Claims for Patent: 12,144,792

« Back to Dashboard

Summary for Patent: 12,144,792

Title:	Therapeutic agents for neurodegenerative diseases
Abstract:	The present disclosure provides for treating neurodegenerative diseases comprising administering acetyl-leucine or a pharmaceutically acceptable salt thereof.
Inventor(s):	Michael Strupp
Assignee:	Intrabio Ltd
Application Number:	US16/324,353
Patent Claims:	1. A method of treating a neurodegenerative disease or one or more symptoms associated with a neurodegenerative disease in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration of at least 3 months, wherein the neurodegenerative disease is not cerebellar ataxia or Niemann-Pick Type C, wherein the neurodegenerative disease is chosen from Alzheimer's disease, amyotrophic lateral sclerosis (ALS), multiple system atrophy type P (MSA-P), multiple system atrophy type C (MSA-C), frontotemporal dementia with parkinsonism, progressive supranuclear palsy, corticobasal degeneration, Lewy Body dementia, Parkinson's Disease, and ataxia telangiectasia (Louis Barr disease), and wherein the therapeutically effective amount of acetyl-leucine or pharmaceutically acceptable salt thereof is administered at least two times a day to achieve a total daily dose of from about 500 mg to about 15 g. 2. The method according to claim 1, wherein the neurodegenerative disease is associated with lysosomal dysfunction. 3. The method according to claim 2, wherein the neurodegenerative disease associated with lysosomal dysfunction is chosen from ALS, MSA-P, MSA-C, frontotemporal dementia with parkinsonism, progressive supranuclear palsy, Alzheimer's disease, and Louis Barr disease. 4. The method according to claim 1, wherein the neurodegenerative disease is chosen from progressive supranuclear palsy, Parkinson's Disease, and Louis Barr disease. 5. The method according to claim 1, wherein the subject has been found to have a genetic and/or biochemical marker of the neurodegenerative disease. 6. The method of claim 1, wherein treating is delaying onset of the neurodegenerative disease or one or more symptoms of a neurodegenerative disease that would otherwise be expected to manifest according to typical disease progression in the subject. 7. A method of treating a neurodegenerative disease or one or more symptoms associated with a neurodegenerative disease in a subject in need thereof comprising: administering a therapeutically effective amount of the acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years, wherein the neurodegenerative disease is not cerebellar ataxia or Niemann-Pick Type C, and wherein the neurodegenerative disease is chosen from Alzheimer's disease, amyotrophic lateral sclerosis (ALS), multiple system atrophy type P (MSA-P), multiple system atrophy type C (MSA-C), frontotemporal dementia with parkinsonism, progressive supranuclear palsy, corticobasal degeneration, Lewy Body dementia, Parkinson's Disease, and ataxia telangiectasia (Louis Barr disease). 8. A method of delaying progression of a neurodegenerative disease or one or more symptoms associated with a neurodegenerative disease in a subject in need thereof over time as compared to typical disease progression comprising: administering a therapeutically effective amount of the acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years, wherein the neurodegenerative disease is not cerebellar ataxia or Niemann-Pick Type C, and wherein the neurodegenerative disease is chosen from Alzheimer's disease, amyotrophic lateral sclerosis (ALS), multiple system atrophy type P (MSA-P), multiple system atrophy type C (MSA-C), frontotemporal dementia with parkinsonism, progressive supranuclear palsy, corticobasal degeneration, Lewy Body dementia, Parkinson's Disease, and ataxia telangiectasia (Louis Barr disease). 9. A method of reversing progression of a neurodegenerative disease or one or more symptoms associated with a neurodegenerative disease over time in a subject in need thereof comprising: administering a therapeutically effective amount of the acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years, wherein the neurodegenerative disease is not cerebellar ataxia or Niemann-Pick Type C, and wherein the neurodegenerative disease is chosen from Alzheimer's disease, amyotrophic lateral sclerosis (ALS), multiple system atrophy type P (MSA-P), multiple system atrophy type C (MSA-C), frontotemporal dementia with parkinsonism, progressive supranuclear palsy, corticobasal degeneration, Lewy Body dementia, Parkinson's Disease, and ataxia telangiectasia (Louis Barr disease). 10. The method according to claim 1, wherein the method comprises initiating administration of a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject in need thereof when the subject is asymptomatic. 11. The method according to claim 10, wherein the initial administration occurs after the subject has been found to have a genetic and/or biochemical marker of the neurodegenerative disease. 12. The method according to claim 1, wherein the method comprises administering a therapeutically effective amount of the acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years. 13. The method according to claim 1, wherein the acetyl-leucine is acetyl-DL-leucine. 14. The method according to claim 1, wherein the acetyl-leucine has an enantiomeric excess of the L-enantiomer or the D-enantiomer. 15. The method according to claim 1, wherein the therapeutically effective amount of acetyl-leucine or pharmaceutically acceptable salt thereof ranges from about 1 g to about 15 g per day, from about 1 g to about 10 g per day, from about 1.5 g to about 7 g per day, from about 4 g to about 6 g per day, or from about 4 g to about 5 g per day. 16. A method of reducing the severity of a neurodegenerative disease or reducing the severity of or eliminating one or more existing symptoms associated with a neurodegenerative disease in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration of at least 3 months, wherein the neurodegenerative disease is not cerebellar ataxia or Niemann-Pick Type C, wherein the neurodegenerative disease is chosen from Alzheimer's disease, amyotrophic lateral sclerosis (ALS), multiple system atrophy type P (MSA-P), multiple system atrophy type C (MSA-C), frontotemporal dementia with parkinsonism, progressive supranuclear palsy, corticobasal degeneration, Lewy Body dementia, Parkinson's Disease, and ataxia telangiectasia (Louis Barr disease). 17. A method of providing neuroprotection in a subject having, suspected of having, or at risk of having a neurodegenerative disease comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years, wherein the neurodegenerative disease is not cerebellar ataxia or Niemann-Pick Type C, and wherein the neurodegenerative disease is chosen from Alzheimer's disease, amyotrophic lateral sclerosis (ALS), multiple system atrophy type P (MSA-P), multiple system atrophy type C (MSA-C), frontotemporal dementia with parkinsonism, progressive supranuclear palsy, corticobasal degeneration, Lewy Body dementia, Parkinson's Disease, and ataxia telangiectasia (Louis Barr disease). 18. The method according to claim 1, wherein the acetyl-leucine is acetyl-L-leucine.