A safer asparaginase treatment for liver (hepatocellular) cancer

Expanding the efficacy of asparaginase to solid tumors

NIH-funded research Jesse Brown VA Medical Center · NIH-11130942

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.

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 IDNIH-11130942 on NIH RePORTER

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.

Who could benefit from this research

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

Not a fit: 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.

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 performed: 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

Chicago, United States

Researchers

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 below.
Last reviewed 2026-06-13 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.