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April Rubeck, Roman's mother: They said hat Roman had hypoplastic left heart syndrome and the best thing that we could do is take him home and it'd probably be 3 or 4 days and he would pass away on his own. I was 25 weeks pregnant when we went back home just absolutely devastated. We decided to come to Mayo. They gave us hope.
1 in 100 babies are born with a heart defect.
It is the No. 1 birth defect.
Dr. Joseph A. Dearani, M.D., Chair, Cardiovascular Surgery, Mayo Clinic: The focus of this initial trial is on the diagnosis of hypoplastic left heart syndrome and that is a congenital heart defect where half of the heart is not really formed. Now the ventricle that the child is left with is a ventricle that now has a much bigger responsibility than what it was initially designed for, and so over time, the function of this ventricle gradually can get reduced to the point that the child or maybe a young adult will start to develop symptoms of heart failure. Once the function of the ventricle goes down, we don't have anything that we can do for that. Stem cell therapy has the ability to improve the function of any of these ventricles that are failing whether it be a child, whether it be an adult, whether it be a congenital defect, whether it be an adult defect.
Mayo Clinic is conducting the first human trials using stem cells to improve congenital heart defects.
Dr. Tim Nelson, M.D., Ph.D., Director, Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, Mayo Clinic: The study here at Mayo Clinic with hypoplastic left heart children is really the first FDA-approved clinical trial using stem cells to try to regenerate and cure congenital heart disease. So the study is really designed to identify children with this disease before they are born, collect umbilical cord blood at the time of their birth, so these children typically need three surgeries — first starting in the first few days of life, few months of life and a few years of life. These three open-heart surgeries give us an opportunity at the second one to deliver these stem cells into their heart with the intent of trying to make their heart bigger better and stronger. So Roman went through the procedure three months ago and received the stem cells and since that time he has done extremely well. What we believe is happening is that these cells are probably stimulating the underlying regenerative capacity of the heart.
There is 40,000 children born in the United States with congenital heart disease and a good percentage of them develop heart failure so this type of therapy may allow us or help us to delay or ideally prevent the need for cardiac transplantation.
Ryan Rubeck, Roman's father: I hope that Roman, Roman's future has no setbacks and nothing holds him back.
April: My hope for Roman's surgeries is that it'll benefit people in the future.
Dr. Nelson: The reality that we're facing today in biomedical research is funding is being pushed down at the national level, so it really creates this Valley of Death between innovation and applications.
Dr. Dearani: If you look at the major milestones in medicine, I mean, we had antibiotic therapy for the life-threatening infections, then we had the inception of heart surgery using the heart-lung machine, then we had transplantation where we were able to come up with medicines that you know minimized rejection. I mean that was probably the last major miracle in medicine so I mean this is the next potential miracle in medicine where it has the potential to impact the largest number of patients in healthcare in heart failure.
Today, over 5 million American suffer from heart failure.
So we can make a difference, so we should make a difference.
By 2030, heart failure is projected to impact 8 million Americans.
Join us. Make a difference.
Thank you.
American Heart Association/American Stroke Association
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