One Great Idea Tour: SO CAL PBS

The “One Great Idea Tour” wraps in Southern California with a look at breakthrough medical treatments for patients suffering with Alzheimer’s and cancer.

Dr. Clive Svendsen heads up the Svendsen Laboratory at Cedars-Sinai. He currently works closely with neurosurgeons, neurologists and other scientists to develop novel ways of using stem cells modified to treat patients with neurological diseases such as ALS, Huntington’s, Alzheimer’s and Parkinson’s.  Dr. Howard Federoff joined UC Irvine as Vice Chancellor for Health Affairs and Dean of the School of Medicine in 2015. Dr. Federoff is a nationally renowned clinical and research leader formerly at Georgetown University and a groundbreaking investigator for neurological disorders. Adrienne Shapiro is an advocate for sickle cell patients and their families.


Tavis Smiley: Good evening from Southern California in the studios here at PBS SoCal. I’m Tavis Smiley.

Tonight, we’re wrapping up our look at what cities are doing to meet some of the challenges this country faces, what we’ve called “One Great Idea Tour”, with a conversation about medical advances in what’s known as regenerative medicine courtesy of a statewide taxpayer initiative that poured billions into scientific research, much of it centered here in Southern California.

We’re glad you’ve joined us. A conversation about scientific breakthroughs coming up right now.

[Walmart Sponsor Ad]

Tavis: “One Great Idea Tour” is made possible by Enterprise. Pick Enterprise, we’ll pick you up.

Announcer: The California Endowment. Health happens in neighborhoods. Learn more.

Announcer: And by contributions to your PBS station from viewers like you. Thank you.

Tavis: When the voters here in California passed Proposition 71, billions of dollars were poured into stem cell research bringing some of the world’s best scientists to Southern California, all working together to find therapies for diseases like Alzheimer’s, ALS, cancer, and sickle cell anemia, to name a few.

Joining me now are Adrienne Shapiro, a fourth generation carrier of sickle cell disease and the cofounder of Axis Advocacy that works on behalf of those with the disease, Dr. Clive Svendsen with the Cedar-Sinai Regenerative Medicine Institute, and Dr. Howard Federoff, Vice Chancellor of Health Services at the University of California Irvine. I am honored to have you all on this program. Thanks for your time.

Dr. Svendsen, let me start with you because this was unprecedented what happened here in California that the voters who cannot agree on much of anything all decided that this was something worth putting $3 billion dollars behind. Before we jump into what’s happened over the last 10 years, what do you make of that decision by the voters to actually advance this kind of idea?

Dr. Clive Svendsen: It was an amazing decision. It made a huge change to the State of California. I think they call it the stem cell state now because of the $3 billion dollars that went into this. And it was triggered a little bit by the ethics of embryonic stem cell work that was around 10 years ago and it allowed the freedom for the scientists to actually work on these interesting stem cells in the State of California.

Tavis: That was the debate 10 years ago whether or not stem cell research was an ethical thing to be doing. Take me back to what that debate was 10 years ago and tell me where you think that debate is today.

Svendsen: Yeah. 10 years ago, it was all about where life starts. And when IVF clinics were popping up creating embryos by putting sperm and egg together, you create a living organism. And the debate was, was it ethical to take cells from a 10-day-old living embryo and use them in research? That created enormous amounts of discussion.

Now if you wind forward 10 years, the science has actually overtaken the ethics. We discovered ways to take adult cells, so I could take your cells from your skin or from your blood, from any age person, give them one protein in the petri dish–you take the cells and put them in the petri dish–and that protein sends that cell back in time to an embryonic state.

So we can now create embryonic stem cells in the dish without using embryos, and that’s changed the whole face of that field and reduced a lot of the ethical concerns. Because now it’s adult stem cells that–we call it reprogramming and send them back in time to a very powerful embryonic state.

Tavis: The other debate, Dr. Federoff, as you know, that was looming 10 years ago in the minds of some still has not been resolved as evidence by the wild success of the book, “The Immortal Life of Henrietta Lacks” is the question of who actually owns these stem cells. Where are we at on that debate?

Dr. Howard Federoff: Yeah. I think clinical investigation has now made clear to us that we have to inform our patients when we’re soliciting informed consent. And in the current environment, being clear about what we’re asking patients to do when they’re going to provide a cell or any tissue, for that matter, is requisite to being able to do high-quality clinical investigation.

I think the instance with regard to the Lacks issue was that we were at a more, if  you will, embryonic time in our development of clinical bioethics. I think that, as Dr. Svendsen has said, has really progressed in parallel and we’re much more sophisticated, always including clinical bioethics as we think about clinical protocol development.

Tavis: The other issue is, when you take that $3 billion dollars we referenced earlier that voters approve and you add the interest and the other contributions, donations, that were made, you’re talking about $6 billion dollars basically that we have poured into this research over the last 10 years. We’ll get in a moment to what we think we’ve accomplished over the 10 years.

But there’s another fundamental question about who owns all this research. When there is the kind of breakthrough, as there will be, that makes the kind of money that people get crazy about, how does that work? Who’s going to own this research?

Federoff: So I think this is a really important area and I think it’s one that continues to be important to patients and their families. Successful research, however it is to be funded, needs to be ultimately able to provide a therapy to a patient and/or their family. And in that context, the only way that really translates into larger scale clinical use is to have a vehicle for commercialization.

So there has to be a path between the discovery, the clinical trial demonstration of a clinical benefit, and ultimately then the approval as it would be from the FDA so that a prescription can be written for whatever the therapy is. The only way to do that historically and probably going forward is that you need a commercial development partner to take that forward.

Tavis: Which means the money goes where?

Federoff: Well, the money is going to flow to several inventors at universities, such as the University of California. If they make an invention that ultimately leads to a royalty consequence, to some licensing of a therapy, they will benefit.

University of California itself will benefit, and I think ultimately the big beneficiaries of this are the patients and their families because you’ve had commercial development now leading to an available, hopefully scalable, technology that could be prescribed anywhere.

Tavis: But that’s the scary part for me, Dr. Svendsen, because Dr. Federoff is right in that eventually we, the people, the demos, benefits from this research if it can save lives.

But if this happens the way everything else in medicine happens, you’ll end up having a breakthrough and the industry will charge so much money for the access to the technology that most fellow citizens can’t afford it anyway after investing $6 billion dollars to make it happen.

Svendsen: Well, I think CIRM–California Institute for Regenerative Medicine–CIRM, we call it CIRM, have pretty good procedures in place. So if they give you money for your funding and you develop a product that goes into patients, the state has a part of that, so the state will benefit from that product. And that’s the unique part of CIRM is it’s a partnership really.

It’s a partnership between the state. It’s a partnership with donors and givers to CIRM. It’s a partnership of universities and it’s a partnership with industry. And a lot of the programs that they fund are actually co-funded by, you know, big pharma, sometimes of industry, and the university, and CIRM.

So this is a partnership agreement. That’s what’s unique about CIRM, actually. They really foster the industry because, as Howard said, it’s never going to get to widespread patient use unless a company comes in to take it to the big time.

Tavis: So before I get to Adrienne and hear her personal story of the journey that she’s on, her family’s been on for four generations, let me give us a chance here in Southern California to beat our chest a bit and brag about what we have done over the last 10 years. Dr. Federoff, you first. Tell me the good news of what we’ve discovered over the last 10 years.

Federoff: Well, I think there are many great stories and I’ll just cite one of them that actually came out locally from the University of California Irvine. A physician scientist was interested in a rare class of disease that cause blindness, retinitis pigmentosa.

So the work of his team was to identify a stem-like cell, the retinal progenitor cell, and show that they could take that cell, transplant it into the eyes of, first, animals with the model of the disease.

And now they’re conducting a clinical trial of testing whether they actually will show safety and eventually, if safety is ascertained, then it will hope to demonstrate benefit. That’s a big, big deal. If someone is blind and this is not an uncommon family of disease, one might actually have a hope for blindness.

Tavis: Dr. Svendsen?

Svendsen: Yeah. I think the eye has actually been leading the way. In Cedar-Sinai and some of the other institutions, we have programs where we’re injecting stem cells behind the eye. They migrate and they protect the cells from dying in diseases like retinitis pigmentosa and also macular degeneration.

Federoff: Absolutely.

Svendsen: The other big area that I’ve been very interested in is Lou Gehrig’s Disease, or ALS. And through some funding and really a lot of collaboration between Cedar-Sinai and the neurologists and neurosurgeons there, we’re on the point of doing a clinical trial for ALS where we’re putting stem cells into the spinal cord of patients that have the disease.

And the interesting thing about that is we’ve actually engineered the cells to make a drug. So not only do you put a stem cell in, but now it’s a stem cell that makes the drug that you can’t normally get into the spinal cord of the patient. So we’re using the stem cells as Trojan horses to deliver drugs.

There’s lots of examples we could go through from around the state like that and CIRM has really pushed the barrier between–there’s this thing called the valley of death where you have an idea in science. You want to take it to the patient.

There’s a valley of death in the middle which is very hard and complex regulatory. CIRM has kind of filled that valley and built a bridge across, so we’re actually going from the research to the patients, thanks to the CIRM funding.

Tavis: One of the persons who championed this years ago to get it passed in California didn’t make it across that bridge. The great actor, Christopher Reeve, lost his courageous battle eventually. Nancy Reagan fought courageously to get this passed on behalf of her husband, late President Ronald Reagan. She, of course, just recently passed.

I raise that–and Michael J. Fox, of course, is, thank God, still with us and he was a big champion of this as well–but these are celebrity persons. What kind of buy-in are we getting 10 years later from everyday people, Dr. Federoff, given the advances that are being made?

Federoff: Yeah. My general impression is that patients really who have high-on medical need and are not responsive to conventional therapy see this as a great hope. And the more that we can refine the understanding of the biology and with all due rigor, but expeditiously move into clinical trials, they are really enthusiastic.

They want to volunteer to be research subject participants and they also want to be able to catalyze interest among others who suffer with their same disease.

Tavis: So speaking of everyday people, enter Adrienne in this conversation. Tell me–I mentioned at the top, of course, that you are a fourth generation sickle cell patient. Tell me about this disease which, as we all know, impacts African American families more than any other group, but tell me about this journey that your family’s had to navigate with this disease.

Adrienne Shapiro: Well, my elder brother had sickle cell and, at three, he was given a blood transfusion. At that point, this was over 50 years ago, so the science wasn’t very good and he ultimately had a stroke from that transfusion. My mom said, “We’re not putting him away” which, you know, that was the practice in those days.

So all my life, we had Teddy and he was this beautiful person, but he suffered. And my mom said, “He’s going to stay home with us and we’re going to take care of him.” So she had trained all of us to look after him. Did he have water? Was he in pain? You know, make sure he had a sweater on.

And she involved all of us in the looking after of him because he was the first generation where they actually had the science, where they had the talk. The previous ones, they didn’t. They just categorized all the symptoms and what happened.

So when it became available, she had all of us kids tested and said, “We’re going to be the last. The Jews stopped it. We can stop it.” So we were all tested and my other elder brother had the trait and I was told that I didn’t have the trait and my younger brother didn’t have the trait.

So you fast-forward years later. I marry a Cuban and I have a child and she’s diagnosed. And we’re like, “No, that can’t be possible.” Everybody said, no, it couldn’t be possible. And that’s where I learned that, you know, like I say, cheap science is bad science, and it was possible.

So my family said, “You will take care of this child. This child will live. We will prove to them that this child will live.” And I started the journey at first of just trying to keep her alive because everywhere you went, they would say, “The child’s not going to live”. When she was really tiny, so young, she was very cute. She had these big beautiful eyes.

We didn’t really have many barriers unless she went to a place that didn’t understand the disease. And I found myself constantly trying to teach the medical staff basically on how to take care of her. And we had one point where she was at one hospital and the medical staff knew what to do and they were doing a great job, but the social workers in the hospital didn’t know and didn’t understand.

So I got into this situation where they were telling me I couldn’t take her home because the social workers had decided that, at seven, she was too dependent on the morphine which she needed in order to relieve her pain.

It was the strangest kind of thing because here I was in the situation where the people were supposed to help us and understand. I think I was kind of oblivious to a lot of what went on in the medical community about our disease until she became a young adult.

Tavis: How old is she now?

Shapiro: She’s 38 now.

Tavis: She’s 38?

Shapiro. She’s 38 now.

Tavis: So she survived, obviously.

Shapiro: She survived.

Tavis: How is she doing at 38?

Shapiro: Well, she’s had a lot of problems. Again, going to a hospital where there wasn’t good training, she had a bad transfusion. Her body’s ultra sensitive. She catches every infection in the world, that she can get any opportunistic one. So for me, stem cells were the only answer because it had to be very personalized medicine.

Tavis: So your family thought they had beaten this back a generation ago.

Shapiro: Yes. We were going to beat it. Well, my mom was going to be the last.

Tavis: Your mama’s going to be the last. Her granddaughter ends up contracting it. How hopeful are you then at this point about the research?

Shapiro: I am such a believer. I supported this work over 20 years ago early on. Because of our genetic makeup, we are how I used to say as a kid, not as a young adult, we were genetically engineered. There was a reason we jump higher, run faster, right? And I didn’t realize that that really was true.

Because of our heritage, it’s very hard to find a match for us genetically. So stem cells, using our own tissue, is really the only way we’re going to be able to heal ourselves or be healed in ways that other people are.

When I talk to these scientists and now have gone many places and talked to scientists about it, I have to say to them and I have to thank them for the work they’re doing because, for my child, and I have a child with the trait who could conceivably have a child with the disease, it is the hope and it is my belief that we’re the last.

Tavis: Let me ask you a question that is impolitic, but I want you to give me an honest answer, not that you would lie to me, but…

Shapiro: No, never.

Tavis: Cancer affects a lot more people than sickle cell does. How hopeful are you that sickle cell, in this process of stem cell research in trying to cure these diseases, will be given the kind of priority, the kind of importance that it deserves, given that it impacts a–most often, not always–but a particular slice of our community which just happens to be African Americans?

Shapiro: Well, it’s the science, right? And it’s a technology and that’s what makes it unique. So you have to understand in the scope of things, as a rare disease, sickle cell anemia is one of the largest communities, all right? So there are 100,00, 110,000 in our country. It’s a worldwide disease, but actually fixing it technology-wise is very simple because it’s single gene fix.

Now you take that and you fix us, then you can apply that to all sorts of things. So that’s why–I mean, that really is why they’re looking at it in, again, the larger population for testing and being able to prove that it works.

Tavis: Dr. Svendsen, let me take that question I asked Adrienne and tweak it just a bit. So it is the science and I’m glad that you guys are in all of these things. When I asked you for examples, you gave one or two particular examples.

You could have given me many others, but how do we know what’s being prioritized or is it just the science? And if you discover one breakthrough, then every other bit of research benefits? Is that how that works or not?

Svendsen: Well, I think it’s a very good point that Adrienne just made. Sickle cell anemia is a single point mutation in the gene. And if we can prove that we can stem cells out, change mutation, just repair it–there’s this new technology called CRISPR which allows you to repair mutations–grow the cells up and then put them back in.

Now they’re reintroduced and, for the blood system, it’s quite nice. They integrate into the bone marrow and they’ll make a whole new blood system. That’s what we call proof of concept that stem cell therapy can work.

It’s actually being done for another rare disorder, SCID disease, at UCLA actually by someone called Don Kohn. That’s the doctor that also has got a grant from CIRM to do sickle cell. I think it’s a proof of concept and very, very important.

And it doesn’t matter that it’s one slice of the community because, if we can do it there, it might open up to all of the genetic kind of diseases as sickle cell. Just one point on the blood. Doctors love to work on blood diseases because the blood is unique.

You can eliminate a lot of the stem cells in the blood marrow where are all the blood stem cells are and replace the whole community and repair it. So now your blood goes back to normal. And that’s a remarkable system in the body, so we’re very hopeful for sickle cell that one could produce stem cell therapies for those diseases.

Tavis: There’s that word, hopeful, Dr. Federoff, that I wanted to pick up on. So, thank you, Dr. Svendsen, for the segue here. How hopeful should fellow citizens be when they watch a program like this and hear this kind of good news? How hopeful should we be that we’re getting close to some major breakthroughs on not just treating, but eradicating some of these illnesses?

Federoff: So I think hope should be harbored by all for three reasons. First is that the stem cell biology is revealing to a greater extent really what causes and how diseases progress, so that’s number one.

And will then too lead to a more precise understanding of how to intervene, whether it’s gene editing, as Clive said, or other, it will be built on the back of a technology, but also bone marrow transplantation, as Clive was saying, is something that we do routinely, so it will be added on.

And third, there are diseases that have no treatments and they’re often rare and they don’t rise to actually being significantly important in the pharmaceutical industry to be targets.

These technologies, when you think about their application because they are foundational technologies, allow us to take on those rare diseases and actually make a difference in the lives of individuals who otherwise would not be treated because there has been no great economic or financial motive to make that a target disease to develop a therapy for.

Tavis: Adrienne, is your mother still living?

Shapiro: No, no.

Tavis: Okay. In your lifetime, how hopeful are you then?

Shapiro: Oh, it’s going to happen.

Tavis: You think so?

Shapiro: It’s going to happen, yeah. And all we have to do is keep our loved ones alive until it happens. You said why did I fight for my organization? That’s why. It’s here.

Tavis: What do you do in your organization?

Shapiro: Well, we advocate for people with sickle cell–well, generally adults because we found our adults, many of them are only children. They have no one to go to the hospital with them, and because opiates are a large part of our treatment.

There is continued resistance in the medical field to give people the amount of pain meds that they need, so we want to give our adults the best chance to be here when it comes.

Tavis: Dr. Svendsen, I assume there must be–I can only imagine how good a feeling it must be to wake up every day to go to work, you and Dr. Federoff, to know that the stuff you’re working on is not just going to fundamentally change lives, but save lives.

Svendsen: It’s an incredible feeling and it’s extremely exciting at the moment. I’ll say on the discovery side, scientists are the worst people to predict how long something’s going to take because we’re always asking, “How long till the next one?” The reality is, things that you think might take years and years and years, you solve very quickly with one new innovative technology. Suddenly, that’s solved.

Things that you think should be easy could take a year and a half, two years. We call them the science gods. They keep at you. You just think you’re getting close and then they make it more difficult for you. It’s interesting. It’s a hard thing to do, but it’s exciting for sure.

Tavis: I got 30 seconds to go, Dr. Federoff. We are really at the end of that 10-year period that California voters initially funded a decade ago. $3 billion turns to $6 billion. Have we made enough progress now to justify to the voters of California and, for that matter, across the country reinvesting?

Federoff: Absolutely. I think unequivocally. This has been a demonstration for the nation and, I would argue, for the world. The progress made is so substantial that it needs to be further accelerated. And whether the state and its electorate determine that this is important, I can’t say, but I would strongly favor another proposition.

Tavis: I’ve said many times that what happens here in California casts either a long shadow or a long sunbeam across the nation. This is a perfect example of a long sunbeam being cast across America because of the work being done here in California, particularly in Southern California, which I’m pleased to call home, as are you. Thank you for coming on the program.

Shapiro: Thank you.

Tavis: Let me thank my hometown station, PBS SoCal, for having us here tonight in Costa Mesa to put the finishing touches on this week of shows about “One Great Idea” across the nation. Thank you for watching. Back in my studio tomorrow night. Until then, thanks for watching and, again, as always, keep the faith.

Announcer: For more information on today’s show, visit Tavis Smiley at

[Walmart Sponsor Ad]

Tavis: “One Great Idea Tour” is made possible by Enterprise. Pick Enterprise, we’ll pick you up.

Announcer: The California Endowment. Health happens in neighborhoods. Learn more.

Announcer: And by contributions to your PBS station from viewers like you. Thank you.

Last modified: October 6, 2016 at 5:42 pm