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Note: Top 10 ALS news stories of 2024
March 3, 2025 at 11:04:28 AM GMT+8 * - permalink -Top 10 ALS news stories of 2024
Of most interest to readers was news on clinical trials for potential treatments
讀者最感興趣的是關於潛在治療方法的臨床試驗的新聞
by Lindsey Shapiro, PhD | December 30, 2024
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Throughout 2024, ALS News Today brought our readers daily coverage of the latest in scientific breakthroughs, treatment advances, and clinical trial updates related to amyotrophic lateral sclerosis (ALS).
在整個 2024 年，ALS 新聞每天都為我們的讀者帶來與 ALS (ALS) 相關的科學突破、治療進展和臨床試驗更新的最新肌萎縮性嵴髓側索硬化症。Here is a list of the top 10 most-read articles published in 2024, along with a brief description. We look forward to continuing to be a trusted resource for the ALS community in 2025.
以下是 2024 年最受讀者歡迎的 10 篇文章，並附有簡要介紹。我們期待在 2025 年繼續成為 ALS 社區可信賴的資源。No. 10 – TPN-101 benefits ALS/FTD patients with C9orf72 gene mutations
No. 10-TPN-101 對 C9orf72 基因突變的 ALS/FTD 患者有益
Data from a Phase 2a clinical trial showed that Transposon Therapeutics‘ oral therapy candidate TPN-101 reduced disease-related biomarkers and slowed respiratory decline in people with ALS or frontotemporal dementia (FTD) associated with C9orf72 gene mutations. TPN-101 is designed to block an enzyme involved in activating pieces of ancient viral DNA that are believed to contribute to neurodegeneration in ALS and FTD. Based on the interim results after six months, and other findings from the trial, the developer indicated its plans to advance the therapy to a registration Phase 3 study for C9orf72-related ALS.
來自 2a 期臨床試驗的資料顯示，轉座子治療公司的口服治療候選藥物 TPN-101 降低了與疾病相關的生物標誌物，並減緩了與 c9orf72 基因突變相關的 ALS 或額顳葉型失智症 (fTD) 患者的呼吸衰退。TPN-101 被設計用來阻斷一種與啟動古代病毒 DNA 片段有關的酶，這種 DNA 片段被認為與 ALS 和 FTD 中的神經退行性疾病有關。基於六個月後的中期結果，以及試驗的其他發現，開發商表示，他們計畫將該療法推進到 C9orf72 相關 ALS 的註冊 3 期研究。No. 9 – IGFBP7 gene mutation linked to rare cases of ALS reversal
9 號 IGFBP7 基因突變與罕見 ALS 逆轉的關係
U.S. researchers identified a mutation in the IGFBP7 gene that’s associated with very rare reversals in ALS disease progression. Typically, as ALS is a progressive disease, its symptoms continuously get worse. However, in a minority of patients, the signs and symptoms of ALS ease or disappear over time. To learn more, the researchers analyzed genetic data from 22 people who had experienced ALS reversal and compared it with data from patients with a typical disease trajectory. A particular mutation in the IGFBP7 gene was associated with up to a 12 times greater likelihood of ALS reversal. The mutation results in lesser production of the gene’s protein product, so researchers believe — though more research still is needed — that inhibiting the protein or the pathway it participates in may be of therapeutic benefit in ALS.
美國研究人員發現了一種 IGFBP7 基因的突變，這種突變與 ALS 疾病進展中非常罕見的逆轉有關。典型地，由於 ALS 是一種進行性疾病，其症狀持續惡化。然而，在少數患者中，ALS 的體徵和症狀隨著時間的推移而減輕或消失。為了瞭解更多 訊息，研究人員分析了 22 名經歷 ALS 逆轉的患者的基因資料，並將其與典型疾病軌跡患者的資料進行比較。IGFBP7 基因中的一個特殊突變與 ALS 逆轉的可能性高達 12 倍有關。這種突變導致該基因的蛋白質產物產量減少，因此研究人員認為 (儘管還需要更多的研究) 抑制該蛋白質或其參與的途徑可能對 ALS 有治療作用。No. 8 – COYA 302 immune-modulating therapy shows promise in small early trial
No. 8-COYA 302 免疫調節療法在小規模早期試驗中顯示出前景
Coya Therapeutics’ investigational therapy COYA 302 was found to slow ALS disease progression and lower levels of disease-related biomarkers in a small Phase 1 clinical trial. COYA 302 is a combination of an immune signaling molecule called interleukin-2 and a fusion protein called CTLA4-Ig that together are intended to ease inflammation that’s believed to contribute to neurodegeneration in ALS. The clinical trial involved four ALS patients who received injections of COYA 302 over nearly a year. The results showed the treatment was well tolerated and generally slowed patients’ functional declines relative to the trial’s start. Improvements in relevant biomarkers also were observed. Based on the study’s findings, Coya indicated plans to conduct a placebo-controlled trial.
在一個小型的 1 期臨床試驗中，科亞治療公司的研究性療法 COYA 302 被發現可以減緩 ALS 疾病的進展和降低疾病相關生物標誌物的水平。COYA 302 是一種名為白細胞介素 -2 的免疫訊號分子和一種名為 CTLA4-Ig 的融合蛋白的組合物，它們共同用於緩解炎症，這種炎症被認為與 ALS 中的神經退行性疾病有關。臨床試驗涉及四名 ALS 患者，他們在接近一年的時間裡接受了 COYA 302 的注射。結果表明，治療耐受性良好，相對於試驗開始時，通常減緩了患者的功能下降。相關生物標誌物的改善也被觀察到。根據研究結果，科亞表示計畫進行一項安慰劑對照試驗。No. 7 – FDA clears Phase 1/2 trial of once-daily SPG302 for ALS
第 7 名： FDA 通過了 ALS 每日一次 SPG302 的 1/2 期臨床試驗
The U.S. Food and Drug Administration gave Spinogenix the green light to launch a Phase 1/2 clinical trial to test its oral treatment candidate SPG302 in ALS patients. The planned trial aims to evaluate the once-daily treatment’s safety, tolerability, and pharmacological properties. SPG302 is designed to trigger the formation of synapses, which are the junctions through which nerve cells communicate — the loss of which is believed to contribute to ALS symptoms. The experimental therapy has demonstrated an ability to regenerate synapses and improve motor and cognitive function in animal models of ALS. It’s now being tested in another Phase 1/2 trial in Australia.
美國食品和藥物管理局批准 Spinogenix 啟動一項 1/2 期臨床試驗，以測試其口服治療候選藥物 SPG302 在 ALS 患者中的應用。計畫中的試驗旨在評估每日一次的治療的安全性、耐受性和藥理 屬性。SPG302 被設計用於觸發突觸的形成，突觸是神經細胞交流的連接點，神經細胞的缺失被認為與 ALS 症狀有關。在 ALS 動物模型中，實驗治療已經證明了再生突觸和改善運動和認知功能的能力。現在正在澳大利亞進行另一個半期試驗。No. 6 – High-dose vitamin B12 approved in Japan for treating ALS
日本批准用於治療肌萎縮性側索硬化症的大劑量維生素 B12
Japanese regulators approved an ultra-high dose of mecobalamin — a naturally occurring version of vitamin B12 — for treating ALS. It’s being sold by Eisai under the brand name Rozebalamin. Vitamin B12 is found in foods such as meats, fish, and dairy, and plays a role in supporting nerve cell health. The approval was based largely on data from a Phase 3 trial involving people with early-stage ALS and moderate disease progression. Data showed that Rozebalamin significantly slowed disease progression, by 43% compared with a placebo, when given alone or combined with standard ALS therapies. The treatment also was well tolerated.
日本監管機構批准了一種用於治療 ALS 的超高劑量甲鈷胺 (一種天然維生素 B12)。衛材以 Rozebalamin 的品牌出售。維生素 B12 存在於肉類、魚類和奶製品等食品中，並在支援神經細胞健康方面發揮作用。該批准主要基於一項涉及早期 ALS 患者和中度疾病進展患者的 3 期臨床試驗的資料。資料顯示，與安慰劑相比，單獨使用或與標準 ALS 療法聯合使用時，Rozebalamin 顯著減緩了疾病進展，減少了 43% 。治療的耐受性也很好。No. 5 – Prilenia plans for Phase 3 trial of pridopidine for ALS
Prilenia Therapeutics indicated its plans to launch a Phase 3 clinical trial of pridopidine in ALS patients after promising Phase 2 results in the HEALEY ALS platform trial. Pridopidine intends to slow ALS progression by activating the sigma-1 receptor, a protein involved in processes important for nerve cell function and survival. Data from the pridopidine arm of HEALEY — a pioneering trial seeking ALS treatments over a faster timeframe — showed that while pridopidine failed to slow disease progression across the entire study group, benefits were observed in a subgroup of patients who were still early in the disease course. These benefits included slower declines in functional ability, respiratory function, and quality of life as compared with a placebo, as well as a possible survival benefit. Pridopidine also was associated with speech improvements across all treated patients.
Prilenia 治療公司表示，在 HEALEY ALS 平台試驗中取得了有希望的第二階段結果之後，它計畫在 ALS 患者中啟動一個第三階段的臨床試驗。普利多定試圖通過啟動 sigma-1 受體來減緩 ALS 的進展，sigma-1 受體是一種參與神經細胞功能和存活過程的蛋白質。HEALEY (一項在更短時間內尋求 ALS 治療的開創性試驗) 的普利多定部門的資料顯示，儘管普利多定未能減緩整個研究組的疾病進展，但在仍處於疾病早期的患者亞組中觀察到了益處。與安慰劑組相比，這些益處包括功能能力、呼吸功能和生活質量下降較慢，以及可能的生存益處。在所有接受治療的患者中，普利多定也與語言能力改善有關。No. 4 – Some recreational activities linked to greater ALS risk
第四名 - 一些娛樂活動與肌萎縮側索硬化症風險增加有關
Participating in certain recreational activities, including golfing, was associated with an increased risk of developing ALS in a study by U.S. researchers. Over the years, scientists have identified environmental risk factors for ALS, including workplace exposures to things like toxic chemicals and pesticides. In this study, researchers surveyed 400 ALS patients and nearly 300 people without the disease to learn more about how recreational activities might influence ALS risk. Several activities, including gardening and woodworking, as well as golfing, were significantly associated with ALS, especially among men. The researchers indicated that more work is needed to validate their findings and to better understand how these activities raise ALS risk.
根據美國研究人員的一項研究，參加某些娛樂活動，包括高爾夫，與患 ALS 的風險增加有關。多年來，科學家已經確定了 ALS 的環境風險因素，包括工作場所接觸有毒化學品和殺蟲劑。在這項研究中，研究人員調查了 400 名 ALS 患者和近 300 名非 ALS 患者，以瞭解娛樂活動如何影響 ALS 風險。一些活動，包括園藝和木工，以及高爾夫球，與肌萎縮側索硬化症顯著相關，尤其是在男性。研究人員指出，需要更多的工作來驗證他們的發現，並更好地瞭解這些活動如何增加 ALS 的風險。No. 3 – Amylyx considers discontinuing Relyvrio after trial failure
No. 3-Amylyx 考慮在試驗失敗後停用 Relyvrio
After Relyvrio (sodium phenylbutyrate and taurursodiol) failed to slow disease progression for ALS patients in a Phase 3 clinical trial, developer Amylyx Pharmaceuticals paused its promotion of the therapy and eventually discontinued it altogether. The therapy had initially been approved in the U.S. and Canada based on Phase 2 trial data showing it could slow functional declines in people with recently diagnosed and rapidly progressing ALS. That finding, however, could not be replicated in the Phase 3 study. The treatment also failed to meet other secondary trial goals related to quality of life, lung function, and survival. Amylyx continues to develop Relyvrio for other neurodegenerative conditions.
在一項 3 期臨床試驗中，Relyvrio (苯丁酸鈉和牛磺二醇) 未能減緩 ALS 患者的疾病進展，開發商 Amylyx 製藥公司暫停了對該療法的推廣，並最終完全停止了該療法。這種療法最初在美國和加拿大獲得批準是基於第二階段的試驗資料，這些資料顯示它可以減緩最近確診並迅速發展的 ALS 患者的功能下降。然而，這一發現在第三階段的研究中無法複製。治療也未能達到與生活質量、肺功能和生存率相關的其他二次試驗目標。Amylyx 繼續開發 Relyvrio 治療其他神經退行性疾病。No. 2 – Early trial data show promise of vet medication monepantel for ALS
第 2 名 - 早期試驗資料顯示獸醫藥物治療 ALS 的前景
Monepantel, a repurposed veterinary medication that’s now named NUZ-001, was found to slow disease progression for people with ALS or motor neuron disease (MND) in a small Phase 1 clinical trial. Used in veterinary practices as a deworming agent, the therapy is also able to block a signaling pathway that scientists believe could help clear the toxic protein clumps that contribute to ALS progression. Findings from the trial, which involved 12 adults with ALS or MND, showed that daily oral treatment was well tolerated and slowed the rate of disease progression by up to 58% relative to an external group of untreated ALS patients. These results and other long-term findings prompted developer PharmAust — now Neurizon Therapeutics — to plan for the launch of a Phase 2/3 study, which will run as part of the HEALEY ALS trial.
Monepantel 是一種重新利用的獸藥，現在被命名為 NUZ-001，在一項小型的第一階段臨床試驗中，研究人員發現它可以減緩 ALS 或運動神經元疾病 (MND) 患者的疾病進展。在獸醫實踐中作為驅蟲劑，該療法也能夠阻斷訊號通路，科學家認為可以幫助清除有毒蛋白質團塊，有助於 ALS 進展。來自涉及 12 名 ALS 或 MND 成年人的試驗結果顯示，每日口服治療耐受性良好，相對於未經治療的 ALS 患者的外部組，疾病進展速度減慢高達 58% 。這些結果和其他長期研究結果促使開發商 PharmAust (現在的 Neurizon 治療公司) 計畫啟動 2/3 階段研究，該研究將作為 HEALEY ALS 試驗的一部分。No. 1 – PrimeC prolongs patient survival without complications in trial
No. 1-PrimeC 在試驗中延長患者生存期且無併發症
The most-read story on ALS News Today in 2024 reported that the ALS therapy candidate PrimeC extended complication-free survival and improved quality of life relative to a placebo among patients in a Phase 2b trial. PrimeC contains a fixed-dose combination of two agents that together are expected to block processes believed to drive ALS progression. Top-line trial data showed that the treatment slowed disease progression in patients who adhered to the trial’s established rules. This analysis demonstrated that PrimeC extended complication-free survival — survival without respiratory failure, ALS-related hospitalizations, or advances in disease stage — by 53% compared with a placebo. Treatment also was associated with positive effects on physical and mental aspects of quality of life. Developer NeuroSense has since met with U.S. regulators to discuss plans for a Phase 3 clinical trial, and is in the process of seeking early approval for PrimeC in Canada.
2024 年《今日 ALS 新聞》(ALS News Today) 上閱讀量最多的報導稱，在 2b 期臨床試驗中，ALS 治療候選藥物 PrimeC 延長了患者的無併發症生存期，改善了患者的生活質量。PrimeC 包含兩種藥物的固定劑量組合，這兩種藥物一起被認為可以阻斷驅動 ALS 進展的過程。頂線試驗資料顯示，該療法減緩了遵守試驗既定規則的患者的疾病進展。這項分析表明，與安慰劑相比，PrimeC 延長了無併發症生存期ーー無唿吸衰竭、 ALS 相關住院治療或疾病分期進展的生存期ーー 53% 。治療還與對生活質量的身心方面的積極影響有關。此後，開發商 NeuroSense 與美國監管機構會面，討論第三階段臨床試驗的計畫，目前正在尋求加拿大 PrimeC 的早期批准。
At ALS News Today, we hope our reporting throughout 2024 had a meaningful impact on those affected by ALS. We look forward to serving the ALS community in 2025, and wish all of our readers a very happy new year!
在今日 ALS 新聞，我們希望我們的報告在 2024 年對那些受 ALS 影響的人產生有意義的影響。我們期待著在 2025 年為 ALS 社區服務，並祝願我們所有的讀者新年快樂！-
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