After receiving both his medical degree and doctorate in molecular physiology from the University of Virginia, Timothy S. Olson, MD, PhD, completed his internship, fellowship and residency at Children’s Hospital of Philadelphia (CHOP). Dr. Olson is now director of CHOP’s Blood and Marrow Transplant Program, which is set to celebrate 50 years of providing blood or marrow transplants to the children who need them.
Recently, the program passed significant milestones: 2,500 unique patients for transplant and 700 unique patients for CAR T-cell therapy. In this Q&A, Dr. Olson discusses the program’s half century of care, long-term clinical and research goals, as well as the upcoming FDA approval of yet another groundbreaking gene therapy developed, in part, at CHOP.
Question: As director of CHOP’s Blood and Marrow Transplant Program, what does it mean to you to lead a program celebrating 50 years of providing lifesaving care to children and families?
It's a privilege for me to be able to lead this program. It has evolved tremendously over the course of the last 50 years. When programs like these were started back in the 1970s, there was a lot of skepticism surrounding whether or not bone marrow or stem cell transplantation was really a curative therapy.
In fact, the pioneers of this approach were highly criticized for putting patients and potential donors at a lot of risk for unproven therapy. And so, transplant has gone from a therapy that was really at the fringe of pediatric oncology specifically, to a therapy that has so much to offer, not only for patients with high-risk cancers, but patients with a variety of blood, immune and metabolic disorders.
Q: The program recently surpassed 2,500 unique transplant patients and 700 unique CAR T-cell therapy patients. What do these milestones say about the evolution of pediatric cellular therapies at CHOP over the past five decades?
It's just tremendous to watch how it's evolved over the last couple of decades. When the program started here in the late 70s and the mid 80s, it was really a narrowly targeted therapy for patients who had high-risk leukemia, a few patients with a disease called aplastic anemia, and a few patients who had severe combined immunodeficiency. We treated only a handful of patients over that first decade or so.
The modern incarnation of our program was started and built by my predecessor and mentor, Nancy Bunin, MD, in 1988. At that time, we really only had the ability to do transplants for patients who happened to have a fully matched sibling donor, which is only about 20% of the population. Thus, a patient’s ability to access one of these therapies was dependent upon having a donor.
In the 1980s, the federal government supported the creation of the National Marrow Donor Program, which since then allowed us to access unrelated donors that have joined this national registry and that were well-matched to patients. That expanded the donor pool even further. In the 1990s, cord blood transplants evolved and so we could use transplants using unrelated cord blood stored in donor banks across the country.
Starting in the early 2000’s, one of the huge breakthroughs for which CHOP has been an international leader, thanks to the work of Dr. Bunin, is the evolution of techniques that make it safe to do transplants for patients who don't have a matched donor. We have learned how to manipulate the immune cells present in the infused donor stem cell transplant through a process we call graft engineering in which we specifically take out the cells that can cause a devastating hyperinflammation condition that's known as graft versus host disease. With the ongoing enhancements we are testing in clinical trials, now in 2026 we can identify a donor in 99% of patients.
We've gone from being able to provide curative therapies for only 20% of patients who had select diseases to now offering curative therapies across 60 to 70 different disease indications.
The other major breakthrough in the past decade is that we can provide gene therapy for patients with select genetic disorders, such as sickle cell disease, a process during which we gene correct their own stem cells, and engraft these cells in the setting of a gene modified autologous transplant. These technologies that we are now harnessing, I don't think that anybody back in the 1970s and 1980s would have predicted would be possible.
Q: You completed your internship, residency and fellowship at CHOP, then spent a decade as a CHOP faculty member before becoming Program leader. How has your personal journey shaped your vision for the future of blood and marrow transplantation at the institution?
I have a background in research, having completed an MD-PhD program when I came to CHOP for residency. During my PhD research, I had done work looking at how white blood cells traffic throughout the body both to maintain health and in the process of inflammatory disease development. When I came to CHOP, I was contemplating a couple of different subspecialty fields in which to pursue fellowship. During my intern year as a resident here at CHOP, I did a rotation on the bone marrow transplant service, and I was immediately hooked.
I thought the medicine was fascinating and the ability to provide truly curative therapies for patients across a wide range of conditions convinced me this was really the direction I wanted my career to go in. From the beginning of my fellowship and through the establishment of my faculty career, I have personally focused on the non-oncologic indications of bone marrow transplant. My work has included developing curative therapies for patients with diseases that are called bone marrow failure disorders and those who have hemoglobin disorders, such as sickle cell disease and thalassemia.
Q: How have advances in transplantation, gene therapy and CAR T-cell therapy changed outcomes for children with cancer, immune disorders and other serious diseases, and where do you see the greatest opportunities for progress in the next decade?
Over the nearly two decades that I have been part of our transplant program, we have gone from performing 70 to 80 transplants per year mostly for children with hematologic malignancies to performing nearly 150 transplants this past year, with half of our patients having non-malignant diseases. Gene therapy is a major part of the expansion of our program, and we are currently projecting to perform 30 to 40 gene therapy transplants per year for diseases including sickle cell disease, thalassemia and leukodystrophies.
CAR T-cell therapy has been paradigm-shifting for patients who have high-risk leukemia, particularly a type of leukemia known as B-cell acute lymphoblastic leukemia, which is the most common leukemia in children. For patients who relapsed after standard chemotherapy or maybe after standard transplants for that form of leukemia, there used to be no options to continue to pursue a cure.
Stephan A. Grupp, MD, PhD, and the Cellular Therapy and Transplant Section (CTTS) team have led, nationally and globally, engineered T-cell therapy, where the T-cells bind to, attack and destroy that refractory leukemia. It's given life to hundreds of patients who in previous eras would not have had a path to curative transplant and would not have been able to survive their leukemia.
It's been very rewarding to see the evolution in technology and how many patients we're able to provide curative therapies for now.
Q: How do you see treatments like CASGEVY™ influencing the future of care for patients with sickle cell disease and other genetic disorders at CHOP?
CHOP has now been a part of the research, in one form or another, for all the stem cell-based gene therapies that are approved and in active use in the United States. Dr. Grupp played a leading role in the clinical trials that led to the approval of CASGEVY™. Other collaborators and members of our program, including Dr. Janet Kwiatkowski, MD, MSCE, have played a critical role in the development of other products as well, like ZYNTEGLO™ for beta thalassemia. We are partnering with companies for gene therapy in rare neurologic disorders called leukodystrophies, and we've playing active roles in the post-approval research on those gene therapies, which are called LENMELDY™ and Skysona®. In the immediate future, there are several additional gene therapies that are coming down the pike that we're playing an active role in as well.
The complexity of the different products that we offer to patients is staggering. Bone marrow transplant started as a field where you collected bone marrow directly from the backs of the hips of a donor, and you transplanted it right away into the patient. Now what we do is so different. The donor product, whether it's the patient's own stem cells or it's a different donor, might undergo gene modification; it might undergo expansion. As another example, we now have expanded cord blood products that can increase the number of stem cells in cord blood units, which has always been the limitation of cord blood transplant. We conduct highly sophisticated graft engineering of our traditional allogeneic transplant products, taking stem cells from a different donor and giving them to a patient. We can now perform two to three sets of graft manipulations on those cells to get rid of all the cells that are going to cause toxicity but bring back some immunologic memory to the graft to help prevent infections after transplant.
The complexity of graft engineering and the stem cell products we're giving is incredible; I doubt the folks who founded this field 30, 40 years ago would have ever imagined we'd have that at our fingertips.
Q: What are your top clinical priorities for the Blood and Marrow Transplant Program over the next five to 10 years?
I hope that our current therapies get better.
I hope that there comes a day where nobody relapses after a bone marrow transplant or CAR T-cell therapy for malignancy. And I hope we can greatly reduce both the short- and long-term side effects of going through our therapies. I hope we continue to discover novel approaches to curing patients. I hope we come up with more novel gene therapies and more novel uses of allogeneic transplant to help children with diseases for which stem cell transplant isn't currently offered.
In the CAR T-cell space, there are a lot of exciting efforts focused on providing immune resets for patients with severe immunologic diseases such as lupus and juvenile arthritis, and many others. I hope that use of those CAR T-cell therapies and novel traditional transplant strategies continue to expand for patients who have immune dysregulation.
Q: What research areas are you most excited to advance through CHOP’s Blood and Marrow Transplant Program?
I think the next frontier is figuring out a way to be able to do transplants that no longer require traditional chemotherapy, allowing patients to avoid the short- and long-term side effects of chemotherapy and radiation.
There are a number of researchers around the country who are working on what we call non-genotoxic conditioning regimens, where with antibodies, small molecules or antibodies conjugated to targeted payloads, we can very selectively eliminate the stem cells of the bone marrow and prevent the immune system of the patient from rejecting the donor's cells thereby allowing us to stably maintain the donor graft and correct the underlying disease.
I could envision a world 10 to 15 years from now where, at least for the non-cancer conditions for which we perform transplants, we may be able to provide these stem cell therapies with no chemotherapy and no radiation at all.
Q: Looking ahead, what are the biggest challenges facing pediatric transplant and cellular therapy programs, and how is CHOP positioned to address them?
There are certainly challenges. Programmatically, we want to expand our reach and our catchment to bring families in from other states with rare diseases, particularly those diseases where we at CHOP have specialized expertise that might not exist in many other hospitals around the country.
One of the critical elements for some of the rare genetic disorders of early childhood is oftentimes the disorder has been discovered too late to be able to help. Now, through national initiatives focused on expanding newborn screening, more of these patients are being identified to have the disease in early infancy. We now want to make sure that early detection is coupled with the ability to provide a cure. So, figuring out ways that we can expand our outreach so that regardless of where you are in this country, if you need curative therapy and we have the specialized expertise to provide it, we will be able to help your child.
We need to have the expertise and personnel to be able to engage across state lines to make sure that we're able to help those patients. A major component of this outreach starts with understanding that these are very high-cost therapies. Many members of our center have been quite active advocating at the federal level to try to break down barriers to be able to get patients to be seen at centers that have the expertise to treat their disorders. We're going to have to continue those advocacy efforts as we continue moving forward.
Q: When the program celebrates its 60th anniversary a decade from now, what accomplishments or breakthroughs would you hope to define its next chapter?
I wouldn't have thought, even just a few years ago, that we would be treating patients with five different gene therapies that are commercially approved, as well as be in the process of onboarding a handful of additional gene therapies that are either close to or just recently approved as well. So, I think the future is very bright, but we have a lot of work to do. There are so many survivors from traditional transplants and from CAR T-cell therapy, but there are diagnoses where our curative therapies still aren't curing enough patients.
We need to figure out how to improve CAR T-cell therapies for other types of leukemia that are not B-cell ALL, for solid tumors and for brain tumors. We need to figure out how to improve the efficacy of traditional transplants for leukemia. We've gotten much better at reducing the toxicity of transplants, but we must figure out ways where we can reduce that toxicity and continue to maximally prevent recurrence of leukemia.
CHOP just supported a brand-new program built on an integrated multi-modal precision approach to cellular therapy for immune regulatory disorders, led by two outstanding junior investigators, Susan E. McClory, MD, PhD, and Caitlin Elgarten, MD, MSCE. The goal of that program is to reach more of these pediatric patients who are followed in rheumatology, gastroenterology and immunology for disorders of immune regulation, and be able to enhance and personalize approaches to curative cell therapies for those patients. I can't wait to see what they accomplish with that support from CHOP over the next 5 to 10 years.
Q: For a family trying to decide where to get these complicated therapies, what would you want them to know about choosing treatment at CHOP?
I think families would be hard-pressed to find a curative therapies team more dedicated to coming up with an individualized set of recommendations and a therapy plan that would help their child regardless of what their underlying disease is. We have outstanding professionals and people throughout our program. Not only are our physician faculty outstanding, but we have the most experienced nurse navigators and advanced practice providers, including nurse practitioners and physician assistants, that help us provide state-of-the-art care. We have outstanding coordinators. We have outstanding social workers, psychologists and child-life specialists who understand the complexities of what it's like for a family to go through one of these therapies, whether the family is from Philadelphia or somewhere else across the world. We understand what the needs are, and we're prepared to help address those needs to be able to make it feasible to come to us for treatment.
We have so many wonderful people who are fully invested in treating your family and treating your child. So, it would be an honor and privilege for us to provide therapy.
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After receiving both his medical degree and doctorate in molecular physiology from the University of Virginia, Timothy S. Olson, MD, PhD, completed his internship, fellowship and residency at Children’s Hospital of Philadelphia (CHOP). Dr. Olson is now director of CHOP’s Blood and Marrow Transplant Program, which is set to celebrate 50 years of providing blood or marrow transplants to the children who need them.
Recently, the program passed significant milestones: 2,500 unique patients for transplant and 700 unique patients for CAR T-cell therapy. In this Q&A, Dr. Olson discusses the program’s half century of care, long-term clinical and research goals, as well as the upcoming FDA approval of yet another groundbreaking gene therapy developed, in part, at CHOP.
Question: As director of CHOP’s Blood and Marrow Transplant Program, what does it mean to you to lead a program celebrating 50 years of providing lifesaving care to children and families?
It's a privilege for me to be able to lead this program. It has evolved tremendously over the course of the last 50 years. When programs like these were started back in the 1970s, there was a lot of skepticism surrounding whether or not bone marrow or stem cell transplantation was really a curative therapy.
In fact, the pioneers of this approach were highly criticized for putting patients and potential donors at a lot of risk for unproven therapy. And so, transplant has gone from a therapy that was really at the fringe of pediatric oncology specifically, to a therapy that has so much to offer, not only for patients with high-risk cancers, but patients with a variety of blood, immune and metabolic disorders.
Q: The program recently surpassed 2,500 unique transplant patients and 700 unique CAR T-cell therapy patients. What do these milestones say about the evolution of pediatric cellular therapies at CHOP over the past five decades?
It's just tremendous to watch how it's evolved over the last couple of decades. When the program started here in the late 70s and the mid 80s, it was really a narrowly targeted therapy for patients who had high-risk leukemia, a few patients with a disease called aplastic anemia, and a few patients who had severe combined immunodeficiency. We treated only a handful of patients over that first decade or so.
The modern incarnation of our program was started and built by my predecessor and mentor, Nancy Bunin, MD, in 1988. At that time, we really only had the ability to do transplants for patients who happened to have a fully matched sibling donor, which is only about 20% of the population. Thus, a patient’s ability to access one of these therapies was dependent upon having a donor.
In the 1980s, the federal government supported the creation of the National Marrow Donor Program, which since then allowed us to access unrelated donors that have joined this national registry and that were well-matched to patients. That expanded the donor pool even further. In the 1990s, cord blood transplants evolved and so we could use transplants using unrelated cord blood stored in donor banks across the country.
Starting in the early 2000’s, one of the huge breakthroughs for which CHOP has been an international leader, thanks to the work of Dr. Bunin, is the evolution of techniques that make it safe to do transplants for patients who don't have a matched donor. We have learned how to manipulate the immune cells present in the infused donor stem cell transplant through a process we call graft engineering in which we specifically take out the cells that can cause a devastating hyperinflammation condition that's known as graft versus host disease. With the ongoing enhancements we are testing in clinical trials, now in 2026 we can identify a donor in 99% of patients.
We've gone from being able to provide curative therapies for only 20% of patients who had select diseases to now offering curative therapies across 60 to 70 different disease indications.
The other major breakthrough in the past decade is that we can provide gene therapy for patients with select genetic disorders, such as sickle cell disease, a process during which we gene correct their own stem cells, and engraft these cells in the setting of a gene modified autologous transplant. These technologies that we are now harnessing, I don't think that anybody back in the 1970s and 1980s would have predicted would be possible.
Q: You completed your internship, residency and fellowship at CHOP, then spent a decade as a CHOP faculty member before becoming Program leader. How has your personal journey shaped your vision for the future of blood and marrow transplantation at the institution?
I have a background in research, having completed an MD-PhD program when I came to CHOP for residency. During my PhD research, I had done work looking at how white blood cells traffic throughout the body both to maintain health and in the process of inflammatory disease development. When I came to CHOP, I was contemplating a couple of different subspecialty fields in which to pursue fellowship. During my intern year as a resident here at CHOP, I did a rotation on the bone marrow transplant service, and I was immediately hooked.
I thought the medicine was fascinating and the ability to provide truly curative therapies for patients across a wide range of conditions convinced me this was really the direction I wanted my career to go in. From the beginning of my fellowship and through the establishment of my faculty career, I have personally focused on the non-oncologic indications of bone marrow transplant. My work has included developing curative therapies for patients with diseases that are called bone marrow failure disorders and those who have hemoglobin disorders, such as sickle cell disease and thalassemia.
Q: How have advances in transplantation, gene therapy and CAR T-cell therapy changed outcomes for children with cancer, immune disorders and other serious diseases, and where do you see the greatest opportunities for progress in the next decade?
Over the nearly two decades that I have been part of our transplant program, we have gone from performing 70 to 80 transplants per year mostly for children with hematologic malignancies to performing nearly 150 transplants this past year, with half of our patients having non-malignant diseases. Gene therapy is a major part of the expansion of our program, and we are currently projecting to perform 30 to 40 gene therapy transplants per year for diseases including sickle cell disease, thalassemia and leukodystrophies.
CAR T-cell therapy has been paradigm-shifting for patients who have high-risk leukemia, particularly a type of leukemia known as B-cell acute lymphoblastic leukemia, which is the most common leukemia in children. For patients who relapsed after standard chemotherapy or maybe after standard transplants for that form of leukemia, there used to be no options to continue to pursue a cure.
Stephan A. Grupp, MD, PhD, and the Cellular Therapy and Transplant Section (CTTS) team have led, nationally and globally, engineered T-cell therapy, where the T-cells bind to, attack and destroy that refractory leukemia. It's given life to hundreds of patients who in previous eras would not have had a path to curative transplant and would not have been able to survive their leukemia.
It's been very rewarding to see the evolution in technology and how many patients we're able to provide curative therapies for now.
Q: How do you see treatments like CASGEVY™ influencing the future of care for patients with sickle cell disease and other genetic disorders at CHOP?
CHOP has now been a part of the research, in one form or another, for all the stem cell-based gene therapies that are approved and in active use in the United States. Dr. Grupp played a leading role in the clinical trials that led to the approval of CASGEVY™. Other collaborators and members of our program, including Dr. Janet Kwiatkowski, MD, MSCE, have played a critical role in the development of other products as well, like ZYNTEGLO™ for beta thalassemia. We are partnering with companies for gene therapy in rare neurologic disorders called leukodystrophies, and we've playing active roles in the post-approval research on those gene therapies, which are called LENMELDY™ and Skysona®. In the immediate future, there are several additional gene therapies that are coming down the pike that we're playing an active role in as well.
The complexity of the different products that we offer to patients is staggering. Bone marrow transplant started as a field where you collected bone marrow directly from the backs of the hips of a donor, and you transplanted it right away into the patient. Now what we do is so different. The donor product, whether it's the patient's own stem cells or it's a different donor, might undergo gene modification; it might undergo expansion. As another example, we now have expanded cord blood products that can increase the number of stem cells in cord blood units, which has always been the limitation of cord blood transplant. We conduct highly sophisticated graft engineering of our traditional allogeneic transplant products, taking stem cells from a different donor and giving them to a patient. We can now perform two to three sets of graft manipulations on those cells to get rid of all the cells that are going to cause toxicity but bring back some immunologic memory to the graft to help prevent infections after transplant.
The complexity of graft engineering and the stem cell products we're giving is incredible; I doubt the folks who founded this field 30, 40 years ago would have ever imagined we'd have that at our fingertips.
Q: What are your top clinical priorities for the Blood and Marrow Transplant Program over the next five to 10 years?
I hope that our current therapies get better.
I hope that there comes a day where nobody relapses after a bone marrow transplant or CAR T-cell therapy for malignancy. And I hope we can greatly reduce both the short- and long-term side effects of going through our therapies. I hope we continue to discover novel approaches to curing patients. I hope we come up with more novel gene therapies and more novel uses of allogeneic transplant to help children with diseases for which stem cell transplant isn't currently offered.
In the CAR T-cell space, there are a lot of exciting efforts focused on providing immune resets for patients with severe immunologic diseases such as lupus and juvenile arthritis, and many others. I hope that use of those CAR T-cell therapies and novel traditional transplant strategies continue to expand for patients who have immune dysregulation.
Q: What research areas are you most excited to advance through CHOP’s Blood and Marrow Transplant Program?
I think the next frontier is figuring out a way to be able to do transplants that no longer require traditional chemotherapy, allowing patients to avoid the short- and long-term side effects of chemotherapy and radiation.
There are a number of researchers around the country who are working on what we call non-genotoxic conditioning regimens, where with antibodies, small molecules or antibodies conjugated to targeted payloads, we can very selectively eliminate the stem cells of the bone marrow and prevent the immune system of the patient from rejecting the donor's cells thereby allowing us to stably maintain the donor graft and correct the underlying disease.
I could envision a world 10 to 15 years from now where, at least for the non-cancer conditions for which we perform transplants, we may be able to provide these stem cell therapies with no chemotherapy and no radiation at all.
Q: Looking ahead, what are the biggest challenges facing pediatric transplant and cellular therapy programs, and how is CHOP positioned to address them?
There are certainly challenges. Programmatically, we want to expand our reach and our catchment to bring families in from other states with rare diseases, particularly those diseases where we at CHOP have specialized expertise that might not exist in many other hospitals around the country.
One of the critical elements for some of the rare genetic disorders of early childhood is oftentimes the disorder has been discovered too late to be able to help. Now, through national initiatives focused on expanding newborn screening, more of these patients are being identified to have the disease in early infancy. We now want to make sure that early detection is coupled with the ability to provide a cure. So, figuring out ways that we can expand our outreach so that regardless of where you are in this country, if you need curative therapy and we have the specialized expertise to provide it, we will be able to help your child.
We need to have the expertise and personnel to be able to engage across state lines to make sure that we're able to help those patients. A major component of this outreach starts with understanding that these are very high-cost therapies. Many members of our center have been quite active advocating at the federal level to try to break down barriers to be able to get patients to be seen at centers that have the expertise to treat their disorders. We're going to have to continue those advocacy efforts as we continue moving forward.
Q: When the program celebrates its 60th anniversary a decade from now, what accomplishments or breakthroughs would you hope to define its next chapter?
I wouldn't have thought, even just a few years ago, that we would be treating patients with five different gene therapies that are commercially approved, as well as be in the process of onboarding a handful of additional gene therapies that are either close to or just recently approved as well. So, I think the future is very bright, but we have a lot of work to do. There are so many survivors from traditional transplants and from CAR T-cell therapy, but there are diagnoses where our curative therapies still aren't curing enough patients.
We need to figure out how to improve CAR T-cell therapies for other types of leukemia that are not B-cell ALL, for solid tumors and for brain tumors. We need to figure out how to improve the efficacy of traditional transplants for leukemia. We've gotten much better at reducing the toxicity of transplants, but we must figure out ways where we can reduce that toxicity and continue to maximally prevent recurrence of leukemia.
CHOP just supported a brand-new program built on an integrated multi-modal precision approach to cellular therapy for immune regulatory disorders, led by two outstanding junior investigators, Susan E. McClory, MD, PhD, and Caitlin Elgarten, MD, MSCE. The goal of that program is to reach more of these pediatric patients who are followed in rheumatology, gastroenterology and immunology for disorders of immune regulation, and be able to enhance and personalize approaches to curative cell therapies for those patients. I can't wait to see what they accomplish with that support from CHOP over the next 5 to 10 years.
Q: For a family trying to decide where to get these complicated therapies, what would you want them to know about choosing treatment at CHOP?
I think families would be hard-pressed to find a curative therapies team more dedicated to coming up with an individualized set of recommendations and a therapy plan that would help their child regardless of what their underlying disease is. We have outstanding professionals and people throughout our program. Not only are our physician faculty outstanding, but we have the most experienced nurse navigators and advanced practice providers, including nurse practitioners and physician assistants, that help us provide state-of-the-art care. We have outstanding coordinators. We have outstanding social workers, psychologists and child-life specialists who understand the complexities of what it's like for a family to go through one of these therapies, whether the family is from Philadelphia or somewhere else across the world. We understand what the needs are, and we're prepared to help address those needs to be able to make it feasible to come to us for treatment.
We have so many wonderful people who are fully invested in treating your family and treating your child. So, it would be an honor and privilege for us to provide therapy.
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