Updates in Hematology

🧫 AGING

📜 “Aging and Adult Stem Cells” (Cell Stem Cell, 2025). https://lnkd.in/dzvCQ25F

  • Aging alters stem cell quiescence and differentiation across tissues, driving functional decline and tissue‑specific lineage bias.
  • Somatic mutations and clonal selection, combined with niche remodelling and chronic inflammation, link stem cell aging to degenerative diseases and cancer risk.

     

    💡NOVELTY💡: Highlights how intrinsic stem cell aging and extrinsic niche/inflammatory signals converge to impair regeneration and promote age‑related disease.
🧬 NEURO-REGENERATION

📜 “Stem cell therapies advance in Parkinson’s disease and beyond” (Nature Medicine, 2025). https://lnkd.in/d25kZipa

  • Parkinson’s trials in the USA and Japan show graft survival, dopamine release, symptom improvement, and favorable safety; one US program is advancing to phase 3.
  • Early efforts in ALS, Alzheimer’s, and epilepsy show initial signals but face long, complex, and costly development.

     

    💡NOVELTY💡: Marks a shift in neurodegenerative stem‑cell therapy toward late‑stage efficacy trials.
🩸HEMATOLOGY

📜 “Irradiation- and Busulfan-Free Stem Cell Transplantation in Fanconi Anemia” (Nature Medicine, 2025). https://lnkd.in/dGYsQngV

  • Anti‑CD117 antibody (briquilimab) enables stem cell transplantation in Fanconi Anemia without irradiation or busulfan.
  • In a phase 1b trial, three children achieved durable engraftment with minimal toxicity and no acute GVHD for up to two years.

💡NOVELTY💡: First proof that non‑genotoxic, antibody‑based conditioning can safely achieve engraftment in DNA‑repair‑deficient patients.

📜 “Microbial Metabolite Drives Ageing-Related Clonal Haematopoiesis via ALPK1” (Nature, 2025) https://lnkd.in/dsn7xNU4

  • The gut metabolite ADP-heptose accumulates with age and activates ALPK1 in pre-leukemic HSCs, triggering TIFA–NF-κB signaling and expansion of DNMT3A-mutant clones.
  • Genetic deletion of ALPK1 or TIFA blocks this effect in mice, confirming a microbiome-driven mechanism of clonal hematopoiesis.

    💡NOVELTY💡: Reveals a gut microbiome-bone marrow axis where ADP-heptose directly drives mutant HSC expansion, linking aging microbiome changes to leukemia risk.

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