The Fourth Conference on Stem Cell Gene Therapy was held in Athos Hotel Halkidiki Greece and was sponsored by the Hellenic Society of Hematology. The Conference was attended by all the international leaders in the field of stem cell gene therapy who presented the more recent advances in the field .16 speakers were from US Institutions, 22 from European Institutions ,3 from Greece , one from Canada and two from Japan.
Stem Cell Gene Therapy represents the most advanced field of gene therapy. Professor Aiuti ( Milano ) Dr Candotti ( NIH, USA ) and Prof Ariga ( Japan ) presented in the conference new data on the cure of patients with severe immunodeficiency due to complete deficiency of the enzyme Adenosine Deaminase. These patients were destined to die because of their severe defect in their immune system but they now live a normal life after transfer of the normal adenosine deaminase gene in their hemopoietic stem cells. Considerable progress has been achieved in the treatment of the X-linked severe combined immunodeficiency ( X-SCID ) . Initial studies summarized by Prof Cavazzana-Calvo ( Paris ) have shown that this lethal disease can be cured by stem cell gene therapy but patients develop keukemia due to integration of the therapeutic genes near genes that can cause cancer ( oncogenes ). In the conference long term cures of such patients without the appearance of leukemia were reported by Prof Thrasher ( London ) following changes in the therapeutic protocols. Two sessions of the conference were devoted in the current status of the development of gene therapy for thalassemia. New developments in therapeutic approaches and novel vectors were presented by Drs Sadelain ( New York ), Ferrari ( Milano ) , Bodine ( NIH ), Malik ( Cincinnatti ), and Persons ( Memphis ) and plans for upcom ing clinical trials were outlined. The problems of bone marrow conditioning of patients receiving stem cell gene therapy was addressed by Drs Fassas ( Thessaloniki ) anf Roncarolo ( Milano ). The initial results of stem cell mobilization trial in thalassemia in progress in Thessaloniki Greece were presented by Dr Yannaki ( Thessaloniki ). New data were also presented in the development og stem cell gene therapy in Chronic Granulomatous Disease, Wiscott-Aldrich Syndrom,Fanconi Anemia,AIDS, GVH,and for bone marrow chemoprotection.
In addition to the emphasis to the clinical application of stem cell gene therapy the Conference gave considerable emphasis on the molecular and cell biology of stem cell gene transfer. New revolutionary approaches were presented by prof Naldini ( Milano ) . The biology of vector integration into the genome was dealt with by several speakers ( von Kalle, Heidelberg;Baum,Hannover;Dunbar NIH,USA; Mavillio,Milano;Emery,Seattle USA ; Montini,Milano ). A session was devoted on the biology of stem cells as it relates to stem cell gene therapy with speakers Gerson ( Cleveland),Karlsson ( Lund ) , Papayannopoulou ( Seattle , USA) ,Bonnet ( London ) and von Laer ( Frankfdurt ).Stem cell gene therapy is currently based on addition of therapeutic genes in the patients stem cell but it s expected that technological developments will eventually allow the in vivo correction of abnormal genes by gene targetting. Novel approaches on gene targeting were presented by Profs Russell and Lieber ( Seattle ,USA) . Another major issue of stem cell gene therapy is the in vivo expansion of the genetically modified stem cells. Very encouraging results on stem cell expansion in primates were presented by Prof Kiem ( Seattle ).
In general the conference revealed the very satisfactory progress of this field and it made clear that new clinical applications and cures of diseases are expected in the near future.