New clinical trial results show how personalized medicine will alter treatment of genetic disorders

One of the nation’s pre-eminent genetic researchers, Eric Hoffman, PhD, of Children’s Research Institute at Children’s National Medical Center, predicts that in relatively short order, medicine’s next innovation–individualized molecular therapies–will have the unprecedented ability to treat muscular dystrophies, and other disorders.

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Researchers reveal missing link in a heart disease pathway

University of Michigan scientists and their colleagues have helped characterize a previously unknown link in the chain of biochemical reactions implicated in some forms of heart disease.

The finding provides a new target for future drug therapies.

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Light powered platinum more targeted and 80 times more powerful than similar cancer treatments

Researchers from the Universities of Warwick, Edinburgh, Dundee and the Czech Republic’s Institute of Biophysics have discovered a new light-activated platinum-based compound that is up to 80 times more powerful than other platinum-based anti-cancer drugs and which can use “light activation” to kill cancer cells in much more targeted way than similar treatments.

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‘Micro’ livers could aid drug screening

MIT researchers have devised a novel way to create tiny colonies of living human liver cells that model the full-sized organ. The work could allow better screening of new drugs that are potentially harmful to the liver and reduce the costs associated with their development.
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Obesity and lack of exercise could enhance the risk of pancreatic cancer

Obesity and aversion to exercise have become hallmarks of modern society – and a new study suggests that a blood protein linked to these lifestyle factors may be an indicator for an increased risk of developing pancreatic cancer. Researchers from the Dana Farber Cancer Institute report their findings in the August 15 issue of Cancer Research, a journal of the American Association for Cancer Research.

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Scientists turn mouse into factory for human liver cells

Oregon Health & Science University researchers have figured out how to turn a mouse into a factory for human liver cells that can be used to test how pharmaceuticals are metabolized.

The technique, published in the journal Nature Biotechnology, could soon become the gold standard not only for examining drug metabolism in the liver, which helps scientists determine a drug’s toxicity. But it also can be used as a platform for testing new therapies against infectious diseases that attack the liver, such as hepatitis C and malaria.

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Green tea boosts production of detox enzymes, rendering cancerous chemicals harmless

Concentrated chemicals derived from green tea dramatically boosted production of a group of key detoxification enzymes in people with low levels of these beneficial proteins, according to researchers at Arizona Cancer Center.

These findings, published in the August issue of Cancer Epidemiology, Biomarkers & Prevention, a journal of the American Association for Cancer Research, suggest that a green tea concentrate might help some people strengthen their metabolic defense against toxins capable of causing cancer.

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Wonderful Medical Animation Reel

This 3D animation shows various surgical (mostly orthopedic) medical procedures performed to fix the problems in our bodies. Very nice done. 

New Drug May Help Treat Crohn’s Disease

Two new studies show that a new drug called Cimzia may ease symptoms of Crohn’s disease.

Cimzia hasn’t been approved by the FDA yet. Patients would give themselves injections of the drug, which targets an inflammatory chemical called tumor necrosis factor (TNF) alpha.

The two new studies, published in The New England Journal of Medicine, highlight Cimzia’s clinical trials in Crohn’s disease patients.

Read rest of this article at WebMD site

 

Scientists develop a model that could predict cells’ response to drugs

MIT researchers have developed a model that could predict how cells will respond to targeted drug therapies. Models based on this approach could help doctors make better treatment choices for individual patients, who often respond differently to the same drug, and could help drug developers identify the ideal compounds on which to focus their research.

In addition, the model could help test the effectiveness of drugs for a wide range of diseases, including various kinds of cancer, arthritis and immune system disorders, according to Douglas Lauffenburger, MIT professor of biological engineering and head of the department. Lauffenburger is senior author of a paper on the new model that will appear in the Aug. 2 issue of Nature.

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New technique to ‘see’ and protect transplants successful in diabetic animal model

Researchers at Johns Hopkins have found a way to overcome a major stumbling block to developing successful insulin-cell transplants for people with type I diabetes.

Traditional transplant of the cells, accompanied by necessary immune-suppressing drugs, has had highly variable results, from well- to poorly tolerated. Part of the problem, the Hopkins researchers say, is an inability to track the cells—so-called pancreatic beta cells—once they’re inside the body.

Now a new technique encapsulates the insulin-producing cells in magnetic capsules, using an FDA-approved iron compound with an off-label use, which can be tracked by magnetic resonance imaging (MRI). The product, tested in swine and diabetic mice, also simultaneously avoids rejection by the immune system, likely a major reason for transplant failure. The work will be published online next week in Nature Medicine.

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Cholesterol drug hits diabetes with one-two punch

Patients with type 2 diabetes may soon be able to control their glucose and their cholesterol levels with a single drug, according to a study led by Vivian A. Fonseca, professor of medicine and pharmacology at Tulane University School of Medicine and chief of the Tulane University Health Sciences Center Diabetes Program.

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Super 3D animation that shows the mode of action of an HIV drug

In this cool animation the mode of action of a novel HIV drug, a protease inhibitor, is explained. Protease inhibitors revolutionized treatment of HIV infection by enabling drug combinations with inhibitors of another HIV enzyme the reverse transcription. Thus, this made it more difficult for virus to develop multiple mutations simultaneously to escape the effects of a single drug.

Amazing chemistry and rational drug design is involved in creating these new drugs.

Omega-3 fatty acids protect eyes against retinopathy

Increasing intake of the omega-3 fatty acids DHA and EPA, found in popular fish-oil supplements, may protect against blindness resulting from abnormal blood vessel growth in the eye, according to a study published online by the journal Nature Medicine on June 24. The study was done in mice, but a clinical trial at Children’s Hospital Boston will soon begin testing the effects of omega-3 supplementation in premature babies, who are at risk for vision loss.

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Faulty cell membrane repair causes heart disease

During vigorous exercise, heart muscle cells take a beating. In fact, some of those cells rupture, and if not for a repair process capable of resealing cell membranes, those cells would die and cause heart damage (cardiomyopathy)

Researchers at the University of Iowa Roy J. and Lucille A. Carver College of Medicine have discovered a specific repair mechanism in heart muscle and identified a protein called dysferlin that is critical for resealing heart muscle cell membranes.

The study, led by UI researcher and Howard Hughes Medical Institute investigator Kevin Campbell, Ph.D., also shows that loss of dysferlin causes cardiomyopathy in mice. Furthermore, heart damage in these mice is exaggerated by vigorous exercise or by inherent muscle weakness caused by a muscular dystrophy defect.

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Remarkable advance in muscle restoration in an animal model of Duchenne muscular dystrophy

Using a new type of drug that targets a specific genetic defect, researchers at the University of Pennsylvania School of Medicine, along with colleagues at PTC Therapeutics Inc. and the University of Massachusetts Medical School, have for the first time demonstrated restoration of muscle function in a mouse model of Duchenne’s muscular dystrophy (DMD). The research appears ahead of print in an advanced online publication of Nature.

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Gleevec decreases cancer recurrence for patients with primary gastrointestinal stromal tumor

Preliminary results from a large, randomized, placebo-controlled clinical trial for patients with primary gastrointestinal stromal tumor (GIST), a type of tumor usually found in the stomach or small intestine, showed that patients who received imatinib mesylate (Gleevec ®) after complete removal of their tumor were significantly less likely to have a recurrence of their cancer compared to those who did not receive imatinib. The clinical trial was sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), and conducted by a network of researchers led by the American College of Surgeons Oncology Group (ACOSOG).
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Green tea and COX-2 inhibitors combine to slow growth of prostate cancer

Drinking a nice warm cup of green tea has long been touted for its healthful benefits, both real and anecdotal. But now researchers have found that a component of green tea, combined with low doses of a COX-2 inhibitor, could slow the spread of human prostate cancer.

In the March 1 issue of Clinical Cancer Research, researchers from University of Wisconsin-Madison demonstrate that low doses of the COX-2 inhibitor celecoxib, administered with a green tea polyphenol called pigallocatechin-3-gallate (EGCG), can slow the growth of human prostate cancer. Their experiments were performed in cell cultures and in a mouse model for the disease.
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Gene profiling predicts resistance to breast cancer drug Herceptin

Using gene chips to profile tumors before treatment, researchers at Harvard and Yale Universities found markers that identified breast cancer subtypes resistant to Herceptin, the primary treatment for HER2-positive breast cancer. They say this advance could help further refine therapy for the 25 to 30 percent of breast cancer patients with this class of tumor.

In the February 15 issue of Clinical Cancer Research, the researchers found that HER2-positive tumors that did not respond to Herceptin expressed certain basal markers, growth factors and growth factor receptors. One of these, insulin-growth factor receptor 1(IGF-1R), was associated with a Herceptin response rate that was half that of tumors that did not express IGF-1R.
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‘Bridge’ protein spurs deadliest stages of breast cancer

A protein known for its ability to “bridge” interactions between other cellular proteins may spur metastasis in breast cancer, the disease’s deadliest stage, a study from Burnham Institute for Medical Research has found.
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Researchers identify stem cells in pancreatic cancer

Researchers have discovered the small number of cells in pancreatic cancer that are capable of fueling the tumor’s growth. The finding is the first identification of cancer stem cells in pancreatic tumors.

Cancer stem cells are the small number of cancer cells that replicate to drive tumor growth. Researchers believe current cancer treatments sometimes fail because they are not attacking the cancer stem cells. By identifying the stem cells, researchers can then develop drugs to target and kill these cells.

This is particularly crucial for pancreatic cancer, which has the worst survival rate of any major cancer type. Nearly everyone who develops pancreatic cancer dies from the disease.
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Molecular link between inflammation and cancer discovered

A team led by biochemists at the University of California, San Diego has found what could be a long-elusive mechanism through which inflammation can promote cancer. The findings may provide a new approach for developing cancer therapies.

The study, published in the January 26 issue of the journal Cell, shows that what scientists thought were two distinct processes in cells–the cells’ normal development and the cells’ response to dangers such as invading organisms–are actually linked. The researchers say that the linkage of these two processes may explain why cancer, which is normal growth and development gone awry, can result from chronic inflammation, which is an out-of-control response to danger.
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Novel EGFR antibody outperforms cetuximab in mouse model of lung cancer

Antibodies that selectively bind and destroy cancer cells represent some of the most promising cancer therapy approaches being developed today. Several of these antibodies have reached the market, including cetuximab (Erbitux®, ImClone Systems), which targets the epidermal growth factor receptor (EGFR) protein. However, a study conducted at the Dana-Farber Cancer Institute and the Ludwig Center at Dana-Farber/Harvard Medical School now suggests that antibodies binding a particular protein conformation, caused by hyperactivation, might have distinct therapeutic advantages over antibodies, like cetuximab, that bind to wild-type (normal) target proteins.
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Brain tumor researchers find their ‘niche’

Brain tumors appear to arise from cancer stem cells (CSCs) that live within microscopic protective “niches” formed by blood vessels in the brain; and disrupting these niches is a promising strategy for eliminating the tumors and preventing them from re-growing, according to results of a study by investigators at St. Jude Children’s Research Hospital. CSCs are cells that continually multiply, acting as the source of tumors.

“The finding that brain CSCs exist in protective vascular (blood vessel) niches helps explain the origin of brain tumors and suggests a new strategy for eliminating them,” said Richard Gilbertson, M.D., Ph.D., co-director of the Neurobiology and Brain Tumor Program at St. Jude. Gilbertson is senior author of a report on this work that appears in the January issue of Cancer Cell.
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Pivotal kidney cancer study shows doubling of disease-free survival

Bayer and Onyx Pharmaceuticals announced that the New England Journal of Medicine has published their pivotal Phase III trial demonstrating that Nexavar® (sorafenib) tablets doubled median progression-free survival (PFS) in patients with advanced renal cell carcinoma (RCC), or kidney cancer. The trial is the largest randomized controlled trial ever conducted in advanced RCC.
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Researchers demonstrate ability of new therapy to treat severely elevated cholesterol levels

Researchers at the University of Pennsylvania School of Medicine have demonstrated the potential of a new type of therapy for patients who suffer from high cholesterol levels. The findings are in the January 11 issue of the New England Journal of Medicine (NEJM). In this study, patients with homozygous familial hypercholesterolemia (FH), a high-risk condition refractory to conventional therapy, had a remarkable 51% reduction in low-density lipoprotein (LDL) or “bad cholesterol” levels.
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Targeting leukemic stem cells by Bcl-2 inhibition

Researchers at The University of Texas M. D. Anderson Cancer Center have found, in laboratory studies, that the experimental drug ABT-737 which has shown promise in some cancers, can destroy acute myeloid leukemia (AML) blast, progenitor and even stem cells that are often resistant to standard chemotherapy treatment.

The drug was powerful in its own right, the researchers say, but they found that some AML cells were themselves resistant to ABT-737, so they added another drug that knocked out this secondary resistance. Together, these agents may provide a powerful therapy against AML, and could form the basis of a new way to treat the cancer, say the scientists, whose study was published in the November 14 issue of the journal, Cancer Cell.
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Vaccine for brain tumors shows promising results

A vaccine for treating a recurrent cancer of the central nervous system that occurs primarily in the brain, known as glioma, has shown promising results in preliminary data from a clinical trial at UCSF Medical Center.

Findings from the first group of six patients in the study, being conducted at the UCSF Brain Tumor Research Center, showed that vitespen (trademarked as Oncophage), a vaccine made from the patient’s own tumor, was associated with tumor-specific immune response in patients with recurrent, high-grade glioma.
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Study finds potential ovarian cancer stem cells

Massachusetts General Hospital (MGH) researchers have identified potential ovarian cancer stem cells, which may be behind the difficulty of treating these tumors with standard chemotherapy. Understanding more about the stem-like characteristics of these cells could lead to new approaches to treating ovarian cancer, which kills more than 16,000 U.S. women annually and is their fifth most common cause of cancer death. The report will appear in the July 25 Proceeding of the National Academy of Sciences (PNAS) and has received early online release.
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Progress being made in exploring potential use of stem cells to treat heart disease

Scientists are making headway in exploring the potential future use of stem cells to treat heart disease, according to a review article in the current issue of Nature (June 29, 2006).

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Reprogramming Biology

Visionary futurist Ray Kurzweil, whose remarkable ideas on technological progress have been an inspiration for Biosingularity blogs, have a wonderful concise article on biological advances in recent issue of Scientific American

As a scientist working on biological systems I fully agree and whole heartedly support Kurzweil's observations that: " Biology is now in the early stages of an historic transition to an information science, while also gaining the tools to reprogram the ancient information systems of life ….. We are now beginning to understand biology as a set of information processes, and we're developing realistic models and simulations of how the processes involved in disease and aging progress. Moreover, we are developing the tools to reprogram them."

In the article Kurzweil predicts that tinkering with our genetic programs will extend human lifespan beyond the current limits. He also reiterates that biological systems are also subject to the "law of accelerating returns", which had tremendous impact on information technologies. Indeed, the cost of sequencing and synthesizing gene base pairs have decreased more than 10,000 fold over the last 15 years, and this exponential progress is currently accelerating as predicted by Kurzweil in his recent book. 

Read rest of the article at Scientific American web site.
 

Drug that battles resistance to leukemia pill Gleevec ‘extremely effective’ against cancer

An experimental therapy that battles drug resistance in Chronic Myeloid Leukemia (CML) has proved "extremely effective" in fighting cancer, giving patients for whom all conventional therapies have failed another option, researchers at UCLA's Jonsson Cancer Center reported.

The Bristol-Myers Squibb therapy, Sprycel (dasatinib), treats CML that has mutated and becomes resistant to the leukemia pill Gleevec, said Dr. Charles Sawyers, a professor of hematology/oncology, a Jonsson Cancer Center researcher and lead author of the study, published in the June 15, 2006 issue of the peer-reviewed New England Journal of Medicine.
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Cure found for Huntington disease in mice offers hope for treatment in humans

Researchers at the University of British Columbia's Centre for Molecular Medicine and Therapeutics (CMMT) have provided ground-breaking evidence for a cure for Huntington disease in a mouse offering hope that this disease can be relieved in humans.

Published today in Cell journal, Dr. Michael Hayden and colleagues discovered that by preventing the cleavage of the mutant huntingtin protein responsible for Huntington disease (HD) in a mouse model, the degenerative symptoms underlying the illness do not appear and the mouse displays normal brain function. This is the first time that a cure for HD in mice has been successfully achieved.
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Researchers discover how a high-fat diet causes type 2 diabetes

Howard Hughes Medical Institute researchers have discovered a molecular link between a high-fat, Western-style diet, and the onset of type 2 diabetes. In studies in mice, the scientists showed that a high-fat diet disrupts insulin production, resulting in the classic signs of type 2 diabetes.

In an article published in the December 29, 2005, issue of the journal Cell, the researchers report that knocking out a single gene encoding the enzyme GnT-4a glycosyltransferase (GnT-4a ) disrupts insulin production. Importantly, the scientists showed that a high-fat diet suppresses the activity of GnT-4a and leads to type 2 diabetes due to failure of the pancreatic beta cells.

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New Drug Points Up Problems in Developing Cancer Cures

FDA approved Nexavar, a drug that officials described as “a major advance” in treating kidney cancer.
The manufacturer of Nexavar, Bayer, used X-rays to determine that the drug doubled the time, to 167 days from 84, before tumors grew substantially in number or size, a finding called “progression-free survival.”
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