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Seminar luncheon

Stem cell gene therapy to cure diseases: overview, a success story and current advances


Speakers: Maria Grazia Roncarolo, MD
Organizers: Yalia Jayalakshmi
Date: 2017-01-27
Time: 11:00-13:30 Pacific Time
Registration fee: (USD): Food: $0.
Location: Crowne Plaza Hotel, Foster City
Major Sponsor:
Vendor show vendors registered to date: (4)DyChrom; JL Powers & Co; Waters Corp.; XenoTech
Registration: http://www.PBSS.org
Registration deadline:2017-01-25  (it will close sooner if the seating cap is reached)

About the Topic

Millions of people worldwide have serious genetic and acquired diseases for which there is no cure.Yet exciting advances in stem cell and gene therapy are creating opportunities to not only treat, but actually cure these diseases.The rate of scientific discovery has accelerated at such a pace that outcomes considered science fiction just ten years ago are now being seriously considered and pursued by experts around the globe. Genetic diseases of the blood, the immune system and the nervous system are now cured by inserting a normal copy of the relevant gene into somatic cells.   

Primary immunodeficiencies (the bubble boys), were originally considered good candidates for gene therapy because they were well understood at the molecular level and because the target cells, the blood stem cells, are easily accessible and can be explanted, genetically corrected in the laboratory, and then retransplanted. Gene therapy with blood stem cells has been successfully developed as therapeutic strategy for several forms of primary immunodeficiency, including severe combined immunodeficiency (SCID)-X1, adenosine deaminase (ADA)-deficient SCID, and chronic granulomatous disease (CGD). Initial approaches were based on ex vivo retroviral vector mediated gene transfer of the therapeutic gene into the patient’s blood stem cells. The inclusion of reduced-dose conditioning in the ADA-SCID has allowed the engraftment of multipotent gene-corrected HSC at substantial level. Results of the ADA-SCID gene therapy trials have demonstrated safety, long-term restoration of immune competence and clinical benefit. Based on the data in the first 18 patients the product has been licensed to GSK that recently received European marketing authorization for Strimvelis.

The use of self-inactivating lentiviral vectors is providing significant advantages in gene regulation and reduction in the potential for adverse mutagenic events. Lentiviral vectors are currently used in clinical trials to cure SCID, Wiskott Aldrich syndrome and other primary immunodeficiencies but also blood disorders, such as thalassemia and sickle cell anemia, and metabolic diseases.

The prospects for continuing advancement of gene therapy to wider applications remain strong. Emerging methods for direct gene replacement by genome editing will broaden the use of gene therapy to other genetic and acquired diseases. In summary, in the near future the continuing advancement of stem cell and gene therapy will make it possible “to cure what is curable and make curable what is not”.


About the Speakers

Maria Grazia Roncarolo, MD
George D. Smith Professor of Pediatrics and Medicine
Division Chief, Pediatric Stem Cell Transplantation and Regenerative Medicine
Co-Director, Bass Center for Childhood Cancer and Blood Diseases
Co-Director, Institute for Stem Cell Biology and Regenerative Medicine
Department of Pediatrics, Stanford School of Medicine


Dr. Roncarolo has made outstanding contributions to translational research in the fields of immunology and gene therapy. Her translational research studies have led to greater understanding of the mechanisms underlying immune tolerance and have been of paramount importance to the development of novel therapies for patients with genetic and acquired diseases of the hematopoietic and immune systems.

She contributed to the elucidation of the mechanisms underlying the induction and breaking of tolerance in stem cell transplantation/organ grafts/autoimmune diseases and gene therapy. Specifically, she discovered a new subset of human T regulatory cells with immune regulatory and suppressor functions, named Type 1 (Tr1) cells. She established that these cells were present in tolerant patients following hematopoietic stem cell transplantation. Subsequently, she isolated these cells from both mice and man, demonstrating that they are responsible for induction and maintenance of tolerance to allo- and self-antigens and food- and environmental- antigens. Recently, she discovered that the surface molecules CD49b and LAG3 are specific biomarkers for this subset of T regulatory cells, which allows for their isolation for therapeutic purposes and in vivo tracking in patients. She was the principal investigator of the first clinical trial using these ex-vivo generated donor-derived Tr1 cells to prevent the occurrence of severe graft-versus-host disease in leukemia patients undergoing haploidentical hematopoietic stem cell transplantation.

Dr. Roncarolo also discovered that Rapamycin favors expansion of another subset of human T regulatory cells, CD25+FOXP3+ Treg cells, allowing for their therapeutic use. She further demonstrated that treatment with Rapamycin and IL-10 induces tolerance in vivo by promoting differentiation of Tr1 cells and expansion of CD25+FOXP3+ T regulatory cells. She showed that specific Ag-targeting to the hepatocytes results in immunological tolerance mediated by CD25+FOXP3+ T regulatory cells.

Parallel to her studies on immunological tolerance, Dr. Roncarolo has investigated the pathological mechanisms responsible for genetic diseases of the hematopoietic and immune system in order to design therapies that will ultimately cure afflicted patients.

Dr. Roncarolo was the principal investigator for the first lentiviral-vector based gene therapy trial for patients with Wiskott-Aldrich syndrome, which demonstrated safety and efficacy of this therapy. In addition, she was the principal investigator for the first successful gene therapy trial for Severe Combined Immunodeficiency (SCID) patients lacking adenosine deaminase (ADA), a purine metabolism disorder that results in severe immunodeficiency and death. In this trial she introduced a new therapeutic conditioning regimen for the host, which favored the outgrowth of the gene corrected cells, resulting in the most successful clinical outcome for this formerly non-treatable genetic disease. Based on these results, gene therapy for ADA-SCID has obtained Orphan drug status from both the FDA and EMEA and recently has received European Commission approval to market under the name of Strimvelis. This is the first stem cell gene therapy product, which received market authorization.


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