(upbeat music)
- Dear Dr. Roboz,
we have been on this journey together
for almost eight years now.
I genuinely believe my story would be vastly different
if our paths had not crossed.
- My mom lost her mother when she was my age.
I remember coming home and sitting at the kitchen table
and my mom said, I have this blood disorder.
- I was diagnosed in 2013 with a form of leukemia.
- She had been seen already by several other doctors.
They were perplexed.
You need your bone marrow to live,
and hers was very abnormal.
- [Cheryl] They told me
that I needed a stem cell transplant.
- This is the best chance.
Well, the only chance to get rid of it.
- [Narrator] After the stem cell transplant,
we started seeing signs of recurrence
of the original problem.
- And the only real plan was traditional chemotherapy,
where I would become really, really sick
maybe for the last four months of my life.
- So we asked Precision Medicine,
can you look at these cells?
And can you find a pathway that gives me a shot?
- Precision Medicine allows this deep understanding
of a disease that is very unique.
We scan entire genomes in just a few hours
to tailor the treatment to each patient.
- Through computational bio-sciences
we discovered that there was a novel mutation
in Cheryl's bone marrow
that could be susceptible for a new drug.
- One of them was a drug called PU-H71.
We were collaborating with the team
that had actually developed that drug.
- Gabriela Chiosis and Memorial Sloan-Kettering.
Together we published back in 2016
that there's very unique characteristics on certain tumors.
And these cause extreme dependencies
for survival in the tumors.
If you take it out,
the whole system collapses, just like a Jenga tower.
We developed this test,
and that test told us that she had very high levels.
And because we have a drug that can target this protein,
we suggested to Dr. Roboz,
maybe this will be a great option.
- And I said, well, I wanna try it.
- We put forward a request to the FDA
to get access to this drug.
I said, I've got a really compelling story from the lab
and I've got a little bit of data
and I have absolutely no idea what this is gonna do.
- Dr. Roboz was creating the protocol as we went along.
- [Dr. Gail] We were watching very carefully,
literally running back and forth
between the clinic room and the laboratory.
Four weeks, five weeks,
six weeks we've started seeing blood work
go in the right direction.
- I went into complete remission.
- To say that we were thrilled
with the initial bone marrow biopsy results,
that is an understatement.
- We were screaming in the halls,
hugging each other and laughing and crying.
My children now don't have to be motherless daughters.
It's a miracle.
- Precision medicine is transforming patient care
at a very rapid pace.
- We're seeing that this particular drug,
PU-H71 has other beneficial effects.
It opened a new area of research
on other different cell types
that help your immune system, for example.
- Precision medicine, I think really is changing medicine.
- What we achieved here is a miracle.
What we really are hoping to do moving forward
is to bring miracles to every patients.
- At Weill Cornell there is a true commitment
to the collaboration that's gonna be needed
in some of these very, very difficult to treat cancers.
- We, as a team can make a difference.
- We've been given a lot more time.
I'm grateful for that.
(upbeat music)