Methods for treatment of autism spectrum disorder
Inventors
Tierney, Elaine • Porter, Forbes D.
Assignees
Johns Hopkins University • National Institutes of Health NIH • Kennedy Krieger Institute Inc • US Department of Health and Human Services
Publication Number
US-8987246-B2
Publication Date
2015-03-24
Expiration Date
2033-07-18
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Abstract
The present invention relates to the field of autism. More specifically, the present invention provides methods for treating individuals with autism spectrum disorder. Accordingly, in one aspect, the present invention provides methods for treating patients with autism spectrum disorder. In one embodiment, a method for treating an autism spectrum disorder (ASD) in a patient comprises the step of administering a therapeutically effective amount of cholesterol to the patient. In more specific embodiments, the ASD is autism, Asperger's disorder, pervasive developmental disorder-not otherwise specified (PDD-NOS), Rett's syndrome and childhood disintegrative disorder. In one embodiment, the patient has autism.
Core Innovation
The present invention provides methods for treating individuals with autism spectrum disorder (ASD) by administering a therapeutically effective amount of cholesterol. The invention encompasses treatment of patients with various forms of ASD, including autism, Asperger's disorder, pervasive developmental disorder-not otherwise specified (PDD-NOS), Rett's syndrome, and childhood disintegrative disorder. Cholesterol supplementation is proposed as a therapeutic approach based on the discovery that a subgroup of children with ASD exhibits low cholesterol levels.
The problem being addressed is the need for treatments for autistic individuals, as ASD comprises a broad group of neurodevelopmental disorders characterized by impairments in social and communication interactions, repetitive behaviors, and restricted interests. The background highlights that these disorders affect as many as 1 in 88 children in the United States, and current treatments are insufficient.
The invention is based on the hypothesis that cholesterol and other sterol abnormalities may contribute to the cause of certain ASD subtypes and that cholesterol supplementation may improve behavioral and communicative functions. A clinical trial is described to determine the frequency of low cholesterol in ASD patients and to test the efficacy of cholesterol supplementation in improving core symptoms of ASD in those with low cholesterol. Methods for administering cholesterol to ASD patients without Smith-Lemli-Opitz syndrome (SLOS) are also disclosed, with therapeutically effective amounts ranging from about 20 to about 300 mg/kg body weight per day.
Claims Coverage
The patent includes 14 claims featuring methods for treating ASD patients through cholesterol administration. Two independent claims focus on treating ASD patients without SLOS by administering therapeutically effective cholesterol amounts.
Treatment of ASD patients lacking SLOS with cholesterol
A method for treating a patient having autism spectrum disorder but not Smith-Lemli-Opitz syndrome by administering a therapeutically effective amount of cholesterol to the patient.
Treatment of ASD patients without SLOS and with normal cholesterol levels
A method for treating a patient having ASD with normal cholesterol levels and who does not have SLOS by administering a therapeutically effective amount of cholesterol to the patient.
Treatment of ASD patients without SLOS and with low cholesterol levels
A method for treating a patient having ASD with low cholesterol levels and who does not have SLOS by administering a therapeutically effective amount of cholesterol to the patient.
The independent claims consistently disclose methods of administering therapeutically effective amounts of cholesterol to patients with ASD who do not have SLOS, covering patients with both normal and low cholesterol levels. The dosage range is specified as about 20 to about 300 mg/kg body weight/day, and specific ASD subtypes are included within the treatment methods.
Stated Advantages
Cholesterol supplementation improves social and communication abilities in individuals with ASD, especially in subtypes associated with low cholesterol.
The treatment approach provides essential evidence to evaluate cholesterol supplementation as a potential therapy for the core symptoms of ASD in youths.
Cholesterol is essential to multiple biological functions relevant to brain development and neurotransmission, making supplementation a plausible therapeutic strategy.
Documented Applications
Treatment of autism spectrum disorder including autism, Asperger's disorder, pervasive developmental disorder-not otherwise specified, Rett's syndrome, and childhood disintegrative disorder by administering cholesterol.
Clinical trial evaluation of cholesterol supplementation in children with ASD and hypocholesterolemia to assess improvement in behavioral and communicative functions.
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