BreakThrough Digest Medical News |
- Can gene therapy cure fatal diseases in children?
- Why does Alzheimer’s disease affect twice as many women as men?
- Experts propose ‘cyber war’ on cancer
- New study shows promise in using RNA nanotechnology to treat cancers and viral infections
- Preeclampsia poses a significant long-term health risk according to new research from Ben-Gurion U.
- Vitamin D supplements do not improve cholesterol as previous research suggested
| Can gene therapy cure fatal diseases in children? Posted: 04 Sep 2012 09:00 PM PDT In order for the body to function, a balance is necessary between the cells that build up the bones in our skeletons and the cells that break them down. In the disease malignant infantile osteopetrosis, MIOP, the cells that break down the bone tissue do not function as they should, resulting in the skeleton not having sufficient cavities for bone-marrow and nerves.
“Optic and auditory nerves are compressed, causing blindness and deafness in these children. Finally the bone marrow ceases to function and, without treatment, the child dies of anaemia and infections”, explains Carmen Flores Bjurström. She has just completed a thesis which presents some of the research at the division for Molecular Medicine and Gene Therapy in Lund. The researchers’ work focuses on finding alternatives to the only treatment currently available against MIOP, namely a bone-marrow transplant. This treatment can be effective, but it is both risky and dependent on finding a suitable donor. Gene therapy requires no donor, as stem cells are taken from the patients themselves. Once the cells’ non-functioning gene has been replaced with a healthy copy of itself, the stem cells are put back into the patient. Great hopes have been placed on gene therapy as a treatment method but the work has proven to be more difficult than expected. The method is used today for certain immunodeficiency diseases, and has also been applied to a blood disorder called thalassemia. “So far, the method is not risk-free. Since it is impossible to control where the introduced gene ends up, there is a certain risk of it ending up in the wrong place and giving rise to leukaemia. This is why gene therapy is only used for serious diseases for which there is no good treatment”, says Carmen Flores Bjurström. The Lund researchers have conducted experiments with gene therapy in both patient cells and laboratory animals. The next step is to conduct trials on patients. The trials will probably take place at the hospital in Ulm, Germany, which currently treats the majority of children in Europe suffering from MIOP. MIOP is a rare disease: in Sweden a child is born with the condition approximately once every three years. Worldwide, the incidence of the disease is one case for every 300 000 births. It is, however, more common in Costa Rica where 3-4 children per 100 000 births have the disease. “But there are several other genetic mutations that lead to other osteopetrosis diseases. If we manage to treat MIOP, it may become possible to treat these other conditions as well”, hopes Carmen Flores Bjurström along with her supervisor, Professor Johan Richter. ### The thesis is entitled “Targeting the hematopoietic stem cell to correct osteopetrosis” and it will be presented on 6 September. Contact: Carmen Flores Bjurström |
| Why does Alzheimer’s disease affect twice as many women as men? Posted: 04 Sep 2012 09:00 PM PDT New Rochelle, NY, September 5, 2012?A group of experts has developed consensus recommendations for future research directions to determine why nearly two-thirds of Americans with Alzheimer’s disease (AD) are women. The recommendations are published in a Roundtable discussion in Journal of Women’s Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women’s Health website at http://www.liebertpub.com/jwh.
An estimated 5.4 million Americans are affected by AD and related dementias, and that number will likely rise to 11-16 million people by the year 2050 if no effective cures or preventive measures are developed. The main risk factors for AD are age and sex, with affected women outnumbering men 2 to 1. This may be due at least in part to the fact that women tend to live longer. An interdisciplinary roundtable of experts convened by the Society for Women’s Health Research (Washington, DC) led to a set of recommendations to help guide future AD research and make the evaluation of sex and gender differences a component of future studies. The consensus recommendations encompass seven themes, including the need to assess the link between sex and AD incidence, raise awareness of sex differences among the research community, and to take into account sex-based differences in the experimental design and data analysis of studies on disease risk, early diagnosis, and drug discovery. “There are still major gaps in our knowledge of the role of sex and gender in the onset and progression of Alzheimer’s disease, and these recommendations will provide a useful guide for future research in this area,” says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women’s Health, Executive Director of the Virginia Commonwealth University Institute for Women’s Health, Richmond, VA, and President of the Academy of Women’s Health. ### About the Journal Journal of Women’s Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women’s healthcare issues. Tables of content and a sample issue may be viewed on the Journal of Women’s Health website at http://www.liebertpub.com/jwh. Journal of Women’s Health is the Official Journal of the Academy of Women’s Health. About the Academy Academy of Women’s Health (http://www.academyofwomenshealth.org/) is an interdisciplinary, international association of physicians, nurses, and other health professionals who work across the broad field of women’s health, providing its members with up-to-date advances and options in clinical care that will enable the best outcomes for their women patients. The Academy’s focus includes the dissemination of translational research and evidence-based practices for disease prevention, diagnosis, and treatment of women across the lifespan. About the Society for Women’s Health Research The Society for Women’s Health Research (SWHR), a national non-profit organization based in Washington, DC, is widely recognized as the thought leader in research on sex differences and is dedicated to improving women’s health through advocacy, education, and research. About the Publisher Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Breastfeeding Medicine, Population Health Management, Thyroid, and Metabolic Syndrome and Related Disorders. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 70 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website at www.liebertpub.com. Mary Ann Liebert, Inc. 140 Huguenot Street, New Rochelle, NY 10801 www.liebertpub.com Phone: (914) 740-2100 (800) M-LIEBERT Fax (914) 740-2101 Contact: Cathia Falvey |
| Experts propose ‘cyber war’ on cancer Posted: 03 Sep 2012 09:00 PM PDT In the face of mounting evidence that cancer cells communicate, cooperate and even engage in collective decision-making, biophysicists and cancer researchers at Rice University, Tel Aviv University and Johns Hopkins University are suggesting a new strategy for outsmarting cancer through its own social intelligence.
“We need to get beyond the notion that cancer is a random collection of cells running amok,” said Herbert Levine, co-director of Rice’s Center for Theoretical Biological Physics (CTBP) and co-author of the cover article in this week’s Trends in Microbiology that pulls together dozens of recent discoveries about the social behavior of cancer cells. “These cells lead sophisticated social lives.” Article co-author Eshel Ben-Jacob, a senior investigator at CTBP, said, “Cancer is a sophisticated enemy. There’s growing evidence that cancer cells use advanced communications to work together to enslave normal cells, create metastases, resist drugs and decoy the body’s immune system.” Ben-Jacob, Levine and Donald Coffey, a noted cancer researcher at Johns Hopkins, suggest in the article that cancer researchers act like modern generals and go after their enemy’s command, control and communication capabilities. The article is in volume 20, issue 9, pages 403-410 of the journal. “It’s time to declare a cyber war on cancer,” said Ben-Jacob, who, along with Coffey, is speaking today at a workshop titled “Failures in Clinical Treatment of Cancer” at Princeton University. Ben-Jacob said cancer cells have been shown to cooperate to elude chemotherapy drugs, much like bacteria that communicate and act as a team to resist attacks from antibiotics. He said some cancers appear to sense when chemotherapy drugs are present and sound an alarm that causes cells throughout a tumor to switch into a dormant state. Similar signals are later used to sound the “all clear” and reawaken cells inside the tumor. “If we can break the communication code, we may be able to prevent the cells from going dormant or to reawaken them for a well-timed chemotherapeutic attack,” Ben-Jacob said. “This is just one example. Our extensive studies of the social lives of bacteria suggest a number of others, including sending signals that trigger the cancer cells to turn upon themselves and kill one another.” The article cites numerous examples of similarities between the behavior of bacterial colonies and cancerous tumors. “The parallels between the communal behaviors of bacteria and cancer cells suggest that bacteria can serve as a valuable model system for studying cancer,” said Coffey, professor of urology, oncology, pathology and pharmacology and molecular sciences at the Johns Hopkins University School of Medicine. “We believe this approach could be particularly valuable for investigating intractable problems like metastasis, relapse and multiple drug resistance.” Levine, Rice’s Karl F. Hasselmann Professor in Bioengineering, and fellow CTBP co-director José Onuchic were recruited to Houston last year, thanks in part to a grant from the Cancer Prevention and Research Institute of Texas (CPRIT) that was designed to spur new thinking about cancer and foster collaborations between CTBP scientists and cancer specialists in the Texas Medical Center. “This opinion article reflects the multidisciplinary strategy of the CTBP — to communicate and work together with researchers across disciplines for solving the biomedical challenges of our time,” said Onuchic, Rice’s Harry C. and Olga K. Wiess Professor of Physics and Astronomy and professor of chemistry. Ben-Jacob, the Maguy-Glass Chair in Physics of Complex Systems and professor of physics and astronomy at Tel Aviv University, worked previously with Levine and Onuchic on a number of groundbreaking studies about the underlying biophysics of bacterial social behavior. He joined Rice University this summer as senior investigator of the CTBP and adjunct professor of chemistry and cell biology. ### A high-resolution image is available for download at: http://news.rice.edu/wp-content/uploads/2012/09/0907_CANCER_color-lg.jpg A copy of the research article is available at: http://www.sciencedirect.com/science/article/pii/S0966842X12001011 Contact: Jade Boyd |
| New study shows promise in using RNA nanotechnology to treat cancers and viral infections Posted: 03 Sep 2012 09:00 PM PDT
A new study by University of Kentucky researchers shows promise for developing ultrastable RNA nanoparticles that may help treat cancer and viral infections by regulating cell function and binding to cancers without harming surrounding tissue.
The study, published in Nano Today, was carried out in the laboratory of Peixuan Guo, the William S. Farish Endowed Chair in Nanobiotechnology at the UK Markey Cancer Center, in collaboration with Dr. Mark Evers, director of the UK Markey Cancer Center. The study uses RNA (ribonucleic acid) as a building block for the bottom-up fabrication of nanostructures. Using the RNA nanotechnology pioneered by Guo, the researchers constructed ultrastable X-shaped RNA nanoparticles using re-engineered RNA fragments to carry up to four therapeutic and diagnostic modules. Their RNA nanoparticles can include small interfering RNA for silencing genes, micro-RNA for regulating gene expression, aptamer for targeting cancer cells, or a ribozyme that can catalyze chemical reactions. The study demonstrated that regulation of cellular functions progressively increased with the increasing number of functional modules in the nanoparticle. “RNA nanotechnology is an emerging field, but the instability and degradation of RNA nanoparticles have made many scientists flinch away from the research in RNA nanotechnology,” Guo said. “We have addressed these issues, and now it is possible to produce RNA nanoparticles that are highly stable both chemically and thermodynamically in the test tube or in the body with great potential as therapeutic reagents.” The RNA nanoparticles displayed several favorable attributes: polyvalent nature, which allows simultaneous delivery of multiple functional molecules for achieving synergistic effects; modular design, which enables controlled self-assembly with defined structure; thermodynamically stable, which keeps the RNA nanoparticles intact in animal and human circulation systems, where they exist at very low concentrations; and chemically stable, which makes the nanoparticles resistant to RNase (an enzyme, which cleaves RNA) digestion in the blood serum. “A major problem with cancer treatments is the ability to more directly and specifically deliver anti-cancer drugs to cancer metastases,” Evers said. “Using the nanotechnology approach that Peixuan Guo and his group have devised may allow us to more effectively treat cancer metastasis with fewer side effects compared to current chemotherapy.” ### In addition to Evers and Markey team member Dr. Piotr Rychahou, Guo’s research team at UK also includes Farzin Haque, first author on the paper; Dan Shu; Yi Shu; and Luda Shlyakhtenko. Contact: Allison Perry |
| Preeclampsia poses a significant long-term health risk according to new research from Ben-Gurion U. Posted: 03 Sep 2012 09:00 PM PDT
Ben-Gurion University of the Negev (BGU) researchers have determined that preeclampsia is a significant risk factor for long-term health issues, such as chronic hypertension and hospitalizations later in life. The findings from the retrospective cohort study were just published in the Journal of Maternal-Fetal and Neonatal Medicine.
Thousands of women and their babies die or get very sick from preeclampsia; it affects approximately 5 to 8 percent of all pregnancies. It is a rapidly progressive condition characterized by high blood pressure and the presence of protein in the urine, typically occurring after 20 weeks gestation and up to six weeks postpartum. According to the study, the BGU researchers found patients with preeclampsia had significantly higher rates of chronic hypertension diagnosed after pregnancy. Patients with preeclampsia were also more likely to be hospitalized at least once. Exposed women had .28 hospitalization per patient rate, while the non-exposed patients had a lower .23 hospitalization per patient rate. The study included women who gave birth between the years of 1988 to 1998, and had a follow-up until December 2009. It assessed 2,072 patients with mild or severe preeclampsia in one or more of their pregnancies, while the comparison group of 20,742 patients did not have preeclampsia. The study was conducted to evaluate long-term morbidity of patients with hypertensive disorders during pregnancy. Patients with chronic hypertension and pre-gestational diabetes before the pregnancy were excluded. ### The researchers, all from BGU’s Faculty of Health Sciences, include Guy Shalom, Prof. Ilana Shoham-Vardi, Ruslan Sergienko, Prof. Arnon Wiznitzer, Michael Sherf, and Prof. Eyal Sheiner. About American Associates, Ben-Gurion University of the Negev American Associates, Ben-Gurion University of the Negev (AABGU) plays a vital role in sustaining David Ben-Gurion’s vision, creating a world-class institution of education and research in the Israeli desert, nurturing the Negev community and sharing the University’s expertise locally and around the globe. With some 20,000 students on campuses in Beer-Sheva, Sede Boqer and Eilat in Israel’s southern desert, BGU is a university with a conscience, where the highest academic standards are integrated with community involvement, committed to sustainable development of the Negev. AABGU is headquartered in Manhattan and has nine regional offices throughout the U.S. For more information, visit www.aabgu.org. Is preeclampsia a significant risk factor for long-term hospitalizations and morbidity? Contact: Andrew Lavin |
| Vitamin D supplements do not improve cholesterol as previous research suggested Posted: 03 Sep 2012 09:00 PM PDT Vitamin D has been touted for its beneficial effects on a range of human systems, from enhancing bone health to reducing the risk of developing certain cancers. But it does not improve cholesterol levels, according to a new study conducted at The Rockefeller University Hospital. A team of scientists has shown that, at least in the short term, cholesterol levels did not improve when volunteers with vitamin D deficiency received mega-doses of vitamin D. The finding is published in the journal Arteriosclerosis, Thrombosis and Vascular Biology.
The researchers, led by Manish Ponda, an assistant professor of clinical investigation in Jan Breslow’s Laboratory of Biochemical Genetics and Metabolism, studied 151 people with vitamin D deficiency. The study participants were given either 50,000 internationals units of vitamin D3 or a placebo weekly for eight weeks. Participants’ cholesterol levels were measured before and after treatment. Correcting vitamin D deficiencies with high doses of oral vitamin D supplements did not change cholesterol levels, Ponda and his colleagues found, despite effectively increasing vitamin D to recommended levels. Vitamin D levels nearly tripled in the group that received supplements, but were unchanged in the placebo group. “Our study challenges the notion that replenishing vitamin D improves cholesterol,” says Ponda. “In fact, a biologic response to vitamin D was correlated with an increase in LDL cholesterol.” Ponda and his colleagues also tested the effect of vitamin D supplementation on lipoprotein particle size and number, biomarkers of cholesterol not typically measured in clinical practice, and found no change in response to increases in vitamin D. These clinical trial results confirm those from a recent data mining study, published in July in the journal Circulation, conducted by the Breslow lab in collaboration with scientists at Quest Diagnostics. In that study, the researchers examined a de-identified dataset for 8,592 patients and showed that raising vitamin D levels from deficient to optimal levels had no statistically significant effect on LDL (bad) cholesterol, or triglycerides. Increasing vitamin D had a small, but clinically minimal impact on total and HDL (good) cholesterol. Both studies were supported by a Clinical and Translational Science Award from the National Center for Advancing Translational Science. “This study raises questions about the use of vitamin D supplements to improve cholesterol,” Ponda says. “Longer-term studies on the impact of vitamin D supplementation are needed to make stronger recommendations.” Contact: Joseph Bonner ### |
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