Die Trying - The Battle For ALS Treatment (VICE on HBO: Season 4, Episode 16)

Summary

The Vice documentary explores the urgent search for an ALS cure, highlighting the personal stories of patients like Beth and Matt, who face the devastating progression of the disease. It delves into the challenges of clinical trials, the FDA's cautious drug approval process, and the desperate measures patients take, such as seeking experimental treatments abroad. The documentary underscores the need for a reevaluation of drug approval protocols to expedite access to potential treatments and give hope to those battling this fatal disease.

Takeaways

• 🕒 The urgency of finding a cure for ALS is underscored by the fact that patients are losing their abilities rapidly, and time is of the essence.
• 💉 Despite the Ice Bucket Challenge raising over 220 million dollars for ALS research, no one in the U.S. is receiving the experimental drug discussed in the script due to FDA safety concerns.
• 📉 The disease progresses quickly, leading to unrelenting muscle loss and eventual paralysis, while cognitive function remains largely intact.
• 🧬 ALS patients face significant barriers to accessing experimental treatments, with clinical trials being the primary way to access potential drugs, but strict eligibility criteria often exclude patients.
• 🌐 Some patients are forced to travel abroad to access experimental treatments, highlighting the limitations of the U.S. healthcare system in providing cutting-edge care for ALS.
• 🔬 Research centers like the ALS Therapy Development Institute are working tirelessly to understand the disease and find treatments, focusing on pharmacology and testing various compounds.
• 💼 The FDA's cautious approach to drug approval can be detrimental to ALS patients who are willing to take risks on unproven treatments in the hope of slowing or stopping their disease.
• 💊 Even when effective treatments are found, the high cost and regulatory hurdles can prevent patients from accessing them, as illustrated by the story of a drug company unable to provide their treatment in the U.S. due to financial constraints.
• 🌟 The potential for ALS research to benefit other neurodegenerative diseases like Alzheimer's, Parkinson's, and Huntington's is highlighted, emphasizing the broader implications of finding a cure.
• 🚫 The script calls for a reevaluation of the FDA's drug approval process for diseases with no viable treatment, advocating for a more patient-centered approach that acknowledges the desperate need for solutions.

Q & A

What is the primary focus of the documentary 'Vice' this week?

The primary focus of the documentary 'Vice' this week is the search for a cure for ALS (Amyotrophic Lateral Sclerosis) and the challenges faced by patients and researchers in finding effective treatments.

How much money was raised for ALS research through the ice bucket challenge in 2014?

The ice bucket challenge raised more than 220 million dollars for ALS research in 2014.

What criticism did the ice bucket challenge face?

Critics of the ice bucket challenge dismissed it as viral narcissism, suggesting that people were more interested in showing off than actually helping the cause.

What is the current situation regarding the availability of the drug discussed in the script in the U.S.?

The drug discussed in the script is not available in the U.S. due to FDA regulations and concerns about safety. No one in the U.S. is receiving the drug at the moment.

What is the role of the FDA in the approval process for drugs like those for ALS?

The FDA is responsible for ensuring the safety and efficacy of drugs before they are released to the public. It has a standard drug approval process that can be a contentious issue for terminally ill patients who are willing to accept risks associated with unapproved drugs.

What is the significance of the ALS Therapy Development Institute mentioned in the script?

The ALS Therapy Development Institute is the world's first research center solely focused on ALS. It plays a crucial role in researching and developing potential treatments for the disease.

What is the current status of clinical trials for ALS treatments?

Clinical trials for ALS treatments are ongoing, but they are limited in scope and often have strict exclusion criteria, making it difficult for many patients to participate.

Why do some ALS patients travel abroad for treatment?

Some ALS patients travel abroad for treatment because they are unable to access experimental treatments or clinical trials in the U.S. due to strict regulations and limited availability.

What is the 'expanded access' program mentioned in the script?

The 'expanded access' program is an FDA initiative that allows patients to try unapproved drugs. However, the process can be lengthy and may not be feasible for ALS patients who deteriorate rapidly.

What is the potential impact of changing the drug approval process for ALS as suggested by Dr. Andrew Lo?

Changing the drug approval process for ALS, as suggested by Dr. Andrew Lo, could lead to faster approvals and more trials, potentially increasing access to treatments and accelerating the development of cures for ALS and other progressive neurological diseases.

What is the current reality for ALS patients who are severely disabled, as depicted in the script?

The current reality for severely disabled ALS patients is grim. They often require 24/7 care, face the decision of whether to get a tracheotomy and use a ventilator, and live with the constant threat of choking or suffocating due to their inability to move or communicate effectively.

Outlines

00:00

💉 The Quest for an ALS Cure

The paragraph introduces the personal journey of a Vice journalist, Angelina, who is diagnosed with ALS. It discusses the urgency of finding a cure as the disease progresses rapidly, affecting not just the patient but also their families. The script also touches on the Ice Bucket Challenge that raised significant funds for ALS research, yet the FDA's stringent safety regulations prevent patients from accessing experimental treatments. The narrative is interspersed with emotional accounts from patients and their families, highlighting the devastating impact of ALS.

05:05

🧬 ALS Research and Clinical Trials

This section delves into the research efforts at the ALS Therapy Development Institute, where Dr. Perrin explains the disease's progression and the challenges in finding a cure. The script discusses the Institute's focus on pharmacology and the use of mice to test potential treatments. It also addresses the limited number of clinical trials and the stringent criteria that exclude many patients, like Matt Bolena, who are desperate to participate in trials to access potential treatments.

10:05

🌐 Global Struggle for ALS Treatment

The paragraph discusses the difficulties faced by ALS patients in accessing experimental treatments due to stringent regulations. It features Wissam Majid, who had to travel to Lebanon for an experimental stem cell treatment not available in the US. The narrative criticizes the FDA's cautious approach, which, while prioritizing safety, also delays potentially life-saving treatments. The script also includes the perspective of a drug company CEO, who explains the financial barriers to providing unapproved drugs and the emotional toll of denying patients access to potentially life-changing treatments.

15:05

🏥 FDA's Role in ALS Treatment Access

This section explores the FDA's stance on drug approval for ALS, emphasizing the need for a balanced approach between safety and accessibility. Dr. Janet Woodcock explains the FDA's expanded access program, which allows patients to try unapproved drugs, but the process is time-consuming and often not feasible for rapidly progressing diseases like ALS. The script also introduces an economist's proposal for a disease-specific risk assessment model that could potentially speed up drug approvals and encourage pharmaceutical investment in treatments for severe diseases.

20:07

🌿 The Harsh Reality of Living with ALS

The paragraph portrays the harsh realities of living with ALS, focusing on the physical and emotional toll it takes on patients and their caregivers. It features Erik Valor, who is completely paralyzed and relies on a machine for breathing and eye-tracking technology for communication. The narrative discusses the difficult decisions patients must make, such as getting a tracheotomy, which ends natural speech but ensures survival. The script also touches on the broader implications of ALS research for other neurological diseases.

25:07

🚨 The Urgency for ALS Treatment

The final paragraph is a call to action, emphasizing the urgency of finding effective treatments for ALS. It highlights the Ice Bucket Challenge's success in raising awareness and funds but stresses that more needs to be done to improve access to experimental treatments. The script concludes with a powerful message from patients, urging the FDA to act quickly and grant them the right to try potentially life-saving treatments.

Keywords

💡ALS

ALS, or Amyotrophic Lateral Sclerosis, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to loss of muscle control and eventually death. In the video, ALS is the central theme, as it discusses the search for a cure and the challenges faced by patients and researchers.

💡Ice Bucket Challenge

The Ice Bucket Challenge was a viral fundraising campaign that raised over 220 million dollars for ALS research. It is mentioned in the script as a pivotal event that brought ALS into the national spotlight and funded research efforts, despite criticism from some who saw it as more about self-promotion than actual help.

💡FDA

The FDA, or Food and Drug Administration, is a United States federal agency responsible for ensuring the safety of drugs and treatments. In the context of the video, the FDA's strict approval process for drugs is discussed as a potential barrier to patients accessing experimental treatments for ALS.

💡Clinical Trials

Clinical trials are experiments conducted to determine the safety and effectiveness of medical treatments. The video discusses the limited access to clinical trials for ALS patients and the challenges they face in participating, which is critical as there is currently no cure or effective treatment for the disease.

💡Neurone

Neurone is a clinical trial mentioned in the script that showed promising results in slowing down the progression of ALS in a majority of the patients who received the full treatment. It represents hope for patients but also the frustration of limited trial availability.

💡Stem Cell Treatment

Stem cell treatment is a therapeutic approach that uses stem cells to repair damaged tissues or cells. In the video, some ALS patients travel abroad for experimental stem cell treatments, highlighting the desperation and the limitations of treatment options within the United States.

💡Tracheotomy

A tracheotomy is a surgical procedure that creates an opening in the windpipe to help patients breathe. In the video, the decision to get a tracheotomy is portrayed as a difficult one for ALS patients, as it signifies the loss of natural speech and the need for permanent ventilation.

💡Neurologist

A neurologist is a medical professional specializing in disorders of the nervous system. In the script, a neurologist plays a critical role in diagnosing ALS and guiding patients towards potential treatments, emphasizing the importance of their role in patient care.

💡Paralysis

Paralysis is the loss of muscle function in part of the body. The script describes the progression of ALS leading to paralysis, illustrating the devastating physical toll the disease takes on patients and the urgency of finding a treatment.

💡Cognitive Function

Cognitive function refers to the mental processes involved in thinking, learning, and memory. The video notes that while ALS patients experience physical decline, their cognitive function often remains intact, leaving them fully aware of their deteriorating condition.

💡Expanded Access

Expanded access is a program that allows patients to try unapproved drugs. The script discusses the FDA's expanded access program as a potential solution for ALS patients who are willing to take the risk of using experimental drugs to treat their disease.

Highlights

The search for a cure for ALS is urgent, with patients fighting against time.

No one in the US is receiving a particular drug due to FDA safety concerns.

The Ice Bucket Challenge raised over $220 million for ALS research in 2014.

Critics view the Ice Bucket Challenge as narcissistic rather than beneficial.

Angelina, a journalist at Vice, shares her personal connection to ALS.

ALS symptoms can start subtly, like difficulty in typing or applying suntan lotion.

The progression of ALS can lead to complete paralysis and death.

There is currently no effective treatment or cure for ALS.

ALS TDI received only a fraction of the Ice Bucket Challenge funds.

Research at ALS TDI focuses on pharmacology and using mice to understand the disease.

Clinical trials are the only chance for patients to access potential treatments.

Patients face significant barriers to entry in clinical trials due to strict criteria.

Some patients are willing to take risks with unapproved drugs to potentially slow ALS progression.

The FDA's approval process is criticized for being too slow and restrictive for terminal diseases like ALS.

Patients are forced to travel abroad for experimental treatments due to limited options in the US.

Drug companies cannot charge for unapproved drugs in the US, limiting access to potential treatments.

A new mathematical framework is proposed to judge risk on a disease-by-disease basis for drug approval.

The current system may deter pharmaceutical companies from investing in treatments for ALS.

Patients with ALS face difficult decisions regarding their quality of life and medical interventions.

The urgency of ALS requires the FDA to reevaluate its drug approval process to save lives.

Transcripts

this week on Vice the search for a cure

for ALS we're fighting the clock what I

don't know is if there's enough time for

my daughter how many people in the US

are getting your drug in the u.s. no one

the FDA is worried about safety every

day that passes he's losing more of

himself

[Music]

[Applause]

[Music]

in 2014 the ice bucket challenge raised

more than 220 million dollars for

research into ALS now it put this fatal

neurological disease in the national

spotlight for the first time since Lou

Gehrig's famous diagnosis in 1939 but

for us your advice

this was already a very personal story

[Music]

my name is Angelina 4 News and I work at

vise I wrote the article for vise calm

the worst part of the ice bucket

challenge is the people criticizing it

critics of the ice bucket challenge

dismissed it as viral narcissism where

people were more interested in showing

off than helping out I wrote the article

because the campaign raised hundreds of

millions of dollars and it brought

attention to a cause that had been

widely ignored for over 75 years but

that was in the only reason I was just

having trouble typing at first turning

on my blow-dryer you know the little

things and I didn't think anything of it

until one day I went to spray suntan

lotion and the suntan lotion completely

slipped out of my hand and I remember

looking down and thinking oh my god this

is what paralysis looks like within a

week the best case scenario went from

shoulder surgery to spinal surgery to

something we can't treat and I didn't

know what something we can't treat was

like what is that like would I just have

like a really up left hand for

the rest of my life until my neurologist

gave me a phone number to call for a

follow-up

he's like you need to call this for a

second opinion so the next day I called

and the woman who answered said thank

you for calling the ALS and Lou Gehrig's

disease Center at Columbia and I dropped

the phone and I just sat in my bed

people now know what ALS is but most

don't understand how quickly the disease

destroys your body and how hard it is to

find any kind of treatment so I set out

to do a non-traditional piece of

journalism reporting from the patient's

perspective and showing firsthand the

challenges we face I started with one of

the first commenters on my story

Robert Hepburn and his daughter Beth hi

my name is Beth a friend I'm here is

doing the ice bucket challenge

[Music]

oh my god diagnosed with ALS

I mean the misery is why my life was

very active

it was working in Washington DC as a

paralegal and then on the weekends I

went and go out at night no do your 3:00

in the morning like I need on the 1d is

something does living in this living I

didn't want just I'm pouring old chair I

wanted it to look good agency in the

dark

[Music]

I need to get some like Exodus the your

Islam thank you

you know it started out with she needs

help cutting her food okay

you get over it you deal with it you

move on and then it's like okay she

needs help

unzippering her pants to go to the

bathroom okay

learn how to do it deal with it move on

to lose that autonomy and your

independence nobody gives that obviously

you hang on to that as long as you can

[Applause]

[Music]

do anything no no me and it's like the

way they're change was that me my money

a year ago

I can still lift my arm and I can put on

sunscreen I can wash my hand I really

believe this will be cured someday you

know what I don't know is if there's

enough time for my daughter and I really

you know kind of hope and pray there is

but we're we're fighting the clock

Ellis researchers are fighting the clock

to racing to find an effective

to see what progress they've made I

visited the world's first Research

Center solely focused on this disease

the ALS therapy Development Institute

dr. C Perrin gave me a candid

explanation of what ALS patients are up

against what happens when you get this

disease unrelenting muscle loss from

wherever you get your first symptoms to

spreading across the body the motor

neurons that control your movements the

brain of the cell is up in your spinal

cord it sends these processes down out

of your spinal cord into your muscles

tour actually innervates the tissue when

that nerve pulls back the muscle no

longer has any signaling from the brain

if you will and how to move and your

muscles progressively just atrophy

throughout your entire body and that's

ultimately what ends up causing

paralysis and eventually death that your

central nervous system in your brain is

for the most part in most patients

unaffected meaning you have full

cognitive function so you know exactly

what's going on around you and yet you

have no ability to communicate speak or

move

is there even a viable treatment for it

right now

there is no effective treatment or a

cure today there's some on the horizons

we're optimistic we're gonna get there

als TDI received only four out of the

220 million dollars raised by the ice

bucket challenge which they immediately

channeled into the most promising drugs

a big part of our research program at

als TDI is pharmacology so basically

that's trying to understand how

therapeutics are working in a whole

animal system we focus on mice and the

way we use the animals is number one to

try to understand the disease but also

and maybe more importantly we're trying

to find treatments the first Mouse that

we're looking at it has the gene in it

that would cause the disease but it

hasn't had onset yet so you can see he's

a little spunky

so how far progress is this this one is

probably about 3/4 of the way through

its disease progression it doesn't have

a normal gait with its hind limbs

anymore it starts to drag its toes a

little bit and ultimately it won't be

able to use its hind limbs at home a lot

of our effort is to test therapeutics to

make this Mouse basically have a better

longer life we've tested a lot of drugs

probably somewhere on the order of 300

different compounds we've had three show

efficacy up until now in those are our

clinical trial candidates that we're

moving forward right now clinical trials

are how drugs are tested before their

release to the public and because

there's currently no proven treatment

for ALS these trials have become the

only chance for patients to access drugs

that could help them but for patients

like Matt Bolena just getting into a

trial is a battle in itself you don't

want me to push you gonna do it all on

your own that's good I really think I

like the kids and my wife is the victim

to the disease because you know they're

the ones that have to pick up a slack

and change their lives buddy you know I

can't please dad see it's like the

saddest thing in the world isn't it like

you say I just come round with me I'm

like sorry dude

[Music]

I was flying for the Navy I was fine

prowlers and then I started having

symptoms and the Navy didn't know what

it was so they they grounded me they

said you can't fly anymore what was the

plan of action for treatment did you

look at experimental stuff I had already

started like about a year before that

like taking stuff because I had a

feeling that I had ALS so I had been

taking a bunch of experimental stuff

already

what about clinical trials from the day

I was diagnosed I had already had too

many years worth of tests to make I

can't get into any except two years

since onset so I can't get into a

clinical trial oh wow so you were

diagnosed and you were like immediately

outside of the exclusion criteria it's

miserable because like you want to be

part of the solution too like you want

to be providing data to the scientists

so we can't do it it's ridiculous a guy

I'm like I I've been you know on and off

risking my life for ten years let me try

some drugs that are gonna potentially

help I think the risk and I'm not scared

of it for most rapidly fatal diseases

the standard drug approval process is a

hotly contested issue with many

terminally ill patients willing to

accept the risks that come with an

unapproved drug one of the most coveted

trials is called neurone it slowed down

the progression of the disease in 92

percent of the patients who received the

full treatment

the brainstormed trial at Mayo accepted

16 patients we had almost a thousand

patients on the waiting list

like many ALS patients I applied to this

trial and didn't get in hey Kevin Haase

was one of the very few did when they

called and said that I can have that

I just couldn't believe it because they

thought how many tens of thousands of

people are praying and hoping for the

same chance one of the important steps

in the brainstorm trial is that the

cells come out of the bone marrow and

then they're treated in a way that makes

them protective for nerve cells so

they're kind of enhanced stem cells

he'll have a spinal turn and that allows

us to inject the stem cells into the

fluid around the spinal cord sorry okay

yes and when you have an injury anywhere

in your body those cells will move out

of the bone marrow and they go to the

area that's injured and they aid the

healing process now we're taking these

healing cells and we're putting them

into the nervous system so we're putting

them into a place where they don't

usually go I don't know it's kind of

like surreal

I remember sending my paperwork to get

into this trial

[Music]

don't feel like crying on camera today

so I think we should probably cut due to

the limited scope of these trials many

ALS patients like Wissam majid are left

with no other option than to go abroad

to seek treatment Oh about five seconds

bureaucracy and all that is getting in

the way right now and the FDA is worried

about safety I mean the side-effect of

ALS is death it's not right I mean every

day that passes he's losing more of

himself

and that goes for every als patient yeah

he's willing to take the risk which as

far as it was not yeah my wonder I have

none the Pythagorean

if there were options here in America

we'd prefer to do it here but there are

no options right now Wissam was forced

to leave the country his family and

friends raised $30,000 to send him to

Lebanon for an experimental stem cell

treatment that works in a similar way as

neurone we're in Beirut and we are on

our way to see wissam the day before his

big stem-cell procedure what are your

expectations for tomorrow time no that's

my goal - and then you can kind of see

it in my left hand also in my right hand

of atrophied a little bit no I know

we followed with some as he prepared for

treatment I'm good how are you feeling

[Music]

hopefully they find a cure soon before

anything happened I wanted to understand

why patients like Bassam who are

severely disabled are forced to travel

halfway across the world just to access

treatment so I met with dr. Janet

Woodcock the director of the FDA Center

for Drug Evaluation and Research for a

disease like ALS where a drug can work

for one person and not necessarily the

other because no two ALS patients are

alike what's the harm in just releasing

a drug if it's safe first of all most

drugs have liabilities if FDA were just

released a lot of ineffective drugs out

there we would never find out we'd never

find out which ones are harmful we'd

never find out which ones are beneficial

we know this from long and bitter

experience but if a patient is willing

to take the risk if I spoke to a navy

jet pilot who has the disease and he's

willing to sign off on it I'm willing to

sign off on it again what is the harm of

releasing a drug that we're willing to

take the risk before well we don't want

to stand between people trying

investigational drugs okay so there's a

large access programs that FDA runs

where we agree that people who wish to

try investigational products can sign on

to that we agree with that philosophy

the

da program is called expanded access and

it allows patients to try unapproved

drugs but in the months it often takes

to complete the paperwork ALS patients

can lose their ability to walk and speak

or breathe on their own exhausted by the

process patients often give up and as we

learn from rich KC the CEO of a company

that developed a highly promising drug

known as NP double O one even innovative

drug companies need revenue to keep

their research going but in the u.s.

drug companies are not allowed to charge

for unapproved drugs and that leaves

American patients out in the cold how

many people in the US are getting your

drug through compassionate use and how

many people are getting it Europe in the

u.s. nobody and the reason it's

available there and not here is that

their laws allow us to be reimbursed for

our costs because we obviously don't

have the financial resources to just

give it away and in Europe we can get

reimbursed for our costs here in the US

you can't it's against the law to sell

an unapproved drug how did it feel when

ALS patients who responded to your drug

came to you and asked you for the drug

this is horrible horrible feeling I mean

you feel like less than human because

these people were doing well and of

course I wanted to give it to him but

there was no way we had no money at that

time there was questions about whether

we would survive as a company it's a

really heart-wrenching you know

discussion with these patients

and then of course you know most of them

end up dying so it's a various tragic

MIT economist dr. Andrew Lowe thinks the

real issue here is that the FDA's

current approval system treats all

diseases the same so he developed a

complex formula that judges risk on a

disease by disease basis you're saying

that not all diseases should be treated

equally als is a good example patients

might be willing to take a bigger risk

of getting a drug that may not work if

there is some chance that that drug

might work whereas somebody who's taking

acne medication may not be willing to

take any kind of chance for adverse

consequences so the idea is to use this

mathematical framework that we developed

and to be able to come up with a single

number that says here this is the

threshold you ought to use for approving

drugs for this disease whereas for this

other disease that's maybe not as

serious you can use a different

threshold a more conservative threshold

lowering that threshold would not only

increase access for patients but also

incentivize pharmaceutical companies to

invest in these treatments drug

companies when they think about

investing in a particular drug

development program they've got to

factor in how much they're going to put

into it versus how much they can get out

of it and if there's a lot of

uncertainty about whether or not they

can get a drug approved they may not

invest dollars initially if on the other

hand we change the standards for these

terrible illnesses then drug companies

were willing to invest more we might get

cures sooner rather than later if this

theory is correct the adoption of the

technique would lead to faster approvals

and more trials which would open up the

possibility of treating or even curing a

whole host of diseases that first

effective treatment for a loss is going

to be the biggest game-changer we've

ever seen and ultimately because ALS

does look like other progressive

neurological diseases Alzheimer's

Parkinson's Huntington's some of these

drugs will also work there so the

investment that was made to do this hard

work in a loss is also going to reap

benefits in other patients with

significant unmet needs as well without

reevaluating how we approve drugs for

diseases but no viable treatment the

only option patients have is how to

spend the remaining days afternoon and

welcome to the critical command center

Erik valor is paralyzed from the neck

down a machine pumps oxygen into his

lungs and eye tracking technology as his

only means of communicating the decision

to get a tracheotomy was worrisome

because it would mean the end of natural

speech I delayed as long as possible but

after a nearly fatal choking incident I

consented

[Music]

Oh because he doesn't burp sometimes

when we opened the tube at Bert's

soaring three times a day

Erik receives his meals through a

feeding tube which is inserted directly

into his stomach look at his arms just

just literally skin and bones same with

the legs so we have to fight to keep

wait aren't you still Erik is in

constant danger when he triggers this

alarm by scrunching his cheek

it means he's choking on his own saliva

or mucus when Erik needs to be suctioned

it's immediate because he can choke

together because you have to remember he

can't talk he can't sneeze he can't

hiccup he can't burp that's why the

caregivers are so close

Eric has to have 24/7 care he cannot be

left alone

at all not even for a minute

this isn't just Eric this is a decision

all ALS patients will eventually have to

make and this disease is so aggressive

in the short time since we filmed with

Beth she too is faced with the decision

of whether or not to get a tracheotomy

lose what's left of her natural speech

and indefinitely breathe through a

ventilator what kind of life are you

really living on that drink

I just don't know that's the thing I you

will have the meds Oh straight to go

through like day after day year after

year

what are you thinking what's going on in

your brain Rachel's gone this ring is

just enjoy the time I have you know and

when my drives up my time son you know

but the problem it's the fried cheese

I'm what it's like I joke or the shriek

and I got a new year later he bites of

things here ALS and I didn't wait the

one year for it to happen I would feel

guilty Robert leaving that for now my

family that they knew there would have

been something they could have done for

me by mr. C was you know get over not

being able to handle going on a trach

what I learned from doing the story is

that the FDA's one-size-fits-all system

doesn't work for neurodegenerative

diseases like ALS we need wider and

better access to clinical trials and

experimental medicine here in the United

States the ice bucket challenge is

proved people want to find a solution

and we want to be part of this process

too every 90 minutes someone with ALS

dies and another is diagnosed the FDA

needs to understand that this is urgent

and it needs to act ALS slowly tortures

its victims and all we're asking for is

the fundamental right to save our own

lives

[Music]

[Applause]

[Music]

[Music]

you

you