The development of disease-modifying therapies (DMTs) for neurological disorders is an important goal in modern neurology, and the associated challenges are similar in many chronic neurological conditions. Major advances have been made in the multiple sclerosis (MS) field, with a range of DMTs being approved for relapsing MS and the introduction of the first DMTs for progressive MS. By contrast, people with Parkinson disease (PD) still lack such treatment options, relying instead on decades-old therapeutic approaches that provide only symptomatic relief. To address this unmet need, an in-person symposium was held in Toronto, Canada, in November 2022 for international researchers and experts in MS and PD to discuss strategies for advancing DMT development. In this Roadmap article, we highlight discussions from the symposium, which focused on therapeutic targets and preclinical models, disease spectra and subclassifications, and clinical trial design and outcome measures. From these discussions, we propose areas for novel or deeper exploration in PD using lessons learned from therapeutic development in MS. In addition, we identify challenges common to the PD and MS fields that need to be addressed to further advance the discovery and development of effective DMTs.

开发针对神经系统疾病的疾病修饰疗法 （DMT） 是现代神经病学的一个重要目标，在许多慢性神经系统疾病中，相关挑战是相似的。多发性硬化症 （MS） 领域取得了重大进展，一系列 DMT 被批准用于复发性 MS，并推出了第一个用于进行性 MS 的 DMT。相比之下，帕金森病 （PD） 患者仍然缺乏此类治疗选择，而是依赖几十年前仅提供症状缓解的治疗方法。为了解决这一未满足的需求，2022 年 11 月在加拿大多伦多举行了一次面对面的研讨会，让 MS 和 PD 领域的国际研究人员和专家讨论推进 DMT 开发的策略。在这篇 Roadmap 文章中，我们重点介绍了研讨会上的讨论，重点关注治疗靶点和临床前模型、疾病谱和亚分类以及临床试验设计和结果测量。从这些讨论中，我们利用从 MS 治疗开发中吸取的经验教训，提出了 PD 中新的或更深入的探索领域。此外，我们还确定了 PD 和 MS 领域需要解决的常见挑战，以进一步推进有效 DMT 的发现和开发。