Treating Niemann-Pick Type C with a Special Sugar Mixture

This patent describes specific mixtures of modified beta-cyclodextrin molecules designed for direct delivery to the brain to treat the rare genetic disorder Niemann-Pick disease Type C.

What it covers

This patent focuses on specific mixtures of beta-cyclodextrin molecules, which are ring-shaped sugar molecules. These molecules are modified by adding 'hydroxypropyl' groups to one or more of their hydroxyl positions. The mixture can also include some unmodified beta-cyclodextrin. These special mixtures are intended to be used as an active ingredient in medicines. The patent also covers how to make these mixtures, how to test them to ensure they are safe for use, and how to use them in pharmaceutical compositions. Specifically, these compositions are designed for delivery directly into the spinal fluid (intrathecal) or brain ventricles (intracerebroventricular) to treat Niemann-Pick disease Type C.

What it doesn't cover

• —Does not cover beta-cyclodextrin mixtures that are not modified with hydroxypropyl groups, unless they are included as an optional component.
• —Does not cover using these compositions for diseases other than Niemann-Pick disease Type C.
• —Does not cover drug delivery methods other than intrathecal or intracerebroventricular administration.
• —Does not cover other types of cyclodextrin molecules, such as alpha-cyclodextrin or gamma-cyclodextrin.
• —Does not cover compositions that do not include at least some hydroxypropyl beta-cyclodextrin molecules.

The clever bit

The novelty lies in the specific composition of hydroxypropyl beta-cyclodextrin mixtures, carefully formulated and qualified for direct administration into the central nervous system, to effectively address the unique challenges of treating Niemann-Pick disease Type C.

Why it matters

Niemann-Pick disease Type C is a rare, severe genetic disorder that causes cholesterol to build up in cells, leading to progressive neurological damage and often early death. Current treatments are limited, and delivering drugs directly to the brain or spinal fluid is crucial for many neurological conditions. This patent aims to provide a specific, safe, and effective formulation for this challenging delivery route and disease.

Real-world examples

• 1.Pharmaceutical formulations for rare neurological diseases
• 2.Drugs delivered directly into the spinal fluid
• 3.Therapies for lysosomal storage disorders