Swedish Center for Blood Disorders and Stem Cell Transplantation
The Swedish Cancer Institute focuses on treating all blood-related diseases at the Center for Blood Disorders and Stem Cell Transplantation.
Our team of renowned specialists offers cutting-edge therapies, including novel immunotherapies and stem cell transplantation, while providing access to a wide range of clinical trials. The center is active in the institute’s personalized medicine program, using gene sequencing of the patient’s unique tumor to understand which treatment would be best for each individual patient.
Leukemia (acute and chronic)
Lymphoma (Hodgkin and non-Hodgkin)
Benign hematologic conditions
Stem Cell Transplant
Stem cells are immature cells found in the bone marrow that “grow up” to be blood cells. There are three types of blood cells: white blood cells, which fight infection; red blood cells, which carry oxygen to and remove waste products from organs and tissues; and platelets, which enable the blood to clot. In a stem-cell transplant, cells are taken from a patient’s bone marrow or peripheral blood, cleansed of any cancer cells and frozen until they are ready to be used.
Stem Cell Pre-Collection and Mobilization
The first step in the stem cell transplant process is referred to as pre-collection. During this time, patients undergo mobilization. In this video, Dr. Pagel, medical oncologist, answers some frequently asked questions about the pre-collection process.
Stem Cell Collection
After mobilization, a patient’s care team will decide when a patient is ready to begin the next step in the stem cell transplant process, known as collection. In this video, Dr. Pagel, medical oncologist, answers some frequently asked questions about the collection process.
Stem Cell Transplant
The final step of the stem cell transplant is the transplant itself. Stem cells that were previously mobilized and then collected will be returned to the patient’s body. In this video, Dr. Pagel, medical oncologist, answers some frequently asked questions about the transplant process.
Medical oncologists are physicians who specialize in treating cancer with a variety of cancer-fighting medications. Our medical oncologists meet with patients and their families to determine an individualized treatment plan working with other cancer specialists and oncology nurses.
If your doctor believes you are a good candidate to participate in a clinical trial evaluating a new treatment or more effective combinations of treatments — and you agree — you will have access to the very latest in research treatments.
What Cancer Types Can Be Treated With a Stem Cell Transplant?
For a few types of cancer including leukemia, lymphoma – Hodgkins and non-Hodgkins and multiple myeloma, stem-cell transplantation may be effective.
Risks & Side Effects
Patients who undergo a stem-cell procedure may experience short-term side effects such as nausea, vomiting, fatigue, loss of appetite, mouth sores, hair loss, and skin reactions. Additionally, these patients are at high risk of infection during the seven to ten days that their blood counts are low.
Coordination of Insurance Benefits
Stem-cell transplant procedures are very expensive. Many health insurance companies cover some of the costs of transplantation for certain types of cancer. Insurers may also cover a portion of the costs if special care is required when the patient returns home. Federal government programs and local service organizations may also be able to help.
Editing the genome without breaking it.
Universal Cells use recombinant adeno-associated virus (rAAV)-mediated gene editing to efficiently edit chromosomal genes without the use of genotoxic nucleases. rAAV vectors are effective and safe and have been used in numerous clinical trials. We have licensed a stem cell-tropic rAAV vector serotype for engineering human pluripotent stem cells. Our technology allows us to produce customized stem cells that contain deletions, insertions, or point mutations at any genomic position.
Unlike nuclease-based genome editing, our approach is not genotoxic. It does not require a double-strand break, generate off-target alterations to the genome, or produce unwanted mutations at the target site. It also does not introduce nuclease genes into the cell that may have unintended effects.
A major advantage of universal donor cells is that a single cell line can be differentiated into a therapeutic cell type, tested for regulatory approval, and used in multiple recipients, creating an off-the-shelf product. We can provide our engineered stem cells, or our gene-editing technology can be applied to a stem cell line of choice to create customized donor cells.
Universal Cells addresses the allogeneic rejection problem by manipulating human leukocyte antigen (HLA) expression in human stem cells. Multiple HLA class I and class II proteins must be matched for histocompatibility in allogeneic recipients. We eliminate the expression of these polymorphic HLA proteins by gene editing, express specific non-polymorphic HLA molecules in order to provide essential class I signals that block lysis by Natural Killer cells, and introduce suicide genes for enhanced safety.
Significant evidence supports the concept that these HLA-engineered cells will function normally and avoid allogeneic rejection after transplantation.
1) Rare individuals who are HLA class I-negative or HLA class II-negative are relatively healthy, demonstrating that HLA-negative human cells can form all essential organs.
2) Mice lacking major histocompatibility complex (MHC) class I and class II antigens (the murine equivalent of HLA) have been extensively studied and are healthy except for a lack of CD4+ and CD8+ T cells. Importantly, transplantation experiments have shown that organs or cells from class I-negative mouse donors survive longer in allogeneic recipients (sometimes persisting indefinitely), including liver cells, kidneys, hearts, and pancreatic islets. These mouse experiments suggest that HLA-engineered human cells will also survive longer than allogeneic cells in many of the clinical settings being considered for pluripotent stem cells.
HLA class I engineering
HLA-A, B and C are polymorphic class I proteins expressed by most nucleated cells that must be matched for histocompatibility. The Beta-2 Microglobulin (B2M) gene encodes a common subunit essential for cell surface expression of all HLA class I heterodimers (the other subunits are the heavy chains for HLA-A, B, C, E, F, or G), so B2M-/- cells are class I-deficient. We edit both B2M genes to create human pluripotent cells that lack polymorphic class I proteins. These editing steps also introduce a Thymidine Kinase (TK) suicide gene to allow for in vivo elimination of transplanted cells.
For some cell types, a lack of class I expression leads to lysis by Natural Killer (NK) cells. To overcome this “missing self” response, we simultaneously knock in the heavy chain of the non-polymorphic HLA-E gene using HLA single-chain technology (see below). This provides a class I-positive signal to inhibit NK cells. These class I-engineered stem cells can serve as universal donor cells in applications where the differentiated cell product does not express HLA class II.
HLA class II engineering
Unlike HLA class I, class II proteins lack a common subunit that can be edited to prevent surface expression. Therefore our approach is to edit both copies of the RFXANK transcription factor gene required for class II expression. Patients with RFXANK mutations do not express HLA class II molecules on their antigen-presenting cells. Combining class I and class II engineering create a universal donor stem cell line appropriate for deriving any differentiated cell product.
If desired, specific HLA class II alpha and beta chain transgenes can be expressed in RFXANK-/-cells from a RFXANK-independent promoter.