Stem Cells and Genetic Diseases Explained - Discover the Truth

Joy Kong, MD explains what stem cell therapy can and cannot do for genetic diseases. Gene therapy has real promise but isn't a cure-all, and managing expectations matters as much as the science itself.
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This is the exact reason why some doctors or scientists in the field does not like the word stem cells, because they feel that this is giving some kind of connotation that we're providing this magical cure that would put the cells in the body and they will magically appear where they're needed and then start replicating and replenishing local tissue. Hello. Welcome back, everybody. I'm coming back to answering your questions about stem cell therapy. So there are so many questions, but one thing does pop up is about genetic diseases. So there are a lot of genetic diseases. Some happen very early in life and some later. Cystic fibrosis earlier on onset, and Huntington's disease happening start happening in midlife. More common, more famous, the sickle cell anemia, hemophilia, et cetera. So what about stem cells? Can it go in and magically fix everything? Well, not really. At least not yet. And this is the exact reason why some doctors or scientists in the field does not like the word stem cells, because they feel that this is giving some kind of connotation that we're providing this magical cure that would put the cells in the body and they will magically appear where they're needed and then start replicating and replenishing local tissue. Right. Repair and making new tissue. In theory, it sounds great, but we're really not. We can put certain cells, for example, mesenchymal stem cells, locally and help with local regeneration. We can even put cells in IV form that help rejuvenate your immune system and that can travel to places where they're needed. But the majority of the time, their function is not to replace local tissue. It's really to tell your own tissue to regenerate so that they're signaling your immune system to come on board to help you calm inflammation and promote regeneration. And. And then they're triggering local stem cells to come alive and start to replenish tissue. So this is one of the major functions. Of course, we have other forms of stem cell therapy, like bone marrow transplant or cord blood transplantation, accomplishing very similar things. This is actually one of the only FDA indications for stem cell therapy, which is to replenish the whole blood and immune system after a person's immune system is wiped out through chemotherapy in cases of autoimmun and cancer. So the only other FDA approval is for acute graft versus host disease that's still steroid resistant. So, so these are indications. But yes, it can replenish the blood system and it can be curative in certain conditions, like the ones that you know Cancer, autoimmune diseases, so it can change your immune system. But as far as genetic diseases like what we're talking about, maybe single gene, right. We think that it should be easy to fix. However, stem cell therapy, that's not really how it works. It promotes regeneration, promotes repair, and only a fraction of the cells, if any at all, actually stay in the body. There's wide degree of, I think, variant variation of opinion. Some will say that none of them survive. And of course, research shows that there can be a fraction or even larger fraction when a person is more depleted. There, there can be some kind of chimerism. So it means two organisms in one body, right? Two, two type of tissues and DNA. So it can happen, but can it reverse genetic diseases? The quick, you know, answer that may not encompass everything, but if you really want an answer, I would just say no right now. Because the degree of incorporation into the new, the new person, right? So the person that wants healing, the degree of incorporation of the DNA is still very low. What we can do is to prevent damages or reduce damages, and that's downstream from the genetic defect. So just the faulty genes themselves doesn't mean that you, you are not going to do well. Right. That's why Huntington's disease, it takes some time to manifest or certain metabolic diseases, well, it, it takes time for the toxic metabolites to build up and then you're noticing symptoms. So it's not just the gene itself, right? The genes can be turned on and off also. But if the genes are being manifested, and it still takes time for your end organ to accumulate damage, now stem cell therapy can come in, help reverse the damage in the tissue so people can feel better if they have, let's say, kidney damage, the kidney damage can be reversed to some degree. The tissue can be healthier, and the overall inflammation of the body as a result of all these damages can calm down. So we can help the person to some degree. Of course, there are various genetic defects. So some, for example, bone marrow transplant, if you have sickle cell anemia, it could be curative, but for a lot of these genetic mutations, unfortunately, right now, just stem cell therapy that we can get from either the US or abroad probably is not going to do, you know, have a major impact on the gene, you know, those genetic material. However, you can do gene therapy, but gene therapy is still at early stages. So there are a few ways that people can get gene therapy. One is through the CRISPR method, right? It's been talked about a lot, but there are various drawbacks still. And it's very expensive. And there's another method which use plasmid and that could be a little easier on the body, but that's still new. There's still not as many conditions that actually has a, a modification method. So we're, you know, still at this place that we're hoping for more advances. But you know, that said, I also want to mention a particular category of genetic disease which is mitochondria disease. So yes, your mitochondria, it's like an organ organism that has symbiosis with your own cells. So all your cells are mammalian cells will have these mitochondria that's inside producing ATP energy, but that's really a bacteria. So they carry their own DNA material and that only is passed down by the mother. So it's through the maternal line because the eggs will have the mitochondria. And interestingly the that when there's a reset process, the mitochondria is also reset. So when the baby's formed, there's fresh new mitochondria. Well, we all know our mitochondria declines as we age. That's one of the biggest reasons for chronic illness. So that's why there are a lot of therapies geared toward mitochondria enhancement. And, and you know, there's all the rage these days, mitochondria. And there are testing also developed for. Exactly. For mitochondrial function. So for mitochondria disease, the degree you can do mitochondria transfer, what's interesting was if you do, let's say umbilical cord derived stem cell treatment, these are young stem cells, right? And with young mitochondria, very, very young. So, so young that is in between the embryonic stem cell and the baby's stem cell. So even though the baby is zero day old, right, Day one freshly born, but the stem cells that's in that baby's body is not as young as what's in the umbilical cord. Because what happened was when the baby was forming, the fetus forming, the cells were trapped in the umbilical cord and that's why it's retained a lot of its primitive characteristics. So that's why it' and those cells will contain these fresh new mitochondria and it can help replenish mitochondria to some degree. So now you've got these healthy mitochondria without the genetic defect. So this is kind of the, a little bit of the breakdown about genetic condition and stem cell therapy. So it's very important that we don't give people false hopes. What we can and what we cannot bring about needs to be spelled out to, you know, the public and the patient so we know where we're at. And yeah, I wish we have all have magic wands and we can cure everybody, but maybe at one point we would. So science is advancing at an incredible rate. It has become very, very exciting, even just in the area of gene therapy using plasmid to start producing certain proteins like Follistatin. Right. So Brian Johnson went over and some celebrities also and people, other people have gotten, I've known people who have gotten the gene therapy inserting a gene that expresses Follistatin, which promotes muscle growth and help with body recomposition and stamina and maybe even mental health, cognitive improvement. So, yes. So things are changing rapidly and it's very exciting. Make sure you drive safe, stay alive, so that you can reap the benefit of all these new technology. And that's it. I have for you today and thank you so much for spending this time with me.










