Alzheimer's disease detection and treatment based on ceramide and sphingolipid levels

This technology is a method to treat Alzheimer’s disease by reducing the level of the cellular biomarker APP-C99.

Unmet Need: Therapeutic tool for Alzheimer's disease detection and treatment

Currently, there is no cure for Alzheimer’s disease (AD) and few FDA-approved drugs available to alter disease progression. Since AD diagnosis involves obtaining a complete medical history often including expensive diagnostic scans, performing cognitive tests, and excluding other possible causes of dementia, there is already great potential for delays in treatment. Early diagnosis, effective tracking of disease progression, and finally, therapeutics to prevent and treat AD would lessen the burden of cognitive decline.

The Technology: Early Alzheimer’s disease biomarker for detection and treatment

This technology reduces a patient’s level of endoplasmic reticulum-mitochondrial-associated membrane (ER-MAM) localized APP-C99 in cells using a variety of different pharmacological approaches, including but not limited to γ-secretase activators. Patients with Alzheimer’s disease have heightened levels of APP-C99, a byproduct of the pathway that drives the buildup of β-amyloid (Aβ). This technology aims to treat AD by reducing the function and concentration of APP-C99 in cells.

This technology has been validated with human patient cell lines.

Applications:

• Method for the reduction of ER-MAM localized APP-C99
• Treatment for Alzheimer’s disease (AD)
• Tool for screening other putative therapeutics for treatment of AD
• Method for treating AD based on ratio of ceramide to sphingomyelin, ratio of cholesterol esters to free cholesterol, or ratio of C99 to total Aβ
• Method for early AD detection via ceramide or sphingomyelin detection
• Method for longitudinal AD progression tracking

Advantages:

• Early neurodegenerative disease treatment
• Method of determining a patient’s risk for Alzheimer’s disease (AD)
• Uses patients’ blood, skin biopsy, or urine samples for initial screening prior to treatment
• Uses relevant biomarkers to assess and treat AD

Lead Inventor:

Eric A. Schon, Ph.D.

Patent Information:

Patent Pending (US20220395486)

Patent Pending (EP3702470)

Patent Issued (WO/2019/173795)

Related Publications:

• Pera M, Larrea D, Guardia‐Laguarta C, Montesinos J, Velasco KR, Agrawal RR, Xu Y, Chan RB, Di Paolo G, Mehler MF, Perumal GS. “Increased localization of APP‐C99 in mitochondria‐associated ER membranes causes mitochondrial dysfunction in Alzheimer disease” EMBO J. 2017 Nov 15; 36(22): 3356-71.

Tech Ventures Reference:

• IR CU15147, 2312, 2312-a

• Licensing Contact: Kristin Neuman