阿尔茨海默病协会部分云拨款500万美元用于开发创新的基因靶向治疗

IF 13 1区 医学 Q1 CLINICAL NEUROLOGY
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引用次数: 0

摘要

部分云,通过阿尔茨海默氏症协会,宣布向四项研究提供总计500万美元的资助,以开发阿尔茨海默氏症的潜在疗法。这些拨款主要用于基因靶向治疗:改变基因表达的治疗。由慈善家Michaela“Mikey”Hoag和阿尔茨海默氏症协会(Alzheimer's Association)创立的Part the Cloud以其严格而稳健的审查程序而闻名,鼓励其他投资者从初始资金中跟进,扩大这些创新试验。迄今为止,部分云资助的研究已经获得了超过16亿美元的后续资金支持。云的一部分提供战略资金,以推进有前途的研究性阿尔茨海默病治疗进入临床试验。治疗目标多种多样,这一点很重要,因为越来越多的人认为,有效治疗和预防阿尔茨海默氏症可能是针对该疾病不同方面的多种干预措施的个性化组合。新获奖者是云翻译基因靶向挑战赛的第一批获奖者。超过100个基因与患阿尔茨海默氏症和其他痴呆症的风险或抵抗力有关。因此,个性化基因编辑和基因相关治疗具有巨大的潜力。阿尔茨海默氏症协会首席科学官和医学事务负责人Maria C. Carrillo博士说:“有了这些实质性的、有针对性的奖项,研究团队将大大扩大遗传学如何为阿尔茨海默氏症和其他痴呆症的个性化治疗提供信息的研究,以解决每个人独特的生物环境。”“我们的云计划继续发挥其全球领导作用,播种广泛的治疗管道,推动该领域向前发展,为来自所有社区和疾病所有阶段的每个人提供有效治疗。现在比以往任何时候都更有必要资助像这样有远见的、前瞻性的赠款。Part the Cloud始于2012年,专门用于加速将实验室研究结果通过试验转化为可能的治疗方法——通过为早期临床试验提供必要的支持,填补阿尔茨海默病药物开发的空白。“阿尔茨海默氏症和其他导致痴呆症的疾病影响着全球5000万人,仅在美国就有700多万人。它直接,出乎意料地,残酷地影响了我的家庭,也可能影响到你的家庭。我们必须确定并测试能够减缓、预防或逆转这些疾病造成的脑细胞损伤的治疗方法。”Hoag说。霍格补充说:“我相信,针对阿尔茨海默病和相关痴呆症的遗传基础量身定制的治疗方法,极有可能推动该领域取得重大突破。”部分云已经筹集了近9000万美元,用于资助最有可能减缓、停止或最终治愈阿尔茨海默病的临床试验。这些拨款确实发挥了作用,我们正努力朝着实现没有阿尔茨海默病的世界的愿景迈进一步。”要了解更多关于Part the Cloud的信息,包括当前和未来的融资机会,请访问alz.org/partthecloud.The阿尔茨海默氏症协会推出了一款新的移动应用程序,旨在帮助新诊断的早期患者及其护理伙伴导航他们与阿尔茨海默氏症和其他痴呆症的旅程。免费的“我的ALZ之旅”应用程序提供个性化的指导、教育、规划工具、互动活动和当地社区资源。阿尔茨海默氏症协会社区项目和服务高级主管伊丽莎白·埃杰利说:“痴呆症的诊断可能会让人不知所措,感到孤独。”“我的ALZ之旅提供了值得信赖、易于获取的指导,帮助处于疾病早期阶段的个人和家庭感受到支持,并对接下来的事情充满信心。通过在正确的时间提供正确的信息,我们希望这款应用能够促进联系,消除耻辱,并使个人及其护理伙伴能够自信地采取行动。”目前,有超过700万美国人患有阿尔茨海默病。2024年阿尔茨海默氏症协会的一项调查发现,66%的痴呆症护理人员报告在痴呆症诊断后难以找到资源和支持。痴呆症患者,包括阿尔茨海默病协会国家早期咨询小组(ESAG)的现任和前任成员,在这款应用的开发过程中发挥了重要作用——确定了它的需求,帮助设计和测试了它的内容,并最终为它命名。ESAG现任成员布伦特·比斯利(Brent Beasley)在2023年被诊断出患有早期阿尔茨海默病,他说:“对痴呆症患者来说,技术可能是一项挑战,但我发现My ALZ Journey应用程序很容易使用。” “让痴呆症患者为应用程序的开发提供输入和反馈,有助于使其变得实用,并对确诊患者及其护理伙伴的现实需求做出反应。”对于那些已经在日常生活中享受使用应用程序的人来说,My ALZ Journey具有简单,直观的设计,可以引导用户进行个性化体验。主屏幕提供了一个精心策划的路径,逐步指导和资源,随着时间的推移,以反映用户的旅程。该应用程序的图书馆可以方便地访问来自alz.org的基于主题的教育内容,回答有关阿尔茨海默病、护理和未来规划的常见问题。用户可以通过社区部分直接连接到当地的阿尔茨海默氏症协会分会,该分会突出显示了附近的支持团体、活动和项目。活动中心提供互动工具、规划资源和活动,以帮助用户保持参与和支持。“在诊断出阿尔茨海默氏症之后,个人和家庭有很多问题,”Edgerly说。“我们的应用程序提供了一个简单的起点,可以获取信息,并将用户与重要的服务和资源联系起来,特别是针对疾病的早期阶段。我们希望受阿尔茨海默病和其他痴呆症影响的家庭知道,他们并不孤单。”《My ALZ Journey》现在可以在苹果应用商店和b谷歌Play商店免费下载。欲了解更多信息,请访问alz.org/MyALZJourney。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Alzheimer's Association Part the Cloud grants $5 million to develop innovative gene targeting treatments

Part the Cloud, through the Alzheimer's Association, announces the awarding of grants totaling $5 million to four studies to develop potential therapies for Alzheimer's disease. These grants focus on gene targeting therapies: therapies that change the expression of genes.

Started by philanthropist Michaela “Mikey” Hoag and the Alzheimer's Association, Part the Cloud is known for its rigorous and robust review process, encouraging other investors to follow on from its initial funding to expand these innovative trials. To date, Part the Cloud–funded studies have garnered more than $1.6 billion in follow-on funding support.

Part the Cloud provides strategic funding to advance promising investigational Alzheimer's therapies into clinical trials. The treatment targets are varied, which is important given the growing consensus that effective treatment and prevention of Alzheimer's is likely to be a personalized combination of multiple interventions targeting different aspects of the disease.

The new awardees are the first recipients of the Part the Cloud Translational Gene Targeting Challenge. More than 100 genes are associated with risk for or resistance to the development of Alzheimer's and other dementias. As a result, there is great potential for personalized gene editing and gene-related therapies.

“With these substantial and targeted awards, research teams will significantly expand the investigation of how genetics can inform personalized treatments for Alzheimer's and other dementias that address each individual's unique biological circumstances,” said Maria C. Carrillo, PhD, Alzheimer's Association chief science officer and medical affairs lead.

“Our Part the Cloud initiative continues its global leadership in seeding a wide-ranging treatment pipeline that drives the field forward toward effective treatments for everyone from all communities and at all stages of the disease. Now more than ever, it is crucial to fund visionary, forward-looking grants like these,” Carrillo said.

Started in 2012, Part the Cloud is specifically designed to accelerate the translation of findings from the laboratory, through trials, into possible therapies—filling the gap in Alzheimer's drug development by providing essential support for early-phase clinical trials.

“Alzheimer's and other diseases that cause dementia affect 50 million people worldwide, including over 7 million in the U.S. alone. It affected my family directly, unexpectedly, and brutally, and it might impact yours, too. We must identify and test treatments that can slow, prevent, or reverse the brain cell damage inflicted by these diseases,” said Hoag.

“I believe that therapies tailored to the genetic underpinnings of Alzheimer's disease and related dementias have very high potential to supercharge the field toward major breakthroughs,” Hoag added. “Part the Cloud has raised nearly $90 million to fund clinical trials with the highest probability of slowing, stopping or ultimately curing Alzheimer's disease. These grants are truly making a difference as we work to get one step closer to our vision of a world without Alzheimer's disease.”

To learn more about Part the Cloud, including current and future funding opportunities, visit alz.org/partthecloud.

The Alzheimer's Association has launched a new mobile app aimed at helping newly diagnosed, early-stage individuals, and their care partners navigate their journey with Alzheimer's and other dementias. The free “My ALZ Journey” app provides personalized guidance, education, planning tools, interactive activities, and local community resources.

“A dementia diagnosis can be overwhelming and isolating,” said Elizabeth Edgerly, senior director, Community Programs and Services, Alzheimer's Association. “My ALZ Journey offers trusted, easy-to-access guidance that helps individuals and families in the early stages of the disease feel supported and confident about what comes next. By delivering the right information at the right time, we hope the app will foster connection, combat stigma, and empower individuals and their care partners to take action with confidence.”

Currently, there are more than 7 million Americans living with Alzheimer's disease. A 2024 Alzheimer's Association survey found that 66% of dementia caregivers report difficulty finding resources and support following a dementia diagnosis.

People living with dementia, including current and former members of the Alzheimer's Association National Early Stage Advisory Group (ESAG), were instrumental in the app's development—identifying the need for it, helping shape and test its content, and eventually naming it.

“Technology can be challenging for someone living with dementia, but I find the My ALZ Journey app easy to use,” said Brent Beasley, a current ESAG member who was diagnosed with early-stage Alzheimer's disease in 2023. “Having people living with dementia provide input and feedback into the app's development helps make it practical and responsive to the real-life needs of diagnosed individuals and their care partners.”

Ideal for those who already enjoy using apps in their daily lives, My ALZ Journey features a simple, intuitive design that guides users through a personalized experience. The Home screen offers a curated path with step-by-step guidance and resources that evolve over time to reflect the user's journey. The app's Library provides easy access to topic-based educational content from alz.org, answering common questions about Alzheimer's, caregiving, and planning for the future. Users can connect directly to their local Alzheimer's Association chapter through the Community section, which highlights nearby support groups, events, and programs. The Activity Center offers interactive tools, planning resources, and activities to help users stay engaged and supported.

“In the wake of an Alzheimer's diagnosis, individuals and families have so many questions,” Edgerly said. “Our app offers a simple starting point for getting information and connecting users with important services and resources, specifically for the early stages of the disease. We want families affected by Alzheimer's and other dementia to know they are not alone.”

My ALZ Journey is available now for free in the Apple App Store and Google Play Store. For more information, visit alz.org/MyALZJourney.

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来源期刊
Alzheimer's & Dementia
Alzheimer's & Dementia 医学-临床神经学
CiteScore
14.50
自引率
5.00%
发文量
299
审稿时长
3 months
期刊介绍: Alzheimer's & Dementia is a peer-reviewed journal that aims to bridge knowledge gaps in dementia research by covering the entire spectrum, from basic science to clinical trials to social and behavioral investigations. It provides a platform for rapid communication of new findings and ideas, optimal translation of research into practical applications, increasing knowledge across diverse disciplines for early detection, diagnosis, and intervention, and identifying promising new research directions. In July 2008, Alzheimer's & Dementia was accepted for indexing by MEDLINE, recognizing its scientific merit and contribution to Alzheimer's research.
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