Transcript for Family on a Mission to Find a Cure for Daughter's Rare Genetic Disease
We'll begin with robin at the Vatican there for an historic conference on stem cell therapy that could provide hope for millions battling rare diseases and, robin, you're about to introduce us to a very inspiring little girl. I really am. The first day of the conference and the theme is hope for our children. A research center in sioux Falls, South Dakota, has given hope to many children with its work in adult stem cell therapy and you are about to meet one of them. âª It says, Olivia -- It does. How old are you going to be. 4. Good girl. She was precocious. She was an adventurist, she's amazing. âª Happy birthday âª Reporter: On her fourth birthday Olivia was a typical preschooler. Hurry up before the alarm goes off. All right. She wanted to be involved with everything in sports. Very active. Good job, look at me. Reporter: Soon her parents noticed their lively little gir was changing. When did you realize that something wasn't right? Really it was when she was 7 that we had the rapid decline in the vision and we said, this doesn't seem right at all. Reporter: For two years Olivia and her family met with doctors all over the country searching for answers. Finally at age 7 she was diagnosed with juvenile batten disease. It's a rare genetic illness that commonly affects children destroying brain cells and causing neurological disorders including blindness, frequent seizure, dementia and ultimately premature death. How did this time impact the family? Everything is different. Nothing is anywhere near what it used to be. It affects everything. Think about your child if you have a Normal vivacious outgoing 7-year-old daughter who suddenly goes blind and then imagine that's just the beginning. Reporter: Batten disease is just 1 of 7,000 rare illnesses affecting 30 million Americans. More than half of the people impacted are children. It's a game. Reporter: It's a game, yes. Do you like to play games? Uh-huh. Now, 12 years old Olivia's illness doesn't stop her from being the joke ter in the family. Keeping her big sister Sydney smiling. What is it that you're hoping with this new technology that maybe they'll find something to help your sister. Yeah, I'm really hoping we can find a cure soon so we can save her. Reporter: Sioux Falls, south Dakota, is home to one of the country's leading medical systems, Sanford health, the castle themed children's hospital draws people from all over the country. And children like Olivia battling a rare disease have found hope in the cutting-edge work that happens here at Sanford research center. Shall we? Exactly. This way. Reporter: I caught up with Dr. David pierce to get a glimpse of the work that could potentially save lives through adult stem cell therapy. This is extremely impressive. Tell us a little about this facility. What we're working on here is cures for disease, for children. You met a child with batten disease and cells from her brain are dying. We can try to compensate for that with drugs and with gene therapy but ultimately we want to be able to replace those cells and restore her to full function. Reporter: Less than 1% of rare genetic diseases have fda approved treatments, it's a race against time to implement the ultimate cure, adult stem cell therapy which is still in the research phase. Dr. Pierce, how close are you to fda approval? For gene therapy we have fda approval now. The stem cell, it's going to be a slightly slower process because it just hasn't been done before. You kn one year but more realistically three years, quite frankly. When I see the family, they know what you're doing here and it gives them hope. How excited are you right now with this facility, with how close you are to making breaks. The facts now that we actually have clinical trials in progress, treatments in progress, I personally can't think of any more job satisfaction than that. It's not a job, it's a passion, it's a life. It is. I will never forget spending time with Olivia and her family and also Dr. Pierce and all the researchers there and this woman was with me in Sioux Falls. Dr. Robin smith, president and founder of the stem for life foundation, the third year that tem for life and the Vatican have joined forces for this conference and every time I say that, people always feel like, what, what's going on here. Tell us about the conference. Well, it's really exciting. We have politicians and scientists and patient, from all around the world, regulatory bodies really focusing on how we get the cell therapies developed and to patients who are in need. A global initiative as well. It's a global initiative around a very simple concept that the cures for tomorrow are going to come from cells within our bodies today. We need to talk more about that and that's what the purpose of this conference is for because many people who have gone through illnesses, my colleague Amy and myself, we went through it, the number goes on and on and on, but we had to use drugs, that was a first line of defense but you and other, researchers, scientists are saying, hmm, there may be another route to go. Explain that. It's a way to get your own body's immune system to be harnessed towards whatever the disease you're trying to fix so maybe it's to make a vaccine for cancer. Getting your own immune system to kill those cancer cells or top an overactive immune system from killing good tissue so we're learning each day. Immunotherapy and talked about that today and will continue that, so vice president Biden tomorrow. Yes. He's going to talk about his moon shot initiative. Yes and a little about his personal story and what his -- what made him focus on this. An audience with the pope. How did that all -- how did that joint venture come together, robin. I think we all want the same thing, how to help people suffering, sick, the margin aislized. It's the year of mercy and everybody is excited about this global movement to see what we can do to help people. It is a global movement and for people who are wondering at home we're talking about adult stem cell therapy, not Embree yachtic cell. 5,000 clinical trials focused on taking adult stem cells and using them to repair damaged tissue. Everyone I've been talking to here, my doctor, Sergio giralt, my sister, researchers, Dr. Pierce who you just met in that piece will be receiving an award later, the Sanford has just done wonderful things there in sioux Falls and when you talk about it, people are really feeling that we are on the brink of really major breakthrough, major differences. Absolutely. Effective therapies and maybe even cures. It's not that far away. You did say the "C" word, cures. I did, I did. We're all really focused on that and I think that's the difference of using the drugs you talked about and it's very different to restore and to use your own body's immune system, it's much easier to tolerate. Thank you so much. Thank you for having me. For the tireless efforts of your work and this conference. Oh, absolutely. They're having all these
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