Studying Tropical Genetic Blood Diseases

October 16, 2010

Sir David Weatherall, 77, an Oxford researcher-physician, was among the first to use the tools of molecular biology to understand thalassemia. He was in New York to receive the Lasker-Koshland Special Achievement Award for “50 years of international statesmanship in biomedical science.” A condensed version of  conversations with him follows:

Q. YOU GREW UP IN LIVERPOOL. HOW DID TROPICAL GENETIC BLOOD DISEASES BECOME YOUR LIFE’S WORK?

A. In 1956, after I’d finished my medical training, I was drafted for compulsory military service. At the time, there was an insurgency in Malaya, where the Commonwealth forces were fighting the Communists, and I was not anxious to get involved with that. Nonetheless, I soon found myself on a troop ship for Singapore.

When I got there, because I had no pediatric training, the army put me in charge of a children’s ward looking after the families of Commonwealth soldiers. And there I encountered a 2-year-old, the daughter of a Gurkha from Nepal. She had profound anemia. No one understood why. We kept her alive with transfusions.

So in my spare moments, I went to the biochemistry department at Singapore University Hospital, and worked with people there to try to figure it out. Within six months, we had an answer: thalassemia. That was a big surprise. This genetic disease was thought to occur only in the Mediterranean.

Q. WHAT EXACTLY IS IT?

A. It’s a defect in the genes that makes it impossible for hemoglobin to properly form. Of course, in the 1950s, we understood little about hemoglobin’s biochemistry. Thalassemia was, and is, a terrible disease. The children generally don’t live to adulthood — and then only with constant transfusions. In the case of this little girl, her parents eventually took her to their village in Nepal, where she died.

Q. SO SHE WAS THE BEGINNING OF YOUR INTEREST?

A. Yes. The army next sent me up to northern Malaya, where the last of the fighting was going on. I used this time to search for more thalassemia. I’d construct equipment from old car batteries and filter paper, and that’s how I separated the different hemoglobins in the blood samples I’d collect. Whenever I found anything abnormal, I’d post the slides to a good old boy, Herman Lehman, at a laboratory in London. We actually found one or two more cases that way.

Read Complete Interview Here..


Innovative drug design wins medical research prize

May 15, 2010

Courtesy by: news-medical.net

The development of a new way to treat iron overload disease has won the 2010 Sir Zelman Cowen Universities Fund Prize for Medical Research.

Dr Rachel Codd from the Discipline of Pharmacology won the Prize for her discovery of an award-winning compound that has the potential to significantly improve the treatment of the disease.

Inheritable blood disorders arising from single-gene defects are among the most common diseases in the world, with around seven per cent of the population estimated to be carriers. Each year, 300,000 to 500,000 babies are born with severe blood disorders, including sickle-cell anaemia and the thalassemias.

To prevent life-threatening anaemia, patients with beta-thalassemia undergo blood transfusions every two to four weeks. Regular blood transfusions cause an excess of iron to accumulate in the body resulting in iron overload disease. Since humans do not have an active iron excreting mechanism, patients must undergo additional treatment to remove the iron (chelation therapy).

The current treatment for iron overload disease is effective only when administered by intravenous infusion. To improve the quality of life of thalassemia patients, there is a need to develop iron chelation agents that are orally active.

Dr Codd has shown that simple modifications to the currently available iron chelation therapy have the potential to improve treatment options for thalassemia, including oral delivery. In addition, the compounds may have application in neurodegenerative diseases such as Parkinson’s disease, in which irregular iron levels have been implicated as contributing factors.

Dr Codd’s group at the University uses a chemical biology approach to find platforms for drug design and drug discovery. This approach has also led to innovations in the design of compounds as potential anti-cancer agents and antibiotics.

Professor Jonathan Stone, Managing Trustee of the Sir Zelman Cowen Universities Fund congratulated Dr Codd on winning the award.

“In awarding the Prize to Dr Codd, the Prize Committee noted the novelty of the compounds, their clinical applicability to a range of difficult-to-treat diseases, and the strong momentum of Dr Codd’s work,” he said.

The Prize, an award of $10,000 and a medal crafted by renowned Melbourne sculptor, Michael Meszaros, will be awarded to Dr Codd at a function to be held later in the year.


Six Researchers to Receive Prestigious Awards from the American Society of Hematology

July 14, 2009

Courtesy by: us.sys-con.com

WASHINGTON, July 13 /PRNewswire-USNewswire/ — The American Society of Hematology (ASH), the world’s largest professional society of blood specialists, will honor six scientists who have made significant contributions to the understanding of hematologic diseases. These awards, including the newly created Ernest Beutler Lecture and Prize, will be presented at the 51st ASH Annual Meeting taking place December 5-8 in New Orleans.

Past ASH president H. Franklin Bunn, M.D., of the Brigham and Women’s Hospital in Boston, will be presented with the Wallace H. Coulter Award for Lifetime Achievement in Hematology, which was established in 2007. This award, named for Wallace Henry Coulter, a prolific inventor who made important contributions to hematology and to ASH, is bestowed on an individual who has demonstrated a lifetime commitment and made outstanding contributions to hematology, and who has made a significant impact on education, research, and/or practice. Dr. Bunn will receive the award for his leadership in advancing the field of hematology and hematology research for more than 40 years. Throughout his career, Dr. Bunn’s research has represented only a part of his commitment to the field. He has served on many National Institutes of Health advisory groups and councils and as an Associate Editor of Blood, a reviewer and editor of publications about hemoglobin and hemoglobin disorders, and an author of two textbooks. Most importantly, he has been an inspiring teacher of hematology to medical students and a masterful mentor of fellows and junior faculty.

Connie Eaves, Ph.D., of the BC Cancer Agency, University of British Columbia in Vancouver, will be presented with the Henry M. Stratton Medal, which honors an individual whose well-recognized contributions to hematology have taken place over a period of several years. Dr. Eaves will receive this award for her remarkable achievements in the area of stem cell biology for more than two decades. Dr. Eaves has been on the cutting edge of adapting or introducing technologies related to stem cell biology, especially her ground-breaking techniques of using the long-term culture system as means of understanding the proliferative and renewal properties of normal and malignant primitive human hematopoietic stem cells.

Louis M. Staudt, M.D., Ph.D., of the National Cancer Institute’s Center for Cancer Research in Bethesda, MD, will be presented with the William Dameshek Prize, awarded to an individual who has made a recent outstanding contribution to the field. Dr. Staudt will be recognized for his landmark contributions to the diagnosis and treatment of lymphomas. Dr. Staudt pioneered the use of gene expression profiling to delineate clinically distinct lymphoma subtypes and molecular predictors of survival. He further showed that each molecular subtype of cancer has its own “Achilles heel,” which can be discovered by loss-of-function RNA interference-based genetic screens. His work established a new paradigm for understanding the pathways of lymphomagenesis and the interactions of the malignant cell with its environment.

John E. Dick, Ph.D., of the University Health Network in Toronto, will be recognized with the E. Donnall Thomas Lecture and Prize for his pioneering research into the development of human leukemia, which has transformed the view of how leukemia progresses. This prize, named after a Nobel Prize laureate and past Society president, recognizes pioneering research achievements in hematology.

Thomas Maniatis, Ph.D., of Harvard University, Cambridge, MA, and Yuet Wai Kan, M.D., of the University of California – San Francisco, are the inaugural recipients of the Ernest Beutler Lecture and Prize. This award, named for the late Ernest Beutler, M.D., past president of ASH and physician-scientist for more than 50 years, includes a two-part lectureship that recognizes major advances related to a single topic. This year’s lecture will be on the impact of molecular genetic studies of globin genes on the diagnosis and treatment of thalassemia.


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