Thanks very much Nirali so I'm just going to do quick introductions for everyone in
the psychosocial group. Our first speaker is Carlos Sandy. He's a volunteer advocate
representing the Pediatric Oncology Branch, the National Cancer Institute as part of the
St. Baldrick's Stand Up to Cancer Dream Team initiative. And in his second day job, he
advances the world of healthcare technology. And his last but most important hat is dad
hat. And with that, I will hand it off to Carlos.
myself off of mute here Hi, thank you. I'm again Carlos, Sandy parent of one of the early
car T cell patients at the NCI and as you can see from the photo have done a lot of
work with St. Baldrick's. So that's a photo from 2005. The year that is basically spent
as a poster child for St. Paul. And it does, you know, show a very happy, healthy, ridiculously
attractive family, which we totally are, but it's not the complete picture. Next slide.
What's missing from that picture is is this kid this is my daughter, Althea, who was AML
patient from 2006, early part of the year to later part of the year she was diagnosed
with them 716 months made it about two years through about nine months through brutal treatment
and a failed quarter plan. She's the one who really taught us how much quality of life
and you know, other things you're willing to sacrifice to try to keep the kid alive.
We asked her to draw a lot in her short time and treatment, but she did die anyway, just
two months after her second birthday. And that was a pretty rough time to live. But
if she had we would have never met the next guy I'm going to introduce I can say that
pretty definitively because it required a vasectomy reversal to even have the opportunity
to meet him. Next slide. And this is what he looks like to the scientific community.
He was patient number 10. On that first car T trial, the pediatric trial at NCI see on
the chart, he was five years old at the time suffering from primary refractory pre B A
LL, during the course of treatment suffered a grade three cytokine release event. Shortly
after infusion, he ended the trial MRD negative and went on to have a consolidated stem cell
transplant and 54 days after infusion next place. And this is patient 10 before he even
became a patient, so Phineas was four and a half when he developed leukemia. About six
years after his sister died, it was a huge blow for us. But also when we heard that it
was a ll instead of AML. We really thought we were in the clear, I don't think is like
colleges to ever seen a family celebrate an ELL diagnosis before but we really were relieved
at first next slide. But obviously, you know I'm talking to you guys today is that things
didn't go as well as we helped Phineas started off in high risk classification for treatment
because of a super high white count at diagnosis. And throughout the first month. I mean, it
was predictably rough for for a kid that age and the steroids were no fun. He was dogged
by persistent parvo virus infections, but things went from rough to really bad after
induction when he stood about 2% MRD is marrow. So he got put on the very high risk protocol,
which at the time had an arm to that was the CLO FIRA being arm which fortunately got closed
down before he ever got that stuff. But you know, it looked like we were getting into
the kind of chemotherapy that we hope to avoid. And that was not good. So during month two,
during his second PEGASE paradise infusion Phineas went into full on anaphylactic shock.
And that was a perfect example of how three people in the same family equipment can really
perceive one event completely differently. My wife and I both came with him to clinic
that morning, but she was out getting lunch when the whole reaction started. I didn't
even bother to call her because I didn't think it'd be safer to be driving when she knew
that our son was coding. So she completely missed out on all the action. But I remember
it pretty vividly the rapid response team leaping into action you know the look on his
doctor's face. As he was watching this patient go down the the tone of his voice just tried
to talk him back and the shock going for that moment of routine infusion and clinic and
then you know once again watching my child literally dying in front of me. When things
started turning around there in the recovery room, I managed to look up from all the action
items the security guard standing outside the procedure room and realize that that guy
was there to deal with me. If I reacted to my son's death. And a few minutes after that
intense situation and realization my wife shows up with lunch and Styrofoam containers
not having any idea what was going on. I'm soaked with adrenaline. Phineas is asleep
and all he remembered from the event It was feeling itchy one minute and then waking up
in a different room. totally happy. Next slide, please.
The rest of frontline therapy was predictably awful MRD after each month cycle was still
on to 3%. I started looking for some way to get this kid into remission, we knew he needed
a transplant. But in the meantime, we really tried our best to just give him as much of
a childhood as we could with whatever time we had with him. So we did things like setting
up an above ground pool on the driveway. can't quite see it there. But the blue patch is
a ton of gravel I had dumped to level it out. You know, and in this picture, he's having
a great time. And he's have a lot of fun, but my wife and I are completely freaking
out because we knew he needed a transplant. We knew his disease was progressing. We knew
we needed to MRD negative. And really our best hope was this car T trial at NCI we'd
already reached out and provisionally accepted of the trial got the accepted because cog
wanted to try another dose of methotrexate, another round of methotrexate. But, again,
this slide, we knew we had a slot so we were getting ready to go to Bethesda the next month.
Next slide please. And once we got to Bethesda there, of course, more delays. I think he
was just a bad metabolizer of a lot of chemotherapy. He had really high liver enzyme issues even
just on like a diluted maintenance chemo protocol. That cute little puppy backpack you see him
wearing something that we actually bought at Target in Bethesda to hold a battery powered
infusion pump it a bag of saline, he was running around with for a week trying to flush out
all the stuff and his blood, which did work. And two weeks after we got to Bethesda, we
got really accepted for a third time into the trial and get started. Slide please posted
the caringbridge site. So when finished got his T cells. We were told to expect things
to get a little crazy in about a week. So I took my daughter back down to North Carolina
thinking we had time she started school, but three days later he started spiking, very
high fever, losing his ability to speak and within a couple hours of it all starting he
was verbally non responsive and in ICU. At this time, I was the one away from the reaction
when it started but I did manage to drop my daughter off with some friends and hightail
it back to Bethesda and time to spend night two in ICU with Phineas. He was still completely
unresponsive when I got there. But at about three in the morning. Somebody was checking
his lines and he uttered the first words since he wanted to CRS which were tell that stupid
nurse to leave me alone. He'd be Okay, next slide. This was him three days after the CRS
event, and we were a little concerned because, you know, when they did an MRI of his brain
shortly after the peak of the cytokine storm, it was lit up completely in white. And just
to kind of riff back on the last conversation he did have some methotrexate toxicity prior
to going into treatment. The radiologist actually said he was surprised the kid was actually
able to walk looking at that brain scan and he couldn't tell if it was intrathecal or
CRS or something else going on. But we really weren't too worried about it because he wasn't
proving and even the the monkey foot thing wasn't too worried because next slide. We've
seen that behavior before. So not the first time we saw the kid confusing hands and feet,
probably not related to cytokine response. Next slide. Within a week or two, he was feeling
well enough that we snuck out of the NCI for a day trip up to the Catoctin mountains to
go hiking. So issues were resolving quickly. Next slide please. And by October, he was
able to have the transplant we were desperately hoping for. It was a completely PTSD soaked
process for my wife and I had been through that before with our daughter. But clinically
he did phenomenally well. I mean he wouldn't strong, healthier that he'd been since he
got diagnosed and really sailed through with minimal GVHD and almost no adverse events
whatsoever during transplant. So we Credit Car T with putting him in position for that
kind of success. Next slide, please. brief aside so during that last slide, we were on
a trip to the beach he discovered frozen waffles and so when making wish asked him what is
one wish was if he could have anything in the whole world he asked for a four slice
toaster. Fortunately, they talked him out of that he took the Orlando trip instead.
Next slide. And this is Phineas seven years later, with his original syringes taken about
three days ago. Really this this all worked out better than we could have ever hoped for.
For us. The uncertainty was probably the hardest part of it all not knowing if would actually
work or not, but relative to what we experienced with my daughter. and other things you experienced
during his treatment
party I want to say it was a breeze but it was certainly not the worst part of the experience
and didn't last very long. So we're extremely grateful for that.
And I'm grateful for the 10 minutes I've got here today. I see it's running out but thank
you for the time. Thank you for everything you guys are doing and keep on sciencing.
US Department of Health and Human Services, National Institutes of Health National Cancer
Institute cancer.gov One 800 four cancer.