Gene variants linked to survival after HSCT

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DNA helix
Image by Spencer Phillips
New research has revealed a link between rare gene variants and survival after hematopoietic stem cell transplant (HSCT). Researchers performed exome sequencing in nearly 2500 HSCT recipients and their matched, unrelated donors. The sequencing revealed several gene variants—in both donors and recipients—that were significantly associated with overall survival... [Read Article]
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MicroRNAs required for cGVHD development

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Lab mice
Photo by Aaron Logan
New research suggests a family of microRNAs, miR-17-92, plays a key role in chronic graft-versus-host disease (cGVHD). Researchers found miR-17-92 is responsible for the T- and B-cell pathogenicity that causes cGVHD. The team also discovered that pharmacological inhibition of miR-17 alleviated the symptoms of cGVHD in mice. Yongxia... [Read Article]
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MSC product receives orphan designation

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Mesenchymal stem cells
The US Food and Drug Administration (FDA) has granted orphan drug designation to CYP-001 for the treatment of acute graft-versus-host disease (GVHD). CYP-001 is a mesenchymal stem cell (MSC) product manufactured using Cynata Therapeutics Limited’s proprietary Cymerus™ technology. According to Cynata, this technology enables economic manufacture of MSCs at commercial scale... [Read Article]
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UCART19 can bridge to transplant in adults

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T cells
Image from NIAID
Preliminary data from a phase 1 trial suggest UCART19 can serve as a bridge to transplant in adults with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL). This “universal,” donor-derived, chimeric antigen receptor (CAR) T-cell therapy produced complete responses (CRs) in 6 of 9 patients treated. Five of the patients... [Read Article]
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Phase 1 results with UCART19 in kids

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Layla Richards, the first person
treated with UCART19
Photo courtesy of GOSH
Early results from a phase 1, pediatric trial of UCART19 expand upon results observed in children who received the therapy via a compassionate use program. Two patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) received UCART19, a “universal,” donor-derived chimeric antigen... [Read Article]
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Mortality in patients with PTLD

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Cell infected with Epstein-Barr
virus (green/red) among
uninfected cells (blue)
Image courtesy of
Benjamin Chaigne-Delalande
A review of recent studies has revealed a “significant burden” of mortality related to post-transplant lymphoproliferative disorder (PTLD), according to researchers. Of the 120 PTLD patients studied, 64% died and 42.5% died with PTLD. The median time from hematopoietic... [Read Article]
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AdV viral burden linked to mortality in kids

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Transmission electron
micrograph of adenovirus
Image from CDC/
G. William Gary Jr.
Data from the AdVance study revealed a “strong correlation” between adenovirus (AdV) viral load and mortality in pediatric recipients of allogeneic hematopoietic stem cell transplant (allo-HSCT), according to researchers. The team found that patients with the highest AdV viral burden had roughly... [Read Article]
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Manufactured graft deemed safe in blood cancer patients

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Peripheral blood
stem cell graft
Phase 1 results suggest a programmed cellular therapy is safe for use in patients with hematologic malignancies. The therapy, ProTmune, is being developed as a next-generation allogeneic graft intended to reduce the incidence and severity of acute graft-versus-host disease (GVHD) after hematopoietic stem cell transplant (HSCT). Three of 7... [Read Article]
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