NIH-funded research NIH-funded research

A safer asparaginase treatment for liver (hepatocellular) cancer

Official title: Expanding the efficacy of asparaginase to solid tumors

Funded at Jesse Brown VA Medical Center · NIH-11130942 · Data updated 2026-11-12

A human-like, less-toxic form of the cancer drug asparaginase is being developed for adults with hepatocellular (liver) cancer whose tumors need the amino acid asparagine.

More Solid Tumors research and trials

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionJesse Brown VA Medical Center
NIH-funded
Lab location1 site (Chicago, United States)
Project startApril 1, 2013
Project endJune 30, 2027
Funding year2025
NIH project number5I01BX001919-11
Project IDNIH-11130942 on NIH RePORTER

Who could benefit from this research

Could help

Adults (typically 21+) with hepatocellular carcinoma whose tumors test positive for biomarkers indicating dependence on blood asparagine would be the ideal candidates.

Less likely to help

Patients whose tumors have high internal asparagine-production (no biomarker for ASNase sensitivity), those with other cancer types without the biomarker, or those with severe hypersensitivity to protein therapies are unlikely to benefit.

What this research studies

Researchers are developing a human-like version of asparaginase that reduces immune reactions and removes the damaging glutaminase side activity that causes severe side effects in adults. They use biomarker testing to identify tumors that rely on blood asparagine (low or no expression of the tumor's asparagine-synthesizing enzyme) so the drug can target the right patients. Preclinical in vivo studies have shown the engineered enzyme is safer than current bacterial asparaginases while maintaining similar anti-leukemia activity. The goal is to provide proof-of-concept that this approach could be used in adult liver cancer patients and to enable future clinical testing in people whose tumors have the right biomarker profile.

Why it matters

Potential benefit: If successful, this could offer adults with certain liver cancers a new targeted therapy with fewer immune and toxicity problems than existing bacterial asparaginases.

How similar studies have gone: Asparaginase is an established drug for pediatric ALL, but applying a engineered, human-like low-glutaminase form to adult solid tumors is novel and currently supported mainly by preclinical safety and efficacy data.

Where this research is happening

  • Jesse Brown VA Medical Center
    Chicago, United States
    Active

Researchers

  • Principal investigator: Lavie, Arnon — Jesse Brown VA Medical Center
  • Study coordinator: Lavie, Arnon

Related research

Other NIH-funded projects on the same topics.

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.