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COMPOSITION AND METHOD FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES USING SMALL MOLECULE MODULATORS
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ORDINARY APPLICATION
Published
Filed on 28 October 2024
Abstract
COMPOSITION AND METHOD FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES USING SMALL MOLECULE MODULATORS ABSTRACT This invention relates to a composition and method for treating neurodegenerative diseases using small molecule modulators. The composition targets key cellular mechanisms involved in neurodegeneration, including protein aggregation, oxidative stress, and neuroinflammation. The method involves administering the composition to patients diagnosed with neurodegenerative conditions, such as Alzheimer's disease, Parkinson's disease, or ALS, with the aim of slowing disease progression and improving neuronal survival. The small molecule modulators inhibit toxic protein aggregation, reduce oxidative stress, and modulate neuroinflammatory responses, offering a disease-modifying approach to treatment.
Patent Information
Application ID | 202441082127 |
Invention Field | BIO-CHEMISTRY |
Date of Application | 28/10/2024 |
Publication Number | 45/2024 |
Inventors
Name | Address | Country | Nationality |
---|---|---|---|
Dr. Kiranmai Mandava | Professor & Principal St. Pauls Collge of Pharmacy, Sy. No. 603 , 604 & 605 Turkayamjal (V), Abdullapurmet (M), R.R. Dist. - 501510, Telangana, India. | India | India |
Dr. Sneha Thakur | Associate Professor St. Pauls Collge of Pharmacy, Sy. No. 603 , 604 & 605 Turkayamjal (V), Abdullapurmet (M), R.R. Dist. - 501510, Telangana, India. | India | India |
Dr. N. Sarithadevi | Associate Professor St. Pauls Collge of Pharmacy, Sy. No. 603 , 604 & 605 Turkayamjal (V), Abdullapurmet (M), R.R. Dist. - 501510, Telangana, India. | India | India |
Applicants
Name | Address | Country | Nationality |
---|---|---|---|
St. Pauls College of Pharmacy | TURKAYAMJAL, NAGARJUNA SAGAR ROAD, HYDERABAD, TELANGANA 501510 | India | India |
Dr. Sneha Thakur | Associate Professor ST. PAULS COLLEGE OF PHARMACY, TURKAYAMJAL, NAGARJUNA SAGAR ROAD, HYDERABAD, TELANGANA 501510 | India | India |
Specification
Description:COMPOSITION AND METHOD FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES USING SMALL MOLECULE MODULATORS
FIELD OF THE INVENTION
The present invention relates to the field of pharmaceutical compositions and therapeutic methods aimed at treating neurodegenerative diseases. Specifically, it focuses on the use of small molecule modulators to target key pathways associated with the progression of neurodegenerative conditions, including Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS). The invention pertains to compositions that modulate cellular mechanisms such as protein aggregation, neuroinflammation, and oxidative stress to offer potential therapeutic benefits.
BACKGROUND OF THE INVENTION
Neurodegenerative diseases are a class of disorders characterized by progressive degeneration of neurons in the central and peripheral nervous systems. These conditions, including Alzheimer's disease, Parkinson's disease, and ALS, affect millions worldwide, and their incidence is increasing with the aging population. Currently available treatments are largely symptomatic and fail to address the underlying causes of neurodegeneration, leaving patients with limited options for disease modification.
Recent research has identified several molecular pathways involved in neurodegeneration, such as abnormal protein aggregation, mitochondrial dysfunction, oxidative stress, and chronic neuroinflammation. These pathways contribute to neuronal death and the progressive nature of the disease. Small molecule modulators that can target these pathways offer a promising therapeutic approach. Such modulators have the potential to alter disease progression by restoring cellular homeostasis and reducing the accumulation of toxic proteins.
However, despite significant advances in understanding the mechanisms behind neurodegeneration, there remains an unmet need for therapies that can effectively halt or reverse disease progression. The present invention seeks to address this gap by providing compositions and methods using small molecule modulators aimed at treating neurodegenerative diseases at their core.
SUMMARY OF THE INVENTION
In an embodiment, the present invention provides a composition comprising small molecule modulators specifically designed to target key pathways involved in neurodegenerative diseases. These small molecules act on cellular mechanisms such as protein misfolding, oxidative stress, and neuroinflammation to reduce the neurodegenerative effects and improve neuronal survival.
The method of treatment involves administering the composition to a subject suffering from a neurodegenerative disease. The composition includes one or more small molecule modulators that inhibit protein aggregation, enhance mitochondrial function, and suppress neuroinflammation. The invention aims to slow or halt disease progression by reducing the burden of toxic protein species, lowering oxidative damage, and decreasing inflammation in the central nervous system.
In an embodiment, the composition can be administered orally, intravenously, or through intranasal routes, depending on the clinical requirements. The composition is suitable for use in both early and advanced stages of neurodegenerative diseases and can be tailored to address specific disease phenotypes by adjusting the type and dosage of small molecule modulators. The small molecules are designed to cross the blood-brain barrier, ensuring direct therapeutic action on neurons.
BRIEF DESCRIPTION OF THE FIGURES
The accompanying figures where like reference numerals refer to identical or functionally similar elements throughout the separate views and which together with the detailed description below are incorporated in and form part of the specification, serve to further illustrate various embodiments and to explain various principles and advantages all in accordance with the invention.
The figure illustrates a schematic representation of the method for treating neurodegenerative diseases using small molecule modulators. The diagram outlines the key steps of the method, including:
1. Identification of neurodegenerative disease in the subject.
2. Selection of the appropriate small molecule modulator(s) based on the disease phenotype.
3. Administration of the modulator(s) via oral, intravenous, or intranasal routes.
4. Modulation of cellular pathways, including protein aggregation, oxidative stress, and neuroinflammation.
5. Improvement in neuronal survival and reduction in disease progression.
DETAILED DESCRIPTION OF THE INVENTION
While various embodiments of the invention have been shown and described herein, it will be obvious to those skilled in the art that such embodiments are provided by way of example only. Numerous variations, changes, and substitutions may occur to those skilled in the art without departing from the invention. It should be understood that various alternatives to the embodiments of the invention described herein may be employed. It shall be understood that different aspects of the invention can be appreciated individually, collectively, or in combination with each other.
The invention is a composition that contains small molecule modulators capable of modulating neurodegenerative pathways at the molecular level. These small molecules have been carefully selected based on their ability to affect protein misfolding, mitochondrial dysfunction, and neuroinflammation, all of which are critical drivers of neurodegeneration.
In an embodiment, the small molecule modulators include inhibitors of amyloid-beta and alpha-synuclein aggregation. These inhibitors prevent the formation of toxic oligomers and fibrils, which are associated with Alzheimer's and Parkinson's diseases. By reducing the buildup of these aggregates, the modulators help maintain normal cellular function and promote neuronal survival.
Another aspect of the invention involves small molecules that act as antioxidants, targeting mitochondrial dysfunction and reducing oxidative stress. Mitochondria are essential for neuronal energy production, and their dysfunction is a key factor in the progression of neurodegenerative diseases. The small molecules used in the invention are designed to enhance mitochondrial function and reduce the generation of reactive oxygen species (ROS), thereby protecting neurons from oxidative damage.
Additionally, the invention includes small molecule modulators that target neuroinflammation by inhibiting pro-inflammatory cytokines such as TNF-alpha and IL-6. Chronic neuroinflammation contributes to neuronal damage in neurodegenerative diseases, and modulating this response can reduce inflammation and protect neurons.
In an embodiment, the composition is formulated in a pharmaceutically acceptable carrier that allows for effective delivery of the small molecule modulators to the central nervous system. The formulation may include liposomes, nanoparticles, or other delivery vehicles that enhance the bioavailability of the small molecules and facilitate their crossing of the blood-brain barrier.
The method of treatment involves administering the composition to a patient diagnosed with a neurodegenerative disease. The dosage and frequency of administration will depend on the disease stage and the specific small molecules used. Treatment may be initiated at the early stages of the disease or after diagnosis of advanced neurodegeneration, with the goal of slowing disease progression and improving quality of life.
In an embodiment, the treatment is tailored to the patient's specific disease phenotype by adjusting the types and concentrations of small molecule modulators. For example, in Alzheimer's disease, the focus may be on inhibiting amyloid-beta aggregation, while in Parkinson's disease, the treatment may target alpha-synuclein aggregates and mitochondrial dysfunction.
The composition may be used as a monotherapy or in combination with other therapies, such as anti-inflammatory drugs or symptomatic treatments. The invention aims to provide a disease-modifying treatment that addresses the root causes of neurodegeneration, offering patients a more effective approach to managing their condition.
6. Advantages of the Invention
1. Targeted Modulation: The invention offers a targeted approach to treating neurodegenerative diseases by addressing key molecular pathways such as protein aggregation, oxidative stress, and neuroinflammation.
2. Disease Modification: Unlike existing treatments that focus on symptom management, the composition aims to slow or halt disease progression by modulating underlying disease mechanisms.
3. Broad Applicability: The small molecule modulators can be tailored to treat various neurodegenerative diseases, including Alzheimer's, Parkinson's, and ALS.
4. Improved Neuronal Survival: The composition enhances neuronal survival by preventing toxic protein aggregation, reducing oxidative stress, and modulating neuroinflammation.
5. Versatile Administration: The composition can be administered through multiple routes, including oral, intravenous, and intranasal, offering flexibility in clinical application.
, Claims:I/WE CLAIM:
Claim 1
A method for treating neurodegenerative diseases, comprising administering to a subject in need thereof a composition comprising at least one small molecule modulator, wherein the small molecule modulator inhibits protein aggregation, reduces oxidative stress, and modulates neuroinflammation to improve neuronal survival and reduce disease progression.
Claim 2
The method of claim 1, wherein the neurodegenerative disease is Alzheimer's disease, Parkinson's disease, or amyotrophic lateral sclerosis (ALS).
Claim 3
The method of claim 1, wherein the small molecule modulator inhibits amyloid-beta aggregation.
Claim 4
The method of claim 1, wherein the small molecule modulator inhibits alpha-synuclein aggregation.
Claim 5
The method of claim 1, wherein the small molecule modulator reduces oxidative stress by enhancing mitochondrial function.
Claim 6
The method of claim 1, wherein the small molecule modulator inhibits pro-inflammatory cytokines, including TNF-alpha and IL-6.
Claim 7
The method of claim 1, wherein the composition is administered orally, intravenously, or intranasally.
Claim 8
The method of claim 1, wherein the composition is formulated with a pharmaceutically acceptable carrier to enhance delivery to the central nervous system.
Claim 9
The method of claim 1, wherein the treatment is initiated at the early stages of the neurodegenerative disease.
Documents
Name | Date |
---|---|
202441082127-FORM-5 [05-11-2024(online)].pdf | 05/11/2024 |
202441082127-COMPLETE SPECIFICATION [28-10-2024(online)].pdf | 28/10/2024 |
202441082127-DRAWINGS [28-10-2024(online)].pdf | 28/10/2024 |
202441082127-EDUCATIONAL INSTITUTION(S) [28-10-2024(online)].pdf | 28/10/2024 |
202441082127-EVIDENCE FOR REGISTRATION UNDER SSI(FORM-28) [28-10-2024(online)].pdf | 28/10/2024 |
202441082127-FORM 1 [28-10-2024(online)].pdf | 28/10/2024 |
202441082127-FORM FOR SMALL ENTITY(FORM-28) [28-10-2024(online)].pdf | 28/10/2024 |
202441082127-FORM-9 [28-10-2024(online)].pdf | 28/10/2024 |
202441082127-POWER OF AUTHORITY [28-10-2024(online)].pdf | 28/10/2024 |
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