By the time I was 6 months post diagnosis,
I was taking 42 pills a day.
I have so many different medications
that we sometimes joke
that Covid is afraid of me.
ALS is characterized as a progressive
neurological disorder,
meaning your nerves break down over time,
resulting in progressive paralysis.
In my family, it typically starts as weakness, usually in the leg.
My voice is going
and I can barely feed myself.
It's like, do you want to go to hell fast?
Or do you want to go to hell slow?
There is no cure.
There is no effective treatment.
It's one of the most terrifying and hardest to treat diseases
that are out there.
This disease is different.
We have to think about it differently
and we have to use our flexibility
to think about how we approve drugs
for a disease that is 100% fatal.
I am ready to accept the ALS Ice Bucket Challenge.
Where's the water? Where's the ice?
Alright.
Whoa.
The Ice Bucket Challenge in 2014
drew a lot of attention
to this horrible disease
and brought millions of new funding
into disease research.
Because patients die so quickly from the disease,
ALS historically hasn't generated the same level of public attention
as conditions like breast cancer,
which has a large constituency of survivors.
ALS stands for amyotrophic lateral sclerosis,
a fatal and progressive neurodegenerative disease
in which the motor neurons
that control the voluntary muscles
slowly and progressively die.
And the types of muscles that are affected
in ALS, they include everything down,
not just the muscles in our arms and legs,
but the muscles responsible for breathing
and the muscles responsible for eating and swallowing,
which means patients,
they literally are alive and fully conscious
but can't move at all.
The causes of most forms of ALS are not known.
However, there are about 10% of the cases that are familial.
And in recent years,
scientists have started to discover
some of the genes responsible for some of these familial cases.
Another 90% of cases are so-called sporadic cases,
which means that researchers really have no idea
about what causes those cases.
I think this document’s really priceless.
It shows just how many generations
and how many families have been impacted.
My family carries a gene called SOD1
and there’s only about 300 people
in this country that carry that particular gene.
My grandfather, Harold John Lindsey, died of ALS.
His brother, Wesley Lindsey, also died.
And he was quite young.
He was only 30.
We believe my mom is the 33rd person to have gotten
ALS in our family.
After my uncle was diagnosed,
he asked his siblings to please get tested.
My mother's 2 other siblings tested negative
and then my mother tested positive.
Her first symptom was that she couldn't lift her leg up
off the floor to get into bed.
Within a few months, it became apparent
that she wasn't walking very well.
She called me and she said, honey, I need to see someone.
I was at work.
And I walked out to the parking lot
and I screamed.
Because I knew what was coming.
The first thing I did was try and get her into the same trial
that my uncle had been in
because I knew that drug would work for her.
A drug called tofersen
through a company called Biogen.
It was for our specific mutation
and it was the best option out there.
The Biogen drug tofersen is a
very interesting
new approach to ALS.
It is potentially
the first drug
that would target a known genetic cause of ALS.
Biogen is trying to get tofersen approved
based on the fact that it
lowers blood levels of a protein
called neurofilament.
A neurofilament is something
that is produced
when brain or nerve cells die.
And Biogen is saying
that tofersen lowers levels
of neurofilament and that is
an indicator that it's likely to slow progression of ALS
and is doing something good in terms of protecting
the motor neurons in the spinal cord.
However, in the phase 3 trial of Biogen’s drug,
tofersen failed to meet its primary goal
of slowing decline.
But they are seeing promising effects and
this drug could set a precedent for other types of drugs
against specific genetic causes of ALS in the future.
And if we unlock cures for ALS, we will also unlock cures
for Parkinson's, Alzheimer's and beyond.
I was diagnosed with ALS
about 5 years ago when I was 37,
and as you can see,
Sandra is my voice.
The majority of ALS patients
have no access to promising treatments.
I have not been able to qualify for a clinical trial
for over 2 years,
and so what that means,
I'm left to cobble together medicine.
I'm left to wander on my own.
And that does not feel right.
What we are asking for
is not for the FDA to lower the bar for efficacy
but instead to add to their calculation 2 things.
Number 1, the serious nature of ALS.
And number 2, the lack of meaningful treatments.
What I need you to do
is to approve treatments
that are safe and may extend my life.
Give me the ability to choose
whether I try that treatment
or I leave my fate to ALS.
There are only 3 main drugs
approved for ALS and they all have very modest effects.
The first drug called Rilutek or Riluzole
was approved in 1995 in the US.
The second drug was approved in the US in 2017
from a Japanese drug company.
It's called Radicava.
And then just this year,
the third main ALS drug was approved.
It’s from Amylyx Pharmaceuticals.
At least 40 drugs are now in trials,
according to the ALS Association.
Drug development is a long and arduous process.
It can take decades between when researchers
first have an idea for a drug to when it gets on the market.
Historically, the US Food and Drug Administration has demanded that
demanded that drugs be proven both safe and effective
before they're approved for use.
Effective in the past has meant that a drug basically
directly impact the symptoms
and/or effect a disease has on patients’ lives.
But in recent years, the FDA has often been approving drugs
based on lab tests that indicate
a drug is likely to improve symptoms or extend life
or otherwise impact the clinical course of the disease.
The accelerated approvals have been most common for cancer drugs people
cancer drugs.
People advocating for reform of accelerated approval
worry that if you just approve many, many drugs
and we don't prove that they're effective,
you could have many drugs that are on the market
that aren't really effective.
And all you're left with is risk and cost.
Something that you'll hear frequently in the ALS community
is ALS is 100% fatal.
So we have a very high tolerance for risk.
If there is a chance it could help us, we are willing to take it.
July of 2021, my mom started tofersen expanded access.
She immediately noticed a halt in her symptoms.
Things were really good until about Christmas.
She didn't have hardly any progression at all.
And then by January she started to progress more.
In April, she developed blood clots in both lungs, so she
actually had that next tofersen injection delayed by a month.
In May, my mother moved in with my sister
under hospice’s care
and she decided to stop all treatments.
She was tired.
It was just really hard on her.
August, things got very progressive
and she passed away on August 30th.
I think as soon as someone
in your family or someone you love
gets an ALS diagnosis
and you see the fight that we’re up against,
you see the limited options, the lack of hope.
It kind of turns everyone into a warrior.
And we all just want to fight.
Fight for our loved ones, fight for ourselves.
Together, you and I, we will end this legacy of death.
We will continue on with a legacy of hope.
I came up with the idea for I AM ALS in 2018
to put together a nonprofit
that was able to help ALS patients and their families
navigate the disease.
And we show them the power of their voice
and help them tell their story.
For the last 3 years, we have been able to increase
federal funding for ALS research, and we have been able
to pass 2 laws
and we have been able to work with BioPharma
and the FDA and NIH
to rethink how we approach
the development of treatments for ALS patients.
When we launched I AM ALS, I told Sandra that my goal
was to increase money for ALS research
by $100 million in our first 3 years.
And I am happy to say
that we have increased it by over $600 million.
When my mom was first diagnosed, she said, honey,
I don't want ALS to be your life.
But ALS is my life.
I had a 50/50 shot of carrying the gene.
I realized that I needed to be tested as well.
I knew as soon as I saw the geneticist’s face
that my results were positive.
And when she said it out loud, I collapsed.
I just collapsed in thinking,
what about my children?
Each of my children have a 50/50 shot of carrying this gene.
But as a parent, as a mom, to think that you may have
passed something like this onto your children is heartbreaking.
But when I was able to pick myself up
and look at that screen again,
the first words out of the neurologist’s mouth were,
your children will not have to worry about this.
I am filled with hope
that my children will live in a world without ALS.
Numerous drug companies are pursuing ALS drugs
and certainly the standards the FDA develops
for approving ALS drugs could influence
numerous drug applications
that come forward for ALS in the future.
So I may be an optimist,
but I think we are only a few years away
where a doctor can say to a patient,
you have ALS, but the good news is
I have treatments that we can try for you.
Well it’s like that starfish story.
The guy was walking on the beach and
he could only throw a couple in,
but they appreciated it
because they were saved.
Thank you again.
Thank you so much.
Bye.