Channel: Boston Children's Hospital clear
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| 1 | Boston Children's Hospital | induced pluripotent stem (iPS) cells: Part 1 | 7274 | 48.2 | 2:50 | We have two different types of stem cells, the traditional embryonic stem cell, which comes from early human embryos, and this new form called the induced pluripotent stem cell. By calling it pluripotent, we recognize that it has the ability to make any tissue in the body. A property that previously we thought only embryonic stem cells had. Now importantly, they're not identical, and we're still learning whether or not the induced pluripotent cells can be a full replacement for embryonic stem cells. They may one day, but certainly for the foreseeable future, we're going to move forward studying both types of stem cells. A little over two years ago, a Japanese scientist named Shinny Amanaka discovered that if you took a small set of genes, which are normally expressed in embryonic stem cells, and you made them expressed in skin cells, it would turn the skin cells into embryonic stem cells. These are the so-called IPS cells, the induced pluripotent stem cells. There's been a natural evolution in the technique itself. The practice that Shinny Amanaka introduced two and a half years ago was to carry these reprogramming genes in via viruses. The problem is that viruses insert themselves into the cells DNA and can act as mutagens, it can actually induce the cells to become cancerous. So over the last couple of years, we and others have been working on ways of eliminating the viruses from the system. And now we have a new improved approach where we can say use a virus to carry these reprogramming genes into the cell, affect the reprogramming, the reversion to the embryonic state, but then we can remove the viruses. And that's one technique that we practice that leaves us with a viral-free cell, which is a pristine cell that we can now study, and it would be a cell that would be safe to put back into a patient. The real long-term hope, though, is that we don't have to use viruses at all, that we could identify drugs which act on the same pathways as the viruses to reactivate this embryonic or pluripotent potential in every cell. Initially, we expect we'd do it in a petri dish, but maybe in the long run, we'd even be able to learn how to affect these changes or transitions and cell fate in the actual identity of a cell from one type into another in your own body. That would be cell therapy, that would be the realization of one of the great ambitions of regenerative medicine. | ↗ | |||
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| 2 | Boston Children's Hospital | Induced Pluripotent Stem Cells (iPSC) Part 2: Research and Therapy | 1064 | 43.2 | 1:42 | As a physician scientist here at Children's Hospital, I see lots of kids with a variety of both genetic and malignant conditions that affect the blood in the bone marrow. I see kids come in with sickle selenemia. It's a very, very painful illness that's caused by a single abnormal base, a single point mutation out of the three billion pieces of genetic code in their cells. And yet that single abnormal molecule results in tendency for their blood to become thick and sludge in their vessels and cause excruciating pain and adeterioration in organs over time, the heart, the lungs, the kidneys. That's a very devastating disease. And yet we can now study that in a petri dish. We can take sickle cells from a patient, put them into a petri dish, reprogram and repair the sickle cell defect. And in a mouse model, we've actually been able to show that you can affect essentially a cure in that disease. But then there's dozens of others. There are various kinds of anemias in bone marrow failure. Things called fanconees anemia, discaritosis congenitum. A variety of these conditions are all now subject to the same basic plan form. Where we take the patient's cells, we reprogram them into stem cells in a petri dish and then have a tool for research and a possible cell for therapy. | ↗ | |||
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| 3 | Boston Children's Hospital | Induced Pluripotent Stem Cells (iPSC) Part 3: The Future of Stem Cell ... | 755 | 42.3 | 4:21 | We have a very high hope for being able to treat patients with the products of stem cells. I don't know when that's going to occur. I wish I could promise it as soon as five to ten years, but in reality it may take much longer. However, we're hopeful that one of the first successes will be in the blood. So we've known for many decades that we can transplant healthy bone marrow, which is the blood-forming tissue, from a donor into a diseased patient, someone who might have leukemia or might have one of these genetic diseases. The problem is that we don't have a matched donor for every patient. And so some patients go without the ability to have this potentially curative bone marrow transplant. But if we could take from every patient a skin biopsy and make their own stem cells, be able to make blood stem cells that we repair genetically and we can give them back a healthy transplant, we could solve the donor shortage problem for bone marrow transplantation, we could treat any one of dozens of diseases. Here we've gained this success with blood, we want to start expanding to other tissue types. So we now have efforts going with the lung biologists and there are very exciting opportunities for thinking about treating cystic fibrosis with this strategy. We're talking with experts in metabolic disease. We're talking with cardiologists. We're beginning to study the development of beating heart cells in the petri dish. But applications of these pluripotent stem cells are really limitless. It's been unfortunate, but already we've seen medical entrepreneurs typically not within the United States, but in rather unregulated locals like Bermuda or China or Russia, already offering so-called stem cell therapies to patients. Pants are vulnerable. Patients want to believe that there's a cure out there somewhere and they're unfortunately too easily preyed upon by the siren call of clinicians who claim to have the answers. So we're involved in trying to educate people about the realities of stem cells. Whereas we do believe we're making progress, it will take time. And so patients need to be wary. They need to use all of the resources available, including the clinicians and scientists at Children's Hospital to answer their questions. Also, we're yet children's hospital have been leaders of the International Society for Stem Cell Research. And this organization, the leading worldwide professional organization of stem cell scientists, has come up with a set of guidelines for moving this very promising science to prove in clinical therapies. And on the website, www.inscr.org, patients can find information packets to arm them with the information they need and the questions they need to have to ask their doctors whether or not the kind of therapy that they're being offered is stem cell based and is legitimate. Because we do hope to be able to have real stem cell therapies coming online over the next five to ten years. But patients have to be wary of those illegitimate folks who are promoting stem cells way ahead of their being proven. And so we hope in some day in the not-too-distant future that each area of disease, that is studied and treated in the Children's Hospital community, we'll be using in one way or another the fruits of the pluripotent stem cell biology. | ↗ | |||
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| 4 | Boston Children's Hospital | Induced Pluripotent Stem Cells (iPSC) Part 1: Techniques for Deveopmen... | 724 | 42.2 | 2:51 | We have two different types of stem cells, the traditional embryonic stem cell, which comes from early human embryos, and this new form called the induced pluripotent stem cell. By calling it pluripotent, we recognize that it has the ability to make any tissue in the body. A property that previously we thought only embryonic stem cells had. Now importantly, they're not identical, and we're still learning whether or not the induced pluripotent cells can be a full replacement for embryonic stem cells. They may one day, but certainly for the foreseeable future, we're going to move forward studying both types of stem cells. A little over two years ago, a Japanese scientist named Shinny Amanaka discovered that if you took a small set of genes, which are normally expressed in embryonic stem cells, and you made them expressed in skin cells, it would turn the skin cells into embryonic stem cells. These are the so-called IPS cells, the induced pluripotent stem cells. There's been a natural evolution in the technique itself. The practice that Shinny Amanaka introduced two and a half years ago was to carry these reprogramming genes in via viruses. The problem is that viruses insert themselves into the cells DNA and can act as mutagens, it can actually induce the cells to become cancerous. So over the last couple of years, we and others have been working on ways of eliminating the viruses from the system. And now we have a new improved approach where we can say use a virus to carry these reprogramming genes into the cell, affect the reprogramming, the reversion to the embryonic state, but then we can remove the viruses. And that's one technique that we practice that leaves us with a viral-free cell, which is a pristine cell that we can now study, and it would be a cell that would be safe to put back into a patient. The real long-term hope, though, is that we don't have to use viruses at all, that we could identify drugs which act on the same pathways as the viruses to reactivate this embryonic or pluripotent potential in every cell. Initially, we expect we'd do it in a petri dish, but maybe in the long run, we'd even be able to learn how to affect these changes or transitions and cell fate in the actual identity of a cell from one type into another in your own body. That would be cell therapy, that would be the realization of one of the great ambitions of regenerative medicine. | ↗ | |||
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| 5 | Boston Children's Hospital | Induced Pluripotent Stem Cells (iPSC) Part 4: Continued Research | 234 | 39.2 | 2:55 | Operating under restrictive policy for the last eight years certainly has encouraged a degree of outside the box, thinking. To every cloud there's a silver lining and I hesitate to say that the president's restrictions were responsible for these breakthroughs in direct cell reprogramming but no doubt this major breakthrough has in fact emerged in a setting where we were otherwise compromised in the types of science we could do with embryonic cells. But it's important to realize that the breakthroughs in direct cell reprogramming are built upon the foundation of embryonic stem cell biology. The new induced pluripotence stem cells, the ones where we can make any patient skin cells back into an embryonic like state is very exciting but it doesn't answer all of our questions in science. The embryonic stem cells remain the gold standard. We've studied them in mice for 30 years in humans for 10. We've only had the induced pluripotence cells for two years so we have much less experience. We are certainly hopeful that they'll do everything that we know the embryonic stem cells can do but we remain to prove that point. There are also many very important questions that relate to the earliest stages of human development. These are questions that are absolutely germane to issues like birth defects, human fertility and infertility and some kinds of cancer. We'll never get at those questions by studying reprogrammed skin cells. We need to go to the early human embryo to be able to understand those issues. The liberalization of federal funding under the new Obama plan will be exciting and will offer us more support for carrying this vision forward but it won't provide us all the resources that we need. Unfortunately, the National Institutes of Health isn't particularly effective at funding this very risky translation of bench research to the bedside and there are lots of resources that we still need to go to the philanthropic community to fulfill. So with a combination of this enhanced federal support and the visionary support of philanthropists we hope to be able to put together the very, very considerable sums of money. It's going to take us to realize this goal, this $60 million, seven year goal of getting stem cells into patients. | ↗ |