Method for Identifying And/or for Obtaining an Active Substance for the Treatment and Therapy of Familial Amyotrophic Lateral Sclerosis, and Use of an Active Substance for the Treatment of This Disease

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent discloses a new method for identifying and/or developing active substances for treating familial Amyotrophic Lateral Sclerosis (ALS), particularly forms associated with SOD1 gene mutations. The invention describes a cellular screening platform using cells expressing the receptors syndecan-3 and/or syndecan-1, which mediate the uptake of toxic SOD1 protein aggregates linked to ALS progression. The patent also claims the use of pentosan polysulfate sodium (PPS) and heparin-like compounds as inhibitors of this toxic aggregate uptake, proposing them as potential drugs for ALS treatment.

Use CasesContent extracted from patent full text and abstract with AI.

• Screening and discovery of new drug candidates that can prevent SOD1 aggregate uptake in neurons for ALS therapy.
• Development of laboratory kits enabling pharmaceutical companies or research labs to search efficiently for ALS drug candidates targeting the syndecan-1/3 pathway.
• Therapeutic use of pentosan polysulfate sodium (PPS) or heparin-like compounds to inhibit the spread of toxic SOD1 aggregates between neurons in ALS patients.
• Potential adaptation of this screening platform for other neurodegenerative diseases involving aggregate-prone proteins.

BenefitsContent extracted from patent full text and abstract with AI.

• Provides a targeted, mechanistic approach to ALS drug discovery, focusing on a specific cellular uptake pathway (syndecan-1/-3 receptors).
• Enables efficient identification and validation of promising ALS therapeutic candidates in vitro before clinical testing.
• Offers promising repurposing of known substances (PPS, heparin, heparin-like compounds) that could accelerate the development of effective ALS treatments.
• May slow or halt the progression of ALS associated with SOD1 mutations by blocking neuron-to-neuron propagation of toxic protein aggregates.
• Can increase the scientific understanding of ALS disease mechanisms, potentially informing future therapies for related disorders.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The invention relates to a method for identifying and/or obtaining active substances for the alleviation or treatment of ALS, comprising the following steps: a. providing a composition containing cells having the cell surface receptors syndecan-3 and/or syndecan-1, b. contacting the composition from step a) with SOD1 aggregates, c. contacting the composition from step a) with the test substance to be screened, it being possible for step b) and step c) to be performed simultaneously or for step c) to be performed before step b), d. determining the uptake of the SOD1 aggregates into the cells. The invention moreover relates to a kit or a composition for conducting the method according to the invention, containing at least cells expressing the cell surface receptors syndecan-3 and/or syndecan-1 and also SOD1 aggregates. The invention further relates to pentosan polysulfate sodium (PPS) or compounds from the group of the heparins or heparin-like compounds, for use in the preparation of an inhibitor for the uptake of SOD1 aggregates in neurons or neuronal cells of an organism and as medicament in the treatment of ALS.