SCID Fact Sheet

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SCID – also known as ‘bubble boy disease’ - is a rare genetic disorder, effecting one in 30,000 newborns. Left untreated the children die before the age of 2, and the only readily available treatment involves high-risk bone marrow transplants. Because these patients already have a compromised immune system, 10 to 20 percent don’t survive the transplant.

Gene therapy has been used to correct the defect in certain types of SCID, but early gene modifying techniques resulted in some patients developing cancer. Newer gene therapy techniques appear to be safer but have been tried on fewer than 20 patients.

Researchers funded by California’s stem cell agency are looking for a better alternative to help these children. They are trying to improve the safety of bone marrow transplant (BMT), which essentially uses the stem cells in bone marrow to give the children a new immune system that works properly. Most of the risk of current BMT procedures comes from the radiation or chemotherapy given to patients before the transplant to wipe out the patient’s own stem cells that form immune cells. These regimens kill many types of cells beyond those intended and result in numerous toxic side effects.

Disease team award

Stanford School of Medicine

This team proposes to replace SCID patients’ dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). They plan to eliminate the bad cells with an antibody, a protein, that very specifically targets and eliminates blood forming stem cells. If successful, the procedure could open up similar BMT therapies to patients with other auto-immune diseases such as multiple sclerosis, lups or diabetes that are generally not candidates for BMT currently. These diseases, while debilitating, are not immediately life-threatening and generally don’t warrant the risks involved in BMT the way it is done today.

CIRM Grants Targeting Immune Diseases including SCID

Researcher name Institution Grant Title Approved funds
Kenneth Weinberg Stanford University “Engineered immune tolerance by Stem Cell-derived thymic regeneration” $1,271,729
John Zaia Beckman Research Institute of City of Hope ZIinc Finger Nuclease-Based Stem Cell Therapy for AIDS $14,536,969
Tippi MacKenzie University of California, San Francisco Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation $1,230,869
Yang Xu University of California, San Diego Induction of immune tolerance to human embryonic stem cell-derived allografts $1,192,680
Thomas Lane University of California, Irvine Human Embryonic Stem Cells and Remyelination in a Viral Model of Demyelination $368,081
Judith Shizuru Stanford University A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants $90,147
Kenneth Weinberg Stanford University Embryonic stem cell-derived thymic epithelial cells $628,793
Morton Cowan University of California, San Francisco Gene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID $3,931,662
Irving Weissman Stanford University Prospective isolation of hESC-derived hematopoietic and cardiomyocyte stem cells $2,471,386
Judith Shizuru Stanford University A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants $19,068,382
Samuel Pleasure University of California, San Francisco Human stem cell derived oligodendrocytes for treatment of stroke and MS $2,459,235
Donald Kohn University of California, Los Angeles Clinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease $13,145,465
Zack Jerome University of California, Los Angeles Human Embryonic Stem Cell Therapeutic Strategies to Target HIV Disease $2,401,903
Mark Anderson University of California, San Francisco Generation of a functional thymus to induce immune tolerance to stem cell derivatives $1,191,000
Inder Verma The Salk Institute for Biological Studies Curing Hematological Diseases $5,979,252
Mark Anderson University of California, San Francisco Stem cell differentiation to thymic epithelium for inducing tolerance to stem cells $1,314,089
Jeffrey Bluestone University of California, San Francisco Stem cell tolerance through the use of engineered antigen-specific regulatory T cells $1,152,768
Gay Crooks University of California, Los Angeles Engineering Thymic Regeneration to Induce Tolerance $1,235,445
Husein Hadeiba Palo Alto Institute for Research and Education, Inc. Application of Tolerogenic Dendritic Cells for Hematopoietic Stem Cell Transplantation $733,061
Jeanne Loring Scripps Research Institute Thymus based tolerance to stem cell therapies $1,108,921
David DiGiusto Beckman Research Institute of City of Hope Development of RNA-based approaches to stem cell gene therapy for HIV $3,097,160
Anjana Rao La Jolla Institute for Allergy and Immunology Generation of regulatory T cells by reprogramming $1,464,446
Ellen Robey University of California, Berkeley Human Immune System Mouse models as preclinical platforms for stem cell derived grafts $1,005,605
Irvin Chen University of California, Los Angeles HPSC based therapy for HIV disease using RNAi to CCR5. $9,905,604
Judith Shizuru Stanford University Purified allogeneic hematopoietic stem cells as a platform for tolerance induction $1,233,275
Michele Calos Stanford University Safe, efficient creation of human induced pluripotent stem cells without the use of retroviruses $1,406,875
Irvin Chen University of California, Los Angeles Genetic modification of the human genome to resist HIV-1 infection and/or disease progression $616,800

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