Cure Sickle Cell Initiative (CureSCi) - Sickle Cell Hematopoietic Stem Cell Bank (SCBank) - Catalog

Name

Cure Sickle Cell Initiative (CureSCi) - Sickle Cell Hematopoietic Stem Cell Bank (SCBank)

Accession Number

HLB02662222a

Acronym

CureSCi-SCBank

Related studies

BSI Study IDs

CSD

Is public use dataset

False

Keywords

Has Study Datasets

False

Has Specimens

True

Specimen ID Type

Coded

Study Website

https://curesickle.org/

The Framingham Heart Study Group requires that the requestor must obtain full or expedited IRB/Ethics Committee review and approval to obtain these data. Waivers or a determination that the research is exempt from ethical regulations do not suffice.

False

Clinical Trial URLs
N/A
Study type

Epidemiology Study

Collection Type

Open BioLINCC Study

Cohort type

Adult

Interventions

Study Open Date (Data)

None

Study Open Date (Specimens)

2022-10-25

Date materials available

2022-10-25

Last updated

None

Study period

2021-2022

Study Contacts
NHLBI Division

DBDR

Classification

Blood Disease

HIV study classification

non-HIV

COVID study classification

non-COVID

Pre-Website # of Specimens Shipped

None

# of Returned Specimens

None

Primary Publication URLs
N/A
Conditions

Anemia, Sickle Cell
Sickle Cell Disease

Objectives

Many cellular therapies for sickle cell disease (SCD) rely on the in vitro manipulation of cells to effect genetic changes in hope of curative therapies through stem cell transplantation. One of the major impediments in the field is the availability of SCD derived stem cells for testing of gene targeting therapies. The purpose of this collection is to provide a bank of frozen blood derived stem cells that can be made available to scientists in the SCD field. A central repository allows for a standardized cell pool to be used by many different laboratories and thus enable comparisons of in vitro manipulation.

Background

Cellular therapy for treating the hemoglobinopathy associated with SCD is developing at a rapid pace, with multiple approaches in development. Most of the therapeutic approaches that use stem cell transplantation will require preclinical testing of the modified HSC, to establish and validate the in vitro procedures. Emerging therapies include gene addition of beta globin, Bcl11a modification as well as other gene and base editing techniques performed at many academic and industry research laboratories.


To develop these novel cell therapies, there is a need for both healthy and SCD CD34+ cells for use in preclinical development and in validation of these therapeutic approaches. One of the challenges in obtaining SCD CD34+ HSCs is that mobilization and collection of these cells is challenging due to technical limitations of the apheresis procedure. Moreover, peripheral blood mobilization of stem cells in SCD patients is limited to plerixafor as G-CSF is contraindicated in SCD thus obtaining relatively fewer cells at collection. Taken together, there are very few centers that can potentially assemble SCD stem cell donors and have the expertise to successfully collect CD34+ cells for research use.

Participants

Volunteers with SCD were recruited as donors for collection of peripheral blood stem cells by apheresis. Inclusion criteria included: Diagnosis of sickle cell disease with genotype HbSS, HbS/beta thalassemia, age 18-45 years, receiving regularly-scheduled blood transfusions or exchange transfusions as part of existing medical care, adequate hematologic parameters, organ function and performance status. Exclusion criteria included subjects on concurrent hydroxyurea treatment, with uncontrolled illness, known myelodysplasia of the bone marrow, pregnancy or breastfeeding and/or receipt of an investigational study drug or procedure within 90 days of enrollment.

Design

Volunteers were treated with plerixafor, a drug shown to safely mobilize peripheral blood (PB) CD34+ cells in people with SCD. The collected blood cells will be purified by magnetic separation using CD34 beads, validated, frozen. CD34 positive cells are frozen in aliquots immediately after purification. A portion of cells from the CD34 negative fraction were also aliquoted and frozen and are available for request.

Conclusions

The plerixafor-mobilized apheresis units from subjects with sickle cell disease were collected and CD34+ cell isolation was conducted under standard operating procedures described in detail elsewhere (1) and in the manual of operations.



  1. Esrick EB, Manis JP, Daley H, Baricordi C, Trébéden-Negre H, Pierciey FJ, Armant M, Nikiforow S, Heeney MM, London WB, Biasco L, Asmal M, Williams DA, Biffi A. Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Blood Adv. 2018 Oct 9;2(19):2505-2512. doi: 10.1182/bloodadvances.2018016725. PMID: 30282642; PMCID: PMC6177648.

Disease classification

Publications

Mat types

CD34+ Cells
CD34- Cells

Network

Please note that biospecimen availability is subject to review by the NHLBI, BioLINCC, and the NHLBI Biorepository. Certain biospecimens may not be made available for your request. Section 3.0 of the BioLINCC Handbook describes the components of the review process.

  • Material Types
    CD34 Negative Cells, CD34 Positive Cells

    Last Modified: July 26, 2023, 9:50 a.m.
  • General Freeze/Thaw Status
    All vials of CD34 Neg/Pos Cells are unthawed.

    Last Modified: July 26, 2023, 9:50 a.m.
  • Visits (Vials)
    26 July 2023
     
    CD34 Negative Cells CD34 Positive Cells Total
    A00 90 44 134

    Last Modified: Aug. 8, 2023, 8:07 a.m.
  • Visits (Subjects)
    26 July 2023
     
    CD34 Negative Cells
    Total number of subjects Average volume (10e6 cells) per subject
    A00 3 750.00
     
      CD34 Positive Cells
    Total number of subjects Average volume (10e6 cells) per subject
    A00 3 45.10

    Last Modified: Aug. 8, 2023, 8:07 a.m.