Stem Cell Research In San Diego Uses Patient's Own Cells
June 12, 2013 1:27 p.m.
Melissa Houser, Director of Parkinson's Disease and Movement Disorder Center at Scripps Clinic.
Cassandra Peters, participant, Parkinson's research, volunteer Summit-4-Stem Cell
Michael Kalichman, Director of Research Ethics Program at UC San Diego
Related Story: Stem Cell Research In San Diego Uses Patient's Own Cells
CAVANAUGH: Experimental medical research underway at Scripps has the potential for helping a number of patients with Parkinson's disease. But not only that. Researchers say it could also change the way stem cell research is viewed and the way doctors and patients team up to find new treatments for disease. Eight patients at Scripps hope the procedure will allow their own skin cells to be developed into cells that can replace brain cells damaged by Parkinson's. The patients are also involved in helping to raise millions of dollars for the research. My guests, Dr. Melissa Houser is director of the Parkinson's Disease and Movement Disorders center at Scripps. Welcome to the show.
HOUSER: Thank you, Maureen.
CAVANAUGH: And Cassandra Peters is here, a stem cell patient, to support non-embryonic stem cell research.
PETERS: Thank you.
CAVANAUGH: Of all the people with Parkinson's Disease, how did you become one of the eight involved in this?
PETERS: Well, that's a good question. And one I probably should have put to Dr. Houser before the broadcast. I think initially when I saw Dr. Houser and she gave me my diagnosis. I'm sorry, can you --
CAVANAUGH: Sure, I'm wondering how you were selected for this particular trial. Dr. Houser?
HOUSER: Cassandra met our inclusion criteria based on her age, her scoring, had no significant core morbidity, so she didn't have any other illnesses that would preclude her being a candidate.
CAVANAUGH: So what stage have these eight patients have progressed to? Is there a level that you wouldn't have taken them on this program?
HOUSER: Yes, based on the embryonic stem cell trials which used actually fetuses, we found out that the people below the age of 60 did a little bit better than the older aged group, and the earlier stage people did better than the later stage people. So based on that, we set our criteria around the mien age of 60 and a certain level of score.
CAVANAUGH: You use the term embryonic stem cell. And some of the research into Parkinson's patients has involved embryonic stem cells. But this is very different. It uses pluripotent stem cells. Can you tell us what that means?
HOUSER: Yes. By using your own skin cells, we devolve them into what's called a pluripotent stem cell. That is able to convert into any cell we want it to be. A bone cell, brain cell, a heart cell. And once we get the brain primed or the cell primed to become a brain cell, we turn it into a dope mean producing cell. And Dopamine is what's missing in Parkinson's.
CAVANAUGH: What was the procedure like?
PETERS: Well, are the initial procedure to have my skin cells removed was very simple. There was a small plug, I would say the size of a pencil eraser of skin removed from my upper arm. And I don't even think it bled. It was really relatively painless. And frankly sort of incredible that they could do so much with so little. But I now have a little bitty scar, and that's my badge of honor.
CAVANAUGH: Doctor, we lay people have understood for the longest time that mature cells were not as malleable as embryonic stem cells and therefore couldn't be transformed into different kinds of cells. What happened?
HOUSER: Well, the technology changed. We were able to harvest mature cells and learn how to nurture them to become embryonic-type stem cells that have the potential to go back into their original purpose.
CAVANAUGH: That sounds very mysterious. Obviously you don't remove the DNA from the cells. They are these patients' cells. But how do you make them go back in time like that?
HOUSER: With very nutrients, various genetic engineering of the cells, growth factor, enzymes in the petri dish.
CAVANAUGH: And from what I understand, the person came up with the way of changing these cells into stem cells that can morph into brain cells and so forth, that person won a Nobel Prize?
CAVANAUGH: How often has this particular procedure been used in any kind of medical research?
HOUSER: Well, the use of IPS is quite common. There are lots of institutes doing this sort of cell work. But most of them are doing it to check how drugs can work on that cell line. And some of them are doing animal trials. Our trial is trying to take it into the rodent phase with a view toward using it as actual therapy for Parkinson's disease, instead of keeping it in the lab.
CAVANAUGH: And what is the goal of this procedure? You're taking the skin cells from patients like Cassandra, you're manipulating them so they become stem cells that can mirror other cells, really become other cells in the body. So where are you going to put these cells?
HOUSER: Well, first we're going to put them in rats. In their brains, and see if they're safe, and see if they're efficacious in treating rat Parkinson's behavior. Then we're going to go through our regulatory process to get FDA approval. Go through the board to check for the ethics of it. But our main goal is that we'll eventually be able to do a pilot study with the same eight patients and plant those autologous, meaning patient-specific cells, back into the brain.
CAVANAUGH: Michael Kalichman is on the line, Director of the Research Ethics program at UC San Diego. Welcome back to the show.
KALICHMAN: Hi, Maureen.
CAVANAUGH: I spoke with Dr. Houser, and she explained how these cells in this pilot program for Parkinson's disease are not embryonic. They're mature cells that are being manipulated. Since much of the debate over the use of stem cells involves embryonic stem cells, do you think this procedure puts that debate to rest?
KALICHMAN: Well, it certainly doesn't raise the issues that human embryos do because we're not using them in this case. But there are -- almost everything we do has ethical questions, questions about how we should act. And there are still some challenges. The question of talking to the IRB about what's going to be done, one of the challenges is do the people who are participating fully understand what they're getting into? The informed consent process. And we have a system in place to try and ensure that researchers make sure that the subjects in these studies are aware of what we're doing, so when they say yes, they know what they're saying yes to.
CAVANAUGH: Cassandra, how well informed do you feel along the steps of this procedure? Have you been kept up-to-date and worked close with the doctors all the way through?
PETERS: Well, I think that that is part of what sets this entire proposed study apart from others. We are in contact, not just with doctor Houser, but with Dr. Loring, and all the scientists that are doing this, are that are changing our cells into the Dopamine neurons that we need. So I personally feel like I have been kept wonderfully well informed. And any possible question I could have would be answered.
CAVANAUGH: How has this changed or altered the way that you're coping with Parkinson's?
PETERS: Well, I think -- I don't speak it too strongly when I say that it has provided a purpose for my life. When I was given this diagnosis, I think it's fair to say that I felt it was a death sentence. And nothing could be farther from the truth. Parkinson's, obviously, can be controlled with medication, and exercise is wonderful. There are a lot of things that you can do to mitigate the disease. But so far, the one thing that can't be done or hasn't been done up to now is you can make it stop and go away. So I feel extraordinarily blessed to be part of this project. Because I feel that we are literally on the edge of a whole wondrous and new medical age.
CAVANAUGH: And doctor Houser, if -- it sounds as if there's quite a few hurdles that you have to go through in order to see whether or not this procedure is effective. But if it is approved and this trial gets your way, what implications does this research have for other diseases?
HOUSER: Like any investigational device or any medication that's being developed for Parkinson's disease, it will go through a number of hurdles, make sure it's safe, make sure it's effective. Then it could be put into controlled clinical trials, placebo, double blind, and maybe make this treatment available for Parkinson's patients worldwide. If we can establish that this is effective in Parkinson's, also we might be able to translate it into other neurodegenerative diseases, Alzheimer's, ALS.
PETERS: Macular degeneration.
HOUSER: Yeah, should it be effective.
CAVANAUGH: There's another aspect of this particular trial that is underway, I guess you can call it. It's being funded by patients themselves. And perhaps Cassandra you could tell me more of that. There's a Summit for Stem Cell Organization that's sort of developed with this trial. How are you raising the money? This is taking a lot of -- a couple million dollars, right?
PETERS: At least, yeah, 2.5 as I understand it. How are we raising the money? Any way we can. Personally, I'm just trying to make sure that the word gets out about the summit for stem cell. Sherry Gould who is doctor Hauser's nurse practitioner, she is the fireplug. She is the initial impetus behind all this. And she initially proposed that this group climb Mt. Kilimanjaro to raise funds for this project. And they did! There was a group of I believe 16 climbers, I could be wrong about that. Three of which had Parkinson's. And they all made it to the top.
PETERS: So one other way that we were doing it was to continue the mountain climbing idea which is wonderful. And the mountain we all think of first is Mt. Everest. So the summit for stem cell in October is going to take basically I think the same group of people to climb up to base camp on Mt. Everest.
CAVANAUGH: Wow. Let me ask Michael Kalichman, that's an inspiring story, but do you see any ethical challenges with having patients raising the money for the research that's being done? Is this a healthy change or does it pose any ethical challenge?
KALICHMAN: Well, I think the biggest problem with any project is whether people are informed, and whether they know what they're getting into. Research is funded by the government, it's funded by private foundations, in this case it's funded by patients. And in all cases, as long as people know that there are risks, that there are uncertainty, this is all very exciting and very important. Parkinson's can take a tremendous toll on individuals and on their families. And this is an opportunity to do something about it. We won't know until the research is done whether it will make a change. As long as people know that there is that chance. It's not a guarantee that you put the money in, we get a guaranteed result.
PETERS: I can speak as part of this project, and any question, any concern that I've had is immediately addressed by either Dr. Loring, Houser, or one of the scientists in the lab. I feel as well informed as a person can that obviously doesn't have a science background.
CAVANAUGH: And Dr. Houser?
HOUSER: Well, I share the ethical concern about having patients in any way pay for a form of their treatment or research. But what I liken this to is more like the breast cancer world. It's not just patients that are funding a climber or sponsoring a climber. One of my best friends had breast cancer, my daughter and I went on the walk, and we paid to sponsor the walk. So it's just people that love patients that have Parkinson's disease.
CAVANAUGH: And such important research. What do you see your timeframe as?
HOUSER: We already have two cell lines that are ready to be delivered into the rodent brains. We anticipate that'll take about a year. Then we have the language and the data do forward to enter into the conversation with the FDA, and then apply for a multimillion dollar grant that would take us into the next translational phase.
CAVANAUGH: Now, since these procedures are so specific to the individual patient, you've taken individual cells, skin cells, and you're going to try to turn them into cells that can actually help damaged brains repair themselves in Parkinson's patients. How would this be used in a more general way? Is there any sort of general application from what you're going to be finding out specifically with each of these patients?
HOUSER: Well, we could -- right now, it only takes us up to the rat phase. But we can also keep the cells alive in the lab and have them used for specific conditions the patient might get at any time in their lifetime. Heart disease. But are specifically developing these cells to be Dopamine producing cells though. So our point is not to freeze them and use them for embryonic stem cells. We're using them just for this particular disorder.
CAVANAUGH: I have to end it there. I want to thank my guests. Thank you so much for coming in and speaking to us. And good luck!
HOUSER: Thank you.
PETERS: Thank you.
KALICHMAN: Thank you. Bye-bye.