Cladribine

中国临床肿瘤学会（CSCO）白血病、淋巴瘤及骨髓瘤专家委员会工作会议暨2026年CSCO血液肿瘤学术大会于1月16-18日在海口举办。本次大会汇聚国内领域专家与学者，聚焦白血病、淋巴瘤及骨髓瘤的基础研究与临床实践，围绕诊疗实践中的关注焦点与疑难问题展开深入探讨。会议坚持以临床需求为导向，旨在推动科研创新、拓展国际视野、加强学科建设，促进前沿学术成果向临床转化，从而切实提升患者的生存质量。 会议期间，医会宝肿瘤视界特邀中山大学孙逸仙纪念医院方建培教授，就儿童白血病的独特性和治疗挑战，以及脐带血移植预处理方案的关键要点与未来革新，分享了深刻见解与宝贵临床经验 医会宝肿瘤视界 急性淋巴细胞白血病（ALL）是儿童常见的恶性肿瘤，请您谈谈儿童ALL与成人相比有哪些核心独特性？诊疗中面临的主要挑战是什么？ 方建培教授 儿童ALL的临床特点主要可归纳为以下几点： 第一，在生物学特性上，儿童与成人在基因类型上存在较大差异。例如，ETV6、MLL、FLT3、BCR-ABL等基因异常在儿童患者中有其独特的表现率，与成人明显不同。 第二，儿童患者可根据生物学特征和治疗反应将疾病危险度分为低危、中危和高危，而成人患者通常多属于高危类型。 第三，即使使用相同的化疗方案，儿童对药物的耐受性普遍优于成人，因此同一治疗方案下儿童的治疗效果相对较好。 当前儿童ALL治疗面临的主要挑战： 第一，推进精准诊断与精准治疗。目前需要将儿童ALL进一步区分为普通儿童型与婴儿型，两者在生物学特征上存在较大差异。婴儿ALL中MLL基因重排等异常尤为高发，其发病机制符合“二次打击”学说。因此，当前首要解决的问题是提升婴儿ALL的临床疗效，同时让患者及家属了解该疾病的治疗新进展，从而提高其治疗意愿与治疗依从性。 第二，尽管儿童ALL已属可治性疾病，但医保付费仍是重要瓶颈，导致相当一部分患者因费用问题无法完成标准治疗。此外，对于婴儿ALL，公众对其临床疗效的认知度不及普通儿童ALL，导致治疗放弃率相对较高。因此，需要加强行业内宣传与公众科普，提高对婴儿ALL可治性的认识，引导患者坚持完成治疗。 第三，患者坚持治疗需要配套支持体系，包括费用保障与社会援助等。 第四，目前多数新药研发主要针对成人患者，儿童适应证缺乏，导致儿童ALL患者无法及时获益于成人治疗领域的新进展。即使部分靶向药物可用于儿童，也因未被纳入医保报销范围而需完全自费，限制了其广泛应用。 医会宝肿瘤视界 成功的移植离不开精准的预处理。在儿童ALL患者进行脐带血造血干细胞移植时，其预处理方案的设计需要把握哪些关键要点？ 方建培教授 脐带血造血干细胞移植中，预处理是关键环节。除预处理外，其预防体系也同样重要。本次重点探讨预处理相关内容。方案设计需重点考虑以下方面： 第一，脐带血的细胞数量仅为外周血干细胞或骨髓的约1/10。因此，以脐带血作为供体进行移植时，不可直接沿用外周血干细胞或骨髓移植的预处理方案，而需要调整关键药物策略。 例如，抗胸腺细胞球蛋白（ATG）在外周血干细胞和骨髓移植中，因细胞数量充足，常用较大剂量并在预处理中晚期使用，以降低移植物抗宿主病（GVHD）并促进植入。但脐带血干细胞数量仅为上述两种供体的1/10，若使用同等剂量的ATG，约90%的干细胞可能被清除，这会造成植入困难，即便植入成功，大剂量ATG也会明显增加病毒激活风险，进而因感染引发严重并发症，影响患者生存质量。因此，目前认为在脐带血移植的预处理中，应避免使用ATG；如必须使用，需将用药时间提前至-9天或减少剂量，以使脐带血供体细胞能达到与外周血或骨髓相似的临床效果。 第二，对儿童患者选择脐带血作为供体时，需考虑其生长发育需求，优先采用化疗方案，而非放化疗联合。 第三、在某些情况下，放疗仍是清除肿瘤细胞最有效的手段，可显著降低脐带血移植后的复发风险。如T细胞急性淋巴细胞白血病，尤其对于未完全缓解或伴有中枢神经系统白血病的患者，需将放疗纳入预处理方案。 若采用含放疗的预处理方案，需加强肝小静脉闭塞病（VOD）和血栓性微血管病（TMA）等并发症的预防，例如积极选用前列地尔、低分子肝素甚至去纤苷等药物进行干预。 第四，在设计预处理方案时，应优先选用能够透过血脑屏障的化疗药物，同步杀伤颅内的肿瘤细胞，以保障干细胞植入并降低复发风险。例如，近年来逐步应用于临床研究的克拉屈滨、塞替派等新药，纳入预处理方案后可有效提升脐带血移植成功率。 最后，移植预处理强度评分通常采用清髓性方案标准，评分需控制在4分以内，不建议超过4.5分。 医会宝肿瘤视界 对于未来造血干细胞移植技术的革新以及推广使用，您有何建议和展望？ 方建培教授 未来，造血干细胞移植的可及性有望进一步提升，但移植相关并发症，尤其是对患者生育能力的影响，需引起广泛关注。为此，在干细胞移植技术的更新上提出以下几点建议： 第一，尽量选用避免损伤生殖系统的预处理药物，如白消安与环磷酰胺。近年来相关研究显示，苏消安则可能降低对内分泌系统的损伤。 第二，若需采用放疗作为预处理手段，建议优先采用全骨髓照射（TMI），即以局部骨髓腔放疗替代传统全身放疗（TBI）或全淋巴照射（TLI）。此类精准放疗技术可在实现精准清髓的同时减少副作用。 第三，如条件允许，应在移植前对供者细胞与受者细胞进行相互适应与共培养处理，提升两者的兼容性，使供者细胞更顺利植入受者体内。这有助于减轻受者损伤，好比“和平解放”比“强攻占领”更能保护城市，从而有助于术后恢复和生活质量改善。 最后，应进一步研发基因修饰等新技术。通过基因治疗部分替代传统干细胞移植，将有助于降低治疗副作用，并提升患者的长期生存质量。 本期采访嘉宾详细介绍 方建培 教授 中山大学孙逸仙纪念医院 中山大学博士生导师、教授、主任医师 中山大学孙逸仙纪念医院儿童医学中心学术带头人 国家临床重点专科（地中海贫血专科）负责人  第七届中国儿科卓越贡献医师获得者 国家卫生健康委员会儿童血液病专家委员会副主任委员 中华医学会儿科学分会基层儿科发展委员会主任委员 国家卫健委儿童白血病专家组副组长 国家儿童医学中心血液肿瘤医疗联盟副盟主 中国小儿血液与肿瘤杂志副总编辑 中华儿科杂志、中华实用儿科临床杂志、中国实用儿科杂志、国际儿科学杂志等常务编委/编委 声明：本文由医会宝编辑部根据方建培教授采访内容整理，仅用于交流学习，未经授权禁止转载！ 致：读者朋友 为了不让大家错过医会宝肿瘤视界 每个工作日17:30推送的医学知识和前沿资讯 下面教大家如何第一时间接收到我们的推文： 点击文章开头上方“医会宝肿瘤视界”→点击右上角“…” →点选“设为星标 ★ ” 加上星标 这样就不会找不到我们啦！

VANCOUVER, BC / ACCESS Newswire / January 26, 2026 / BioNxt Solutions Inc. ("BioNxt" or the "Company") (CSE:BNXT)(OTCQB:BNXTF)(FSE:BXT), a bioscience innovator specializing in advanced drug delivery systems, is pleased to announce significant progress in the development of its proprietary lead cladribine oral thin film (ODF) program. Recent study results demonstrating a greater than 40% improvement in bioavailability compared to conventional formulations represent a key development milestone and support the Company's transition toward human clinical evaluation. The results strengthen confidence in BioNxt's sublingual oral thin film platform and mark an important inflection point in the Company's development strategy. Development focus now moves toward human clinical study, which is expected to be more streamlined and efficient because cladribine is an already approved active pharmaceutical ingredient, allowing the Company's clinical work to concentrate on demonstrating bioavailability and bioequivalence rather than repeating large-scale safety and efficacy trials. Advancing Toward Human Clinical Study in Multiple Sclerosis With formulation and preclinical validation having exceeded expectations, BioNxt is now progressing its lead cladribine oral thin film program for Multiple Sclerosis toward a first human clinical study. Current activities are focused on clinical planning, manufacturing readiness, and regulatory alignment to support a disciplined transition into human-focused development. The Company is working closely with an experienced clinical research organization (CRO) to support study design, operational execution, and regulatory compliance. This collaboration is intended to de-risk timelines and ensure high-quality execution as the Multiple Sclerosis program advances toward clinical evaluation. Capital-Efficient Path Toward Commercialization BioNxt's development strategy leverages the established clinical and safety pro cladribine, an already approved therapy with significant commercial precedent. By combining a proven active pharmaceutical ingredient with a differentiated oral thin film delivery format, the Company believes it is pursuing a capital-efficient and execution-driven pathway toward potential commercialization and partnering opportunities. The oral thin film platform is designed to improve patient adherence, simplify administration, and enhance real-world treatment usability, particularly in chronic and neurological diseases where swallowing difficulties and treatment fatigue are common. Expansion into Myasthenia Gravis Enabled by Study Results Importantly, the strength of the recent study results also enables the expansion of BioNxt's cladribine ODF strategy into Myasthenia Gravis (MG), a rare, chronic autoimmune neuromuscular disease in which the immune system disrupts communication between nerves and muscles. MG is characterized by fluctuating muscle weakness that commonly affects the eyes, face, throat, and limbs, often leading to difficulty swallowing, speaking, and chewing, as well as severe fatigue. Myasthenia Gravis is a rare but serious autoimmune neuromuscular disease that affects an estimated 1.4 million people worldwide, based on global prevalence analyses published in peer-reviewed epidemiology studies. These symptoms can make conventional oral tablet administration challenging for many patients, contributing to treatment burden and adherence issues. BioNxt believes its sublingual, needle-free oral thin film delivery format may be particularly well suited for MG patients by simplifying administration and reducing reliance on swallowing intact tablets. BioNxt is repurposing the same cladribine drug and oral thin film delivery technology for MG, allowing the Company to build on existing formulation knowledge and development progress. Because both indications utilize the same active ingredient and delivery platform, BioNxt believes the MG program may follow an accelerated and capital-efficient development pathway relative to a de novo drug program. According to GlobalData, the Myasthenia Gravis market across the seven major markets (7MM) is forecast to reach approximately USD 6.7 billion by 2032, driven by improved diagnosis rates and the introduction of newer, disease-modifying therapies. Addressing Large Autoimmune Markets BioNxt's lead product candidate, BNT23001, is being developed as a sublingual cladribine oral thin film for Multiple Sclerosis (MS), with the objective of providing a swallow-free alternative to oral cladribine tablets such as Mavenclad®. Merck KGaA reported 2024 Mavenclad® net sales of more than USD 1 billion (€1,062 million), and GlobalData estimates the Multiple Sclerosis market generated USD 32.8 billion in 2024 across 68 markets and is projected to reach USD 41.2 billion by 2034. Together, the MS and MG programs highlight the scalability of BioNxt's oral thin film platform and its potential to support multiple high-value autoimmune indications using a focused, platform-driven development strategy. Strategic Outlook With its lead cladribine program in Multiple Sclerosis accelerating toward human clinical study and expansion into Myasthenia Gravis, BioNxt is entering a phase increasingly defined by execution, timelines, and platform-driven value creation. The Company believes this approach positions it well to advance clinical development while maintaining capital discipline and strategic flexibility. About BioNxt Solutions Inc. BioNxt Solutions Inc. is a bioscience innovator focused on next-generation drug delivery platforms, diagnostic screening systems, and active pharmaceutical ingredient development. Its proprietary platforms include sublingual thin films, transdermal patches, oral tablets, and a new targeted chemotherapy platform designed to deliver cancer drugs directly to tumors while reducing side effects. With research and development operations in North America and Europe, BioNxt is advancing regulatory approvals and commercialization efforts, primarily focused on European markets. BioNxt is committed to improving healthcare by delivering precise, patient-centric solutions that enhance treatment outcomes worldwide. BioNxt is listed on the Canadian Securities Exchange: BNXT, OTC Markets: BNXTF andtrades in Germany under WKN: A3D1K3. To learn more about BioNxt, please visit . Investor Relations & Media Contact Hugh Rogers, Co-Founder, CEO and Director Email: investor.relations@bionxt.com Phone: +1 780-818-6422 Web: LinkedIn: Instagram: Cautionary Statement Regarding " Forward-Looking" Information This press release contains forward-looking information within the meaning of applicable Canadian securities laws. Forward-looking information includes, but is not limited to, statements regarding the interpretation and significance of the Company's preclinical study results; the potential advantages of BioNxt's sublingual oral dissolvable film (ODF) technology; the planned progression into human pharmacokinetic and bioequivalence studies; the potential applicability of the Company's drug-delivery platforms to additional therapeutic indications; and statements regarding future development, regulatory, commercialization, licensing, or partnering activities. Forward-looking information is based on management's current expectations, assumptions, and beliefs as of the date of this press release. Such information is subject to a number of risks, uncertainties, and other factors, many of which are beyond the Company's control, that may cause actual results to differ materially from those expressed or implied. These risks and uncertainties include, but are not limited to, scientific and preclinical development risks; the possibility that results observed in animal studies may not be predictive of human outcomes; the timing, cost, conduct, and results of future studies or clinical trials; manufacturing and scale-up risks; reliance on third-party service providers; regulatory and approval risks; intellectual property risks; competitive developments; and general economic and capital market conditions. Readers are cautioned not to place undue reliance on forward-looking information. Except as required by applicable securities laws, BioNxt undertakes no obligation to update or revise any forward-looking information, whether as a result of new information, future events, or otherwise. Mavenclad® is a registered trademark of Merck KGaA. BioNxt Solutions Inc. is not affiliated with, sponsored by, or associated with Merck KGaA. SOURCE: BioNxt Solutions Inc. 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