Revolutionizing stroke therapeutics

[Music]

now my grandfather and i have always

been close

from starting out taking naps on his

chest as an infant

to spending every day at his house as a

preschooler and toddler

learning my shapes and colors as i’ve

grown up

we’ve tried to stay close which can

sometimes be difficult when you live

over 700 miles apart from each other

so my grandparents and i we skype

a lot

lot which has allowed me to keep up with

the important things

such as what flavor of ice cream they

each got from dairy queen that day

or how tech support won’t help them with

their computers

keeping in touch so frequently has

allowed me to pick up on some of the

more subtler things as well

for example when i noticed some changes

to my granddad’s personality and memory

as well as some apathy to things he used

to love

with these changes my granddad began to

also have problems with his stability

and began to fall more

now most people might chalk this up to

normal aging

but as a medical student and a

neuroscience phd

i was afraid that something more serious

was actually happening

i was afraid that all of these memory

and personality changes

were actually signs of a greater

neurological base problem

i’m sure we can all sympathize with not

being able to remember where we put our

keys one day

but there’s a difference between that

and being able to remember what you had

for breakfast

i encouraged my granddad to see a

physician and some neuro imaging

revealed that my granddad was

experiencing

tiny restrictions in blood flow to

certain areas of his brain

or small strokes which were resulting in

something called

vascular dementia

now to really understand what was

happening in my granddad’s brain i want

you all to imagine a network of

sprinklers

watering a lawn on a hot summer day you

could see how if any of the sprinklers

became clogged or blocked

the grass in that area wouldn’t be able

to get the water or the nutrients it

needs

eventually over time this would cause

the grass in that area

to wither and die

now that was exactly what was happening

in my granddad’s brain

as certain blood vessels became clogged

or blocked the brain cells in that area

weren’t able to get the oxygen or

glucose they needed

eventually over time that caused those

cells in that area to die

and this cell death resulted in changes

to his personality

his memory and even his ability to walk

now i’ve memorized the signs and

symptoms of stroke

learned how to measure strokes on

neuroimaging and even work to develop a

therapeutic

to help the recovery of stroke sufferers

despite all of this knowledge i realized

how powerless i was to help

all of these hundreds of hours of

research and training

didn’t matter when i needed it the most

because there was nothing i could hand

him and say

here take this this will make it better

instead everything i and others had been

working on in the lab

while promising was still years away

from clinical trial

clinical implementation and making it

into the hands of the patients who

needed it

now my grandad is one of 15 million

people who experience a stroke

worldwide and don’t have time to wait

for a therapeutic either

what’s even more troubling is stroke is

not only

a leading cause of death and long-term

disability worldwide

but it hits us especially hard here in

georgia

if you take a look at the map behind me

no

that’s not a map of waffle house

locations

that’s actually a map of stroke

incidents

see we’re all living in a region called

the stroke belt

where the incidence of stroke is 30 to

50 percent greater than the rest of the

country

this means that 20 000 people in georgia

will have a stroke this year

and one in 18 people in georgia will

have a stroke in their lifetime

as you can see stroke is not only a

globally

devastating disease but it’s affecting

our friends

our families and our neighbors

we have to work together on strategies

to help those suffering today

now the biggest bottleneck to helping

those suffering today

is the divide between therapeutic

development and getting it to the

patients who need it

this divide is referred to as the gap

between the bench

and the bedside this means that while

we’re making great strides developing

new therapeutics and techniques in the

lab

they’re not making it to the patients

who need it

now why does this gap exist

well the drug development process is

long

difficult and expensive first

from drug must complete studies and

animal models

then it must move through three phases

of clinical trials

phase one is safety testing

if no adverse side effects are seen

after a couple months of testing in

human patients

then a drug can move to phase two or

efficacy testing where we ask

does the drug work this phase can last a

couple months to years and involves

hundreds of patients

only about thirty percent of drugs make

it past phase two

in phase three there’s large-scale

testing

in hundreds of thousands of patients

over multiple years

if a drug is one of the 14 that

successfully completes

all three phases of these clinical

trials only

then can it be requested for fda

approval

this arduous process takes an average of

12 years and 1 billion dollars to

complete

now these fda approval and clinical

trial steps are absolutely necessary and

i’m not advocating we do anything to

change them

i am however advocating we make this a

two-fold process

in order to help patients the best of

our abilities present

and future we must one continue

therapeutic development

but two simultaneously work on

immediately

implementable techniques frankly because

people are suffering and dying of this

disease today

and it’s our obligation to help them to

the best of our ability

during my phd i wanted to test out this

two-fold approach in practice

in order to focus on something with

immediate potential i set my eye on

stroke recovery prediction models

while the ability to predict someone’s

recovery after stroke has improved in

recent years

there’s still room for refinement

i wanted to see if we could use narrow

imaging a patient was already undergoing

such as mri and use that to predict

their outcome down the line

now the goal of this work isn’t to

develop something that’s going to

immediately enhance a person’s ability

to walk or talk after a stroke

the goal of this research is however to

develop something that physicians can

use to make

targeted and personalized rehabilitation

plans for patients

in order to make these targeted plans i

took an mri

and made a bunch of measurements that

were related to the brain after stroke

i then looked for the relationships

between these measurements and long-term

outcomes in our models

from this analysis i was able to

identify one measurement out of many

tested

that was significantly correlated to

gait behavior

survival and recovery up to 12 weeks

later

this is pretty awesome we have something

we can easily measure within the first

24 hours of stroke

that can tell us what to expect out of a

person’s walking and recovery

up to three months later and this is

something that doctors can start

implementing for people like my

grandfather

right now

while doing this work i also wanted to

focus on the overarching goal

of this twofold process or developing a

therapeutic

that can aid in the recovery of stroke

patients

currently there is only one fda approved

small molecule therapeutic for stroke

called

tpa now tpa works to break up the clot

think drano for the brain rhino

but it has no direct mechanisms to

preserve protect or regenerate tissue

thinking back to our hose analogy it’s

like finally being able to untangle or

unclog that hose after

hours of decreased water flow

sure it’ll help save some parts of the

dead and dying grass or brain

but for the areas that’s already died

it’s already too late

building off the work of others before

me i worked with my lab mates

to develop a therapeutic with the

potential to heal

this neurological damage this

therapeutic

called neural stem cell-derived

extracellular vesicles

or nsevs are nano-sized packets of dna

rna and protein instead of just working

to reopen the hose

like current approved therapeutics with

these nsevs we’re actually

reseeding parts of the dead and dying

lawn

to salvage what used to be there and

encourage new life

from our experiments we’ve seen that

these nsevs

not only decrease the damage done from

stroke but actually

improve the speed of recovery and even

large detrimental strokes

to really illustrate this i have two

videos to show you

in our large animal pig model of stroke

the first video is of me and an animal

three days after stroke

this animal has not received treatment

for this testing we want the animal down

a track which measures property of their

gait

or walking such as their pressure speed

or stride length

as you can see this animal needs a lot

of encouragement from this rattle to

successfully walk down the track

in contrast here is an animal three days

after stroke

that has received these nscevs or

seeds

as you can see there’s a perceptible

difference

in the speed of recovery of the animal

that has received the nscevs

compared to the animal that hasn’t

based on these experiments we believe

that our nsevs have promising potential

to help the recovery of stroke patients

worldwide

now i truly believe this two-fold

approach

of continuing therapeutic development

while also simultaneously working on

immediately implementable techniques

will be one of the keys to providing the

best care we can for patients

but there’s one last part of this game

plan i’ve left out and that’s step

three or you

see stroke has been a devastating

disease for a long time

and i’m sure you’ve all been personally

affected by it

due to limited success in clinical

trials however funding agencies and

individuals

have started to lose hope in the

possibility of finding a cure for stroke

we want to reinvigorate the field to

believe in techniques for today while

continuing to work towards a cure

for tomorrow in order to bring about

this change

we need your confidence support and

participation

this work is only possible through your

endorsement so we’re actually

asking you to show your support vocally

and monetarily

speak out on social media in

conversations with your communities

through political advocacy and through

contributions to research drives and

taxes

through your passion and support we can

bring awareness to the need for more

stroke research

together through this raised awareness

of stroke and multifaceted research

approaches we can start to re-landscape

the field

maybe one day we’ll be able to talk

about life after stroke

not in months and years of rehab

but maybe in days and weeks

not as a completely debilitating life

event

but as an unfortunate setback

we can start to restructure the emphasis

from an all-or-nothing game of

therapeutic development

to an all or something game that can

help patients today

frankly because i want my granddad back

i want him back to his happy healthy

jokester self

and i want to be able to hand him

something that can make it better before

it’s too late

and i want the same for all of your

loved ones and grandparents

and maybe after today we’ll be one step

closer to making that a reality

thank you

[音乐]

现在我的祖父和我

一直很接近,

从婴儿开始在他的

胸前小睡到

学龄前儿童和蹒跚学步的孩子每天都在他家

学习我的形状和颜色,因为我

长大了,

我们尝试过 当你们相距超过 700 英里时,保持近距离

有时会很困难,

所以我和我的祖父母

经常使用 Skype,这让我能够

跟上重要的事情,

比如他们

每个人都吃什么口味的冰淇淋 那天的乳制品皇后,

或者技术支持如何无法帮助他们与

他们的计算机

保持如此频繁的联系,这

让我也能够了解一些

更微妙的事情

,例如,当我注意到

我祖父的性格和记忆发生了一些变化

时 以及对他

过去喜欢

的这些变化的一些

冷漠 耳鼻喉科和

神经科学博士

我害怕发生更严重

的事情

我们有一天把钥匙放在了

那里,但这

和能够记住

早餐吃了什么是

区别

导致

血管性痴呆的大脑或小中风

现在要真正了解

我祖父的大脑中发生了什么 我希望

你们都想象一个

洒水器网络

在炎热的夏天浇灌草坪 你

可以看到任何洒水器如何

被堵塞或阻塞

该区域的草最终

无法获得所需的水或

营养 我这会

导致该区域的草

枯萎

死亡,这正是

我祖父大脑中发生的事情,

因为某些血管堵塞

或阻塞了该区域的脑细胞

无法获得所需的氧气或

葡萄糖

最终随着时间的推移,

导致该区域的那些细胞死亡

,这种细胞死亡

导致他的性格发生变化,

他的记忆力甚至他的行走能力

现在我已经记住了中风的体征和

症状,

学会了如何在

神经影像学上测量中风,甚至 努力开发一种

治疗方法

来帮助中风患者康复

尽管有这些知识我意识到

我是多么无能为力来帮助

所有这些数百小时的

研究和培训

在我最需要它的时候并不重要因为我

什么都没有 可以

递给他,

说拿这个,这会让它变得更好,

而不是我和其他人在实验室里所做的一切,

而承诺离临床还有几年的时间

我的祖父是全球 1500 万

中风患者中的一员

,他们没有时间

等待治疗,

更麻烦的是中风

不是 只是

全球死亡和长期

残疾的主要原因,

如果你看看我身后的地图,它对格鲁吉亚的打击尤其严重,

,那不是华夫饼屋

位置

地图,实际上是中风事件地图,

看我们 所有人都生活在一个称为中风带的地区,该

地区的中风发病率

比该国其他地区高出 30% 到 50%

这意味着格鲁吉亚

今年将有 20 000 人中风

,格鲁吉亚每 18 人中就有 1 人患有中风

如您所见,中风不仅是一种

全球性的

破坏性疾病,而且它影响着

我们的朋友、

家人和邻居,

我们必须共同努力制定策略

来帮助他们 今天的痛苦 帮助今天受苦的人

的最大瓶颈

是治疗

开发与将其提供给

需要治疗的患者之间的

鸿沟,这种鸿沟被称为

长凳和床边之间的差距,

这意味着虽然

我们做得很好

在实验室中开发新的治疗方法和技术取得了长足的进步

他们现在没有将其提供给现在

需要它的患者

为什么这种差距存在

良好 药物开发过程

长期

困难且昂贵 首先

从药物必须完成研究和

动物模型

然后它必须移动 通过

临床试验的三个阶段,第一

阶段是安全性测试,

如果

在对人类患者进行几个月的测试后没有发现不良副作用,

那么药物可以进入第二阶段或

功效测试,我们

询问药物是否有效,这个阶段可以持续

几个月到几年,涉及

数百名患者,

只有大约 30% 的药物在第三

阶段通过

第二阶段 - 多年来

在数十万患者中

进行大规模测试

如果一种药物是

成功完成

这些临床试验所有三个阶段的 14 种药物之一,

那么它才能申请 FDA

批准

这个艰巨的过程平均需要

12 年和 1 十亿美元

现在完成这些 FDA 批准和临床

试验步骤是绝对必要的,

我不主张我们做任何事情来

改变它们,但

我主张我们将其作为一个

双重过程

,以帮助患者尽

我们所能

未来我们必须一个人继续

治疗发展,

但坦率地说,两个人同时致力于

立即

实施的技术,因为

今天人们正在遭受这种疾病的折磨和死亡,我们有义务

在我的博士学位期间尽我们所能帮助他们,我想测试这

两个 - 实践中的折叠方法,

以便专注于具有

直接潜力的事情,我将目光投向了

中风恢复预测 n 个模型,

虽然近年来预测某人

中风后康复的能力有所提高,

但仍有改进的

余地 这项工作的目标

不是开发能够

立即提高一个人

在中风后走路或说话的能力的

东西,而是开发一些医生可以

用来为患者制定

有针对性和个性化的康复

计划

的东西 为了制定这些有针对性的计划,我

进行了核磁共振检查

并进行了一系列

与中风后大脑相关的测量,

然后在我们的模型中寻找

这些测量与长期结果之间的关系,

从这个分析中我能够

确定一个 许多测试

中与

步态行为

存活和恢复显着相关的测量结果长达 12 周

后 这是非常棒的,

我们可以在中风的前

24 小时内轻松测量一些东西,

它可以告诉我们三个月后一个

人的行走和恢复的预期

这是医生可以开始

为像我这样的人实施的东西

祖父

现在

在做这项工作时,我还想

专注于

这个双重过程的总体目标,或者开发

一种可以帮助中风患者康复的

治疗方法

目前只有一种 FDA 批准

的中风小分子治疗方法,

称为

tpa 现在 tpa 有效 为了打破凝块,

想想大脑犀牛的德拉诺,

但它没有直接的机制来

保护或再生组织

回想我们的软管类比,

就像在水流减少数小时后终于能够解开或

疏通软管

肯定它会 帮助拯救

死亡和垂死的草或大脑的某些部分,

但对于已经死亡的区域,

建立 w 已经为时已晚 在我之前的其他人中,

我和我的实验室伙伴

一起开发了一种有

可能治愈

这种神经损伤的

治疗方法,这种治疗方法

称为神经干细胞衍生的

细胞外囊泡

或 nsev,是纳米大小的 dna

rna 和蛋白质包,而不仅仅是

致力于 用这些 nsev 重新打开软管,

就像当前批准的治疗方法一样,

我们实际上是在

重新种植部分死亡和垂死的

草坪,

以挽救曾经存在的东西,并

从我们的实验中鼓励新的生命我们已经看到,

这些 nsev

不仅减少了造成的损害

中风,但实际上

提高了恢复速度,甚至是

大的有害中风

来真正说明这一点

对此测试的治疗,我们希望动物

沿着测量其步态或行走特性的轨道行驶,

例如压力速度

或 st 骑行长度,

如您所见,这只动物需要

从拨浪鼓中得到很多鼓励才能

成功地走下赛道

,相比之下,这是一只

在中风三天后

收到这些 nscev 或

种子

的动物,您可以看到速度上有明显的

差异 与未

接受 nsevs

的动物相比,接受 nsevs 的动物的康复率

基于这些实验,我们

相信我们的 nsevs 具有

帮助全世界中风患者康复的巨大潜力,

现在我真的相信这种

持续治疗的双重方法 开发

同时致力于

立即实施的技术

将是

我们为患者提供最佳护理

的关键之一,但

我遗漏了这个游戏计划的最后一部分,那就是第三步,

否则你会

看到中风是毁灭性的

疾病很长一段时间

,我敢肯定,由于临床试验的成功有限,你们都受到了个人的

影响。

r 资助机构和

个人

已经开始

对找到治愈中风的可能性失去希望

我们希望重振该领域,

相信今天的技术,同时

继续努力

争取明天的治愈,以实现

这一改变,

我们需要你的 信心支持和

参与

这项工作只有通过您的支持才能实现,

因此我们实际上

要求您

在社交媒体上

通过政治宣传和通过

对研究驱动和

税收的贡献

通过您的热情与您的社区进行对话,以口头和金钱方式表达您的支持 和支持 我们可以

通过提高

对中风的认识和多方面的研究

方法使人们意识到需要更多的中风研究 我们可以开始重新规划

该领域

也许有一天我们将能够

谈论中风后的生活

而不是几个月 和多年的康复,

但也许在几天和几周内

不会像完全虚弱的生活

一样 耳鼻喉科,

但作为一个不幸的挫折,

我们可以开始将重点

治疗

发展的全有或全无游戏调整为可以帮助今天的患者的全有或全无游戏

坦率地说,因为我希望我的祖父回来,

我希望他回到他快乐健康的状态

开玩笑的自己

,我希望能够

在为时已晚之前给他一些可以让它变得更好的东西

,我希望你所有的

亲人和祖父母都一样

,也许今天之后我们将更

接近实现这一目标

谢谢