{"title":"AB025. Efficacy of 5-ALA brightness analysis for malignant brain tumor surgery.","authors":"Takashi Kon, Yosuke Sato, Yusuke Kobayashi, Katsuyoshi Shimizu, Tohru Mizutani","doi":"10.21037/cco-24-ab025","DOIUrl":"https://doi.org/10.21037/cco-24-ab025","url":null,"abstract":"<p><strong>Background: </strong>For fluorescence-guided neurosurgery, 5-aminolevulinic acid (5-ALA) is widely used for intraoperative tumor visualization. We quantified the brightness of 5-ALA by Image J and report the pathological results of glioma, and non-tumor lesions.</p><p><strong>Methods: </strong>From 2019 to 2023, we investigated 27 high-grade glioma patients who underwent surgery with 5-ALA. Twenty-three cases of glioblastoma (GBM) and four cases of anaplastic astrocytoma (AA) were examined. The pathological diagnosis was based on the classification of World Health Organization (WHO) 2016. The 5-ALA was administered before surgery, and the 5-ALA brightness was quantified. Other than high-grade glioma, four low-grade gliomas (LGGs), two radiation necrosis, and one inflammation patients were evaluated by Image J.</p><p><strong>Results: </strong>In GBM, the mean brightness was 134.5±65.4, except for one negative case. AA showed the mean brightness with 180.5±51.6. All LGGs showed negative in the brightness. In two radiation necrosis cases, the mean brightness was 139.5±37.4. In one inflammatory case, the brightness was 239, but after the lesion removed, the adjacent brain parenchyma showed bright, and the border was not clear. In one GBM case, the ventricle was opened, and the brightness difference between the tumor and the ventricular wall was observed.</p><p><strong>Conclusions: </strong>5-ALA brightness analysis by Image J would be helpful to distinguish between malignant glioma and LGG, and other non-tumor lesions to support rapid pathological diagnosis. Also, it would be useful to distinguish between the tumor and the ventricle wall. As for radiation necrosis and inflammation, border of the lesion is unclear.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 Suppl 1","pages":"AB025"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281064","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jia Jia Teo, Razan Nossier, Angad Chauhan, Tuan Zea Tan, Vincent Diong Weng Nga
{"title":"AB050. Novel treatment vs. standard of care in melanoma-associated leptomeningeal metastases: a systematic review & network meta-analysis.","authors":"Jia Jia Teo, Razan Nossier, Angad Chauhan, Tuan Zea Tan, Vincent Diong Weng Nga","doi":"10.21037/cco-24-ab050","DOIUrl":"10.21037/cco-24-ab050","url":null,"abstract":"<p><strong>Background: </strong>Melanoma stands as a prevalent instigator of leptomeningeal disease (LMD) within the realm of cancer. Given the poor prognosis accompanying this condition, ongoing trials explore a spectrum of treatment modalities in pursuit of more effective interventions. To ascertain the most effective therapeutic strategies, we aim to compare novel treatments against the current standard of care for melanoma-associated LMD.</p><p><strong>Methods: </strong>A comprehensive search was conducted across multiple databases, including PubMed/Medline, EMBASE, Scopus, ScienceDirect and Web of Science for relevant studies published from January 2014 to January 2024. We included primary research studies, including observational studies, randomised control trials, quasi-experimental design studies, clinical trials, and experimental studies focusing on LMD caused by metastatic melanoma. Data extraction was conducted according to PRISMA guidelines and quality assessment/risk of bias is performed individually using the GRADE method. A network meta-analysis is conducted to evaluate the effects of multiple interventions within the study. Overall survival outcomes were quantified using log hazard ratio.</p><p><strong>Results: </strong>Out of 680 records screened for eligibility, seven carefully chosen studies, meeting our specific inclusion criteria, provide insights into the management of 397 patients grappling with LMD due to metastatic melanoma. These studies vary in design: one observational cohort study with 29 participants, a clinical trial with 25 patients, four retrospective cohort studies ranging from 39 to 190 participants and one experimental study with 24 patients.</p><p><strong>Conclusions: </strong>Despite the escalating breakthroughs of treatment options in melanoma-associated LMD, further studies may be imperative to conclusively determine whether the newer therapeutic options yield superior outcomes compared to the current standard of care treatments.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 Suppl 1","pages":"AB050"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281081","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Keith Gerard Cheng, John Emmanuel Torio, Elmer Jose Meceda
{"title":"AB057. A rare case of anterior skull base metastasis secondary to follicular thyroid carcinoma presenting as proptosis: a systematic review and illustrative case.","authors":"Keith Gerard Cheng, John Emmanuel Torio, Elmer Jose Meceda","doi":"10.21037/cco-24-ab057","DOIUrl":"10.21037/cco-24-ab057","url":null,"abstract":"<p><strong>Background: </strong>Skull base metastasis from follicular thyroid carcinoma (FTC) is uncommon. A single study, encompassing 473 cases of thyroid carcinoma, revealed a mere 2.5% incidence of such metastasis. Much is unknown about FTC skull base metastasis, and a streamlined algorithm is yet to be created.</p><p><strong>Methods: </strong>We present a case of a 63-year-old female, with a history of a non-toxic goiter, complaining of a chronic history of progressive L proptosis, associated with headache and vision loss. History and radiologic findings were incompatible, hence, a biopsy was performed, revealing FTC metastasis. With this experience, we performed a systematic review of available literature for FTC skull base metastasis to help guide management for future cases. Using PRISMA guidelines, a systematic search across PubMed, Google Scholar, and Cochrane Library using MeSH keywords \"Skull base\", \"Metastasis\", and \"Follicular Thyroid Carcinoma\", identified 18 records. Fifteen articles were assessed for eligibility, but only eight studies met the inclusion criteria for qualitative analysis, including demographics, pathological characteristics, surgical approaches, clinical outcomes, and follow-up data.</p><p><strong>Results: </strong>Included studies showcased a consistent age range (43 to 69 years) among patients diagnosed with FTC, with variability in management for the primary malignancy. Metastatic presentation varied depending on tumor location, with symptoms including dysphagia, proptosis, epistaxis, facial dysesthesia, and visual impairment. Tumor size ranged from 3 cm × 3 cm × 2 cm to 6.8 cm × 3.9 cm × 5.3 cm, greatly influencing surgical management strategies, from punch biopsy to complete resection and reconstruction. Adjuvant therapies included combinations of intensity-modulated radiation therapy (IMRT) with immunotherapy, I-131 therapy, oral radioiodine ablation, and radiotherapy alone, with outcomes showing improvement in most cases. Follow-up duration varied from 12 to 60 months, reflecting a wide range of outcome results.</p><p><strong>Conclusions: </strong>FTC skull base metastasis remains to be an uncommon entity in neurosurgery. Its rarity creates a lack of established guidelines and treatment algorithms. A high index of suspicion as well as good history and physical examination skills are necessary to achieve an adequate diagnosis. Multi-disciplinary teams form the cornerstone of a patient-tailored approach to its management.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 Suppl 1","pages":"AB057"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281088","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mohammad Hamza Bajwa, Altaf Ali Laghari, Sufiyan Sufiyan, Wajiha Amin, Arsalan Ahmed, Syed Hani Abidi, Ahmed Gilani, Nouman Mughal, Syed Ather Enam
{"title":"AB059. Molecular signatures for survival prediction in glioma: a prospective, real-world data analysis.","authors":"Mohammad Hamza Bajwa, Altaf Ali Laghari, Sufiyan Sufiyan, Wajiha Amin, Arsalan Ahmed, Syed Hani Abidi, Ahmed Gilani, Nouman Mughal, Syed Ather Enam","doi":"10.21037/cco-24-ab059","DOIUrl":"https://doi.org/10.21037/cco-24-ab059","url":null,"abstract":"<p><strong>Background: </strong>Glioma characterization and follow-up are underreported from low-and-middle-income country centers within the literature. With the recent emphasis on molecular markers for survival prediction, there is a need for robust data exploring molecular epidemiology in these countries. In Pakistan particularly, there is a significant gap in glioma outcomes reporting and survival analysis.</p><p><strong>Methods: </strong>One hundred and sixty-five consecutive glioma patients were enrolled from 2019 onwards; histopathological and molecular analysis was performed on archived formalin-fixed paraffin-embedded (FFPE) blocks for isocitrate dehydrogenase (IDH), P53, α-thalassemia retardation X-linked (ATRX) and Ki-67 immunohistochemical (IHC) markers. Survival analysis was calculated using the Kaplan-Meier method; hazard ratios are reported through a multivariate Cox regression model.</p><p><strong>Results: </strong>Fifty-seven (35%) histopathological diagnoses were revised according to the updated criteria; 30% (n=16) glioblastoma were converted to a new category on re-analysis. IDH wild type (IDH-WT) gliomas had a significantly worse overall survival (log-rank =0.002), with a 2-year survival rate of 60% for IDH-mutant (IDH-M) and 38% for IDH-WT. Significant survival differences were seen for the Ki-67 index (log-rank =0.001) and methylguanine methyltransferase (MGMT) promotor methylation [log-rank =0.027, 2-year survival rate: 100% (methylation detected), 33% (methylation not detected)]. On Cox proportional hazards regression, gross total resection (P<0.001), IDH mutation (P<0.001), and updated histopathological diagnosis (P<0.001) were significant predictors of survival, with good sensitivity and specificity as seen on receiver operating characteristic (ROC) analysis [area under the curve (AUC) =0.86].</p><p><strong>Conclusions: </strong>In our cohort, the revised World Health Organization (WHO) classification shows significant implications on prognosis and implications for treatment. Although these markers are not commonly used in low-and-middle-income country centers, our results strongly support their greater implementation for improved prognostication and reclassification.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 Suppl 1","pages":"AB059"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281090","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kiran Aftab, Salma Asif, Ansar Rahman, Ummul Wara, Faryal Raees, Ahmad Raza Shahid, Amna Farrukh, Ceemal Fareed, Manal Nasir, Rabeet Tariq, Muhammad Sameer, Meher Angez, Zeba Saleem, Komal Naeem, Muhammad Nouman Mughal, Fatima Mubarak, Syed Ather Enam
{"title":"AB093. Pixel-wise classification of glioma using deep learning for accurate tumour mapping on magnetic resonance imaging.","authors":"Kiran Aftab, Salma Asif, Ansar Rahman, Ummul Wara, Faryal Raees, Ahmad Raza Shahid, Amna Farrukh, Ceemal Fareed, Manal Nasir, Rabeet Tariq, Muhammad Sameer, Meher Angez, Zeba Saleem, Komal Naeem, Muhammad Nouman Mughal, Fatima Mubarak, Syed Ather Enam","doi":"10.21037/cco-24-ab093","DOIUrl":"https://doi.org/10.21037/cco-24-ab093","url":null,"abstract":"<p><strong>Background: </strong>Central nervous system (CNS) tumours, especially glioma, are a complex disease and many challenges are encountered in their treatment. Artificial intelligence (AI) has made a colossal impact in many walks of life at a low cost. However, this avenue still needs to be explored in healthcare settings, demanding investment of resources towards growth in this area. We aim to develop machine learning (ML) algorithms to facilitate the accurate diagnosis and precise mapping of the brain tumour.</p><p><strong>Methods: </strong>We queried the data from 2019 to 2022 and brain magnetic resonance imaging (MRI) of glioma patients were extracted. Images that had both T1-contrast and T2-fluid-attenuated inversion recovery (T2-FLAIR) volume sequences available were included. MRI images were annotated by a team supervised by a neuroradiologist. The extracted MRIs thus obtained were then fed to the preprocessing pipeline to extract brains using SynthStrip. They were further fed to the deep learning-based semantic segmentation pipelines using UNet-based architecture with convolutional neural network (CNN) at its backbone. Subsequently, the algorithm was tested to assess the efficacy in the pixel-wise diagnosis of tumours.</p><p><strong>Results: </strong>In total, 69 samples of low-grade glioma (LGG) were used out of which 62 were used for fine-tuning a pre-trained model trained on brain tumor segmentation (BraTS) 2020 and 7 were used for testing. For the evaluation of the model, the Dice coefficient was used as the metric. The average Dice coefficient on the 7 test samples was 0.94.</p><p><strong>Conclusions: </strong>With the advent of technology, AI continues to modify our lifestyles. It is critical to adapt this technology in healthcare with the aim of improving the provision of patient care. We present our preliminary data for the use of ML algorithms in the diagnosis and segmentation of glioma. The promising result with comparable accuracy highlights the importance of early adaptation of this nascent technology.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 Suppl 1","pages":"AB093"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Alexander Grabenbauer, Tiuri E Kroese, Matthias Guckenberger
{"title":"ESO-Shanghai 13: another milestone in the treatment of oligometastatic disease?","authors":"Alexander Grabenbauer, Tiuri E Kroese, Matthias Guckenberger","doi":"10.21037/cco-24-25","DOIUrl":"10.21037/cco-24-25","url":null,"abstract":"","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 4","pages":"62"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142139400","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Daniele Castellani, Leonard Perpepaj, Demetra Fuligni, Giuseppe Chiacchio, Pietro Tramanzoli, Silvia Stramucci, Virgilio De Stefano, Vanessa Cammarata, Simone Cappucelli, Valerio Pasarella, Stefania Ferretti, Davide Campobasso, Vineet Gauhar, Andrea Benedetto Galosi
{"title":"Advancements in artificial intelligence for robotic-assisted radical prostatectomy in men suffering from prostate cancer: results from a scoping review.","authors":"Daniele Castellani, Leonard Perpepaj, Demetra Fuligni, Giuseppe Chiacchio, Pietro Tramanzoli, Silvia Stramucci, Virgilio De Stefano, Vanessa Cammarata, Simone Cappucelli, Valerio Pasarella, Stefania Ferretti, Davide Campobasso, Vineet Gauhar, Andrea Benedetto Galosi","doi":"10.21037/cco-24-52","DOIUrl":"10.21037/cco-24-52","url":null,"abstract":"<p><strong>Background: </strong>Robotic-assisted radical prostatectomy (RARP) is currently a first-line treatment option for men with localized prostate cancer (PCa), at least 10 years of life expectancy, and candidate for curative treatment. We performed a scoping review to evaluate the role of artificial intelligence (AI) on RARP for PCa.</p><p><strong>Methods: </strong>A comprehensive literature search was performed using EMBASE, PubMed, and Scopus. Only English papers were accepted. The PICOS (Patient Intervention Comparison Outcome Study type) model was used; P: adult men with PCa undergoing RARP; I: use of AI; C: none; O: preoperative planning improvement and postoperative outcomes; S: prospective and retrospective studies.</p><p><strong>Results: </strong>Seventeen papers were included, dealing with prediction of positive surgical margins/extraprostatic extension, biochemical recurrence, patient's outcomes, intraoperative superimposition of magnetic resonance images to identify and locate lesions for nerve-sparing surgery, identification and labeling of surgical steps, and quality of surgery. All studies found improving outcomes in procedures employing AI.</p><p><strong>Conclusions: </strong>The integration of AI in RARP represents a transformative advancement in surgical practice, augmenting surgical precision, enhancing decision-making processes and facilitating personalized patient care. This holds immense potential to improve surgical outcomes and teaching, and mitigate complications. This should be balanced against the current costs of implementation of robotic platforms with such a technology.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 4","pages":"54"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142139465","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Rachmat Andi Hartanto, Daniel Agriva Tamba, Rusdy Ghazali Malueka, Sri Sutarni
{"title":"AB071. Predictive value of plasma microRNA-10b and microRNA-21 on chemotherapy toxicity, recurrence, and overall survival in high-grade glioma patients treated with temozolomide.","authors":"Rachmat Andi Hartanto, Daniel Agriva Tamba, Rusdy Ghazali Malueka, Sri Sutarni","doi":"10.21037/cco-24-ab071","DOIUrl":"https://doi.org/10.21037/cco-24-ab071","url":null,"abstract":"<p><strong>Background: </strong>The low level of median survival rate after complete therapy (i.e., surgery and concomitant chemotherapy and radiotherapy) in high-grade glioma (HGG) patients reflects the needs for a better understanding about HGG pathogenesis, including the role of epigenetic in glioma. MicroRNA (miRNA), a small chain non-coding RNA, has been increasingly utilized in the management of other oncology cases and might possess an immense potential in HGG. The expression of miRNA-10b and miRNA-21 (i.e., two miRNAs that are frequently studied due to its involvement in glioma) are higher in HGG patients and their role in regulatory mechanism of glioma has been established. However, the influence of those miRNAs in toxicity, recurrence, and overall survival of HGG patients is still unclear. We aim to assess the predictive value of plasma miRNA-10b and miRNA-21 in the chemotherapy toxicity, recurrence, and overall survival of HGG patients.</p><p><strong>Methods: </strong>This is an observational analytic study using hospital-based mixed cohort approach. The study is conducted in RSUP Dr. Sardjito, Yogyakarta, from January 2021 to December 2024. We prospectively assess the plasma miRNA level from HGG patients who met the inclusive and exclusive criteria. The consecutive sampling is used until the sample size is met. Statistical analysis will be conducted for temozolomide toxicity using Spearman's rank correlation, for recurrence using logistical regression, and for overall survival test.</p><p><strong>Results: </strong>In this ongoing study, we plan to collect samples from 155 HGG patients. As of April 2024, we managed to collect 96 samples (median age of 49 years and 55% of male patients). Most of the patients were diagnosed with World Health Organization (WHO) grade IV tumors (69.3%), with the most common diagnosis was glioblastoma (62%). Most of the patients had unmethylated O6-methylguanine-DNA methyltransferase (MGMT) and wild-type isocitrate dehydrogenase (IDH) status (62% and 57%, respectively). There was no difference in miRNA-21 expression based on MGMT status (methylated or unmethylated), nor IDH status (wild type or mutant), with P=0.39 and P=0.25, respectively. Moreover, we found no significant difference in miRNA-10b expression in both MGMT status and both IDH status (P=0.19 and P=0.09). As for the data regarding toxicity, recurrence, and overall survival was still on the process of data collection.</p><p><strong>Conclusions: </strong>MiRNA is a promising epigenetic modulator that might be utilized in HGG management. A better understanding on the role of miRNA in HGG patients might be able to improve clinical outcome.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 Suppl 1","pages":"AB071"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281002","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jiney Jose, Peter Choi, Maria Tsoli, Anjana Gopalakrishnan, Carina Lee, Thomas I I Park, David Ziegler, William Denny
{"title":"AB005. Development of tumour specific therapy for treatment of diffuse intrinsic pontine glioma (DIPG).","authors":"Jiney Jose, Peter Choi, Maria Tsoli, Anjana Gopalakrishnan, Carina Lee, Thomas I I Park, David Ziegler, William Denny","doi":"10.21037/cco-24-ab005","DOIUrl":"https://doi.org/10.21037/cco-24-ab005","url":null,"abstract":"<p><strong>Background: </strong>Diffuse intrinsic pontine glioma (DIPG), is an aggressive form of paediatric high-grade glioma (pHGG) that affects children below the age of 10 months. The survival period for a child suffering from DIPG has not changed in decades (approximately 10 months). This pattern is similar for most pHGG; even though the survival period is more extended, tumour recurrence and death are almost inevitable. This is primarily due to the presence of the blood-brain barrier (BBB), which blocks the entry of most therapeutics into the brain, and also due to tumour heterogeneity associated with central nervous system (CNS) tumours that blunt the efficacy of targeted therapy. The development of a meaningful cure for paediatric brain cancer hinges on discovering chemotherapy agents that (I) can cross the BBB; (II) accumulates explicitly in tumour tissues; and (III) can block pathways leading to the escape of cancer stem cells, promoting recurrence.</p><p><strong>Methods: </strong>This project aims to develop therapeutics that can cross the BBB, a significant hindrance to delivering medicines across the brain, and specifically target cancer cells without affecting normal brain cells. We will accomplish this by attaching novel dyes possessing tumour specificity to various classes of chemotherapy agents. The compounds will be tested on patient-derived paediatric brain cancer cell lines and the most potent compounds will be progressed to an animal model of DIPG.</p><p><strong>Results: </strong>Several drug-dye conjugates were designed and synthesized to target various aberrant pathways involved in disease initiation and progression of DIPG. These were tested first in patient-derived DIPG cell lines. Several of these drug-dye conjugates showed potent antiproliferative effect in various DIPG cell lines. One of these conjugates is currently undergoing maximum tolerated dose study in an animal model of DIPG.</p><p><strong>Conclusions: </strong>The present work details an effort to develop BBB crossing tumour specific therapeutic agents for the treatment of DIPG. The work has resulted in several promising drug-dye conjugates showing antiproliferative activity in various patient-derived DIPG cell lines, enabling the progression of such conjugates into animal models of DIPG. Such studies will inform the utility of such drug-dye conjugates for application in difficult to treat pHGGs such as DIPG.</p>","PeriodicalId":9945,"journal":{"name":"Chinese clinical oncology","volume":"13 Suppl 1","pages":"AB005"},"PeriodicalIF":2.1,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281036","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}