MULTIPLE SCLEROSIS: Recovery of UK's strong lead in protecting the MS Brain by securing UK-Based formulation of LIFNano™

多发性硬化症(MS)患者的大脑保护*MS**是一种攻击大脑的脱髓鞘自身免疫性疾病，脑容量逐年减少，从十几岁开始，并在30岁末变得明显。没有治愈的方法。目前的治疗方法只是免疫抑制。*新冠肺炎大流行暴露了脆弱的年轻人，包括英国的多发性硬化症患者(患有多发性硬化症的人)，随着封锁的解除，他们需要继续防护。*MicroSME**LIFNanoRx(LNT)**是剑桥大学的衍生产品，由科学家苏·梅特卡夫创立，治疗多发性硬化症。意识到保护大脑的必要性，她捕捉到了生命周期因子--一种对大脑健康至关重要的干细胞生长因子--深刻的神经保护特性--使用美国食品和药物管理局批准的凝胶纳米颗粒，为持续将生命周期因子运送到大脑提供一个归巢装置。耶鲁大学的合作者Tarek Fahmy为制造含有LIF的纳米颗粒贡献了非凡的能力，也是Metcalfe拥有的技术专利的共同发明者。*临床前数据令人信服，证实了多发性硬化症模型的有效性和安全性，并在治疗4天内部分逆转了瘫痪。值得注意的是，治疗性纳米颗粒“LIFNano(Tm)”跨越了血脑屏障(BBB)--这是一种治疗中枢神经系统(CNS)疾病的备受追捧的产品。*通过保护大脑，LIFNano(Tm)为患有多发性硬化症的患者提供了一种低成本、高价值、安全的解决方案。在以前的政府拨款的支持下，LIFNanoRx现在是一支主要力量--是巨人中的大卫：与那些多发性硬化症市场利润丰厚、付款人成本高、供应商回报高的巨人不同，LNT为患者带来了一种独特的颠覆性、低成本和高价值的方法。在MS社区中有许多LNT的追随者，他们知道LIF不仅是一种保护和治愈大脑的无药物的自然解决方案，而且可能会消除MS的自身免疫根本原因。CoVID-19*计划于2020/2021年进行IB期临床试验，由英国领先的MS神经学家Gavin Giovannoni领导，LNT的临床进展因新冠肺炎而停滞不前。封锁使我们无法获得由耶鲁大学定制的LIFNano(Tm)产品。*这次对i-UK可持续创新基金的竞标将把LIFNano(Tm)的生产转移到英国，由另一家增长强劲的英国中小企业Pharmidex承担。*该项目的预算还包括一个临床试验小组--从我们之前的I-UK BMC奖(2017-2019年)已经到位--在预算内及时为这个SIF项目带来附加值。该小组的专家将监督IMPD的准备工作，为与MHRA监管当局接触做好准备。简单的清洁技术*作为一种完全合成的产品，与基于细胞的疗法相比，LIFNano(Tm)大大减少了对环境的影响。通过利用LIF，LNT为目前无法治疗的CNS疾病带来了新一代治疗方法，包括30%涉及CNS的罕见疾病。LNT是创新的领导者，对英国推动增长的能力产生了影响。对于MS来说，迫切需要的是能够保持LNT在人体试验中迈向第一的高轨迹的CTA。该项目不仅将我们简单的清洁技术引入英国，克服新冠肺炎对LNT治疗MS患者进展的不利影响，还为英国经济作为纳米医学领域的领导者增加了商业实力。

PROTECTING THE BRAIN IN PEOPLE WITH MULTIPLE SCLEROSIS (MS)* **MS** is a demyelinating autoimmune disease that attacks the brain, with year-on-year loss of brain volume, starting late teens and becoming manifest late twenties. There is no cure. Current therapies are immunosuppressive only.* The COVID-19 pandemic has spotlighted the vulnerable young including the UK's MS-ers (people with MS) who need continued shielding as lockdown eases.* The microSME **LIFNanoRx (LNT)** is a spin out from the University of Cambridge, founded by scientist Su Metcalfe to treat MS. Recognising the need to protect the brain, she captured the profound neuro-protective properties of LIF - a stem cell growth factor essential for brain health - using nanoparticles of FDA-approved gel to provide a homing device for sustained delivery of LIF cargo to the brain. Collaborator Tarek Fahmy at Yale University contributed remarkable ability to manufacture the LIF-containing nanoparticles and is co-inventor of the technology patents owned by Metcalfe.* The preclinical data is compelling, confirming efficacy and safety in models of MS, and partially reversing paralysis within 4 days of treatment. Notably the therapeutic nanoparticles "LIFNano(tm)" cross the Blood-Brain Barrier (BBB) - a highly sought after property for treatment of diseases of the central nervous system (CNS).* By protecting the brain, LIFNano(tm) provides a _low cost, high value, safe solution_ for people suffering from MS. Supported by previous government grants, LIFNanoRx is now a major force - a David amongst Goliaths: unlike the Goliaths - where the MS market is lucrative with high costs to payers and high rewards to Suppliers - LNT brings a uniquely disruptive, low costs high value to patients approach. There are many followers of LNT within the MS community who understand LIF is not only a _drug-free_ natural solution to protect and heal the brain, but also may quench the autoimmune root-cause of MS.COVID-19* With a Phase IB clinical trial planned for 2020/2021, to be led by UK's leading MS neurologist Gavin Giovannoni, LNT's progress to clinic was stalled by COVID-19\. Lockdown has stopped our access to the LIFNano(tm) product that is custom made by Yale University.* This bid to the Sustainable Innovation Fund of I-UK will transfer manufacture of LIFNano(tm) to the UK, to be undertaken by Pharmidex, another UK SME with robust growth providing services to evaluate medicines that treat the brain.* The Project's budget also accommodates a Clinical Trial Panel - already in place from our previous I-UK BMC award (2017-2019) - bringing added value to this SIF Project within budget and on time. The Panel's experts will oversee IMPD preparation ready for engaging with the MHRA regulatory authorities. THE SIMPLE CLEAN TECHNOLOGY* As an entirely synthetic product, LIFNano(tm) vastly reduces impact on the environment compared to cell-based therapies. By exploiting LIF, LNT brings a new generation of treatments for currently untreatable diseases of the CNS, including rare diseases where 30% involve the CNS.LNT is an innovation leader, with impacts on UK's ability to drive growth. For MS, the immediate need is CTA that will maintain the high trajectory of LNT towards first in human trials.This Project not only brings our simple clean technology to the UK, to overcome the adverse impact of COVID-19 on LNT's progress to treat patients who suffer from MS. It also adds commercial strength to the UK economy as a leader in NanoMedicines.