Sezione di Medicina Genomica, Dipartimento di Scienze della Vita e Sanità Pubblica, Università Cattolica del Sacro Cuore, 00168 Roma, Italy.
Ocular Oncology Unit, Fondazione Policlinico Universitario A. Gemelli-IRCCS, Università Cattolica del Sacro Cuore, 00168 Roma, Italy.
Genes (Basel). 2023 Nov 8;14(11):2055. doi: 10.3390/genes14112055.
Personalized medicine aims to develop tailored treatments for individual patients based on specific mutations present in the affected organ. This approach has proven paramount in cancer treatment, as each tumor carries distinct driver mutations that respond to targeted drugs and, in some cases, may confer resistance to other therapies. Particularly for rare conditions, personalized medicine has the potential to revolutionize treatment strategies. Rare cancers often lack extensive datasets of molecular and pathological information, large-scale trials for novel therapies, and established treatment guidelines. Consequently, surgery is frequently the only viable option for many rare tumors, when feasible, as traditional multimodal approaches employed for more common cancers often play a limited role. Sebaceous carcinoma of the eyelid is an exceptionally rare cancer affecting the eye's adnexal tissues, most frequently reported in Asia, but whose prevalence is significantly increasing even in Europe and the US. The sole established curative treatment is surgical excision, which can lead to significant disfigurement. In cases of metastatic sebaceous carcinoma, validated drug options are currently lacking. In this project, we set out to characterize the mutational landscape of two sebaceous carcinomas of the eyelid following surgical excision. Utilizing available bioinformatics tools, we demonstrated our ability to identify common features promptly and accurately in both tumors. These features included a Base-Excision Repair mutational signature, a notably high tumor mutational burden, and key driver mutations in somatic tissues. These findings had not been previously reported in similar studies. This report underscores how, in the case of rare tumors, it is possible to comprehensively characterize the mutational landscape of each individual case, potentially opening doors to targeted therapeutic options.
个性化医学旨在根据受影响器官中存在的特定突变,为个体患者开发定制的治疗方法。这种方法在癌症治疗中已被证明是至关重要的,因为每个肿瘤都携带有独特的驱动突变,这些突变可以对靶向药物产生反应,在某些情况下,还可能对其他治疗方法产生耐药性。特别是对于罕见疾病,个性化医学有可能彻底改变治疗策略。罕见癌症通常缺乏大量的分子和病理信息数据集、新型疗法的大规模试验以及既定的治疗指南。因此,对于许多罕见肿瘤,手术往往是唯一可行的选择,特别是在可行的情况下,因为传统的多模式方法对于更常见的癌症往往作用有限。眼睑的皮脂腺癌是一种极其罕见的眼部附属组织癌症,最常发生在亚洲,但即使在欧洲和美国,其发病率也在显著增加。唯一确定的治疗方法是手术切除,这可能导致严重的毁容。对于转移性皮脂腺癌,目前缺乏有效的药物选择。在这个项目中,我们着手研究了两个经手术切除的眼睑皮脂腺癌的突变景观。利用现有的生物信息学工具,我们展示了我们能够迅速而准确地识别两个肿瘤中的常见特征的能力。这些特征包括碱基切除修复突变特征、显著高的肿瘤突变负担以及体细胞中的关键驱动突变。这些发现以前在类似的研究中没有报道过。本报告强调了在罕见肿瘤的情况下,如何全面描述每个个体病例的突变景观,从而有可能为靶向治疗选择打开大门。