# CCR5 Analyses in HIV Infected Hematology Patients undergoing Umbilical Cord Blood Allogeneic Transplantation

> **NIH NIH R34** · FRED HUTCHINSON CANCER RESEARCH CENTER · 2020 · $1

## Abstract

Patients with hematologic disorders who are HIV infected are generally excluded from allogeneic bone
marrow/stem cell transplant (alloSCT) protocols. Anti-retroviral therapy (ART) is highly effective, safe, and
convenient, however often patients maintain low circulating CD4 counts, and an increased pro-inflammatory
state, with associated morbidity and mortality. AlloSCT for hematologic malignancies is curative in the majority
of patients with otherwise lethal hematologic diseases via immune mediated effects termed graft vs. leukemia
that reflect ultimate elimination of all transplant patient hematopoietic cells during establishment of full donor
engraftment. This translational research program and clinical trial is designed to investigate whether the
infusion of CCR5∆32 homozygous or heterozygous cord blood (CB) units, identified on unrelated CB registry
search, may eliminate HIV infected recipient hematopoietic cells via allogeneic donor immunologic clearance of
recipient hematopoietic cells in patients with hematological malignancies and HIV infection in need of an
alloSCT. To reduce the risk of morbidity and mortality associated with prolonged cytopenia after cord blood
transplantation, we will also use NLA101, which is a cryopreserved cell therapy composed of ex-vivo
expanded CD34+ hematopoietic stem and progenitor cells originally isolated from umbilical CB units. Ten
patients will be enrolled and treated at the following study sites: FHCRC (with its Cancer Consortium partners,
University of Washington and Seattle Children’s Hospital), Case Western Reserve University – University
Hospitals (Marcos de Lima, PI), Children’s Research Institute/Children’s National Medical Center (Blachy
Davila-Saldana, PI), Memorial-Sloan Kettering Cancer Center (Juliet Barker, PI), and University of California-
San Francisco (Timothy Henrich, PI). We will utilize the platform of alloSCT to gain further insights into
potential clearance of HIV reservoir by allogeneic donor immune cell elimination of recipient hematopoietic
cells, and examine immune reconstitution in these patients. We will use state of the art highly sensitive assays
to monitor viral RNA and cell associated RNA and DNA to measure the decay of the HIV reservoir; an
integrated immune monitoring approach that combines several high dimensional platforms (Transcriptomics,
systems serology, multiparametric flow cytometry) will allow us to identify correlates and innate and adaptive
immune mechanisms that are associated to control and decay of the HIV reservoir. This may allow HIV-1
infected patients to discontinue antiretroviral therapy (ART) for a sustained period without expected viral
rebound, as has been observed over a 9 year time period in an HIV infected patient with leukemia treated with
allogeneic stem cell transplant incorporating a CCR5∆32 homozygous graft (aka ‘Berlin patient’). It is expected
that data acquired under this R34 over the next 36 months will inform the design and execution of...

## Key facts

- **NIH application ID:** 9978092
- **Project number:** 5R34HL142322-03
- **Recipient organization:** FRED HUTCHINSON CANCER RESEARCH CENTER
- **Principal Investigator:** FILIPPO MILANO
- **Activity code:** R34 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1
- **Award type:** 5
- **Project period:** 2018-09-15 → 2022-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9978092

## Citation

> US National Institutes of Health, RePORTER application 9978092, CCR5 Analyses in HIV Infected Hematology Patients undergoing Umbilical Cord Blood Allogeneic Transplantation (5R34HL142322-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9978092. Licensed CC0.

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