Thailand Thalassemia Gene Therapy

Thai Doctors Plan to Conduct Thalassemia Gene Therapy Trial
January 6, 2011 - An 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 AND GENE THERAPY   
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

Bangalore turns to gene therapy
Jayashree Nandi May 11, 2011, 01.20am IST
BANGALORE: Altering genes to cure diseases has moved from sci-fi flicks to a major research investment in India. And Bangalore is also looking at it with hope. The latest in labs here is gene therapy for thalassemia and haemophilia, which affect Indians in large numbers. Doctors and researchers are working to make it a reality.

Though treatment could be a long way off, the gene has been found and so too the vector to introduce it into the body. A team of doctors at the recently launched Mazumdar Shaw Centre for Therapeutics is exploring thalassemia treatment. Gene therapy became popular because of its potential in curing genetic blindness but could be a potential saviour in terminal conditions like cancer.


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"Both haemophilia and thalessemia are single gene defects and congenital diseases which unfortunately have no cure. The only option is blood transfusion for life. The beta globin gene that has been identified for thalassemia can be transferred to the body through gene alteration. But when this transfer is being done, the gene doesn't settle permanently but is transient. At our centre, we're collaborating with genomics companies and institutions to study how it can be done permanently and then applied to cure thalassemia," said consultant haemotoglogist Dr Sharat Damodar. Thalassemia is an inherited blood disorder in which the body makes an abnormal form of haemoglobin, while haemophilia is a bleeding disorder and blood takes a long time to clot.

Initially, the team will start with animal tests.

Thalassemia was chosen for gene therapy because the number of patients is the highest in India and the Mediterranean but not so much in the West. About 10,000 children are born in India with thalessemia major every year. Almost 3,000 of them can be cured with bone marrow transplant but even if 10% of them were to get transplant, India has to do at least 300 transplants every year.

Medical director and vice-president, Mazumdar Shaw Cancer Center and Narayana Hrudayalaya Multi-Specialty Hospital, Dr Paul C Salins, told TOI: "There is no doubt that the future is in gene therapy. If we can alter the gene itself it'll have far-reaching implications. Through our research, we're trying to find out how we can deal with diseases that affect the common man."

NEXT-GENERATION SEQUENCING

Associated with gene therapy is exploring what in the gene sequence causes cancer. You either inherit or develop cancer as you grow. The incidence of the latter is higher and is caused by various genetic changes. Head of Institute of Bioinformatics and Applied biotechnology and Ganit Labs, Binay Panda, is collaborating with the advanced therapeutics centre to do genetic analysis and next generation sequencing.

"We want to dissect changes associated with cancer in the human genome -- that's the key to finding solutions. The human genome is 3 billion letters long. We have to read the letters and see how the building blocks of DNA change. An entire individual can be decoded in 10-15 days through this technology," he said. Binay is currently focusing on oral cancer genomics in India.

Biomarkers, an associated technology, is becoming popular. It helps in highlighting various markers in an individual for various diseases.

GENE THERAPY

* Experimental treatment involves introducing genetic material into a person's cells to fight or prevent disease

* Genes are biological units of heredity; they determine traits like colour of hair and eye as well as ability of blood to carry oxygen

* Scientists need to identify more efficient ways to deliver genes to the body. To treat cancer and other diseases effectively with gene therapy, researchers must develop vectors that can be injected into patients and focus on target cells in body