# Acoustic separation of peripheral blood for cellular therapy of sickle cell anemia

> **NIH NIH R21** · BOSTON CHILDREN'S HOSPITAL · 2020 · $238,807

## Abstract

Project Summary
Novel cell therapies, including gene therapy for Sickle Cell Disease (SCD) require a relative large number of
stem cells collected from peripheral blood for cell manipulation and subsequent transfusion. Current methods
rely on centrifugal apheresis, which is associated with poor collection efficiency owing to the altered migration
patterns of SCD red cells. Even when collected, products are highly contaminated with red cells requiring
further processing and the corresponding cell loss alters transduction strategies and the net therapeutic dose.
Poor collections limit the ability of SCD patients to fully participate in clinical cell therapy trials, and there is a
great need to overcome this problem. SCD patients more likely require repeated collections using plerixafor
alone, increasing costs and morbidity. To improve PBSC collections, we will test an alternative method of cell
separation, acoustic apheresis, for the ability to further separate and enrich stem cells from peripheral blood to
allow for the treatment of SCD patients. Current collections from SCD patients yield large volume products
with high hematocrits and acoustophoresis can improve this obstacle. In the first aim of the proposal, we will
test acoustophoresis on normal blood and apheresis products to understand how various peripheral blood cells
can be fractionated and collected. We will then test acoustophoresis on sickle blood and characterize the
functional dynamics of red cells following the separation procedure. These studies are geared toward the goal
of successfully collecting and enriching stem cells from SCD patients, and will furthermore elucidate the
heterogeneity of red cells and their functional dynamics in SCD with correlates for iron deficiency anemia and
other hemoglobinopathies. In the second aim of the proposal, we will develop instrumentation to perform
separations at higher flow rates, with the vision that acoustophoresis could in the future supplement
conventional apheresis in real time as products are collected. This technology could safely collect both cellular
and red cell fractions for clinical use. Acoustophoresis is best studied and introduced clinically by Transfusion
Medicine as this is where patients intersect with those who collect cells and those who treat. As more trials of
cell therapy launch, Transfusion Medicine has the ability to transform the field and directly aid patients to
achieve therapy.

## Key facts

- **NIH application ID:** 9851923
- **Project number:** 5R21HL145636-02
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** JOHN P MANIS
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $238,807
- **Award type:** 5
- **Project period:** 2019-02-01 → 2023-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9851923

## Citation

> US National Institutes of Health, RePORTER application 9851923, Acoustic separation of peripheral blood for cellular therapy of sickle cell anemia (5R21HL145636-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9851923. Licensed CC0.

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