Frontotemporal Dementia

SLU ID 19-027 | Compounds for Treating Progranulin-Deficient Frontotemporal Dementia

Background

Progranulin mutations cause frontotemporal dementia, which is a devastating disease with a mean survival of 3.8 years from diagnosis. Currently there is no cure available. Given that frontotemporal dementia is a disease of haploinsufficiency, strategies aimed at increasing progranulin levels are feasible therapeutic approaches to treating it. A number of academic labs and pharmaceutical companies are carrying out compound screens and genetic screens to identify ways to increase progranulin levels.

Overview

Researchers at Saint Louis University have developed antisense oligonucleotides (ASOs) that can increase levels of a protein called progranulin. Progranulin mutations (in one of the two alleles) lead to an approximately 50% decrease in progranulin protein level and cause frontotemporal dementia. The ASOs may be able to increase progranulin levels in people with progranulin mutations and thus provide a potential therapy for frontotemporal dementia, which currently has no cure.

Benefits

The potential advantages of this technology over existing solutions include:

• Minimizing the dosage required.
• Increasing treatment efficacy.
• Minimizing off-target effects.

Applications

Potential applications of this technology include:

• Treating frontotemporal dementia

Opportunity

Saint Louis University is seeking partners to further develop and commercialize this technology.