Sodium Stibogluconate in the MDS/AML With One of the 65 Defined p53 Mutations
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- STATUS
- Recruiting
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- End date
- Feb 1, 2024
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- participants needed
- 5
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- sponsor
- First Affiliated Hospital of Jinan University
Summary
To evaluate the safety and effectiveness of Sodium Stibogluconate in the treatment of myelodysplastic syndrome/acute myeloid leukemia (MDS/AML) with p53 mutation from a defined list. The list includes 65 p53 mutations that were experimentally confirmed to be pharmacologically restored with tumor-suppressive function by antimonials.
Description
p53 is inactivated by over hundreds of diverse mutations in cancer. The investigator purposefully selected the phenotype-reversible temperature-sensitive (TS) p53 mutations for pharmacological rescue, and pertinently used p53 thermostability as readout to screen for TS p53 mutation rescue compounds. By this, the investigator identified antiparasitic drug antimonials efficiently thermostabilized the TS mutants by noncovalent binding. The antimonials met the three go-to criteria as a targeted drug-availability of co-crystal structure, structure model-compatible Structure-Activity Relationship, and target (p53)-specificity in cells, and consequently extended survival of xenograft mouse with TS p53 mutant.
Under the clinical antiparasitic dosage, the antimonials effectively rescued TS p53 mutant in patient-derived primary acute myeloid leukemia cells. Scan of the most frequent 815 p53 missense mutations identified 65 of them, predominantly TS mutations, as the antimonial-treatable mutations. Thus, the trial aims to evaluate the safety and effectiveness of the approved antimonial-Sodium Stibogluconate-in the treatment of myelodysplastic syndrome/acute myeloid leukemia (MDS/AML) with the 65 antimonial-treatable p53 mutants.
Details
| Condition | myelodysplastic syndromes, Bone Marrow Disorder, Temperature-Sensitive p53 Mutation, P53 Mutation, Myelodysplastic Syndromes (MDS), TP53 Gene Mutation, Bone marrow disorder, acute myelogenous leukemia, Myeloid Malignancy, myelodysplastic syndrome (mds), anll, acute myeloblastic leukemia, Acute Myeloid Leukemia, Preleukemia, Myeloid Neoplasm, Acute myeloid leukemia, MYELODYSPLASTIC SYNDROME, sodium stibogluconate, Pentostam, Acute Myelogenous Leukemia (AML) |
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| Treatment | Sodium stibogluconate |
| Clinical Study Identifier | NCT04906031 |
| Sponsor | First Affiliated Hospital of Jinan University |
| Last Modified on | 18 June 2021 |
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