Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study

NCT03724136 | Enrolling By Invitation DMC

• 1 other identifier: MDSC-ACIST
• Study Type: interventional
• Target: 100
• Locations: 2 countries
• Sites: 3

Brief Summary

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.

Conditions

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; Dementia, Multi-Infarct; Autism; Autism Spectrum Disorder; Autistic Behavior; Autistic Disorder, Current or Active State; Cadasil; LATE Limbic-predominant Age-related TDP-43 Encephalopathy

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (1)

• Activities of Daily Living

  1,3,6,and 12 months post treatment

Study Arms (3)

Arm 1

ACTIVE COMPARATOR

Intravenous Bone Marrow Stem Cell (BMSC) Fraction

Procedure: Intravenous Bone Marrow Stem Cell (BMSC) Fraction

Arm 2

ACTIVE COMPARATOR

Intravenous Bone Marrow Stem Cell (BMSC) Fraction combined with Near Infrared Light exposure .

Procedure: Intravenous Bone Marrow Stem Cell (BMSC) Fraction; Procedure: Near Infrared Light

Arm 3

ACTIVE COMPARATOR

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; Procedure: Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction

Interventions

Intravenous Bone Marrow Stem Cell (BMSC) Fraction PROCEDURE

14 cc of BMSC fraction separated from bone marrow aspirate and filtered with 150 micron filter and administered intravenously.

Arm 1; Arm 2; Arm 3

Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction PROCEDURE

Approximately 1 cc of BMSC fraction separated from bone marrow aspirate and administered to the nasal mucosa topically.

Arm 3

Near Infrared Light PROCEDURE

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.

Arm 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• MD Stem Cells: lead

Study Sites (3)

MD Stem Cells

Westport, Connecticut, 06880, United States

Location

MD Stem Cells

Coral Springs, Florida, 33065, United States

Location

Medcare Orthopaedics & Spine Hospital

Dubai, United Arab Emirates

Location

Related Publications (8)

• Danielyan L, Schafer R, von Ameln-Mayerhofer A, Buadze M, Geisler J, Klopfer T, Burkhardt U, Proksch B, Verleysdonk S, Ayturan M, Buniatian GH, Gleiter CH, Frey WH 2nd. Intranasal delivery of cells to the brain. Eur J Cell Biol. 2009 Jun;88(6):315-24. doi: 10.1016/j.ejcb.2009.02.001. Epub 2009 Mar 25.

  PMID: 19324456 BACKGROUND

• Duncan T, Valenzuela M. Alzheimer's disease, dementia, and stem cell therapy. Stem Cell Res Ther. 2017 May 12;8(1):111. doi: 10.1186/s13287-017-0567-5.

  PMID: 28494803 BACKGROUND

• Johnstone DM, Moro C, Stone J, Benabid AL, Mitrofanis J. Turning On Lights to Stop Neurodegeneration: The Potential of Near Infrared Light Therapy in Alzheimer's and Parkinson's Disease. Front Neurosci. 2016 Jan 11;9:500. doi: 10.3389/fnins.2015.00500. eCollection 2015.

  PMID: 26793049 BACKGROUND

• Robbins JP, Price J. Human induced pluripotent stem cells as a research tool in Alzheimer's disease. Psychol Med. 2017 Nov;47(15):2587-2592. doi: 10.1017/S0033291717002124. Epub 2017 Aug 14.

  PMID: 28805182 BACKGROUND

• Park SE, Lee NK, Na DL, Chang JW. Optimal mesenchymal stem cell delivery routes to enhance neurogenesis for the treatment of Alzheimer's disease: optimal MSCs delivery routes for the treatment of AD. Histol Histopathol. 2018 Jun;33(6):533-541. doi: 10.14670/HH-11-950. Epub 2017 Nov 29.

  PMID: 29185257 BACKGROUND

• Shroff G. Human Embryonic Stem Cells in the Treatment of Autism: A Case Series. Innov Clin Neurosci. 2017 Apr 1;14(3-4):12-16. eCollection 2017 Mar-Apr.

  PMID: 28584692 BACKGROUND

• Chez M, Lepage C, Parise C, Dang-Chu A, Hankins A, Carroll M. Safety and Observations from a Placebo-Controlled, Crossover Study to Assess Use of Autologous Umbilical Cord Blood Stem Cells to Improve Symptoms in Children with Autism. Stem Cells Transl Med. 2018 Apr;7(4):333-341. doi: 10.1002/sctm.17-0042. Epub 2018 Feb 6.

  PMID: 29405603 BACKGROUND

• Weiss JN, Levy S. Neurologic Stem Cell Treatment Study (NEST) using bone marrow derived stem cells for the treatment of neurological disorders and injuries: study protocol for a nonrandomized efficacy trial. Clin Trials Degener Dis. 2016 [cited 2019 Jun 18];1:176-80.

  BACKGROUND

Related Links

• Report from CNN on Duke University stem cell autism study

MeSH Terms

Conditions

Alzheimer Disease; Dementia, Vascular; Lewy Body Disease; Mixed Dementias; Progressive supranuclear palsy atypical; Chronic Traumatic Encephalopathy; Korsakoff Syndrome; Brain Injuries, Traumatic; Dementia, Multi-Infarct; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; CADASIL; limbic-predominant age-related TDP-43 encephalopathy; Dementia; Asperger Syndrome

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Cerebrovascular Disorders; Intracranial Arteriosclerosis; Intracranial Arterial Diseases; Leukoencephalopathies; Arteriosclerosis; Arterial Occlusive Diseases; Vascular Diseases; Cardiovascular Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Brain Injuries; Brain Injury, Chronic; Craniocerebral Trauma; Trauma, Nervous System; Brain Damage, Chronic; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Wounds and Injuries; Memory Disorders; Neurobehavioral Manifestations; Neurologic Manifestations; Signs and Symptoms; Cerebral Infarction; Brain Infarction; Brain Ischemia; Stroke; Infarction; Ischemia; Necrosis; Neurodevelopmental Disorders; Cerebral Small Vessel Diseases; Cerebral Arterial Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Steven Levy, MD

  MD Stem Cells

  STUDY CHAIR

• Jeffrey Weiss, MD

  Coral Springs

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Patients will be assigned to one of 3 arms with monitoring and retesting at 1,3,6 and 12 months following treatment.

Study Record Dates

• First Submitted: October 25, 2018
• First Posted: October 30, 2018
• Study Start: October 24, 2018
• Primary Completion: July 31, 2025
• Study Completion: July 31, 2025
• Last Updated: April 15, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); AE (1)