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MEDIA RELEASE 02 JULY 2014 ______________________________________________________________________________ Unlocking the healing power of stem cells Unique stem cell research facility at the University of Pretoria is a first for South African and will allow researchers to do pioneering clinical trials In a state-of-the art facility at the University of Pretoria, researchers are doing cutting-edge work focused on harnessing stem cells to combat cancer, heart failure and even HIV. Through a number of large funding awards, the facility has been able to significantly increase its resources both in terms of post-graduate students and senior researchers, and as well as highly sophisticated equipment. Some of the high-tech equipment, for example a high-performance cell sorter, is the only one of its kind on the African continent. “With this much-needed cell sorter we will be able to identify and isolate stem cell populations so that they can be studied in more detail,” Prof Michael Pepper, director at the Institute for Cellular and Molecular Medicine (ICMM), explains. “It also makes it possible to keep the cells in a sterile environment for patients who are to be treated.” Stem cells are basic living cells that seemingly have no specific function, but they do have the remarkable potential to turn into many different kinds of cells. Scientists believe that if these cells are tweaked just right, they can be triggered to transform into highly specialised cells with a very specific application in fighting disease. “Stem cells offer potential for the treatment of many life-threatening and infectious diseases,” Pepper, who is also a professor in the Department of Immunology, adds. “Locally this field is still in its infancy, but it holds great potential to alleviate the burden of disease.” Current uses of stem cells in South Africa generally revolve around bone marrow transplants. Pepper, who is also professeur associé in the Department of Genetic Medicine and Development in the Faculty of Medicine at the University of Geneva in Switzerland, is world-renowned for his work on the cellular and molecular components of human disease. He is developing novel stem cell therapies and, together with his team at UP, they are busy preparing for clinical trials for a number of key diseases. The ICMM comprises 20 research groups in seven of the nine faculties at the University of Pretoria that tackle themes such as stem cells, the human genome, cancer, bio-entrepreneurship and human-tissue legislation. Much of the ICMM’s work is done in uncharted territories, such as efforts around HIV eradication. 1 Pepper, working with a global research team, explores ways to help the human immune system to become HIV-resistant. This could have far-reaching implications in the management of the HIV/AIDS pandemic on the African continent. Gene-therapy research is done to create HIV-resistant blood-forming stem cells. This is done by preventing expression of a specific protein found on the surface of white blood cells. The virus uses this protein to enter into the host cell. But some people have a specific gene mutation that prevents expression of this protein and they are therefore immune against certain strains of the virus. The gene therapy project aims to achieve a similar effect. “By applying a specific kind of gene therapy to blood-forming stem cells, we can make them HIV resistant and then transplant them into people who would effectively become HIV-resistant,” he explains. “We are starting to grasp the healing power of stem cells, but we must also recognise that stem cell treatment can be an emotive issue for many people” adds Pepper. Pepper is a passionate proponent of the idea that scientists must engage with the public about their work. “Our work, after all, is funded by tax payers, and the more people understand what we are trying to achieve, the better informed they will be in order to make important decisions for themselves and their families. This also impacts positively on the general acceptance of rapidly-moving and sometime controversial fields,” he adds. He often uses public platforms to tackle complex and controversial issues such as sex determination, gender identity and the human genome. He sees the current regulatory environment pertaining to the use of human tissues in research as “cause for concern”. “Rapidly evolving fields of research, as is the case with stem cells, offer great opportunities, but can also be damaged by unethical practices, causing false hope for often really desperate people,” he says. "It is important to have laws that will protect emotionally vulnerable sick people against bogus therapies.” He believes current South African legislation is flawed and incomplete. “We need more progressive national policy and closely monitored clinical trials to keep us competitive at the cutting edge of this kind of work, while at the same time balancing ethical concerns, scientific progress and public opinion,” he adds. (Pepper is a member of the Minister of Science and Technology’s National Advisory Committee on Innovation.) He believes a public cord blood bank should be established to provide stem cells for therapeutic purposes. Stem cells that are harvested from the umbilical cord and placenta when a baby is born offer an alternative source to bone marrow (from family members of the patient or bone marrow registries). Adult stem cells are more acceptable to people who have objections to the use of stem cells from embryos, says Prof Pepper. Currently, the only cord blood banks operating in South Africa are in the private sector. “The process of collecting cord blood is non-invasive and can increase the pool of local donors significantly,” he explains. Finding bone marrow stem cells that are compatible (genetically similar) to that of the patient remain difficult. Many patients that require bone marrow transplants, especially in the African population in which there is great genetic diversity, therefore go untreated. This leader in medical research and biotechnology has been nominated for the 2014 National Science and Technology Forum (NSTF) Awards, to be announced on 3 July 2014. This is in recognition of his contributions to creating awareness of science, medicine and biotechnology (the use of living things to create useful tools and products). 2 Media enquiries: Nicolize Mulder; Department of University Relations, University of Pretoria; Tel: +27 12 420 3023 / +27 83 709 3041; Email: [email protected] For interviews, contact Prof Michael Pepper at the Institute for Cellular and Molecular Medicine Tel: (012) 3192179/90; Mobile: 072 209 6324; Email: [email protected] HIGH RES PHOTOS – CAPTIONS – DOWNLOAD AT http://bit.ly/1p9HvIO 1. Prof Michael Pepper 2. Prof Michael Pepper with PhD students Ilkadim Kiper (left) and Karlien Kallmeyer (right) 3. Prof Michael Pepper demonstrates the Affymetrix microarray platform and a Dx2 reader, which allows his team to study the genetics of diseases such as leukemia, but also the response of patients to different medications (pharmacogenomics) and gene expression in many settings such as stem cell differentiation. 3