Thai Doctors Plan to Conduct Thalassemia Gene Therapy Trial

January 6, 2011An article published in the January 3, 2011 edition of the Bangkok Post states that doctors in Thailand hope to conduct a human trial on gene therapy in thalassemia by the end of this year.  


The article states that “a team of doctors at Ramathibodi Hospital is studying the gene therapy technology alongside experts in Paris under a collaboration program between the Mahidol University led by Prof Suthat Fucharoen and French-American researcher Philippe Leboulch of Harvard Medical School and the University of Paris.”


CAF has for many years supported the investigation of gene therapy as a potential curative treatment for thalassemia and has funded a number of gene therapy research projects.  For example, in recent years CAF provided funded for a research project entitled “A Phase I Safety Trial of Lentiviral Mediated B-globin Gene Transfer in Patients with Cooley’s Anemia, an important project headed by Michel Sadelain, MD, PhD, of Memorial Sloan-Kettering Cancer Center.

Currently, CAF is funding a research grant specifically focusing on gene therapy.  Derek Persons, MD, PhD, of St. Jude Children’s Research Hospital received renewal funding of $75,000 for the second year of his gene therapy project, “Translations Research in Adult Thalassemia.”  This project focuses on the role of the vector in gene therapy.  Dr. Persons’ work seeks to develop a breakthrough in therapeutic vector production that will increase the probability of success in a human gene therapy clinical trial.

One of CAF’s current fellows is also conducting research in gene therapy.  Daniel Strongin, PhD, of Fred Hutchinson Cancer Research Center, for the project “A new strategy to ensure robust gene expression from gene therapy vectors by positioning the transgene within the nucleus.”  Put in the simplest terms, this study focuses on the role that a vector (the means by which a corrected gene is introduced into a patient’s cells) plays and how it can be modified to help ensure that the new, corrected gene will reproduce in an appropriate way. 


The Thai doctors expect to return to Thailand to conduct a trial around December, said Dr Suradej Hongeng, of Ramathibodi Hospital’s department of pediatrics.

The collaboration came about after the world’s first successful treatment of beta-thalassemia with gene therapy.  A 21-year-old Frenchman treated with the therapy in 2007 now no longer has the need for blood transfusions. He previously had required transfusions every month since birth.  (Click here to read an earlier article about this important trial.)

“This success justifies the hopes placed in the use of gene therapy to treat blood diseases,” Dr Suradej, a haematology specialist, says in the article.  “It is also the first time an effective technology has been developed to improve the quality of life for people with thalassemia.”

According to the Bangkok Post, an estimated 20 million Thais are carriers of thalassemia. About three in 800 children born in Thailand are affected by the most severe form of the disorder, beta-thalassemia.


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