Cellenkos的现成Treg细胞疗法在ALS中显示出临床安全性和初步疗效-动脉网

Cellenkos' Off-the-Shelf Treg Cell Therapy Shows Clinical Safety and Preliminary Efficacy in ALS

CISION 等信源发布 2025-04-23 01:43

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Cryopreserved allogeneic Treg infusions associated with slowed disease progression and decreased plasma neurofilament level in ALS

冷冻保存的同种异体Treg输注与ALS疾病进展减缓和血浆神经丝水平降低相关

HOUSTON

休斯顿

，

April 22, 2025

2025年4月22日

/PRNewswire/ -- New findings published today in

/PRNewswire/ -- 今天发表的新发现

NEJM Evidence

《新英格兰医学杂志证据》

reveal promising clinical outcome for Cellenkos Inc.'s cryopreserved, allogeneic, T regulatory (Treg) cells derived from umbilical cord blood, for the treatment of Amyotrophic Lateral Sclerosis (ALS)—a neurodegenerative disease with limited therapeutic options.

揭示了Cellenkos Inc.的冷冻保存、同种异体、源自脐带血的T调节（Treg）细胞在治疗肌萎缩侧索硬化症（ALS）方面的良好临床结果，这是一种治疗选择有限的神经退行性疾病。

The study reports that patients receiving multiple intravenous infusions of this 'off-the-shelf' Treg therapy showed a measurable slowdown in disease progression. Prior to therapy, participants' ALS Functional Rating Scale-Revised (ALSFRS-R) scores declined at an average rate of −1.66 points per month.

研究报道，接受多次静脉输注这种“现成的”Treg疗法的患者显示出疾病进展有可测量的减缓。在治疗之前，参与者的ALS功能评定量表修订版（ALSFRS-R）评分以平均每月−1.66分的速度下降。

During treatment, that rate slowed significantly to −0.41 points per month, with a modest post-treatment decline of −0.60 points per month—suggesting sustained benefit. Clinical improvements were accompanied by reductions in plasma neurofilament levels, a biomarker correlated with neuronal injury..

在治疗期间，这一速率显著减缓至每月-0.41点，治疗后适度下降至每月-0.60点，表明持续获益。临床改善的同时，血浆神经丝水平（一种与神经元损伤相关的生物标志物）也有所降低。

The Treg therapy was administered in an outpatient setting via peripheral IV, with each patient receiving a standardized fixed dose of 100 million cells. The regimen included weekly infusions for the first month, followed by monthly doses for six months, with optional continued dosing based on physician discretion.

Treg疗法在门诊通过外周静脉注射进行，每位患者接受标准化的固定剂量，为1亿个细胞。该方案包括首月每周一次输注，随后六个月每月一次剂量，根据医生判断可选择继续给药。

The therapy required no lymphodepletion, immunosuppression, interleukin-2, or HLA matching, and patients were discharged the same day. Importantly, no dose-limiting toxicities were reported..

该疗法无需淋巴细胞清除、免疫抑制、白细胞介素-2或HLA匹配，并且患者当天出院。重要的是，没有报告剂量限制性毒性。

Six participants, with a median age of 48.5 years (range: 27–66), and a median baseline ALSFRS-R score of 31.5 (range: 23–43), received a median of 11 infusions (range: 6–22), and all were alive at the time of last follow-up. Among participants with sufficient follow-up data (n=4), the median follow-up duration was 18 months..

六名参与者，中位年龄为48.5岁（范围：27-66岁），基线ALSFRS-R评分中位数为31.5（范围：23-43），接受了中位数为11次的输注（范围：6-22次），在最后一次随访时均存活。在具有足够随访数据的参与者中（n=4），中位随访时间为18个月。

'These results are both encouraging and transformative for those battling ALS,' said Dr.

“这些结果对于那些与ALS斗争的人来说，既令人鼓舞又具有变革性，”博士说。

Simrit Parmar

西姆丽特·帕尔马ർ

, Founder of Cellenkos and faculty member at

，Cellenkos创始人及教职成员于

Texas A&M University

德克萨斯农工大学

. 'Unlike conventional cell therapies that require complex, individualized cell harvesting and hospitalization, our Treg therapy is cryopreserved, ready-to-use, and outpatient-based. It eliminates the need for HLA matching and preconditioning. These findings offer a compelling proof of concept and lay the foundation for our next-generation neurotropic Tregs, CK0803.'.

“与需要复杂、个体化细胞采集和住院治疗的传统细胞疗法不同，我们的Treg疗法是冷冻保存、即用型且基于门诊的。它消除了HLA匹配和预处理的必要。这些发现提供了令人信服的概念验证，并为我们的下一代神经趋向性Tregs（CK0803）奠定了基础。”

CK0803 Tregs, now in clinical trials, are modified to overexpress CD11a and CXCR3, improving their ability to migrate to inflamed brain regions, specifically, inflamed microglia — potentially enabling direct targeting of ALS-related pathology.

CK0803调节性T细胞目前正在进行临床试验，经过修饰后能够过表达CD11a和CXCR3，增强了它们迁移到脑部炎症区域的能力，特别是炎症相关的小胶质细胞——这可能使其直接靶向ALS相关病理成为可能。

These findings offer renewed hope for patients, families, and researchers working toward more accessible, non-invasive, and effective treatments for ALS.

这些发现为患者、家属以及致力于寻找更易获取、非侵入性和更有效的ALS治疗方法的研究人员带来了新的希望。

About Amyotrophic Lateral Sclerosis (ALS)

关于肌萎缩侧索硬化症 (ALS)

ALS is a progressive neurodegenerative disease that affects nerve cells responsible for voluntary muscles. As the disease advances, individuals experience worsening muscle weakness, paralysis, and eventual respiratory failure. Most patients succumb to ALS within three to five years of diagnosis. Currently, there is no cure..

ALS是一种影响负责随意肌的神经细胞的进行性神经退行性疾病。随着疾病的发展，患者会经历日益严重的肌肉无力、瘫痪，并最终导致呼吸衰竭。大多数患者在确诊后的三到五年内死于ALS。目前，该病尚无治愈方法。

About Cellenkos

关于Cellenkos

, Inc.

公司

Cellenkos is a clinical-stage biotech company, advancing allogeneic, off-the-shelf, cord blood-derived T regulatory cell therapies for rare inflammatory and autoimmune diseases. Its pipeline includes:

Cellenkos是一家临床阶段的生物技术公司，致力于推进用于罕见炎症和自身免疫性疾病的同种异体、现成的、脐带血来源的T调节细胞疗法。其研发管线包括：

CK0801

for Aplastic Anemia

针对再生障碍性贫血

CK0802

for Acute Respiratory Distress Syndrome

急性呼吸窘迫综合征

CK0803

for ALS

适用于ALS

CK0804

for Myelofibrosis

针对骨髓纤维化

Using its proprietary CRANE

使用其专有的CRANE

platform, Cellenkos tailors tissue directed Treg therapies that require no HLA or ABO matching, can be administered in outpatient settings, and are optimized for rapid, point of care delivery.

平台，Cellenkos 定制了无需 HLA 或 ABO 配型、可在门诊环境中施用并针对快速、即时治疗优化的组织导向性 Treg 疗法。

Contact

联系

bd@cellenkosinc.com

SOURCE Cellenkos, Inc.

来源：Cellenkos, Inc.

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