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）患者的大脑萎缩 *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，从青少年后期开始，到20多岁后期变得明显。无药可救。目前的治疗方法仅为免疫抑制。* COVID-19疫情凸显了脆弱的年轻人，包括英国的MS-ers（MS患者），他们需要随着封锁的放松而继续保护。microSME **LIFNanoRx（LNT）** 是剑桥大学的一个分支，由科学家苏·梅特卡夫（Su Metcalfe）创立，用于治疗MS。认识到保护大脑的必要性，她利用FDA批准的凝胶纳米颗粒为LIF货物向大脑的持续递送提供了一个归巢装置，从而捕获了LIF的深刻的神经保护特性-一种对大脑健康至关重要的干细胞生长因子。耶鲁大学的合作者Tarek Fahmy为制造含LIF的纳米颗粒贡献了非凡的能力，并且是Metcalfe拥有的技术专利的共同发明人。临床前数据令人信服，证实了MS模型的疗效和安全性，并在治疗4天内部分逆转瘫痪。值得注意的是，治疗性纳米颗粒“LIFNano（tm）”可穿过血脑屏障（BBB）--这是治疗中枢神经系统（CNS）疾病的一种备受追捧的特性。*通过保护大脑，LIFNano（tm）为MS患者提供了一种低成本、高价值、安全的解决方案。在以前政府赠款的支持下，LIFNanoRx现在是一股主要力量-歌利亚中的大卫：与歌利亚不同-MS市场利润丰厚，支付者成本高，供应商回报高- LNT为患者带来了独特的颠覆性、低成本高价值的方法。MS社区中有许多LNT的追随者，他们了解LIF不仅是一种保护和治愈大脑的_无毒_天然解决方案，而且还可以消除MS的自身免疫根源。COVID-19* 由于计划于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COVID-19而停滞。封锁已阻止我们访问由耶鲁大学定制的LIFNano（tm）产品。*此次对I-UK可持续创新基金的竞标将把LIFNano（tm）的生产转移到英国，由英国另一家增长强劲的中小企业Pharmidex承担，该公司提供评估治疗大脑的药物的服务。该项目的预算还包括一个临床试验小组-我们之前的I-UK BMC奖（2017-2019）已经到位-在预算和时间内为这个SIF项目带来附加值。专家组的专家将监督IMPD准备工作，以便与MHRA监管机构接触。简单的清洁技术 * 作为一种完全合成的产品，LIFNano（tm）与基于细胞的疗法相比，大大减少了对环境的影响。通过开发LIF，LNT为目前无法治疗的中枢神经系统疾病带来了新一代的治疗方法，包括30%涉及中枢神经系统的罕见疾病。LNT是创新的领导者，对英国推动增长的能力产生了影响。对于多发性硬化症患者来说，最迫切的需求是CTA，它将保持LNT在人体试验中的领先地位。该项目不仅将我们简单的清洁技术带到英国，以克服COVID-19对LNT治疗多发性硬化症患者的不利影响，还将为英国经济增加商业实力，成为纳米药物的领导者。

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.