NYSCF Scientific Discovery Biobank (NCT06203106)

Overview

The New York Stem Cell Foundation (NYSCF) Scientific Discovery Biobank (NCT06203106) is a large-scale observational repository established to collect and characterize biospecimens from patients with neurodegenerative diseases. This biobank represents one of the most comprehensive stem cell resources for neurodegenerative disease research globally, enabling researchers worldwide to access patient-derived induced pluripotent stem cell (iPSC) lines for disease modeling, drug screening, and mechanistic studies.

Study Details

• NCT Number: NCT06203106
• Status: Recruiting
• Study Type: Observational/Biobank
• Conditions: Progressive Supranuclear Galsy (PSP), Parkinson's Disease (PD), Alzheimer's Disease (AD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), Corticobasal Syndrome (CBS), Huntington's Disease (HD)
• Sponsor: The New York Stem Cell Foundation
• Sites: New York, NY and collaborating academic medical centers
• Enrollment Target: Not specified (ongoing recruitment)

Background and Rationale

Overview

The New York Stem Cell Foundation (NYSCF) Scientific Discovery Biobank (NCT06203106) is a large-scale observational repository established to collect and characterize biospecimens from patients with neurodegenerative diseases. This biobank represents one of the most comprehensive stem cell resources for neurodegenerative disease research globally, enabling researchers worldwide to access patient-derived induced pluripotent stem cell (iPSC) lines for disease modeling, drug screening, and mechanistic studies.

Study Details

• NCT Number: NCT06203106
• Status: Recruiting
• Study Type: Observational/Biobank
• Conditions: Progressive Supranuclear Galsy (PSP), Parkinson's Disease (PD), Alzheimer's Disease (AD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), Corticobasal Syndrome (CBS), Huntington's Disease (HD)
• Sponsor: The New York Stem Cell Foundation
• Sites: New York, NY and collaborating academic medical centers
• Enrollment Target: Not specified (ongoing recruitment)

Background and Rationale

Neurodegenerative diseases including Alzheimer's disease, Parkinson's disease, PSP, ALS, and others share common pathophysiological features including protein aggregation, mitochondrial dysfunction, neuroinflammation, and progressive neuronal loss. However, the mechanistic basis for these diseases remains incompletely understood, partly due to the limited availability of relevant human cellular models.

Patient-derived iPSCs offer a transformative approach to neurodegenerative disease research by enabling researchers to:

The NYSCF biobank addresses the critical need for standardized, well-characterized iPSC lines from patients with diverse neurodegenerative conditions. By providing access to these valuable resources, the NYSCF accelerates the pace of discovery research and facilitates the development of novel therapeutic interventions.

Objectives

Primary Objectives

Secondary Objectives

Sample Collection and Processing

Biospecimen Types

| Specimen Type | Purpose | Processing |
|---------------|---------|------------|
| Peripheral Blood Mononuclear Cells (PBMCs) | Reprogramming to iPSCs | Ficoll density gradient separation |
| Plasma | Biomarker studies | Centrifugation and aliquoting |
| Serum | Protein biomarker analysis | Clotting and centrifugation |
| DNA | Genetic analysis | Extracted from PBMCs |
| RNA | Gene expression studies | Stored in RNA preservation buffers |

Reprogramming Protocol

The NYSCF employs a non-integrative reprogramming methodology to generate iPSC lines. This approach utilizes:

• Sendai virus vectors encoding OCT4, SOX2, KLF4, and c-MYC (the Yamanaka factors)
• Feeder-free culture conditions on defined matrix substrates
• Automated quality control including sterility testing, mycoplasma screening, and pluripotency verification

• Normal karyotype (absence of chromosomal abnormalities)
• Pluripotency marker expression (TRA-1-60, TRA-1-81, SSEA-4, OCT4)
• Ability to differentiate into all three germ layers (ectoderm, mesoderm, endoderm)

Research Applications

Disease Modeling

The iPSC lines in the NYSCF biobank have been used to create disease models for multiple neurodegenerative conditions:

Parkinson's Disease Models:

• iPSC-derived dopaminergic neurons from PD patients have revealed disease-specific phenotypes including:
• Reduced neurite outgrowth and branching
• Increased susceptibility to oxidative stress
• Mitochondrial dysfunction and reduced ATP production
• Alpha-synuclein accumulation and aggregation
• Studies comparing monozygotic twins discordant for PD have identified epigenetic and gene expression differences that may underlie disease susceptibility[@nyscf_ipsc_dopamine]

• NYSCF iPSC lines from patients with familial AD mutations (PSEN1, PSEN2, APP) have enabled:
• Identification of disease-specific molecular subtypes through RNA sequencing[@nyscf_alzheimers_subtyping]
• Demonstration of amyloid-beta and tau pathology in patient-derived neurons
• CRISPR/Cas9-mediated correction of mutations with reversal of phenotypes[@nyscf_psen2_crispr]
• Studies of autophagy and mitophagy defects in AD neurons[@nyscf_bexarotene_autophagy]
• Characterization of PSEN1 familial AD neural progenitors[@nyscf_pasen1_ipsc]

• Motor neurons derived from ALS patient iPSCs have been used to study:
• TDP-43 proteinopathy
• RNA metabolism defects
• Mitochondrial dysfunction
• Axonal transport deficits

Drug Discovery and Screening

The NYSCF biobank supports high-throughput drug screening campaigns by providing:

Mechanistic Studies

Researchers have employed NYSCF iPSC lines to investigate fundamental mechanisms of neurodegeneration:

• Neuroinflammation: Studies on patient-derived astrocytes and microglia have revealed disease-specific inflammatory signatures[@nyscf_reactive_astrocytes_2023]
• Protein aggregation: Direct visualization of alpha-synuclein, tau, and TDP-43 aggregation in living neurons
• Mitochondrial biology: Assessment of mitochondrial function using Seahorse extracellular flux analysis
• Calcium signaling: Investigation of calcium dysregulation as a common pathogenic mechanism

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Significance and Impact

Resource Metrics

The NYSCF biobank has established itself as a critical resource for the neurodegenerative disease research community:

• Over 1,000 patient-derived iPSC lines representing multiple disease categories
• Distribution to over 100 research institutions worldwide
• Contributions to hundreds of peer-reviewed publications advancing understanding of neurodegenerative disease mechanisms
• Partnership with pharmaceutical companies for drug discovery and validation

Scientific Contributions

The biobank has enabled numerous scientific discoveries including:

Collaborative Network

The NYSCF has established partnerships with:

• Academic medical centers specializing in movement disorders and dementia
• Industry partners for drug discovery collaborations
• International stem cell repositories for resource sharing
• Patient advocacy organizations for recruitment and outreach

Data Access and Sharing

Access Process

Researchers interested in obtaining iPSC lines from the NYSCF biobank must:

Data Available

The NYSCF provides the following data with each iPSC line:

| Data Type | Description | Format |
|-----------|-------------|--------|
| Clinical Data | Diagnosis, disease duration, medications, clinical scores | De-identified CSV |
| Genetic Data | Known disease mutations, APOE genotype | VCF or CSV |
| Phenotypic Data | Differentiation efficiency, marker expression | PDF reports |
| Quality Control | Mycoplasma status, karyotype, sterility | Certificate of analysis |

Related Neurodegenerative Resources

Similar Biobanks and Registries

Related Pages

• [Alzheimer's Disease](/diseases/alzheimers-disease)
• [Parkinson's Disease](/diseases/parkinsons-disease)
• [Progressive Supranuclear Galsy](/diseases/progressive-supranuclear-palsy)
• [Amyotrophic Lateral Sclerosis](/diseases/amyotrophic-lateral-sclerosis)
• [Induced Pluripotent Stem Cells](/mechanisms/ipsc-neurodegeneration)
• [iPSC Disease Modeling](/mechanisms/ipsc-disease-modeling)

External Links

• [NYSCF Biobank - ClinicalTrials.gov](https://clinicaltrials.gov/study/NCT06203106)
• [NYSCF Stem Cell Repository](https://www.nyscf.org/partnering/products/)
• [NYSCF Research Institute Publications](https://www.nyscf.org/research-institute/publications/)

References