Jan 16th, 2022 - WAIKOLOA, Hawaii — It is important for physicians to consider “the perils that dwell” in the diagnostic management of patients with uveitis, Steven Yeh, MD, said during a presentation at Hawaiian Eye 2022. “With these patients being very complex oftentimes, we really just don’t want to miss something,” Yeh said. To better identify “the pearls amongst the perils” in uveitis diagnosis, it is impo...

Jan 29th, 2023 - RNA methylation (RM) is a crucial post-translational modification (PTM) that directs epigenetic regulation. It mostly consists of N1-methyladenosine (m1A), 5-methylcytosine (m5C), N3-methylcytidine (m3C), N6-methyladenosine (m6A), and 2'-O-methylation (Nm). The "writers" mainly act as intermediaries between these modifications and associated biological processes. However, little is known about ...

Jan 29th, 2023 - Defects in DNA damage repair (DDR) pathways lead to genomic instability and oncogenesis. DDR deficiency is prevalent in esophageal squamous cell carcinoma (ESCC), but the effects of DDR alterations on mutational processes and tumor immune microenvironment in ECSS remain unclear. Whole-exome and transcriptome sequencing data of 45 ESCC samples from Taizhou, China, were used to identify genomic v...

Jan 28th, 2023 - The chimeric enzyme SETMAR (or Metnase) has been associated with several DNA processes, including DNA damage repair through the non-homologous joining pathway and suppression of chromosomal translocation in mouse fibroblasts. SETMAR overexpression has been reported in certain cancers suggesting that it might contribute to the establishment or progression of these cancers. In leukemia, the SETMA...

Jan 28th, 2023 - Silicosis is one of the most frequent, rapidly developing, and lethal types of pneumoconiosis. However, our understanding of the underlying mechanisms of its pathogenesis and progress remains unclear. We investigated the fundamental processes of silicosis incidence and progression using a combination of lung function testing, histopathology, 16 S rRNA, untargeted metabolomics, and cytokine chip...

Jan 27th, 2023 - PRIMARY OBJECTIVES: I. To determine a maximum tolerated dose or maximum administrated dose of anti-lymphocyte activation gene-3 (LAG-3) antibody (BMS-986016) (anti-LAG-3 monoclonal antibody BMS-986016) and anti-cluster of differentiation 137 (CD137) antibody (BMS- 663513) (urelumab) given independently and in combination with anti-programmed death-1 (PD-1) antibody (nivolumab, BMS-936558) safel...

Jan 27th, 2023 - PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) and dose limiting toxicity (DLT) of ipilimumab in combination with ibrutinib in patients with CLL/small lymphocytic lymphoma (SLL)/RT. (Part A) II. To determine the MTD and DLT of ipilimumab in combination with nivolumab and ibrutinib in patients with CLL/SLL/RT. (Part B) SECONDARY OBJECTIVES: I. To determine the efficacy (res...

Jan 27th, 2023 - Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation of the skin, and precocious pu...

Jan 27th, 2023 - PRIMARY OBJECTIVES: I. To determine the maximal tolerated dose (MTD) of allogeneic bone marrow-derived human mesenchymal stem cells (BM-hMSCs) loaded with the oncolytic adenovirus DNX-2401 (BM-hMSCs-DNX2401) administered by intra-arterial injection (i.e., transfemoral endovascular intracranial injection) in patients with recurrent glioblastoma (GBM), IDH-mutant astrocytoma grade 4, gliosarcoma,...

Jan 27th, 2023 - This is a prospective, multi-center, Phase II study of hematopoietic cell transplantation (HCT) using human leukocyte antigen (HLA)-mismatched unrelated donors (MMUD) for peripheral blood stem cell transplant in adults and bone marrow stem cell transplant in children. Post-transplant cyclophosphamide (PTCy), tacrolimus and mycophenolate mofetil (MMF) will be used for for graft versus host disea...

Jan 27th, 2023 - Primary: To evaluate the long-term safety and tolerability of seladelpar Secondary: To evaluate the long-term efficacy of seladelpar To evaluate the effect of seladelpar on patient-reported outcomes (pruritus)

Jan 27th, 2023 - PRIMARY OBJECTIVES: I. Define the test - retest variance of tumor fludeoxyglucose (FDG) uptake using double baseline 18F-FDG PET imaging (18 to 54 hours apart) in patients with EGFR activated recurrent glioblastoma. II. After defining #1, evaluate whether osimertinib can create a statistically significant decrease in glucose utilization as determined using early, post dosing (24-72 hour) FDG-PE...

Jan 27th, 2023 - PRIMARY OBJECTIVES: I. To determine the recommended phase II dose of alisertib (MLN8237) and bortezomib when combined with rituximab in patients with relapsed/refractory mantle cell and B-cell low grade non-Hodgkin lymphoma. SECONDARY OBJECTIVES: I. To describe the rate of overall response (complete response and partial response) for patients with relapsed/refractory mantle cell and B-cell low ...

Jan 27th, 2023 - PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose/recommended phase 2 dose (MTD/RP2D) of selumetinib sulfate (selumetinib) + vinblastine sulfate (vinblastine) for children with progressive or recurrent low-grade gliomas (LGGs). II. To determine if selumetinib + vinblastine will lead to improved event-free survival (EFS) outcome compared with selumetinib alone for children with prog...

Jan 27th, 2023 - PRIMARY OBJECTIVES: I. To evaluate the safety and tolerability of the blinatumomab given in combination with nivolumab alone, or in combination with both nivolumab and ipilimumab in subjects with poor-risk, relapsed or refractory CD19+ pre-B cell acute lymphoblastic leukemia (ALL) or CD19+ mixed phenotype acute leukemia (MPAL). II. To determine the maximum tolerated dose (MTD) of the combinatio...

Jan 27th, 2023 - This is a multicenter, randomized, placebo-controlled, Phase 2 study to evaluate the pharmacologic effect, efficacy, and safety of SPI-62 in subjects with ACTH-dependent Cushing's syndrome. Each subject who provides consent and meets all inclusion and exclusion criteria will participate in 3 periods: a 28-day screening period (Days -35 to -8), a 7-day baseline period (Days -7 to -1), and a 24-w...

Jan 27th, 2023 - This study is a first-in-human, phase 1/2, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve th...

Jan 27th, 2023 - Study Description: This study will evaluate the clinical and laboratory effects of immunotherapies in patients who recovered from acute mildmoderate COVID-19 infection but still have persistent neurologic symptoms. Despite clinical recovery from the acute infection, some individuals continue to experience ongoing symptoms, at times several months after recovery, and many of these symptoms are n...

Jan 27th, 2023 - The microbiome-derived therapeutic vaccine concept utilized in conjunction with anti-Programmed cell Death protein 1 (PD1) blockade is an innovative option for testing of a rational immunotherapy in colorectal cancer. The concept as such, including the combination with nivolumab, has already been tested in the clinical setting (i.e. in recurrent glioblastoma and adrenal tumors) and shown to be ...

Jan 27th, 2023 - Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) is a chronic and debilitating urological complex of disorders characterized by symptoms of bladder pain or discomfort, mostly upon bladder filling, and often accompanied by lower urinary tract symptoms (LUTS). This study will assess how safe and effective BOTOX (onabotulinumtoxinA) is in treating IC/BPS. Adverse events and change in disease s...