Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study

Study Overview
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Brief Summary
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The purpose of the study is to evaluate the use of autologous Bone Marrow Derived Stem Cells (BMSC) as a means to improve cognitive impairment as occurs in Alzheimer's Disease and other dementias and to improve behavior and socialization issues which occur in adult Autism Spectrum Disorder. The use of Near Infrared Light, in conjunction with the use of BMSC, will also be assessed.

Detailed Description
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Cognition is the process of generating thoughts, recalling memories, processing information, and higher order associations including social interactivity that all take place in the brain. It requires sufficient health and interactivity of neurons in the brain including their ability to form and maintain synaptic connections. Cognitive impairment results from the loss of these abilities. ACIST will test the hypothesis that the delivery of Bone Marrow Derived Stem Cells (BMSC) via the methods in the study with or without the addition of Near Infrared Light will improve cognition through the ability of BMSC to positively affect the health and function of neurons and the brain. Patients enrolling with cognitive impairment will require assessment with the Mini-Mental Status Exam (MMSE). A score of 24 or less will be required. Progressive dementias such as Alzheimer's Disease (ALZ) show a decline of 2 to 4 points per year on MMSE. The goal for ACIST in progressive and stable dementia will be stability over the 1 year follow up and ideally an improvement of 3 points on MMSE. Patients enrolling with Autism Spectrum Disorder (ASD) will be required to be adults (over 18 years of age) and to have a score on the Autism Spectrum Quotient of 20 or above. The goal will be a decrease of 5 or more on the scale over the 1 year follow up period.

Official Title
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Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study

Conditions
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Dementia, Multi-Infarct, Alzheimer Disease, Alzheimer Dementia, Vascular Dementia, Lewy Body Disease, Lewy Body Dementia With Behavioral Disturbance (Disorder), Dementia, Mixed, Parkinson-Dementia Syndrome, Chronic Traumatic Encephalopathy, Huntington's Dementia, Wernicke Korsakoff Syndrome, Traumatic Brain Injury, Autism, Autism Spectrum Disorder, Autistic Behavior, Autistic Disorder, Current or Active State, Cadasil, LATE Limbic-predominant Age-related TDP-43 Encephalopathy

Intervention / Treatment
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* Procedure: Intravenous Bone Marrow Stem Cell (BMSC) Fraction
* Procedure: Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction
* Procedure: Near Infrared Light


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have documented cognitive impairment or diagnosis of disease associated with cognitive impairment such as Alzheimer's Disease, Autism Spectrum Disorder. If under current medical therapy (pharmacologic or surgical treatment) for the condition be considered stable on that treatment and unlikely to have reversal of the associated cognitive impairment as a result of the ongoing pharmacologic or surgical treatment. In the estimation of the investigator have the potential for improvement with BMSC treatment and be at minimal risk of any potential harm from the procedure. Be over the age of 18 Be medically stable and able to be medically cleared by their primary care physician or a licensed primary care practitioner for the procedure. Medical clearance means that in the estimation of the primary care practitioner, the patient can reasonably be expected to undergo the procedure without significant medical risk to health. Exclusion Criteria: All patients must be capable of an adequate neurologic examination and evaluation to document the pathology. Patients must be capable and willing to undergo follow up neurologic exams with the the investigators or their own neurologists as outlined in the protocol. Patients or their designated responsible party for medical decisions must be capable of providing informed consent. Cognitive or memory impairment does not necessarily mean the patient is incapable of giving informed consent. They may simply need more time to process or repetition of the content of the consent to reach understanding and provide informed consent. In the estimation of the investigator the BMSC collection and treatment will not present a significant risk of harm to the patient's general health or to their neurologic function. Patients who are not medically stable or who may be at significant risk to their health undergoing the procedure will not be eligible. Women of childbearing age must not be pregnant at the time of treatment and should refrain from becoming pregnant for 3 months post treatment.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Patients will be assigned to one of 3 arms with monitoring and retesting at 1,3,6 and 12 months following treatment.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Arm 1<br>Intravenous Bone Marrow Stem Cell (BMSC) Fraction | Procedure: Intravenous Bone Marrow Stem Cell (BMSC) Fraction<br>* 14 cc of BMSC fraction separated from bone marrow aspirate and filtered with 150 micron filter and administered intravenously.<br>|
| Active Comparator: Arm 2<br>Intravenous Bone Marrow Stem Cell (BMSC) Fraction combined with Near Infrared Light exposure . | Procedure: Intravenous Bone Marrow Stem Cell (BMSC) Fraction<br>* 14 cc of BMSC fraction separated from bone marrow aspirate and filtered with 150 micron filter and administered intravenously.<br>Procedure: Near Infrared Light<br>* Near Infrared Light will be administered using an FDA cleared medical device on the preoperative day and the first postoperative day as tolerated to the general area of the frontal bone.<br>|
| Active Comparator: Arm 3<br>Intravenous Bone Marrow Stem Cell (BMSC) Fraction combined with Intranasal topical Bone Marrow Stem Cell (BMSC) Fraction. | Procedure: Intravenous Bone Marrow Stem Cell (BMSC) Fraction<br>* 14 cc of BMSC fraction separated from bone marrow aspirate and filtered with 150 micron filter and administered intravenously.<br>Procedure: Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction<br>* Approximately 1 cc of BMSC fraction separated from bone marrow aspirate and administered to the nasal mucosa topically.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mini-Mental Status Exam (MMSE) | In cognitive impairment patients this standard cognitive function test will be administered. The change from pretreatment baseline to each time point post treatment will be assessed. | 1,3,6 and 12 months post treatment. | 
| Autism Spectrum Quotient Exam | Patients with Autism Spectrum Disorder (ASD) or Autism will undergo testing with the Autism Spectrum Quotient (AQ) Exam, an autism assessment for adults. The change in scoring from pretreatment baseline to each time point post treatment will be assessed. | 1,3,6 and 12 months post treatment. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Activities of Daily Living | The patients, friends and family will describe changes in abilities to function on a day to day basis. | 1,3,6,and 12 months post treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Dementia, Alzheimer's Disease, CADASIL, Autism Spectrum Disorder, Adult Autism, High Functioning Autism, Asperger's Syndrome, CTE Chronic Traumatic Encephalopathy, TBI Traumatic Brain Injury, LATE Limbic-predominant Age-related TDP-43 Encephalopathy