NIH-funded research R01 grant

NSD2-NEO1 pathway and CNS spread of childhood ALL

Official title: The role of NSD2-NEO1 axis in CNS infiltration of acute lymphoblastic leukemia

Funded at Thomas Jefferson University · NIH-11447279 · Data updated 2026-11-12

A pediatric leukemia study looks at how a gene mutation helps leukemia cells reach the brain, with the aim of finding targeted, less toxic treatments.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionThomas Jefferson University
NIH-funded
Lab location1 site (Philadelphia, United States)
Project startAugust 18, 2026
Project endJuly 31, 2031
Funding year2026
Award (this year)$492,980
NIH project number1R01CA308034-01A1
Project IDNIH-11447279 on NIH RePORTER

Who could benefit from this research

Could help

Ideal participants are children aged 0-11 diagnosed with acute lymphoblastic leukemia, particularly those at risk for CNS involvement.

Less likely to help

Very young children without CNS involvement or without NSD2-associated pathways may have limited direct benefit.

What this research studies

This project focuses on children with acute lymphoblastic leukemia (ALL) whose disease may invade the central nervous system. Researchers will explore how NSD2 mutations change gene activity and cell behavior to promote CNS entry, using advanced chromatin and sequencing methods to identify key pathways. The goal is to uncover targeted strategies that limit CNS infiltration and reduce long-term side effects from therapy. The work builds on preliminary findings that connect NSD2 mutations to altered adhesion and migration of leukemia cells, potentially revealing new therapeutic targets. Results could pave the way for treatments that are effective against CNS relapse with fewer harmful effects.

Why it matters

Potential benefit: If successful, the study could lead to targeted therapies that prevent CNS relapse and lessen treatment-related toxicity for pediatric ALL patients.

How similar studies have gone: Previous work linking NSD2 mutations to leukemic behavior supports the potential for meaningful advances, though direct CNS-targeted strategies remain under development.

Where this research is happening

  • Thomas Jefferson University
    Philadelphia, United States
    Active

Researchers

  • Principal investigator: Li, Jianping — Thomas Jefferson University
  • Study coordinator: Li, Jianping

Related research

Other NIH-funded projects on the same topics.

  • Caregiver-led mobile tool to support healthy habits for kids treated for ALL

    A caregiver-directed mobile program helps children who were treated for acute lymphoblastic leukemia build healthier daily routines.

    NIH research University of Minnesota · Minneapolis, United States · NIH-11630804

  • Genomics of childhood ALL across global populations

    A wide-ranging study looks at how inherited genetics influence the risk of childhood acute lymphoblastic leukemia around the world.

    NIH research University of Minnesota · Minneapolis, United States · NIH-11608541

  • Genomics of childhood ALL across diverse populations

    A large international study will look at why children everywhere get acute lymphoblastic leukemia and how genetics may influence risk.

    NIH research University of Minnesota · Minneapolis, United States · NIH-11608540

  • Combinatorial targeting for high-risk B-cell ALL treatment

    A research effort at West Virginia University is exploring a combination therapy for adult B-cell acute lymphoblastic leukemia using targeted gene silencing and a small-molecule compound to improve treatment in patients.

    NIH research West Virginia University · Morgantown, United States · NIH-11579281

  • Understanding why Latino children have a higher risk of childhood ALL

    This project looks at why Latino children are more likely to develop acute lymphoblastic leukemia and whether genetic ancestry plays a role, with the goal of improving prevention and outcomes.

    NIH research Beckman Research Institute/city of Hope · Duarte, United States · NIH-11520852

  • Targeting obesity to improve survival for children with ALL

    A new approach explores whether reducing obesity-related factors can make chemotherapy work better for kids with acute lymphoblastic leukemia.

    NIH research Children's Hospital of Los Angeles · Los Angeles, United States · NIH-11474567

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.