January 23, 2009
NIH Podcast Episode #0076
Balintfy: Welcome to the 76th episode of NIH Research Radio with news about the ongoing medical research at the National Institutes of Health—the nation's medical research agency. I'm your host Joe Balintfy. Coming up in this episode, a new genetic target for treating sickle cell disease; and a great way to get a start in biomedical research. But first, better understanding of Hepatitis B and screening of high-risk populations. That's next on NIH Research Radio.
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Panel Advocates Improved Understanding of Hepatitis B and Screening of High-Risk Populations
Balintfy: Hepatitis B is one type of hepatitis, a liver disease, caused by the hepatitis B virus. Hepatitis B spreads by contact with an infected person's blood, semen or other body fluid. Management of hepatitis B is a challenge for physicians and patients due to an incomplete understanding of the disease course, complex treatment indications, and the lack of large studies focusing on important health outcomes. However—
Sorrell: This is a totally preventable disease if you vaccinate.
Balintfy: Dr. Michael Sorrell, Professor of Medicine at the University of Nebraska Medical Center, says there is a tremendous success rate in treating the disease process in the short term.
Sorrell: What we're lacking is long term studies in subsets of populations decide who should be treated and when they should be treated, and when they should not be treated.
Balintfy: Studying hepatitis B is important because it is the leading cause of liver cancer worldwide.
Sorrell: It's an important cause of liver cancer in the United States, however, liver cancer is also caused by hepatitis C which is actually more common than hepatitis B and probably causes more cancer.
Balintfy: Dr. Sorrell, who chaired an NIH Consensus Development Program panel, stressed the importance of vaccination.
Sorrell: One of the things we discussed and spend a lot of time discussing was this question of vertical transmission from mother to child. If the mother is infected with hepatitis B, at least 90% of the time the child whose born will be infected with hepatitis B.Balintfy: Dr. Sorrell adds that infants and children up to five years old have less vigorous immune systems which can result in very high levels of the virus in their bodies. While these patients may have minimal disease symptoms, the virus is still transmissible and some patients will go on to develop chronic liver disease, cirrhosis and cancer. For more information on the panel conclusions, visit consensus.nih.gov. More details on hepatitis B are available at www.niddk.nih.gov.
Researchers Find New Genetic Target for Sickle Cell Disease Therapy
Balintfy: Treatments developed over the past three decades have led to the doubling of the life expectancy of sickle cell disease patients between 1972 and 2002. These treatments include medications, blood and bone marrow transfusions, and other procedures to relieve or prevent complications. Until now, however, scientists could not directly target processes known to affect the severity of sickle cell disease. An article published online in Science last December describes a discovery that could lead to new treatment targets for some inherited blood diseases:
Shurin: The findings in this article are applicable to both people with sickle cell sickle cell disease and thalassemia, or are at least potentially applicable to them.
Balintfy: Dr. Susan Shurin is the National Heart, Lung and Blood Institute deputy director. She says these are genetic disorders that affect approximately seventy-thousand people in the United States, primarily African-Americans.
Shurin: These are diseases that affect many millions of people world wide. These are very big public health problems, particularly in sub-Saharan Africa and the Middle East, in the Mediterranean and also in Southeast Asia.
Balintfy: These disorders also have a very high health burden, making them difficult to deal with in terms of both symptoms and treatment, which can include medications and blood transfusion.
Shurin: For people with sickle-cell disease they tend to have a lot of pain. And for both thalassemia and sickle-cell disease particularly if people are not transfused early in life these may be fatal diseases.
Balintfy: A form of hemoglobin, which is the protein in the blood that makes red blood cells red, and carries oxygen from the lungs to tissues in the body, is involved in both these diseases. Dr. Shurin explains that in sickle cell disease, hemoglobin is abnormal and sticks together. The red blood cells become stiff and sickle-shaped, causing them to block blood vessels and rob tissues of necessary blood and oxygen. In thalassemia, the body has trouble producing adult forms of hemoglobin.
Shurin: So what this study shows, is it lays the groundwork for the development for some targeted therapies which are of potential major benefit for people with both thalassemia and sickle-cell disease.
Balintfy: Researchers are reporting that by suppressing or “turning off” a gene called BCL11A, production of a form of hemoglobin improves dramatically.
Shurin: So there are going to be two really key issues here. The first is to try to find some good ways of targeting this so we can decrease the production of BCL11A, or inactivate it once it’s present in a cell, that would be number one. And number two is to try to do this so we do it as specifically as possible for red blood cells so we get as few side effects in other tissues as possible.
Balintfy: Dr. Shurin adds that both the results and methodology in the study may help move treatment options forward to tests in mice, possibly within the next couple years. For more information on this study and these blood disorders, visit www.nhlbi.nih.gov.Balintfy: When we come back, learn how some of today’s students are getting international training that may help lead to tomorrow’s biomedical discoveries. Stay tuned.
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NIH-Oxford-Cambridge Scholars Program
Balintfy: The National Institutes of Health-Oxford-Cambridge Scholars Program is an accelerated, individualized doctoral training program for outstanding science students committed to biomedical research. Students selected for the program can receive an annual stipend of more than 26-thousand dollars, health insurance and other academic support while studying in the UK and at the NIH. All laboratory research expenses are covered and a 3-thousdand dollar per year travel budget is available. But as Dr. Michael Lenardo, director of the NIH-Oxford-Cambridge Scholars Program explains the program is much more than just money.
Lenardo: This program allows students unparalleled opportunities to carry out international, collaborative projects between investigators at Oxford and Cambridge University, where they’ll have at least one mentor, and in Bethesda at the NIH, where they’ll have another mentor. And those mentors work together with the student to carry out a collaborative project. One of the things we emphasize in the program is to help the students break down barriers, to try new ideas in progress.
Balintfy: Dr. Lenardo, why do you think having multiple mentors is so important to these students and the program?
Lenardo: When you have two people involved, or three people, or four people involved in mentoring, then suddenly the student becomes the focal point, who then has to integrate all this information. And that’s a process actually that is very important in their maturation, because that’s a process that we go through as practicing scientists, where we have to integrate inputs from many different people -- we go out and give a lecture on our research, talk to our colleagues down the hall, people in the lab talking to us about what they feel about the data they’re developing -- that all has to be integrated, and a decision made about how to move forward. So the students in this program begin to learn that very, very early in their scientific education.
Balintfy: How much of this training and learning is about medicine and being a doctor, as opposed to research and being a scientist?
Lenardo: I think the best way to think about it is that during the course of medical school your focus on biology becomes very much a focus on the human body, and all the things that can go wrong with it and do go wrong with it. And so, therefore, doing that at the same time that you’re doing your Ph.D. research, which is training you as a scientist, invariably everything you begin to see as a scientist is through the prism of human biology. And therefore, I think it has a greater tendency to have students focus on aspects of their biomedical research projects that are relevant to human disease, and doing something about human disease. As I mentioned earlier, one of the things that we emphasize as a value in the program, that we try to imbue the students with, is the idea of breaking down barriers. We feel like there’s a wealth of knowledge on medical topics, on medical research, on disease problems, but somehow we’re not getting through that to developing new treatments, cures, and preventative measures. We think that by having the students adopt this new perspective, where they can put together innovative projects with a number of different mentors, they can get beyond traditional disciplines and get beyond some of the traditional barriers. And so having that combined with the medical training, I think makes these individuals particularly powerful in going on in their career, and we hope that many of them will become leaders in new disciplines of the future because they’ve had this very unique training pathway.
Balintfy: Are there other strengths of the program you think are important to share?
Lenardo: One of the things about the program that we feel is especially important is that it is individualized for every student. There’s not one uniform curriculum for all the students, there’s not one individual — there’s not one set of projects or one discipline. All of the students come to us as individuals, with different backgrounds, different depths of scholarship in various areas, and so we act to build on that foundation individually for each student so that they can be prepared to be an outstanding leader in their own area that they choose to work in.
Balintfy: I understand that the program also arranges tutorials for students to help follow their own particular path of education. How does that benefit their learning experience?
Lenardo: Now one of the consequences of that approach is that it has allowed us to actually have an acceleration in the educational training pathway for these students. So, whereas normally in the United States students can take six, seven, or eight years to do their Ph.D., in this program they start on their thesis work on day one, as soon as they’re in the program, and that then allows them to complete the program in just over four years. So it can cut, in some cases, the time down by half in obtaining their Ph.D. degree. As the program has taken root over the last eight years, we’ve found that the students have actually demonstrated to us several other parts of the program that we’ve developed. One that I think is particularly important is that students, as they’ve gotten into their research in the NIH-Oxford-Cambridge Program, have found that they want to take their discoveries to the bedside, and actually develop them as new treatments, cures, or preventative measures. And therefore, they’ve wanted medical training. So we have built a new program, which is a combined degree MD/Ph.D. training pathway that allows students to do their Ph.D. in this program, and then receive medical training at any one of the 41 medical schools around the country that have medical scientist training programs. And these turn out to be some of the most distinguished medical schools in the country.Balintfy: Thanks to Dr. Michael Lenardo, director of the NIH-Oxford-Cambridge Scholars Program. For more information about the program, visit the website: oxcam.gpp.nih.gov. Also, keep an eye out for the NIH vodcast, “I on NIH” – we’ll have more on this topic, including interviews with students, in an upcoming episode.
Balintfy: That’s it for this edition this episode of NIH Research Radio. Please join us again on February 6th when our next edition will be available for download. I'm your host, Joe Balintfy. Thanks for listening. NIH Research Radio is a presentation of the NIH Radio News Service, part of the News Media Branch, Office of Communications and Public Liaison in the Office of the Director at the National Institutes of Health in Bethesda, Maryland, an agency of the US Department of Health and Human Services.