A marrow or cord blood transplant could be the best treatment option, and for some patients, offer the only potential cure, when facing life-threatening diseases.
A FACT accredited (Foundation of the Accreditation of Cellular Therapy) program and a National Marrow Donor Program (NMDP) unrelated and cord blood donor center, Children’s Mercy Kansas City is a leader in research and the treatment of rare and complex diseases using bone marrow, peripheral blood stem cells and cord blood transplants. The program offers autologous, related allogeneic, unrelated allogeneic, and haplo-identical transplants.
Listen as Rakesh Goyal, MD, shares how the Children’s Mercy BMT program is expanding the use of transplants to successfully treat immunodeficiency, metabolic, sickle cell disease and other rare diseases.
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BMT for Treatment of Rare Diseases: New Treatments and Donor Options
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Learn more about Rakesh K. Goyal, MD
Rakesh K. Goyal, MD
Rakesh K. Goyal, MD, is the Chief of Section of Blood and Marrow Transplantation (BMT) at Children’s Mercy Hospital. He also serves as Associate Division Director, Hematology/Oncology/BMT. He is a Professor of Pediatrics at the University of Missouri, Kansas City School of Medicine. Dr. Goyal completed his medical degree and residency in pediatrics in New Delhi, India. He did US residency in pediatrics in at the University of Connecticut Health Center. He then completed a clinical and research fellowship training at Washington University School of Medicine in St. Louis, MO. Dr. Goyal is board certified in Pediatric Hematology/Oncology. He has published and presented extensively on graft vs. host disease, and use of transplant to treat inherited metabolic disorders and immunodeficiency, and other BMT-related topics.Learn more about Rakesh K. Goyal, MD
Transcription:
BMT for Treatment of Rare Diseases: New Treatments and Donor Options
Dr. Michael Smith (Host): Today's topic is bone marrow transplant for the treatment of rare diseases. My guest is Dr. Rakesh Goyal. He’s the Section Chief of bone marrow transplantation and Professor of Pediatrics at University of Missouri, Kansas City School of Medicine. Dr. Goyal, welcome to the show.
Dr. Rakesh Goyal (Guest): Thank you, Dr. Mike. It’s a pleasure to be on your show.
Dr. Smith: So, let’s… Tell us a little bit, I know that you are new to the Children’s Mercy bone marrow transplant program. As the chief, what are your goals for the near and far future for the program at Children’s Mercy?
Dr. Goyal: I am tremendously excited to be a part of Children’s Mercy institution. I consider this as a family and I have worked in this field for quite a while. In fact, in my prior institutions, I worked a number of new areas that are highly promising to cure children with these rare diseases with transplantation. And one of the reasons I came here was that Children’s Mercy, in terms of its supervision, its capability and its research platform offers tremendous opportunity for me to build on my expertize and take this program forward.
Dr. Smith: Let’s talk a little about transplantation, specifically bone marrow transplantation. How has the procedure, in your opinion, improved over the last few years?
Dr. Goyal: Yes, the main excitement in the field is that transplantation is no longer indicated just for children or young adults who have cancer or leukemia, the former diseases, which otherwise would be life threatening or life taking, but there are these new diseases and some old diseases which themselves are not like cancer or leukemia, but have grave implications for quality of life of patient, and sometimes do shorten the life span of these patients. So, the community and researchers such as myself have applied transplantation as a way to fix or cure these patients from their diseases and the list just keeps expanding. So, what has made that possible is a shift in the mindset of the transplant doctors and researchers, that we don’t need to treat these patients as if they have leukemia because we were giving them a ton of chemotherapy, sometimes a lot of radiation to make transplant work. Now, what we have realized is that if the patient doesn’t have leukemia, they have a missing gene product or they have a missing factor that makes them not feel well or be terribly unhealthy, like a weakness in the immune system. Then we can do a transplant and there, we no longer use one size fits all approach. We actually customize what a patient is going to get for transplant recipe. That way the patient sees less toxicity, there are less long-time side effects and we are trying to preserve quality of life including, for a child their growth, their development and, hopefully, the ability to have children themselves later on, to start a family.
Dr. Smith: How exciting. What you just said about the list just keeps on growing on the things that you can treat and even cure through transplantation is just going to continue. Because you’ve really opened a window, haven’t you, with that you’re doing now to really make an impact on diseases we really didn’t have many options for in the past, is that right?
Dr. Goyal: That is true. There are several examples. One large example is the community of children who are affected by Sickle Cell Disease in which the red blood cells that makes hemoglobin which carries oxygen and that is the engine for life is defective and that makes them terribly sick with a variety of organ system issues, pain, poor quality of life, sometimes even infarct and stroke as a child. Then, there is thalassemia, in which the patient is transfusion dependent for the rest of their life. And then eventually may lose their life to iron overload. And then, the other very exciting field is babies who are born with weak immune systems or what is called “bubble-boy syndrome”, where their immune system is so weak that if somebody has a cold, that cold could prove lethal for them. States are now starting to do newborn screening. So, that is another big development, meaning on that filter paper spot from newborn heel stick. Now, the list of diseases that can be diagnosed at birth, before the child has even symptoms, keeps growing. And then, some of them are really transplantable. So, the one big category, even though it’s rare, but highly curable is that category of immune deficiency also called [inaudible 05:14]. The third large category is patients who have these terrible diseases they are born with called “inherited metabolic diseases”, and these include Hurler syndrome, which can be eminently cured with transplant and then, some diseases for which transplant should be considered as a research modality of treatment but highly promising, and those include, diseases I describe is some forms of [inaudible 05:41] and I can go on and on.
Dr. Smith: Right, right. Yes, so, what about the process or the procedure itself? You know, it wasn’t that long ago, Dr. Goyal, that when you heard the word “transplant”, whether it was bone marrow, STEMS or whatever, we all thought of allogenic transplant, right? Where there is a donor, usually a sibling; there was always the rejection that we had to deal with. So, where do we stand with the types of transplants? Are we moving away from allogenic? Are we looking more at the stem cells of the patient? Where do you see that going, the actual procedure?
Dr. Goyal: Great question, Dr. Mike. In fact, we still use, or like to use a donor for the majority of these types of transplants because if you use their own cells, you can’t fix the defects that they have that a lot times they are born with. However, the choice of donor, that menu has expanded tremendously. Often times we have to and continue to find a donor outside the family, given the American family size and structure. Their income challanges for patients who maybe of some ethnic minority or they may have a family background that is relatively uncommon or maybe mixed. Then, we always struggle how to find a good or perfect match. Now, there are two major developments in that field and we are actually actively participating in both. One of them is umbilical cord blood. So, these are not just stem cells as we think of stem cells as in fetus. These are stem cells. They are incredibly powerful. They are double charged and they work extremely well. So, the field of doing transplants from cord blood have taken off in a very good way, especially for children. And, then last but not least – haploidentical transplant, meaning if you can’t find a brother or a sister, or you can’t find a suitable cord blood, then what do you do? There is always a parent, often times two. So, now, we actually have tweaked and developed, there are pioneers, recent pioneers, who have perfected their techniques. I have no hesitation in actually calling up a mom or dad or a brother who’s not even matching and use them. We actually are moving to a field where anybody who needs a transplant, especially a child, would have a donor. So, that is extremely exciting.
Dr. Smith: Yes. That’s really exciting. It must, Dr. Goyal, be very exciting for you personally to be at the forefront of such an exciting field with so much potential. Just for the last question, give us your own personal opinion about where you really see transplantation going into the future. Summarize for us what excites you about the technology.
Dr. Goyal: I think the main, even in my practice time, over last nearly two decades, it’s so gratifying to see that children don’t get as sick when they going to transplantation. That is not always a case. If you have a child with leukemia who has already had a lot of treatment, that’s different. The majority of the time, our focus right now is to make transplant as safe, as non-toxic and to preserve the quality of life for later on so that these transplant survivors, having beaten their underlying terrible diseases can have normal life experiences and can begin to be employed and go to college and do all the good things. So, I see the field of transplantation, in terms of continuing to improve and fine tune and, it’s sort of, I believe it’s going to be personalized and with the discovery in genomic medicine, and how we know everybody’s metabolism and body work, we’ll be able to actually come up with a recipe, no longer one size fits all; no longer one big bang type of an approach.
Dr. Smith: Very exciting stuff. Dr. Goyal, I want to thank you for coming on the show today. Thank you for all the work you are doing. It really is exciting to see what Children’s Mercy is doing and how exciting it is for you to take the reins there. Good luck to you at Children’s Mercy. You’re listening to Transformational Pediatrics with Children’s Mercy Kansas City. For more information, you can go to www.childrensmercy.org. That’s www.childrensmercy.org. I’m Dr. Mike Smith. Thanks for listening.
BMT for Treatment of Rare Diseases: New Treatments and Donor Options
Dr. Michael Smith (Host): Today's topic is bone marrow transplant for the treatment of rare diseases. My guest is Dr. Rakesh Goyal. He’s the Section Chief of bone marrow transplantation and Professor of Pediatrics at University of Missouri, Kansas City School of Medicine. Dr. Goyal, welcome to the show.
Dr. Rakesh Goyal (Guest): Thank you, Dr. Mike. It’s a pleasure to be on your show.
Dr. Smith: So, let’s… Tell us a little bit, I know that you are new to the Children’s Mercy bone marrow transplant program. As the chief, what are your goals for the near and far future for the program at Children’s Mercy?
Dr. Goyal: I am tremendously excited to be a part of Children’s Mercy institution. I consider this as a family and I have worked in this field for quite a while. In fact, in my prior institutions, I worked a number of new areas that are highly promising to cure children with these rare diseases with transplantation. And one of the reasons I came here was that Children’s Mercy, in terms of its supervision, its capability and its research platform offers tremendous opportunity for me to build on my expertize and take this program forward.
Dr. Smith: Let’s talk a little about transplantation, specifically bone marrow transplantation. How has the procedure, in your opinion, improved over the last few years?
Dr. Goyal: Yes, the main excitement in the field is that transplantation is no longer indicated just for children or young adults who have cancer or leukemia, the former diseases, which otherwise would be life threatening or life taking, but there are these new diseases and some old diseases which themselves are not like cancer or leukemia, but have grave implications for quality of life of patient, and sometimes do shorten the life span of these patients. So, the community and researchers such as myself have applied transplantation as a way to fix or cure these patients from their diseases and the list just keeps expanding. So, what has made that possible is a shift in the mindset of the transplant doctors and researchers, that we don’t need to treat these patients as if they have leukemia because we were giving them a ton of chemotherapy, sometimes a lot of radiation to make transplant work. Now, what we have realized is that if the patient doesn’t have leukemia, they have a missing gene product or they have a missing factor that makes them not feel well or be terribly unhealthy, like a weakness in the immune system. Then we can do a transplant and there, we no longer use one size fits all approach. We actually customize what a patient is going to get for transplant recipe. That way the patient sees less toxicity, there are less long-time side effects and we are trying to preserve quality of life including, for a child their growth, their development and, hopefully, the ability to have children themselves later on, to start a family.
Dr. Smith: How exciting. What you just said about the list just keeps on growing on the things that you can treat and even cure through transplantation is just going to continue. Because you’ve really opened a window, haven’t you, with that you’re doing now to really make an impact on diseases we really didn’t have many options for in the past, is that right?
Dr. Goyal: That is true. There are several examples. One large example is the community of children who are affected by Sickle Cell Disease in which the red blood cells that makes hemoglobin which carries oxygen and that is the engine for life is defective and that makes them terribly sick with a variety of organ system issues, pain, poor quality of life, sometimes even infarct and stroke as a child. Then, there is thalassemia, in which the patient is transfusion dependent for the rest of their life. And then eventually may lose their life to iron overload. And then, the other very exciting field is babies who are born with weak immune systems or what is called “bubble-boy syndrome”, where their immune system is so weak that if somebody has a cold, that cold could prove lethal for them. States are now starting to do newborn screening. So, that is another big development, meaning on that filter paper spot from newborn heel stick. Now, the list of diseases that can be diagnosed at birth, before the child has even symptoms, keeps growing. And then, some of them are really transplantable. So, the one big category, even though it’s rare, but highly curable is that category of immune deficiency also called [inaudible 05:14]. The third large category is patients who have these terrible diseases they are born with called “inherited metabolic diseases”, and these include Hurler syndrome, which can be eminently cured with transplant and then, some diseases for which transplant should be considered as a research modality of treatment but highly promising, and those include, diseases I describe is some forms of [inaudible 05:41] and I can go on and on.
Dr. Smith: Right, right. Yes, so, what about the process or the procedure itself? You know, it wasn’t that long ago, Dr. Goyal, that when you heard the word “transplant”, whether it was bone marrow, STEMS or whatever, we all thought of allogenic transplant, right? Where there is a donor, usually a sibling; there was always the rejection that we had to deal with. So, where do we stand with the types of transplants? Are we moving away from allogenic? Are we looking more at the stem cells of the patient? Where do you see that going, the actual procedure?
Dr. Goyal: Great question, Dr. Mike. In fact, we still use, or like to use a donor for the majority of these types of transplants because if you use their own cells, you can’t fix the defects that they have that a lot times they are born with. However, the choice of donor, that menu has expanded tremendously. Often times we have to and continue to find a donor outside the family, given the American family size and structure. Their income challanges for patients who maybe of some ethnic minority or they may have a family background that is relatively uncommon or maybe mixed. Then, we always struggle how to find a good or perfect match. Now, there are two major developments in that field and we are actually actively participating in both. One of them is umbilical cord blood. So, these are not just stem cells as we think of stem cells as in fetus. These are stem cells. They are incredibly powerful. They are double charged and they work extremely well. So, the field of doing transplants from cord blood have taken off in a very good way, especially for children. And, then last but not least – haploidentical transplant, meaning if you can’t find a brother or a sister, or you can’t find a suitable cord blood, then what do you do? There is always a parent, often times two. So, now, we actually have tweaked and developed, there are pioneers, recent pioneers, who have perfected their techniques. I have no hesitation in actually calling up a mom or dad or a brother who’s not even matching and use them. We actually are moving to a field where anybody who needs a transplant, especially a child, would have a donor. So, that is extremely exciting.
Dr. Smith: Yes. That’s really exciting. It must, Dr. Goyal, be very exciting for you personally to be at the forefront of such an exciting field with so much potential. Just for the last question, give us your own personal opinion about where you really see transplantation going into the future. Summarize for us what excites you about the technology.
Dr. Goyal: I think the main, even in my practice time, over last nearly two decades, it’s so gratifying to see that children don’t get as sick when they going to transplantation. That is not always a case. If you have a child with leukemia who has already had a lot of treatment, that’s different. The majority of the time, our focus right now is to make transplant as safe, as non-toxic and to preserve the quality of life for later on so that these transplant survivors, having beaten their underlying terrible diseases can have normal life experiences and can begin to be employed and go to college and do all the good things. So, I see the field of transplantation, in terms of continuing to improve and fine tune and, it’s sort of, I believe it’s going to be personalized and with the discovery in genomic medicine, and how we know everybody’s metabolism and body work, we’ll be able to actually come up with a recipe, no longer one size fits all; no longer one big bang type of an approach.
Dr. Smith: Very exciting stuff. Dr. Goyal, I want to thank you for coming on the show today. Thank you for all the work you are doing. It really is exciting to see what Children’s Mercy is doing and how exciting it is for you to take the reins there. Good luck to you at Children’s Mercy. You’re listening to Transformational Pediatrics with Children’s Mercy Kansas City. For more information, you can go to www.childrensmercy.org. That’s www.childrensmercy.org. I’m Dr. Mike Smith. Thanks for listening.