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DRUG CLASS:

IMPDH inhibitor

16d
Genetic determinants of efficacy of antiviral drugs revealed by genome-wide CRISPR screens. (PubMed, Antiviral Res)
For ribavirin, adenosine kinase (ADK) and adenylsuccinate synthase (ADSS) were critical for nucleotide metabolism and bioactivation. Remdesivir uptake and activation depended on the transporter SLC29A3 and phosphoamidase HINT1, whereas favipiravir resistance was linked to NT5C2-mediated dephosphorylation. Viral replication assays in Huh7 cells validated that knockout of SLC29A3, HINT1, or NT5C2 significantly altered antiviral efficacy. This study delineates the genetic network governing nucleotide analog response, providing mechanistic insights and potential biomarkers for personalized antiviral therapy.
Journal
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NT5C2 (5'-Nucleotidase Cytosolic II) • HINT1 (Histidine Triad Nucleotide Binding Protein 1)
18d
How should immune checkpoint inhibitor myocarditis be treated? (PubMed, Cardiooncology)
In steroid-refractory or severe cases, adjunctive immunomodulators such as mycophenolate mofetil, janus kinase inhibitors, and notably abatacept are utilized, although evidence is largely case-based. By describing the management of three illustrative real-life cases, this article aims to provide a framework for individualized management of ICIrM in adults, integrating current guidelines, literature, and clinical experience. Continued research and prospective trials are critical to refine diagnostic algorithms and improve patient outcomes.
Review • Journal • Checkpoint inhibition • IO biomarker
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CTLA4 (Cytotoxic T-Lymphocyte Associated Protein 4)
1m
Adalimumab in refractory pediatric tubulointerstitial nephritis and uveitis syndrome: a case report of sustained renal-ocular remission and literature review. (PubMed, BMC Nephrol)
Adalimumab can effectively control inflammatory responses and reduce recurrence in pediatric TINU syndrome, with a favorable safety profile, thereby providing a novel and promising biologic alternative for steroid-resistant or refractory cases. It is recommended to focus on ocular lesions in patients with tubulointerstitial injuries to avoid missing the diagnosis of TINU syndrome.
Journal
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TNFA (Tumor Necrosis Factor-Alpha)
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prednisolone
1m
Trial completion date
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IGH (Immunoglobulin Heavy Locus) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
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cyclophosphamide • sirolimus • melphalan • fludarabine IV • mesna • Neupogen (filgrastim)
1m
A Retrospective Study on the Efficacy and Safety of Mizoribine in Anti-Rejection Therapy for Renal Transplant Recipients (ChiCTR2500109624)
P=N/A, N=200, Not yet recruiting, West China Hospital, Sichuan University; West China Hospital, Sichuan University
New trial
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Bredinin (mizoribine)
1m
Sirolimus use in allogeneic hematopoietic cell transplant recipients: assessing its senotherapeutic role in a high risk population. (PubMed, Front Aging)
All patients received an identical conditioning regimen with different GVHD prophylaxis: sirolimus + mycophenolate mofetil (MMF) or cyclosporine + MMF. Our results suggest that the daily administration and dosing used for GVHD prevention are less likely to confer clinical benefits, possibly indicating that the beneficial effects of sirolimus occur within a specific therapeutic window. These findings highlight the need to further investigate senotherapeutic approaches in this setting of accelerated aging.
Journal
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CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • CDKN1A (Cyclin-dependent kinase inhibitor 1A)
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sirolimus • cyclosporine
1m
The safety and short-term efficacy of nivolumab plus ipilimumab for advanced esophageal squamous cell carcinoma. (PubMed, Esophagus)
Our study showed a safety profile comparable to that of CM648 trial. Nivo + Ipi as 2nd or later-line treatment demonstrated promising efficacy.
Journal • PD(L)-1 Biomarker • IO biomarker
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PD-L1 (Programmed death ligand 1)
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PD-L1 expression
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Opdivo (nivolumab) • Yervoy (ipilimumab)
2ms
Selective targeting of HIV infected clones by cognate peptide stimulation and antiproliferative drugs. (PubMed, J Clin Invest)
We stimulated the clone with its cognate peptide and then exposed the cells to paclitaxel and/or carboplatin or the antiproliferative drug, mycophenolate mofetil. The drugs did not affect the proliferation of other CD4+ T cell clones that were not specific for the cognate peptide. This strategy of antigen-specific stimulation followed by treatment with an antiproliferative agent may lead to the selective elimination of clonally expanded HIV-infected cells.
Journal
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CD4 (CD4 Molecule)
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carboplatin • paclitaxel
2ms
Amyopathic Dermatomyositis Presenting With Oral Mucocutaneous Lesion and No Muscle Involvement in an Elderly Woman: A Mimicry of Fungal Infection and Eczema. (PubMed, Cureus)
Systemic corticosteroids and hydroxychloroquine were initiated, followed by mycophenolate mofetil, with limited response. This case illustrates the atypical presentation of ADM and the challenges in establishing an early diagnosis when muscle involvement is absent. It highlights IVIG as an effective option for treatment-resistant disease and underscores the importance of maintaining a high index of suspicion, pursuing thorough diagnostic evaluation, and ensuring long-term monitoring for interstitial lung disease and malignancy, even in patients who present with isolated cutaneous findings.
Journal
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IFIH1 (Interferon Induced With Helicase C Domain 1)
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hydroxychloroquine
2ms
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies-Increasing GVT Effects Without Increasing Toxicity (clinicaltrials.gov)
P2, N=51, Active, not recruiting, Sidney Kimmel Cancer Center at Thomas Jefferson University | Recruiting --> Active, not recruiting
Enrollment closed
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cyclophosphamide
2ms
Ebselen Suppresses Breast Cancer Tumorigenesis by Inhibiting YTHDF1-Mediated c-Fos Expression. (PubMed, Int J Mol Sci)
In addition, ebselen suppressed anchorage-independent growth in vitro and significantly reduced tumor growth in an orthotopic mouse model. These findings highlight YTHDF1 as a promising therapeutic target and support ebselen as a potential small-molecule inhibitor for breast cancer treatment.
Journal
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ANXA5 (Annexin A5) • FOS (Fos Proto-Oncogene AP-1 Transcription Factor Subunit 2) • YTHDF1 (YTH N6-Methyladenosine RNA Binding Protein 1)
2ms
SPI-1005 for the Treatment of Meniere's Disease (Open Label) (clinicaltrials.gov)
P3, N=200, Recruiting, Sound Pharmaceuticals, Incorporated | Not yet recruiting --> Recruiting
Enrollment open