GBA1 as a Radioligand Therapy Target
GBA1, glucosylceramidase beta 1, is a cell-surface protein. In Human Protein Atlas immunohistochemistry, GBA1 staining is highest in thyroid cancer (100% of samples positive), neuroendocrine tumors (100% of samples positive) and pancreatic cancer (100% of samples positive). Clinical status: Clinical-stage (up to phase 2, any modality).
Is GBA1 a good radioligand therapy target?
Scored against the six criteria that decide whether a protein can become a radioligand therapy:
- ✅ Cell-surface accessibility: Localized to the cell surface, so a radioligand can reach it from circulation.
- ✅ Tumor expression: High IHC staining in thyroid cancer, 100% of samples positive.
- ❔ Internalization: Not yet assessed.
- ✅ Clinical precedent: Clinical-stage (up to phase 2, any modality)
- ❔ Shedding: Not yet assessed.
- ❔ Normal-tissue dosimetry: Check kidney, liver, bone marrow and salivary expression in the Human Protein Atlas tissue atlas.
GBA1 expression in cancer
Protein expression of GBA1 across 20 cancer types from Human Protein Atlas immunohistochemistry. The score is a weighted staining intensity from 0 to 1; "% positive" is the share of patient samples with detectable staining.
| Cancer type | IHC score | Level | % positive |
|---|---|---|---|
| Thyroid Cancer | 1.00 | High | 100% |
| Neuroendocrine Tumors | 1.00 | High | 100% |
| Pancreatic Cancer | 1.00 | High | 100% |
| Endometrial Cancer | 1.00 | High | 100% |
| Breast Cancer | 0.97 | High | 100% |
| Melanoma | 0.97 | High | 100% |
| Lung Cancer | 0.97 | High | 100% |
| Prostate Cancer | 0.97 | High | 100% |
| Glioma | 0.97 | High | 100% |
| Liver Cancer | 0.94 | High | 100% |
| Ovarian Cancer | 0.94 | High | 100% |
| Kidney Cancer | 0.94 | High | 100% |
| Colorectal Cancer | 0.92 | High | 100% |
| Head and Neck Cancer | 0.92 | High | 100% |
| Gastric Cancer | 0.91 | High | 91% |
| Bladder Cancer | 0.87 | High | 100% |
| Skin Cancer | 0.86 | High | 100% |
| Cervical Cancer | 0.79 | High | 91% |
| Testicular Cancer | 0.30 | Medium | 56% |
| Lymphoma | 0.19 | Low | 33% |
Is GBA1 internalized?
Nuclens has not yet extracted internalization evidence for GBA1. Internalization matters for radioligands because an internalizing receptor traps the radionuclide inside the tumor cell. Run a full analysis to pull the latest literature.
GBA1 clinical trials
Clinical-stage (up to phase 2, any modality). Nuclens hasn't indexed trials for GBA1 yet. Search ClinicalTrials.gov for GBA1 trials.
GBA1 normal tissue expression
Normal-tissue expression in dose-limiting organs (kidney, liver, bone marrow, salivary glands) drives radioligand dosimetry risk. Nuclens is re-validating its normal-tissue values, so they are not shown here yet. See GBA1 in the Human Protein Atlas tissue atlas, and read how normal-tissue expression predicts radioligand dosimetry risk.
GBA1 gene essentiality (DepMap)
CRISPR knockout effect across 1258 cancer cell lines: -0.06 (not essential). Radioligands kill by radiation, not by blocking the target, so essentiality matters less than for inhibitors. A non-essential target can still be an excellent radioligand target.
Related thyroid cancer radioligand targets
c-MET (MET) · HER3 (ERBB3) · EPCAM · SSTR2 · EGFR · ITGAV · STEAP2 · FAP
See all radioligand therapy targets in thyroid cancer.
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Run a free analysisData sources
Subcellular localization: UniProt via Open Targets (CC BY 4.0 / CC0). Tumor immunohistochemistry: Human Protein Atlas (CC BY-SA 4.0). Clinical trials: ClinicalTrials.gov (public domain). Gene essentiality: DepMap (CC BY 4.0). Internalization and shedding: AI-extracted from PubMed abstracts, so verify against the cited papers before relying on them. Nuclens is a first-pass triage layer, not a substitute for wet-lab validation or clinical dosimetry. Derived expression data on this page is shared under CC BY-SA 4.0.