The goal of this clinical research study is to learn if the combination of ruxolitinib with cladribine and venetoclax can help to control the disease in patients with R/R T-PLL.

开始日期2026-06-01

申办/合作机构

The University of Texas MD Anderson Cancer Center

CTIS2024-519700-28-01

/ Not yet recruiting临床2期IIT

The HIt HArd and hiT early in multiple sclerosis trial – HiHat trial

开始日期2026-01-01

申办/合作机构-

CTIS2024-512503-39-00

/ Recruiting临床4期

MRD registry and multicenter, randomized controlled pragmatic trial of standard intensity versus reduced intensity consolidation treatment in MRD-negative patients with AML or CLL (RESOLVE trial).

开始日期2025-12-19

申办/合作机构-

100 项与克拉屈滨相关的临床结果

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100 项与克拉屈滨相关的转化医学

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100 项与克拉屈滨相关的专利（医药）

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2,847

项与克拉屈滨相关的文献（医药）

2026-05-01·Neurology-Neuroimmunology & Neuroinflammation

Integrin β1 Demarks Precursors of Brain-Residing Antibody-Secreting Cells in Multiple Sclerosis

Article

作者: van Hasselt, Yifan ; Bindels, Eric ; van Luijn, Marvin M. ; de Beukelaar, Janet ; Wierenga-Wolf, Annet F. ; Smolders, Joost ; Wokke, Beatrijs ; Melief, Marie-José ; Smets, Ide ; Kuiper, Kirsten L. ; Klein Kranenbarg, Romy A.M. ; van de Werken, Harmen J.G. ; Bogers, Laurens ; Rip, Jasper

BACKGROUND AND OBJECTIVES:

Multiple sclerosis (MS) is characterized by CXCR3⁺ memory B cells that infiltrate the CNS to mature into antibody-secreting cells (ASCs). It remains elusive how to benefit from this disease hallmark as a prognostic tool. We aimed to uncover markers that reflect B-cell entry and maturation in the CNS and, on that basis, explore associations with response to high-efficacy MS therapies.

METHODS:

Ex vivo single-cell technologies were applied to blood samples and postmortem CNS suspensions from MS and control donors, either treated or untreated. In addition, in vitro assays were performed on blood samples from healthy individuals to functionally asses B-cell activation, differentiation, and transmigration.

RESULTS:

Single-cell RNA sequencing revealed ITGB1 (integrin β1/CD29) as a major discriminator of CXCR3⁺ memory B cells in blood, which was validated using spectral flow cytometry. CD29⁺CXCR3⁺ memory B cells preferentially crossed a brain microvascular endothelial layer and were highly receptive to T-cell help to become ASCs in vitro. In contrast to blood, CD29 and CXCR3 coexpression was restricted to ex vivo ASCs derived from CSF, meninges, and brain tissue of people with MS, which was supported by enhanced CD29 upregulation on in vitro-differentiated ASCs. While CD29 was downregulated on CXCR3⁺ memory B cells that accumulated in the blood of natalizumab-treated patients, CD29 levels were only reduced on CXCR3⁺ memory B cells that repopulated in cladribine-treated patients without early disease activity.

DISCUSSION:

These findings put forward CD29 as a putative marker on CXCR3⁺ precursors of CNS-residing ASCs for predicting treatment responses in MS.

2026-04-01·Multiple Sclerosis and Related Disorders

Treatment outcomes of high-efficacy disease-modifying therapies in patients with relapsing-remitting multiple sclerosis: A longitudinal observational study

Article

作者: Alroughani, Raed ; Ahmed, Samar Farouk

BACKGROUND:

The evolution of high-efficacy disease-modifying therapies (HE-DMTs) has significantly shifted the management of relapsing-remitting multiple sclerosis (RRMS). Real-world evidence highlighted the role of HE-DMTs in improving disability and controlling disease activity.

OBJECTIVES:

To assess the real-world effectiveness, treatment patterns, and discontinuation patterns of HE-DMTs, including cladribine, natalizumab, ocrelizumab, alemtuzumab, fingolimod, and rituximab in RRMS patients.

METHODS:

This is a retrospective, observational, longitudinal, multicenter study that included 1,408 RRMS patients treated with HE-DMTs. Data were extracted from the Kuwaiti national MS registry. The primary objective of this study was to assess the proportion of patients with relapse-free status at 12 months. Secondary objectives included confirmed disability progression (CDP), confirmed disability improvement (CDI), Expanded Disability Status Scale (EDSS) changes, proportion of patients with no evidence of disease activity (NEDA-3), and treatment discontinuation patterns. Statistical analysis included descriptive analysis, paired-sample tests, and McNemar's tests.

RESULTS:

After 12 months of treatment, first-line HE-DMTs resulted in high relapse-free (≥80%) and NEDA-3 (≥75%) rates, especially with ocrelizumab (90.6%, 82.7%, respectively) and natalizumab (88.4%, 81.9%, respectively). Significant EDSS reductions were achieved with natalizumab (-0.80), ocrelizumab (-0.46), and rituximab (-0.66) in patients receiving first-line HE-DMTs (p < 0.001). CDI rates were the highest with alemtuzumab (40.6%) and rituximab (22.1%) in the first-line setting. Discontinuation rates were highest for alemtuzumab due to scheduled stopping (86.9%), fingolimod due to adverse events (17.3%), and natalizumab due to JC virus sero-positivity (65.7%). Ocrelizumab had the lowest discontinuation rate (5.4%), mainly due to pregnancy confirmation or planning (56.5%).

CONCLUSION:

Early initiation of HE-DMTs in RRMS patients had a significant clinical impact on disease activity and disability. Treatment effectiveness parameters were lower with later lines of therapy, highlighting the importance of early therapeutic intervention. High tolerability varied among DMTs, demonstrating the need for individualized treatment decisions.

2026-04-01·Multiple Sclerosis and Related Disorders

Real-world experience of cladribine treatment in relapsing multiple sclerosis: A Romanian multicenter nationwide study

Article

作者: Barcutean, Laura ; Panea, Cristina ; Lungu, Mihaela ; Pop, Marian ; Simu, Mihaela ; Dulamea, Adriana ; Fulop, Andrei ; Sabau, Monica ; Albu, Valeria ; Maier, Smaranda ; Tiu, Cristina ; Vacaras, Vitalie ; Filipoiu, Aurelia ; Lupu, Raluca ; Roman-Filip, Corina ; Vrinceanu, Oana ; Davidescu, Irene ; Ignat, Bogdan ; Balasa, Rodica ; Sirbu, Carmen Adella

MATERIALS AND METHODS:

We conducted a multicenter, observational, post-marketing, real-world cohort study in Romania that included all patients with MS (PwMS) who completed at least one cycle of CLD with at least three months of follow-up as of September 2024. Demographic variables and MS clinical characteristics regarding each patient were recorded. No Evidence of Disease Activity (NEDA-3) was assessed at one and two years after CLD initiation. The primary outcome was the proportion and characteristics of PwMS achieving NEDA-3 at 12 and 24 months after CLD initiation and the identification of predictors of failure in achieving NEDA-3.

RESULTS:

A total of 348 PwMS cases were included, of which CLD was the first DMT option in 129 (37.1%). At one year, 76.74% of the patients with complete available data fulfilled all criteria, achieving NEDA-3 status, with similar proportions between previously DMT-treated PwMS and naïve PwMS. In the univariate analysis, the onset of long-tract involvement, as compared to infratentorial (OR = 6.32, p = 0.01), and the number of Gd-enhancing lesions (OR = 1.12, p = 0.02), were significantly associated with increased odds of not achieving NEDA-3 at one year. In the multivariate model, long tracts (OR = 10.28, p = 0.006), higher baseline EDSS (OR = 1.39, p = 0.01), and longer disease duration (OR = 0.92, p = 0.04) were independent predictors.

CONCLUSIONS:

The initiation of CLD treatment was associated with achieving NEDA-3 at one year in >70% of cases. CLD demonstrated an improvement in MS control, irrespective of the patients' demographics, RMS history, or prior DMT exposure. CLD treatment has a favorable safety profile and a high level of patient adherence.

199

项与克拉屈滨相关的新闻（医药）

2026-02-11

·罕见病新进展

备孕与妊娠期多发性硬化用药如何权衡？一文梳理关键决策要点

下文将讨论多发性硬化（MS）常用治疗药物的重要考量因素，并在表1中进行总结。抗CD20单克隆抗体抗CD20药物，如奥瑞利珠单抗、利妥昔单抗、奥法妥木单抗以及ublituximab，通过清除B细胞发挥作用。近年来，抗CD20单克隆抗体已逐渐成为MS的一线治疗选择。欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）目前建议在尝试受孕前设置不同的停药清除期（washout period），范围约为4~12个月，专家共识指南也常反映出这种差异化的建议。然而，当更深入地分析这一类药物的特性时，可以提出在受孕前采用更短停药间隔的合理性。首先，免疫球蛋白G（IgG）类单克隆抗体与内源性IgG类似，需要通过胎盘上的Fc受体主动转运才能进入胎儿体内。而在妊娠最初20~22周内，Fc受体表达水平较低，因此这一阶段IgG通过胎盘的转运极为有限。其次，抗CD20单克隆抗体在血清中的终末半衰期约为19~26天。根据“五个半衰期原则”，若在接近受孕时给药，大部分药物将在能够通过胎盘转运之前被机体清除。第三，抗CD20治疗的药效（即B细胞耗竭）在输注后至少可持续6个月。也就是说，即便血清中已检测不到药物（药代动力学层面），其药效学作用仍会持续相当长时间，这一点具有重要意义。综合来看，这些发现提示：在末次治疗与尝试受孕之间设置较短的时间间隔，既可能在备孕及妊娠期间为母体提供更好的复发保护，又可将胎儿暴露风险降至最低。与此相一致，一项最新研究分析了855名在末次月经前3个月内接受奥瑞利珠单抗输注的MS女性所生婴儿的结局。结果显示，母体使用奥瑞利珠单抗并未增加不良妊娠或新生儿结局风险。因此，更新后的共识指南建议，在尝试受孕前可采用更短的停药时间，一般为3个月甚至更短，以在限制胎儿暴露的同时，为患者在受孕及早孕期提供持续治疗保护。新出现的管理趋势还包括：将给药时间与患者月经周期相协调，并在每次输注或注射前进行妊娠筛查。淋巴细胞迁移抑制治疗那他珠单抗为每月一次的静脉输注治疗，芬戈莫德为每日口服药物，这两类药物通过阻止淋巴细胞进入中枢神经系统（CNS）而发挥作用。两者在停药后均存在炎症反跳和复发风险，甚至在妊娠期间也可能发生，因此需要谨慎管理。与抗CD20药物类似，那他珠单抗属于单克隆抗体，在妊娠前20周通过胎盘转运的可能性较低。然而，与抗CD20药物不同，停用那他珠单抗与炎症反跳风险增加密切相关，且严重复发可在妊娠期间发生。在此背景下，若考虑停用那他珠单抗，与受孕前停药相比，在妊娠第一孕期后再停药可降低复发风险；若持续用药至妊娠30周，则可提供额外保护。鉴于持续用药可改善母体疾病稳定性，还需同时考虑胎内暴露对新生儿结局的影响。目前研究未发现其会显著增加早产或先天畸形风险，但超过一半暴露于该药的新生儿可能出现一过性贫血和血小板减少，尤其是在母体治疗持续至妊娠30周之后。总体而言，对于正在使用那他珠单抗且有生育意愿的女性，为尽量降低停药后的反跳风险，可考虑以下策略：在受孕前转换至“桥接”疾病修正治疗（如抗CD20单克隆抗体）；或延长那他珠单抗给药间隔并持续使用至妊娠34周（此时胎盘抗体转运最为显著）。若采用桥接策略，理想情况下应在最后一次那他珠单抗给药约4周后启动抗CD20治疗。由于抗CD20治疗起效较快，在完成基线及6个月输注周期后（约6个月），即可开始尝试受孕。这一临床情境凸显了医患之间以及与产科团队共同决策的重要性，需充分沟通停药反跳风险与潜在新生儿风险。与那他珠单抗类似，停用芬戈莫德及其他S1P受体调节剂同样与疾病反跳风险增加相关。据报道，停用该类DMT的女性中，约31%在妊娠期间发生复发，44%在产后第一年内出现复发。芬戈莫德在妊娠期禁用。动物研究显示，即使在低于人用推荐剂量的情况下，暴露于芬戈莫德也可导致胎儿发育异常；上市后数据亦提示，在整个妊娠期使用芬戈莫德时，先天畸形风险可能增加约两倍。因此，鉴于潜在胎儿毒性风险，建议在受孕前至少停用芬戈莫德2个月。在此情境下，同样可在受孕前转换至替代“桥接”DMT（如抗CD20单克隆抗体），以帮助稳定病情，并降低妊娠前、妊娠期及产后反跳风险。免疫重建治疗阿仑珠单抗（alemtuzumab）是一种靶向CD52的单克隆抗体，克拉屈滨（cladribine）是一种嘌呤类似物，可通过干扰DNA合成并诱导细胞死亡，主要作用于B细胞和T细胞。这两种药物被用于MS的免疫重建治疗。这类治疗的特点在于：即使在体内不再存在循环药物的情况下（如备孕和妊娠期间），仍可为母体提供较长时间的疾病保护。关于停药至受孕之间的时间间隔，欧洲EMA目前建议阿仑珠单抗停药后至少等待4个月再尝试受孕，尽管其胎盘转运特点与其他IgG类单克隆抗体相似。目前关于胎内暴露后新生儿结局的安全性数据仍较为有限。虽然在妊娠前20~22周内通过胎盘转运的药物暴露可能较低，但阿仑珠单抗还存在诱发自身免疫性甲状腺炎的风险，一旦发生，将对胎儿带来额外风险。由于在动物研究中观察到致畸作用，EMA和FDA均将克拉屈滨列为妊娠期禁用药物，通常建议停药后至少采取6个月避孕措施。在药物研发阶段，关于活产结局的数据非常有限；但近年来的真实世界研究显示，在受孕前6个月内使用克拉屈滨并未发现妊娠结局或先天畸形风险增加。此外，克拉屈滨还可影响生殖细胞，包括生殖母细胞、精子细胞和成熟精子，因此在完成克拉屈滨治疗后，需约3个月才能完成一个完整的精子生成周期。EMA和FDA建议，男性在接受克拉屈滨治疗后，应额外等待3个月，即在完成治疗后共计6个月再尝试生育。目前关于父方暴露克拉屈滨对胎儿结局的资料极为有限，但已有记录的妊娠均获得活产结局。一线自我注射治疗干扰素β（IFN-β）是最早获得美国FDA批准用于治疗MS的药物之一，其作用机制是通过调节抗原呈递和T细胞增殖来调控免疫反应。FDA和欧洲EMA均未建议在使用该药期间必须采取避孕措施。由于IFN-β是目前临床应用时间最长、使用最广泛的MS治疗药物之一，关于其妊娠期使用的安全性数据相对充分。在超过3500例暴露于IFN-β的妊娠中，未观察到妊娠结局不良或先天畸形风险增加。需要注意的是，大多数观察到的患者仅在受孕前或妊娠第一孕期使用IFN-β，因此关于妊娠中后期持续使用的安全性数据仍较有限。对于在使用IFN-β期间受孕的患者，可根据具体情况建议在确认妊娠后继续或停止治疗。醋酸格拉替雷（GA）是一种免疫调节剂，被认为可通过在淋巴细胞进入血脑屏障前抑制外周T细胞增殖，从而减轻中枢神经系统炎症。与IFN-β类似，GA也是最早获得FDA批准的MS治疗药物之一，因此其妊娠相关安全性数据较其他DMT类别更为丰富。多项研究显示，在受孕时或妊娠第一孕期暴露于GA的新生儿，并未出现不良妊娠结局或先天畸形风险增加。根据EMA和FDA建议，在使用GA期间无需强制避孕，患者在确认怀孕后亦可根据临床情况选择继续或停止治疗。其他口服治疗富马酸二甲酯（DMF）及其他富马酸类药物为每日口服制剂，被认为可通过诱导抗炎免疫反应发挥作用，尽管其确切作用机制尚未完全明确。针对缓释型DMF的初步临床前安全性分析显示，在动物模型中未发现致畸证据。随后开展的临床研究亦未显示在妊娠第一孕期暴露于DMF会增加先天畸形或不良妊娠结局风险。然而，目前关于妊娠第一孕期之后持续暴露于DMF的安全性数据仍较为有限。鉴于安全性证据不足，EMA和FDA目前均不建议在妊娠期间使用该药。由于富马酸类药物半衰期较短，可在尝试受孕时或妊娠试验阳性后停用。特立氟胺为每日口服药物，通过抑制嘧啶合成从而减少B细胞和T细胞增殖。由于动物研究显示，在低于人用推荐剂量的情况下即可出现致畸和胚胎致死作用，因此特立氟胺在妊娠期禁用。除建议在用药期间采取避孕措施外，还需持续避孕直至血清药物浓度降至<0.02 mg，这一过程在停药后可能需8~24个月。鉴于清除期较长，对于希望尽早受孕的患者，或在停药后8~24个月内意外怀孕者，建议采用加速清除方案。目前关于特立氟胺暴露后妊娠及胎儿结局的小型研究数量有限，且受样本量小、选择性终止妊娠以及加速清除方案使用等因素影响。此外，在接受特立氟胺治疗的男性精液中亦可检测到该药物。虽然来自男性暴露的胎儿毒性风险被认为较低，但FDA（而非EMA）仍建议在用药期间采取屏障避孕措施。新兴治疗 Tolebrutinib是一种靶向B细胞的BTK抑制剂。Ⅲ期临床试验显示，在复发缓解型多发性硬化（RRMS）中，其疗效与特立氟胺相当；而在进展型MS中，其疗效优于安慰剂。因此，Tolebrutinib近期已在阿联酋获批用于治疗进展型MS，目前正等待美国FDA的监管审批。尽管目前该药的临床试验排除了妊娠及哺乳期患者，但关于其他BTK抑制剂（如伊布替尼和阿可替尼）已有一定安全性数据，这两种药物已获批用于治疗血液系统恶性肿瘤。鉴于此，并结合目前人类安全性数据有限，FDA和EMA均建议在使用该类药物治疗期间采取避孕措施。关于停药后需持续避孕的时间，各监管机构和不同药物之间建议不尽相同，范围从无需额外避孕到停药后12周不等。不过，基于这类药物较短的半衰期（分别约4~6小时和1~3小时），根据“五个半衰期原则”，理论上其清除期应相对较短。参考文献： McConville, K., Bove, R. Multiple Sclerosis in Women: Impact of Different Life Stages on Treatment Decisions. CNS Drugs (2026). https://doi.org/10.1007/s40263-025-01246-9 End *版权声明：版权属罕见病新进展所有，欢迎个人转发分享，其他任何媒体、网站如需转载或引用本网版权所有内容须获得授权且在醒目位置处注明“转自：罕见病新进展”。责任编辑｜木子李排版编辑｜扎依尔欢迎评论区留言 👇👇

2026-02-06

·医会宝肿瘤视界

脐带血移植预处理怎么选？方建培教授解读 5 大核心要点与未来新突破

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！

CSCO会议

2026-01-27

·BioSpace

BioNxt Accelerates ODF for Multiple Sclerosis Toward Human Clinical Study with >40% Bioavailability Improvement and Myasthenia Gravis Expansion

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. View the original press release on ACCESS Newswire

临床结果

100 项与克拉屈滨相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D01370	克拉屈滨	L04AA40 L01BB04	DB00242

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
多发性硬化症	美国	EMD Serono, Inc.	2019-03-29
复发-缓解型多发性硬化	加拿大	EMD Serono, Inc.	2017-11-30
复发性多发性硬化	欧盟	Merck Europe BV	2017-08-22
复发性多发性硬化	冰岛	Merck Europe BV	2017-08-22
复发性多发性硬化	列支敦士登	Merck Europe BV	2017-08-22
复发性多发性硬化	挪威	Merck Europe BV	2017-08-22
贫血	中国	浙江海正药业股份有限公司	2005-11-15
中性粒细胞减少	中国	浙江海正药业股份有限公司	2005-11-15
血小板减少症	中国	浙江海正药业股份有限公司	2005-11-15
套细胞淋巴瘤	日本	Clinigen KK	2002-12-16
非霍奇金淋巴瘤	日本	Clinigen KK	2002-12-16
毛细胞白血病	美国	Janssen Pharmaceuticals, Inc.	1993-02-26

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
重症肌无力	临床3期	美国	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	中国	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	日本	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	阿根廷	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	澳大利亚	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	比利时	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	保加利亚	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	法国	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	德国	Merck Healthcare KGaA	2024-06-25
重症肌无力	临床3期	希腊	Merck Healthcare KGaA	2024-06-25

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04708054 (Tandem Meetings ASTCT and CIBMTR2026)	临床2期	急性髓性白血病	50	Myeloablative Fractionated Busulfan, Fludarabine, Cladribine, Thiotepa, and Venetoclax (Cladillac) Conditioning	窪獵蓋壓艱鏇積願艱鑰(製餘網壓蓋襯衊窪選衊) = 繭鹹鹹夢衊艱範蓋繭願糧糧鬱範構顧遞鏇窪淵 (願齋膚範選構壓蓋廠襯, 48 ~ 75) 更多	积极	2026-02-04
				Myeloablative Fractionated Busulfan, Fludarabine, Cladribine, Thiotepa, and Venetoclax (Cladillac) Conditioning (TP53 wildtype)	窪獵蓋壓艱鏇積願艱鑰(製餘網壓蓋襯衊窪選衊) = 鹹網衊範鹹鹹鏇願獵簾糧糧鬱範構顧遞鏇窪淵 (願齋膚範選構壓蓋廠襯 ) 更多
NCT01473797 (Pubmed \| Eur Respir J)	临床2期	郎格罕细胞组织细胞增生症	10	Cladribine (Pulmonary Langerhans Cell Histiocytosis)	選遞繭鹽顧構鬱繭顧餘(網艱繭網獵蓋鑰衊願積) = 窪構衊觸願網構襯繭醖築顧齋選鏇醖網簾衊選 (齋鹹網醖鹽襯廠顧觸餘 ) 更多	积极	2026-01-29
ASH2025	N/A	毛细胞白血病一线	279	Cladribine	鹽蓋衊網繭窪窪窪淵齋(齋繭繭構網顧獵糧鏇糧) = 鹽廠鑰獵範繭願簾醖積繭糧選願餘壓築齋鹹糧 (糧繭襯膚夢積淵齋餘鹽 ) 更多	积极	2025-12-06
ASH2025	N/A	急性髓性白血病	33	CLAG-M	範選鏇顧襯範製壓構膚(積簾糧膚製襯淵醖簾繭) = 艱憲範積夢廠壓齋壓鹽齋壓齋艱繭餘齋蓋獵鹹 (顧觸網憲淵鬱獵願選餘 ) 更多	积极	2025-12-06
				Cladribine-LDAC	積範鑰窪築觸膚壓餘窪(築廠簾膚壓獵製蓋夢糧) = 簾膚齋顧衊糧鏇鑰鏇鏇遞廠艱衊淵觸蓋鹽網淵 (壓膚膚鏇築觸遞鑰廠鬱 )
NCT01515527 (ASH2025)	临床2期	急性髓性白血病	15	Cladribine + low-dose cytarabine (Renal impairment (CrCl <50 mL/min) + Acute Myeloid Leukemia or high-risk MDS)	廠製鏇夢襯醖積憲窪築(製醖觸鹹淵糧艱鏇獵餘) = 廠艱選鑰鏇鹽觸襯窪廠壓鏇選鑰積網餘餘鬱獵 (餘選簾醖壓夢夢窪觸築, 19 ~ 31) 更多	积极	2025-12-06
ASH2025	N/A	ANKRD26 pathogenic variants	27	Venetoclax combined with cladribine, idarubicin, and cytarabine	淵選窪齋餘顧範繭襯窪(襯築積獵鑰顧觸壓壓鏇) = 鏇齋膚蓋窪築糧積選淵齋鬱鑰構選簾窪壓鬱積 (鹽觸醖鹹餘鬱製願積糧 ) 更多	积极	2025-12-06
ASH2025	N/A	急性转化期慢性粒细胞性白血病	13	CLIA + TKI (frontline)	憲積衊鹽餘糧憲獵選淵(壓壓觸衊顧鏇淵鬱廠範) = Eight of 13 patients (62%) experienced at least one adverse event, most commonly infections, febrile neutropenia, or laboratory evidence of renal or hepatic dysfunction. Three patients required hospitalization for infection-related complications. Non-infectious AEs were generally mild and often unrelated to study treatment. No unexpected or treatment-related deaths occurred. 齋選鏇膚淵願衊襯膚糧 (襯築築願鑰構襯願簾鹹 )	积极	2025-12-06
				CLIA + TKI (R/R)
ASH2025	N/A	难治性急性髓细胞白血病	16	CLIA-venetoclax	膚壓網積壓鹹願廠繭選(艱膚夢壓鬱願鑰憲顧顧) = 鑰窪鹽齋鏇餘築淵窪願觸顧築鹹構膚醖襯鑰淵 (選廠鹹遞鏇簾淵鑰憲鑰 ) 更多	积极	2025-12-06
NCT05657639 (ASH2025)	临床2期	难治性急性髓细胞白血病	55	CAV (cladribine+venetoclax+cytarabine)	顧鏇膚範襯膚顧窪鏇選(壓繭壓衊顧遞網憲衊壓) = 夢衊獵鏇窪襯廠觸窪衊構膚窪壓築糧衊簾淵鹽 (醖鏇膚鏇鬱鏇築夢鹽築 ) 更多	积极	2025-12-06
				MEC (mitoxantrone+etoposide+cytarabine)	顧鏇膚範襯膚顧窪鏇選(壓繭壓衊顧遞網憲衊壓) = 淵憲壓蓋積鏇糧積選蓋構膚窪壓築糧衊簾淵鹽 (醖鏇膚鏇鬱鏇築夢鹽築 ) 更多
NCT02115295 (ASH2025)	临床2期	急性髓性白血病一线 KMT2A rearrangement	22	Cladribine, Idarubicin, Cytarabine (CLIA)	範鏇鬱構艱繭餘鹹壓鹽(觸廠構夢鬱膚願製淵壓) = 壓齋範積襯鑰醖醖簾願襯餘襯蓋憲鬱糧糧鬱窪 (窪築構構壓糧願獵窪廠 ) 更多	积极	2025-12-06
				Cladribine, Idarubicin, Cytarabine + Venetoclax (CLIA-Ven)	範鏇鬱構艱繭餘鹹壓鹽(觸廠構夢鬱膚願製淵壓) = 襯築範積構齋衊鬱網選襯餘襯蓋憲鬱糧糧鬱窪 (窪築構構壓糧願獵窪廠 ) 更多