among hospitals across the U.S. as part of a new research
consortium is giving hope for the future of babies born with a
rare congenital heart defect called hypoplastic left heart
syndrome. A baby from Alabama named Ryals is one of the first
to benefit from the consortium.
amazing," Andrea Sexton says, beaming about her infant
son. "He is the strongest little boy … If you were just
looking at him, you’d never know anything was wrong."
and her husband, Heath Sexton, never imagined that would even
be possible for their son Ryals after he was diagnosed with a
congenital heart defect while still in the womb.
of a sudden, to see the ultrasound tech blinking and staring
at the monitor, and then getting a little frantic and rushing
out to get the doctor, I knew something was wrong," Heath
diagnosis: hypoplastic left heart syndrome, or HLHS.
Essentially, there was no left side of baby Ryals’ heart.
relatively rare condition. Only about a thousand children are
born with HLHS each year in the U.S.
home from the hospital we were wondering how is this going to
change our lives … Obviously, the care of a child that
possibly would need a lot of help, maybe a lot of long-term
help," Heath Sexton says. "And then, of course, in
the back of your mind, obviously, you’ve read that a lot of
children don’t make it past three months. And, so, just
thinking about, you know, am I going to be prepared … for a
Sextons packed up and temporarily left their home in Alabama
to move to Philadelphia. The plan was to place Ryals’ hopes
of survival in the hands of a team of doctors at Children’s
Hospital of Philadelphia (CHOP), one of the leading medical
centers for HLHS surgeries and treatment.
they met Dr. Joe Rossano, who heads up the cardiac center at
they’re a very complicated surgery," Dr. Rossano says.
"You know, prior to the 1980s and ‘90s, there were
essentially no good surgical options for these patients. But a
number of very innovative surgical techniques were developed
that have allowed many of these children to survive and
born with HLHS go through a series of three surgeries to
essentially rework the plumbing of the heart. The first
surgery comes within days of birth. The second surgery
generally occurs three to six months later. And the third
surgery is usually performed about three years later.
series of surgeries allows children with HLHS to live
relatively normal lives, but it isn’t perfect.
the right side of the heart perform the tasks normally handled
by both sides of the heart puts tremendous stress and pressure
on the right side.
patients with HLHS are aging, doctors are realizing many of
their hearts are unable to continue to function on their own,
and some patients need a heart transplant.
Andrea Sexton found out about a clinical trial going on at
Mayo Clinic aiming to solve that problem.
they set me up with an interview over the phone with Dr.
Nelson," Andrea Sexton says. "He was amazing. He
gave me hope."
Nelson oversees the Todd and Karen Wanek Family Program for
Hypoplastic Left Heart Syndrome. He leads a team of roughly 60
people who research and conduct clinical trials for new HLHS
HLHS program has a mission to … recreate the right ventricle
to make it bigger and stronger," Dr. Nelson says.
"So we’re finding ways of inventing new therapies to
make that right heart stronger by stimulating the growth of
the heart muscle and make the five-horsepower engine, a
10-horsepower engine, a 50-horsepower engine. And if we make
it strong enough, we believe that that has a shot at delaying
and preventing transplant for a significant number of these
to this groundbreaking regenerative therapy clinical trial is
stem cells, but not just any stem cells. Dr. Nelson’s team
uses the babies’ own stem cells derived from umbilical cord
the first product that we’re testing in our clinical trial
is using umbilical cord blood from the baby’s own
body," Dr. Nelson says. "So the child has to be
diagnosed in utero, and we have to be able to collect the cord
blood at birth. We can collect the cord blood from anywhere in
the country and ship it into Rochester, Minnesota, to have it
processed to have a high concentrated product that gets frozen
in low temperatures of liquid nitrogen. That product is frozen
for three months until the child has their second surgery, or
the Glenn operation, which then we can bring those cells back
into the operating room, thaw them, and deliver them directly
into the heart muscle."
hope of this is that it causes a fertilizer type of an effect
where these cells are able to fertilize the right ventricle
muscle and allow it to grow bigger and stronger because it’s
received this cell-based therapy," Dr. Nelson says.
4, 2017, Ryals became the fifth clinical trial patient in Mayo
Clinic’s HLHS team’s research.
Nelson and his team, who had traveled to Philadelphia to
deliver the cells, looked on as a surgeon from CHOP injected
the cells into Ryals’ tiny heart near the end of his
operation. A surgical assistant counted down as the surgeon
injected the cells into different areas of the heart.
several tense hours of waiting during Ryals’ surgery, his
parents were emotional and relieved to hear all went well and
that there were no problems injecting Ryals’ cells.
an emotional hug, tears flowed down Andrea Sexton’s face as
she thanked Dr. Nelson.
welcome," Dr. Nelson says. "He’s a trooper."
felt like we had a superhero come in on this jet plane, and he
had this box of super cells," Heath Sexton says with a
big smile stretching across his face. "And it felt like
you’d come to save my son."
hours later, Dr. Nelson and his team from Mayo Clinic were
back on board Mayo Clinic’s fixed-wing plane to head back to
Minnesota and begin preparations for the next cell delivery in
the clinical trial.
effort to expand the research and help more families, in 2017
the HLHS team at Mayo began creating a hypoplastic left heart
syndrome research consortium.
we’ve built this infrastructure to be able to do clinical
trials, and ultimately, we need to be able to have a large
number of families participating in that," Dr. Nelson
says. "So the consortium really represents our ability to
take the science and technology and teams of people to other
centers of excellence that allows us to interact with more
families at more locations."
joined Mayo Clinic as the first members of the consortium, and
Ryals was the first patient to benefit from it.
few months, Children’s Hospital of Los Angelesbecame the
third institution to join the consortium, followed soon after
by Children’s Minnesota.
Nelson sees the collaboration eventually leading to
breakthroughs that once seemed impossible.
can even imagine where the technology is leading us — to be
able to recreate patches and constructs and maybe even whole
ventricles that can be engineered, that can be transplanted at
some point," he says.
after Ryals received his cells were filled with firsts for the
Sextons: a first smile and a first bottle.
were able to bring Ryals back home to Alabama as a growing,
relatively healthy baby boy.
finally have what they feared they never would: a sense of
normalcy and a chance to dream about Ryals’ future.
guess I see the survivors of the day that are having children
and having careers," Heath Sexton says. "And that’s
what I see my son as. And the hope is that he can see his
see college," Andrea Sexton says. "I see happiness
and I see my boys playing together. I see a happy family. And
I believe that this work, this research that these doctors
have put hard work into is going to — to let us achieve
continues to need minor procedures to maintain the health of
his heart. But now, instead of fear and worry filling the
Sextons’ home, a baby’s laughter does.