Vistide (cidofovir) / Gilead - LARVOL DELTA

Epidemiology, risk factors, and outcomes of cytomegalovirus (CMV) reactivation in allogeneic hematopoietic stem cell transplantation: A retrospective observational single-centre study from western India (ASH 2025) - "While pre-emptive therapy has reduced CMV disease, reactivation remains common, particularly in alternate donor transplants and resource-limited settings where access to newer antivirals like letermovir is limited...Serotherapy type did not impact overall reactivation, but early reactivation was significantly higher with Grafalon vs. Thymoglobulin (90% vs. 33.6%, p<0.001)...Adverse effects included neutropenia (ganciclovir/valganciclovir) and nephrotoxicity (foscarnet/cidofovir)... This single-centre study demonstrates a high CMV reactivation rate (89.7%) in a high-seroprevalence Indian cohort, especially in alternate donor and PTCy-treated patients. While CMV disease was rare due to effective surveillance and pre-emptive therapy, recurrence and antiviral toxicity were common. The type of serotherapy influenced early reactivation risk."

Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Immunology • Neutropenia • Transplantation

Incidence, patterns, and outcomes of adenovirus infections in patients following allogeneic hematopoietic cell transplantation (ASH 2025) - "Cidofovir monotherapy was used in 292 patients (31.7% of those treated), while 2,032 (37.7%) received cidofovir with other agents, and 2,541 (99.6%) received alternative regimens, including preemptive therapy, Cytovir-ADV, rituximab, third-party cytotoxic T lymphocytes, or surgical interventions. These findings underscore the significant burden of AdV infection and its associated mortality in this patient population. Future research should focus on optimizing antiviral strategies and exploring novel therapies to reduce AdV incidence and improve outcomes."

Clinical • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Immunology • Infectious Disease • Leukemia • Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Transplantation

Quantifying the impact of vancomycin on short and long term renal function in a contemporary real-world pediatric stem cell transplant cohort (ASH 2025) - "Commonly used agents such as vancomycin, amphotericin, cidofovir, and others are effective at combating life-threatening infections in transplant recipients, but the tradeoff in short- and long-term renal injury may be significant. Sub-analyses to look separately at allogeneic vs autologous transplant receipients are also underway. This work highlights the need to quantify toxicities and side effects of therapies and paves the way for future efforts to minimize iatrogenic harm without compromising infection control."

Clinical • Real-world • Real-world evidence • Acute Kidney Injury • Chronic Kidney Disease • Infectious Disease • Nephrology • Pediatrics • Renal Disease • Transplantation

Novel use of low-dose cidofovir (2.5 mg/kg Weekly) as CMV prophylaxis in the pre-engraftment Phase of stem cell transplantation: A viable alternative when ganciclovir is contraindicated and letermovir is not available (ASH 2025) - "These include CMV-seropositive recipients (R+) with seronegative donors (D-) who are deemed to be at highest risk of CMV infection due to latent virus reactivation, those with previous CMV infections and in haploidentical BMT cases where Post Transplant Cyclophosphamide (PTCy) was used as GVHD prophylaxis. Low-dose weekly cidofovir represents a novel, cost-effective, and practical approach for CMV prophylaxis and treatment in the pre-engraftment phase of HSCT, particularly in settings where letermovir is unavailable. This strategy may reduce CMV-related graft loss while avoiding the myelotoxicity of ganciclovir. Further prospective studies are warranted to validate its role in global transplant protocols."

Beta-Thalassemia • Bone Marrow Transplantation • Cytomegalovirus Infection • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Transplantation

Administration of off-the-shelf, expanded, most closely HLA matched, third party viral specific T cells for the therapy of adenovirus related disease in adult immunocompromised patients. (ASH 2025) - "While Cidofovir is the primary treatment, its nephrotoxicity and limited efficacy restricts use,especially in post-transplant patients on nephrotoxic drugs...Conclusion CTL therapy for adenovirus infection was safe with no infusion reactions or attributable adverse events inthis study. CTLs appear efficacious in treating both viremia and adenovirus disease."

Clinical • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Malignancies • Hepatology • Hodgkin Lymphoma • Immunology • Infectious Disease • Leukemia • Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome • Myelofibrosis • Nephrology • Non-Hodgkin’s Lymphoma • Pneumonia • Respiratory Diseases • IL15 • IL2 • IL7

Incidence, Risk Factors, and Outcomes of Viral Reactivations in Alternative Donor Hematopoietic Stem Cell Transplants. (PubMed, Blood Cell Ther) - "Four patients (5%) developed ADV disease requiring treatment with cidofovir...The rates of ADV, BK virus, and EBV reactivation requiring intervention are low. Better prophylactic strategies are required to prevent CMV reactivation in alternative donor transplants."

Journal • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Transplantation

AI-driven transfer learning and classical molecular dynamics for strategic therapeutic repurposing and rational design of antiviral peptides targeting monkeypox virus DNA polymerase. (PubMed, Comput Biol Med) - "Three drugs exhibited the strongest binding affinities, outperforming the control drug, Cidofovir diphosphate, and forming stable interactions with key active site residues...Consistent results in MD simulations indicate functional binding without destabilisation. Although ADMET predictions for CFC revealed limitations in permeability and oral bioavailability, its favourable binding profile and reduced predicted toxicity support its potential as a novel antiviral lead."

Journal • Infectious Disease

PRIOH-1: Trial on Efficacy and Safety of Pritelivir Tablets for Treatment of Acyclovir-resistant Mucocutaneous HSV (Herpes Simplex Virus) Infections in Immunocompromised Subjects (clinicaltrials.gov) - P3 | N=158 | Completed | Sponsor: AiCuris Anti-infective Cures AG | Active, not recruiting ➔ Completed

Trial completion • Herpes Simplex • Infectious Disease

In vivo characterization of preclinical efficacy of brincidofovir against glioblastoma (SNO 2025) - "Brincidofovir (BCV) is lipid-conjugated cidofovir characterized by a lack of nephrotoxicity and superior activity compared to cidofovir...In the combination study, mice were administered temozolomide 2 mg/kg p.o. (TMZ) once daily for 5 days, whole brain radiotherapy (WBRT) of 1.5 Gy for 5 days plus TMZ, or bevacizumab 5 mg/kg i.p. BIW alone or concomitant with BCV 10 mg/kg... BCV monotherapy suppressed tumor growth and prolonged survival in PDX models of GBM and improved the efficacy of the standard of care for GBM."

Preclinical • Brain Cancer • Glioblastoma • Solid Tumor

Cidofovir induces antiviral immunity against Bombyx mori nucleopolyhedrovirus by modulating multiple immune pathways in silkworm. (PubMed, Pestic Biochem Physiol) - "Additionally, the 16S rRNA sequencing suggests that both viral infection and Cidofovir treatment alter the composition of gut microbiota in silkworms, with some unique taxa emerging in response to each condition. Together, these findings demonstrate that Cidofovir not only suppresses BmNPV proliferation but also activates multiple innate immune responses, emphasizing Cidofovir's potential as a promising antiviral agent for controlling BmNPV infection in silkworms."

Journal • Infectious Disease • AGO2 • CAT

Synergistic antiviral activity against human adenovirus through combination of itraconazole and brincidofovir. (PubMed, Br J Pharmacol) - "Given the gastrointestinal toxicity associated with brincidofovir, its combination with the host-directed drug itraconazole allowed lower brincidofovir doses to be used to decrease HAdV titres, thereby minimizing adverse drug effects while maintaining antiviral efficacy."

Journal • Gastroenterology • Gastrointestinal Disorder • Infectious Disease

Anti-Infective-Associated AKI: A Narrative Review of the Epidemiology, Mechanisms, Risk Factors, Biomarkers, Clinical Course, Monitoring, Prevention, and Therapeutic Strategies. (PubMed, Antibiotics (Basel)) - "This narrative review addressed and updated different aspects of anti-infective-associated AKI, including amphotericin B, cidofovir, foscarnet, polymyxins, vancomycin, and aminoglycosides. Currently, there is no definitive treatment modalities for the management of AKI with anti-infectives. Therefore, supportive care is the mainstay of treatment."

Biomarker • Journal • Review • Acute Kidney Injury • Critical care • Inflammation • Nephrology • Renal Disease • CST3 • KIM1 • LCN2

Vulval Intraepithelial Neoplasia: A 15-Year Review of Treatment Outcomes in a UK Centre. (PubMed, Cureus) - "Patients received local excision (clear or positive margins), CO₂ laser ablation, or medical therapy (imiquimod/cidofovir) and were stratified into unifocal or multifocal subgroups. By contrast, positive margins and multifocal disease were linked with earlier and more frequent recurrence, underscoring the importance of close, long-term surveillance. These findings may inform follow-up strategies, although further research is needed."

Journal • Oncology

Battle won, war ongoing: A call for mpox vaccination in high-risk dermatologic cases. (PubMed, JAAD Case Rep) - No abstract available

Journal

Use of short-course oral amenamevir (helicase-primase inhibitor) for the treatment of thymidine analogue-resistant herpes simplex lesions. (PubMed, Int J STD AIDS) - "It had also failed to respond to topical therapies and cidofovir. A seven-day course of oral amenamevir provided excellent resolution of the lesions.ConclusionThese two cases highlight the challenges in managing aciclovir-resistant herpes simplex lesions and the swift resolution following treatment with a novel therapeutic agent, amenamevir. Early recognition of resistant HSV lesions allows for prompt outsourcing of alternative, novel anti-viral therapies such as amenamevir."

Journal • Herpes Simplex • Human Immunodeficiency Virus • Infectious Disease • Pain

Lipid-Optimized Sulfone-Bridged Cidofovir Prodrugs as Potent Antivirals Against Orthopoxviruses. (PubMed, J Med Chem) - "Compound 13f exhibited antiviral activity against vaccinia virus (VACV) comparable to that of the reference brincidofovir (BCV), while 13i displayed 8.2-fold enhanced potency. Notably, 13i exhibited a favorable safety profile and significant therapeutic efficacy in both VACV-challenged BALB/c and MPXV-challenged severe combined immunodeficiency mouse models. Through systematic lipid-chain optimization, we identified 13i as a promising antiviral candidate with enhanced efficacy and favorable pharmaceutical properties."

Journal • Genetic Disorders • Immunology • Infectious Disease • Primary Immunodeficiency

In vivo characterization of preclinical efficacy of brincidofovir against glioblastoma (WFNOS 2025) - "Brincidofovir (BCV) is lipid-conjugated cidofovir characterized by a lack of nephrotoxicity and superior activity compared to cidofovir...In the combination study, mice were administered temozolomide 2 mg/kg p.o. (TMZ) once daily for 5 days, whole brain radiotherapy (WBRT) of 1.5 Gy for 5 days plus TMZ, or bevacizumab 5 mg/kg i.p. BIW alone or concomitant with BCV 10 mg/kg... BCV monotherapy suppressed tumor growth and prolonged survival in PDX models of GBM and improved the efficacy of the standard of care for GBM."

Preclinical • Brain Cancer • Glioblastoma • Solid Tumor

Upfront HSCT with an Alemtuzumab Based Conditioning Regimen in Children with Acquired Aplastic Anemia: A Retrospective Study from the SFGM-TC (ASH 2024) - "While pediatric patients with AA respond better than adults to immunosuppressive therapy (IST), the long-term event free survival (EFS) is poor due to relapse, cyclosporine dependence, and clonal evolution...Results : 20 children (8 female; median age 10.4 years (IQR : 7.3-12.6)) with severe AA from 9 centers had received the same conditioning with Fludarabine (150 mg/m2), Cyclophosphamide (120 mg/kg) and Alemtuzumab (0.9 mg/kg)...Only one patient required Ruxolitinib treatment for chronic GvHD of the skin (mild stage with eczema at the elbow) with good evolution...One of them required a 2nd line of treatment with Bortezomib to achieve response...Viral infection requiring therapy occurred in 4 patients : EBV infection without post-transplant lymphoproliferative disorders treated by Rituximab in 3 and a disseminated ADV infection requiring cidofovir therapy and third part T cell in the last. One patient experienced severe hemorrhagic cystic. In conclusion, overall..."

Retrospective data • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Atopic Dermatitis • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Dermatology • Endocrine Disorders • Epstein-Barr Virus Infections • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Bowel Disease • Pediatrics

Severe Native BK Virus Nephropathy in a Patient with Primary Myelofibrosis Following Stem-Cell Transplantation (SCT) (KIDNEY WEEK 2025) - "He was empirically treated with IVIG, reduction of immunosuppressive regimen (tacrolimus, mycophenolate, ruxolitinib discontinued) and eventually starting a BK viral specific T-cell therapy. Despite these interventions, the patient had worsening serum creatinine to 7.54 mg/dl with sC5ba9 grossly positive (>2000ng/ml) concerning for transplant-associated TMA for which he was treated with Eculizumab. Cidofovir was considered but deferred due to its renal toxicity...This case adds to the growing literature of cases and queries the therapy following diagnosis beyond just reduction of immunosuppression. This case also highlights the importance for BKVN to be on the differential diagnosis for patients with dysregulated immune systems."

Clinical • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Myelofibrosis • Renal Disease • Solid Organ Transplantation • Thrombocytopenia • Transplantation

Complete resolution of recalcitrant periungual verruca vulgaris using laser-assisted drug delivery. (PubMed, JAAD Case Rep) - No abstract available

Journal • Dermatology

Preliminary Results of a Phase 2a Clinical Trial to Evaluate Safety, Tolerability and Antiviral Activity of Intravenous Brincidofovir (BCV IV) in Immunocompromised Patients with Adenovirus Infection (ASH 2023) - P2 | "Brincidofovir (BCV) is a lipid conjugate of cidofovir, that crosses target cell membranes by means of facilitated and passive diffusion and has a long intracellular half-life. BCV IV was found to be safe and well tolerated in immunocompromised patients. Notably, the potentially serious gastrointestinal and hepatic toxicities described with the oral BCV formulation were not observed. BCV IV was highly effective in a dose-dependent manner, led to rapid clearance of AdV viremia within a short treatment period and provided sustained virologic response."

Clinical • P2a data • Infectious Disease

Atypical Culprit: BK Virus-Induced AKI in a Native Kidney (KIDNEY WEEK 2025) - "He was maintained on tacrolimus, MMF, and prednisone...Management included discontinuation of sirolimus and administration of IVIG due to hypogammaglobulinemia...While reduction of immunosuppression remains the primary treatment, the benefit of adjunctive therapies such as IVIG, cidofovir, and leflunomide remains unclear and requires further research. Clinicians should be aware of BKPyV induced AKI in native kidneys, particularly when more common etiologies have been excluded."

Acute Kidney Injury • Chronic Kidney Disease • Fibrosis • Immunology • Nephrology • Scleroderma • Systemic Sclerosis

Disseminated Mpox Infection in a Vaccinated Kidney Transplant Recipient (KIDNEY WEEK 2025) - "He was on a belatacept-based immunosuppressive regimen and has received Jynneos approximately two years prior to symptom onset...Given the severity of his lesions, he was treated with IV tecovirimat, vaccinia immune globulin (VIVIg), brincidofovir and topical cidofovir...Additional studies are needed to assess the effectiveness of these vaccines in immunocompromised patients. Figure 1: Perioral lesions before treatment (A) and after treatment ~2 months later (B), abdomen (C), right hand (D), and back lesions with typical desquamation (E)"

Clinical • Chronic Kidney Disease • Human Immunodeficiency Virus • Infectious Disease • Renal Disease • Transplantation

Role of Cytomegalovirus (CMV) Immunoglobulin in Managing Multidrug-Resistant CMV Infection in a Kidney Transplant Recipient (KIDNEY WEEK 2025) - "He received induction with thymoglobulin and maintenance immunosuppression with tacrolimus, MMF, and prednisone. Prophylaxis included valganciclovir (VGCV) and TMP-SMX...Despite VGCV therapy and MMF reduction, viral load rebounded to 94,600 IU/mL with resistance to VGCV and cidofovir. Maribavir (MBV) was initiated, resulting in temporary improvement, but viremia rebounded to 404,000 IU/mL...Foscarnet (FOS) was started but failed to reduce viremia...At seven months, he was discharged on letermovir (LTV), but viremia rose to 40,200 IU/mL by ten months...While not curative, CMV-Ig appeared to modulate disease activity, especially when standard options were exhausted or unavailable. This case underscores the importance of adherence, timely resistance testing, and the potential role of immunotherapeutics like CMV-Ig in refractory CMV management."

Clinical • Cardiovascular • Chronic Kidney Disease • Cytomegalovirus Infection • Diabetes • Fibrosis • Gout • Hematological Disorders • Hepatology • Hypertension • Immunology • Infectious Disease • Inflammatory Arthritis • Leukopenia • Liver Cirrhosis • Metabolic Disorders • Rheumatology • Transplantation

BK Breaks the Rules: AKI Due to BK Virus in a Native Kidney (KIDNEY WEEK 2025) - "This case highlights native kidney BK nephropathy in a patient who received Upadacitinib for Crohn’s disease...Despite medical management of electrolyte abnormalities and metabolic acidosis, persistent uremia led to initiation of hemodialysis while cidofovir was administered to treat the viral burden...Yet, the virus has not been directly linked to Upacitinb in the literature until now. With BK viremia leading to acute renal failure such as in our case, it is reasonable to question whether the virus should be further investigated in non-transplant immunosuppression."

Acute Kidney Injury • Asthma • Cardiovascular • Crohn's disease • Endocrine Disorders • Fatigue • Fibrosis • Gastroenterology • Glomerulonephritis • Heart Failure • Hematological Disorders • Hypertension • Immunology • Inflammation • Inflammatory Bowel Disease • Lupus Nephritis • Metabolic Disorders • Myelofibrosis • Nephrology • Renal Disease • Respiratory Diseases