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In this episode, Dr. Richard Burt shares his insights on hematopoietic stem cell transplant (HSCT) for autoimmune disorders.
For thirty-five years, Dr. Burt dedicated himself to advancing stem cell and cellular therapy, beginning with animal models and, later, some of the world's first clinical trials. He authored over 145 articles, mostly as the first author, and edited four medical textbooks. As the first Autoimmune Committee Chairperson for the International Bone Marrow Transplant Registry (IBMTR), he led the way in developing stem cell clinical trials for autoimmune diseases, securing a National Institute of Health (NIH) $10,000,000 multi-center contract.
Dr. Burt performed America's first hematopoietic stem cell transplant (HSCT) for multiple sclerosis (MS), systemic lupus erythematosus (SLE), Crohn's disease (CD), stiff person syndrome (SPS), and chronic inflammatory demyelinating polyneuropathy (CIDP). He also published the world's first randomized clinical stem cell transplantation trials for systemic sclerosis and multiple sclerosis.
Dr. Burt's pioneering stem cell work earned him recognition as one of the top 50 people in the world for improving humanity by Scientific American in 2006, along with A1 Gore and Steve Jobs. In 2011, Science Illustrated recognized his stem cell work as one of the top 10 advances of the decade.
Key Takeaways:
What is a hematopoietic stem cell transplant?
Hematopoietic stem cell transplantation (HSCT) is a medical procedure where multipotent stem cells are transplanted into a patient, typically from bone marrow, peripheral blood, or umbilical cord blood. These cells can replicate and produce new, healthy blood cells, which can rejuvenate or repair damaged tissue in different organs, and also help fight viruses and foreign tissue.
While leukemia is caused by defects in the blood stem cells themselves, autoimmune diseases arise from defects in the immune cells produced by these stem cells. To treat autoimmune diseases, specialists target the effector cells responsible for the disease and eliminate them, then quickly regenerate healthy cells from the blood stem cell.
By using HSCT to replace damaged or defective cells with healthy ones, physicians hope to mitigate the symptoms of autoimmune disorders and promote long-term recovery. This innovative approach to treatment is still undergoing testing and refinement, but it shows great promise in the fight against these debilitating diseases.
Finding the optimal transplant regimen for autoimmune diseases
If your regimen is too strong, whether it's myeloablative or non-myeloablative, you can have more toxicity and problems. If it's too weak, it's not going to be effective. And so you've got to find that right regimen. When finding the optimal transplant regimen for autoimmune diseases, the aim is to develop a treatment plan that maximizes the benefits of the transplant while minimizing the risks and complications associated with it.
An optimal transplant regimen for autoimmune diseases involves selecting the appropriate donor, considering the type of transplant, choosing the right conditioning regimen, and determining the timing and dosing of immunosuppressive medications.
Financial toxicity in American healthcare systems
Financial toxicity is a growing concern in America, particularly in medical treatments. Unfortunately, this often leads to patients being prescribed expensive treatments that may not be the best option for their specific needs. To achieve successful outcomes with hematopoietic stem cell transplantation, two key factors need to be considered: patient selection and the conditioning regimen used.
However, due to the financial pressures facing healthcare providers and institutions, specialists may feel obligated to prescribe expensive treatments, even if they are not necessarily the most appropriate option for the patient. This can be a major source of frustration for patients, who may struggle to afford these treatments or may not receive the care that they truly need.
Dr. Burt emphasizes the importance of physicians taking responsibility for their patient's care and being mindful of the financial implications of their recommendations. Rather than being driven solely by financial considerations, doctors should focus on providing the best possible care for their patients. By doing so, they can help to mitigate the effects of financial toxicity and ensure that patients receive the treatments that are best suited to their individual needs.
Learn more about Dr. Richard Burt
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