Team maps genetic evolution of T-ALL subtype

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Bone marrow smear of T-ALL
© Hind Medyouf, German
Cancer Research Center
Single-cell analysis has revealed key genetic events in a type of T-cell acute lymphoblastic leukemia (T- ALL), according to researchers. The team tracked the branching pattern of evolution in STIL-TAL1-positive T-ALL and identified mutations that may trigger development of the disease. The researchers... [Read Article]
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Drug nets orphan designation for SCD

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A sickled red blood cell
beside a normal one
Image by Betty Pace
The European Commission (EC) has granted orphan designation to Altemia (formerly SC411) for the treatment of pediatric patients with sickle cell disease (SCD). Altemia gelatin capsules are designed to replenish the lipids destroyed by sickle hemoglobin. Altemia is intended to be... [Read Article]
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Disease, genetics contribute to neurocognitive decline in ALL

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Kevin Krull, PhD (left)
and Yin Ting Cheung, PhD
Photo by Seth Dixon/
St. Jude Children’s
Research Hospital
Chemotherapeutic agents have been associated with neurocognitive side effects in survivors of pediatric acute lymphoblastic leukemia (ALL). Now, researchers have found evidence to suggest that genetics and ALL itself can increase the risk for long-term problems... [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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FDA approves nilotinib for kids with CML

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Nilotinib (Tasigna)
Photo from Novartis
The US Food and Drug Administration (FDA) has expanded the approved indication for nilotinib (Tasigna®) to include the treatment of children. The drug is now approved to treat patients age 1 and older who have newly diagnosed Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase. Nilotinib... [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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Metabolic changes in T cells may limit CAR potential in kids

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David Barrett, MD, PhD
Researchers analyzed peripheral blood T cells from 157 pediatric cancer patients at diagnosis and after chemotherapy and found the potential to produce effective chimeric antigen receptor (CAR) T cells declined with each cycle of chemotherapy. This was also true for acute lymphoblastic leukemia (ALL) and Wilms’ tumor, which had high CAR... [Read Article]
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