Pictured: Illustration of a brain surrounded by a vaccine and a needle/Nicole Bean for BioSpace

图为BioSpace的大脑被疫苗和针头/NicoleBean包围的插图

During the past few years, disease-modifying treatments for Alzheimer’s disease have broken through, but another prevalent neurodegenerative disease, Parkinson’s, has lagged behind. Vaxxinity is one company looking to change that. It published data Thursday showing that its experimental immunotherapy could improve daily movement in Parkinson’s patients and elicit antibodies against a toxic protein involved in the disease process..

在过去几年中，阿尔茨海默氏病的疾病缓解治疗取得了突破，但另一种流行的神经退行性疾病帕金森氏症却落后了。Vaxxinity是一家希望改变现状的公司。它周四公布的数据显示，它的实验性免疫疗法可以改善帕金森病患者的日常运动，并引发针对参与疾病过程的有毒蛋白质的抗体。。

The treatment “could potentially be disease-modifying as shown by its high immunogenicity, and its ability to reduce aggregated [αSyn] in the CSF [cerebral spinal fluid] of [Parkinson’s] patients,” Vinata Vedam-Mai, an assistant professor in the department of neurosurgery and the Fixel Institute at the University of Florida, told BioSpace in an email..

佛罗里达大学神经外科和Fixel研究所的助理教授VinataVedamMai在一封电子邮件中告诉BioSpace，这种治疗“可能会改变疾病，因为它具有高免疫原性，并且能够减少帕金森病患者脑脊液中聚集的[αSyn]。”。。

The article, published in Nature Medicine, describes the results of a Phase I study of UB-312, a synthetic peptide designed to provoke an immune response against toxic clumps of the alpha-synuclein (αSyn) protein. Duplications, point mutations or single nucleotide polymorphisms in the SNCA gene encoding αSyn contribute to Parkinson’s disease, and αSyn aggregates in the form of Lewy bodies are common hallmarks of the condition..

这篇发表在《自然医学》上的文章描述了UB-312的I期研究结果，UB-312是一种合成肽，旨在引发针对α-突触核蛋白（αSyn）蛋白有毒团块的免疫反应。编码αSyn的SNCA基因中的重复，点突变或单核苷酸多态性导致帕金森氏病，路易体形式的αSyn聚集体是该病的常见特征。。

The data—which were presented earlier this year at the AD/PD 2024 International Conference on Alzheimer’s and Parkinson’s Disease—showed that treatment with UB-312 elicited a 20% reduction in clumped αSyn levels for patients given UB-312, compared with a 3% increase in patients given a placebo. Additionally, 12 out of 13 patients (92%) developed antibodies against these harmful clusters, though UB-312 did not lead to the generation of antibodies against full-length αSyn compared with placebo.

今年早些时候在AD/PD 2024阿尔茨海默氏病和帕金森氏病国际会议上提交的数据显示，使用UB-312治疗可使服用UB-312的患者的聚集αSyn水平降低20%，而服用安慰剂的患者则增加了3%。此外，13名患者中有12名（92%）产生了针对这些有害簇的抗体，尽管与安慰剂相比，UB-312并未导致产生针对全长αSyn的抗体。

Patients in the treatment group also reported improved daily movement..

治疗组患者的日常活动也有所改善。。

“We target the root cause of Parkinson’s, not just the symptoms,” Mei Mei Hu, CEO and co-founder of Vaxxinity, told BioSpace in an email. “It offers the first real hope for patients and their families in the fight against Parkinson’s.”

Vaxxinity首席执行官兼联合创始人胡美美（MeiMeiHu）在一封电子邮件中告诉BioSpace：“我们的目标是帕金森氏症的根本原因，而不仅仅是症状。”。“这为患者及其家人抗击帕金森氏症提供了第一个真正的希望。”

In terms of safety, most treatment-emergent adverse effects were mild or moderate, according to the paper, with three severe adverse events—one possibly treatment-related—reported.

根据该论文，就安全性而言，大多数治疗出现的不良反应是轻度或中度的，有三种严重不良事件，其中一种可能与治疗有关。

The fact that the treatment “appears to be well-tolerated” also makes it promising as a disease-modifying therapy, Vedam-Mai said. As a future step, she noted that “it would be desirable to see effects of the therapy on the brain using recent MRI techniques.”

。作为未来的一步，她指出“希望使用最新的MRI技术观察该疗法对大脑的影响。”

Vedam-Mai also said that the study of UB-312 “needs to be scaled up to see better the effects of this treatment.” She additionally recommended that the patient population be more diverse in order to better show efficacy. “The patients should also be chosen at different and later stages of the disease to show that the therapy can ameliorate more advanced patients.”.

Vedam Mai还说，UB-312的研究“需要扩大规模，以更好地观察这种治疗的效果。”她还建议患者群体更加多样化，以更好地显示疗效。“还应在疾病的不同阶段和后期选择患者，以表明该疗法可以改善更晚期的患者。”。

Indeed, Vaxxinity hopes to “reproduce our promising results” in more Parkinson’s patients by moving into a larger Phase II study, Hu said. That study has not yet been scheduled.

事实上，Vaxxinity希望通过进入更大规模的II期研究，在更多帕金森病患者中“重现我们有希望的结果”，胡说。。

Heather McKenzie is a senior editor at BioSpace. You can reach her at heather.mckenzie@biospace.com. Also follow her on LinkedIn.

希瑟·麦肯齐是BioSpace的高级编辑。你可以通过heather.mckenzie@biospace.com.也可以在LinkedIn上关注她。