Die Trying - The Battle For ALS Treatment (VICE on HBO: Season 4, Episode 16)
Summary
TLDRThe 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
💉 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.
🧬 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.
🌐 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.
🏥 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.
🌿 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.
🚨 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.
Mindmap
Keywords
💡ALS
💡Ice Bucket Challenge
💡FDA
💡Clinical Trials
💡Neurone
💡Stem Cell Treatment
💡Tracheotomy
💡Neurologist
💡Paralysis
💡Cognitive Function
💡Expanded Access
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
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