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    DRUG:

    pentostatin

    i
    Other names: CI 825, PD 81565, DCF, NSC 218321, YK 176
    Company:
    Generic mfg.
    Drug class:
    Adenosine deaminase inhibitor
    8d
    Enhancer regulator MLL4 controls skeletal muscle metabolic efficiency by limiting AMPK-mediated fuel catabolism. (PubMed, Nat Commun)
    Pharmacologic inhibition of AMP-metabolizing pathway by Pentostatin activates muscle AMPK, confers resistance to obesity and improves metabolic health. These findings identify an enhancer regulator limiting AMPK-mediated muscle fuel catabolism, offering a potential strategy for treating obesity-related disorders.
    Journal
    |
    KMT2D (Lysine Methyltransferase 2D) • AMPK (Protein Kinase AMP-Activated Catalytic Subunit Alpha 1)
    |
    pentostatin
    3ms
    INCA034176-355: A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy (clinicaltrials.gov)
    P3, N=9, Active, not recruiting, Incyte Corporation | Recruiting --> Active, not recruiting | N=300 --> 9
    Enrollment closed • Enrollment change
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    Imbruvica (ibrutinib) • Rituxan (rituximab) • imatinib • everolimus • pentostatin • Niktimvo (axatilimab-csfr)
    6ms
    Randomized Phase II Trial of Rituximab With Either Pentostatin or Bendamustine for Multiply Relapsed or Refractory Hairy Cell Leukemia (clinicaltrials.gov)
    P2, N=69, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Dec 2025 --> Dec 2027
    Trial completion date
    |
    CD20 (Membrane Spanning 4-Domains A1) • CD22 (CD22 Molecule) • IL2RA (Interleukin 2 receptor, alpha) • ITGAE (Integrin Subunit Alpha E) • ITGAX (Integrin Subunit Alpha X) • ISG20 (Interferon Stimulated Exonuclease Gene 20)
    |
    CD20 positive
    |
    Rituxan (rituximab) • bendamustine • pentostatin
    7ms
    A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy (clinicaltrials.gov)
    P3, N=300, Recruiting, Incyte Corporation | Not yet recruiting --> Recruiting
    Enrollment open
    |
    Imbruvica (ibrutinib) • Rituxan (rituximab) • imatinib • everolimus • pentostatin • Niktimvo (axatilimab-csfr)
    7ms
    Constructing a mitochondrial-related genes model based on machine learning for predicting the prognosis and therapeutic effect in colorectal cancer. (PubMed, Discov Oncol)
    To address the suboptimal treatment outcomes in these patients, we identified PYR-41 and pentostatin as potential therapeutic agents, which are anticipated to enhance therapeutic efficacy in the high-risk group. Additionally, four biomarkers (HSPA1A, CHDH, TRAP1, CDC25C) were validated by quantitative real-time PCR, with significant expression differences between normal intestinal epithelial cells and colon cancer cells. Our prognostic model provides accurate CRC outcome prediction and guides personalized therapeutic strategies.
    Journal • IO biomarker
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    CDC25C (Cell Division Cycle 25C) • HSPA1A (Heat Shock Protein Family A (Hsp70) Member 1A) • GNRP (Ras-Specific Guanine Nucleotide-Releasing Factor 1)
    |
    pentostatin
    7ms
    B Cell Activating Factor Induces Drug Resistance in Hairy Cell Leukemia Variant. (PubMed, Biomedicines)
    Classical hairy cell leukemia (HCL-c) is very sensitive to the standard of care with purine nucleoside analogs (PNAs) cladribine (cDa) and pentostatin. We conclude that BAFF provides chemo-protection in HCL-v cells by activating nonclassical NF-κB signaling, which results in the upregulation of multiple pro-survival or anti-apoptotic genes. Our results highlight an important role of BAFF in HCL-v resistance to chemotherapy and suggest that the BAFF blockade may enhance the chemosensitivity to PNAs in drug-resistant HCL-v patients.
    Journal
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    CXCL8 (Chemokine (C-X-C motif) ligand 8) • PTGS2 (Prostaglandin-Endoperoxide Synthase 2) • CXCL5 (Chemokine (C-X-C motif) ligand 5) • CXCL1 (Chemokine (C-X-C motif) ligand 1) • PACERR (PTGS2 Antisense NFKB1 Complex-Mediated Expression Regulator RNA)
    |
    cladribine • pentostatin
    7ms
    A Blood Stem Cell Transplant for Sickle Cell Disease (clinicaltrials.gov)
    P1, N=3, Active, not recruiting, City of Hope Medical Center | Trial completion date: Mar 2025 --> Feb 2026 | Trial primary completion date: Mar 2025 --> Feb 2026
    Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    cyclophosphamide • pentostatin
    9ms
    Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure (clinicaltrials.gov)
    P2, N=56, Active, not recruiting, National Heart, Lung, and Blood Institute (NHLBI) | Trial completion date: Dec 2024 --> Dec 2027
    Trial completion date
    |
    cyclophosphamide • Campath (alemtuzumab) • sirolimus • pentostatin • Neupogen (filgrastim)
    10ms
    A Blood Stem Cell Transplant for Sickle Cell Disease (clinicaltrials.gov)
    P1, N=3, Active, not recruiting, City of Hope Medical Center | Trial completion date: Nov 2024 --> Mar 2025 | Trial primary completion date: Nov 2024 --> Mar 2025
    Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD4 (CD4 Molecule) • HLA-B (Major Histocompatibility Complex, Class I, B) • FOXP3 (Forkhead Box P3) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    cyclophosphamide • pentostatin
    10ms
    A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy (clinicaltrials.gov)
    P3, N=300, Not yet recruiting, Incyte Corporation
    New P3 trial
    |
    Imbruvica (ibrutinib) • Rituxan (rituximab) • imatinib • everolimus • pentostatin • Niktimvo (axatilimab-csfr)
    10ms
    Rapamycin-resistant polyclonal Th1/Tc1 cell therapy (RAPA-201) safely induces disease remissions in relapsed, refractory multiple myeloma. (PubMed, J Immunother Cancer)
    Consistent with our hypothesis, ex vivo manufacturing using mTOR inhibition and IFN-α polarization consistently yielded a novel RAPA-201 DP that possessed a desirable phenotype relative to cytokine phenotype, memory status, and checkpoint expression. RAPA-201 recipients had preservation of T cell counts and Th1 cytokine secretion yet had increased T cell receptor clonality that associates with antitumor responses in the setting of monoclonal antibody checkpoint therapy. RAPA-201 therapy overcomes previous barriers to effective autologous polyclonal T-cell therapy, as it is feasible to manufacture, exquisitely safe to administer, and mediates remission in patients with RRMM.
    Journal • PD(L)-1 Biomarker • IO biomarker
    |
    PD-1 (Programmed cell death 1) • CD73 (5'-Nucleotidase Ecto) • IFNA1 (Interferon Alpha 1) • LAIR1 (Leukocyte Associated Immunoglobulin Like Receptor 1)
    |
    cyclophosphamide • sirolimus • fludarabine IV • pentostatin • RAPA-201
    1year
    Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure (clinicaltrials.gov)
    P2, N=56, Active, not recruiting, National Heart, Lung, and Blood Institute (NHLBI) | Recruiting --> Active, not recruiting | N=162 --> 56
    Enrollment closed • Enrollment change
    |
    cyclophosphamide • Campath (alemtuzumab) • sirolimus • pentostatin • Neupogen (filgrastim)