#Clinical trial shows first ever positive results in treating primary progressive and relapsing multiple sclerosis Three phase three clinical studies using the drug ocrelizumab to treat patients with multiple sclerosis (MS) have yielded positive results for treating two forms of the disease and the first ever positive results
for a treatment for the less common primary progressive form (PPMS). MS is a condition of the central nervous system in which myelin,
which is characterised by attacks of the disease that interspersed by relatively symptom-free periods.
on 732 patients with PPMS, found that those treated with the drug had a reduction in the progression of clinical disability of 24 per cent.
Professor Gavin Giovannoni, Chair of Neurology at QMUL Blizard Institute, said: he phase III ocrelizumab results for both PPMS and RMS,
These data demonstrate that B-cell targeting can significantly modify the disease, which in effect means a more positive outlook for patients.
and for treatment to be provided as soon after diagnosis as possible to provide optimal outcomes, with the potential to improve patientsquality of life in the long-term. t
The research, funded by the blood cancer charity Bloodwise and the Medical Research Council (MRC
This provides an explanation for how cellular processes go awry in both Shwachman-Diamond syndrome and one in 10 cases of T-cell acute lymphoblastic leukaemia.
open up the possibility that a single drug designed to target this molecular fault could be developed to treat both diseases.
from the Cambridge Institute of Medical Research at the University of Cambridge, said: e are starting to find that many forms of blood cancer can be traced back to defects in the basic housekeeping processes in our cellsmaturation.
Pioneering improvements to electron microscopes pave the way for the creation of a detailed map of the how these diseases develop,
in a way that was never possible before. Single particle cryo-electron microscopy preserves the ribosomes at sub-zero temperatures to allow the collection
ew insights into the biology of blood cancers and disorders that originate in the bone marrow have only been made possible by the latest advances in technology.
the result is a potentially fatal arrhythmia. Now, a team of researchers from Oxford and Stony Brook universities has found a way to precisely control these waves using light.
hen there is scar tissue in the heart or fibrosis, this can cause part of the wave to slow down.
This ideal therapy has remained in the realm of science fiction until now. he team stresses that there are significant hurdles before this could offer new treatments a key issue is being able to alter the heart to be light-sensitised
as gene therapy moves into the clinic and with miniaturization of optical devices, use of this all-optical technology may become possible.
potent way to boost immunity and fight viruses`Many viral infections, such as the common cold, cause mild illnesses that the body immune system eventually defeats.
But when viruses cause severe disease, doctors have few options for effective treatment. Studying mice with a variety of viral infections,
scientists at Washington University School of Medicinein St louis have demonstrated a way to dial up the body innate immune defenses
while simultaneously attacking a protein that many viruses rely on to replicate. The findings, published Oct 19 in Nature Immunology, reveal previously unknown weapons in the body antiviral immune arsenal
the Selma and Herman Seldin Professor of Medicine. t does something that other components don do,
Holtzman and lead author Yong Zhang, Phd, an instructor in pulmonary medicine, suspect that the researchersone-two punch against the virus may explain the large difference in survival rates between control mice
And at 100-fold lower concentrations, all genetically engineered mice survived the infection, compared with only 25-28 percent of the control mice.
Holtzman and his colleagues reported no evidence of autoimmune disease in these mice. Holtzman said a possible explanation for the impressive survival rates
when there is an infection and decrease when there is no infection. Wee not boosting interferon itself,
but the secondary signal that interferon activates, which sets off the rest of the antiviral cascade.
the researchers showed their system was effective in mice with equine encephalitis virus and with strains of influenza virus relevant to public health,
#Mini-kidney organoids re-create disease in lab dishes Stem-cell biology and gene editing advances offer hope for kidney regeneration,
and respond to toxic injury in ways that are similar to kidney tubules in people. major unanswered question was
who led the studies at Brigham and Women Hospital in Boston. He is now an assistant professor of medicine in the Division of Nephrology at the University of Washington and a UW Medicine researcher. nswering this question
he said, as important for understanding the potential of mini-kidneys for clinical kidney regeneration and drug discovery.
To re-create human disease, Freedman and his colleagues used the gene-editing technique called CRISPR.
They engineered mini-kidneys with genetic changes linked to two common kidney diseases, polycystic kidney disease and glomerulonephritis.
The organoids developed characteristics of these diseases. Those with mutations in polycystic kidney disease genes formed balloon like, fluid filled sacks, called cysts, from kidney tubules.
The organoids with mutations in podocalyxin, a gene linked to glomerulonephritis, lost connections between filtering cells. utation of a single gene results in changes kidney structures associated with human disease,
thereby allowing better understand of the disease and serving as models to develop therapeutic agents to treat these diseases,
explained Joseph Bonventre, senior author of the study. He is chief of the Renal Division at Brigham
and Women Hospital and a principal faculty member at Harvard Stem Cell Institute. hese genetically engineered mini-kidneys, Freedman added,
ave taught us that human disease boils down to simple components that can be re-created in a petri dish.
This provides us with faster, better ways to perform linical trials in a dishto test drugs
and therapies that might work in humans. The researchers found that genetically matched kidney organoids without disease-linked mutations showed no signs of either disease.
RISPR can be used to correct gene mutations explained Freedman. ur findings suggest that gene correction using CRISPR may be a promising therapeutic strategy.
In the United states, costs for kidney disease are about 40 billion dollars per year. Kidney disease affects approximately 700 million people worldwide.
Twelve million patients have polycystic kidney disease and two million gave complete kidney failure. Dialysis and kidney transplantation, the only options for patients in kidney failure, can cause harmful side effects and poor quality-of-life. s a result of this new technology,
#Modeling genetic diseases in mini-kidney organoids Harvard Stem Cell Institute researchers at Brigham and Women Hospital have combined cutting-edge,
gene-editing techniques with stem cell science to for the first time successfully model genetic kidney disease in lab-grown, mini-kidneys.
Chief of the Renal Division at Brigham and Women Hospital at Harvard and the study senior author. e were interested in creating disease models using these kidney organoids,
researchers can engineer these mini-kidneys with specific genetic diseases. Using CRISPR technology, Bonventre and colleagues introduced into healthy human pluripotent stem cells either the gene mutations associated with polycystic kidney disease
or those associated with glomerulonephritis. The scientists then coaxed the stem cells to differentiate into mature kidney cells that self-assembled into functional organoids with the physical complications related to their genetic mutations;
for example, organoids with polycystic kidney disease formed cysts, while organoids with changes in a protein that is implicated in glomerulonephritis displayed altered cell to cell interactions,
which could ultimately lead to leaky filters in the mature kidney. utation of a single gene results in changes in kidney structures associated with human disease,
allowing better understanding of the disease and serving as models to develop therapeutic agents to treat these diseases,
Bonventre said. Kidney disease costs the United states 40 billion dollars per year and affects 700 million people worldwide.
Twelve million patients have polycystic kidney disease and two million people have complete kidney failure. Dialysis and kidney transplantation
and poor quality-of-life. hese genetically engineered mini-kidneys have taught us that human disease boils down to simple components that can be re-created in a petri dish,
better ways of testing out drugs and therapies that might work in humans. g
#Snake venom helps hydrogels stop the bleeding Rice university lab employs clotting powers of viper-derived drug,
even in presence of anticoagulants A nanofiber hydrogel infused with snake venom may be the best material to stop bleeding quickly, according to Rice university scientists.
and quickly turns into a gel that conforms to the site of a wound, keeping it closed,
The hydrogel may be most useful for surgeries particularly for patients who take anticoagulant drugs to thin their blood. t interesting that you can take something so deadly
It has been used in various therapies as a way to remove excess fibrin proteins from the blood to treat thrombosis and as a topical hemostat.
This is important because surgical bleeding in patients taking heparin can be a serious problem. The use of batroxobin allows us to get around this problem
The substance used for medicine is produced by genetically modified bacteria and then purified, avoiding the risk of other contaminant toxins.
and injected at the site of a wound, where they reassemble themselves into a gel.
Tests showed the new material stopped a wound from bleeding in as little as six seconds and further prodding of the wound minutes later did not reopen it.
The new work builds upon the Rice lab extensive development of injectable hydrogel scaffolds that help wounds heal
What we did was combine it with the hydrogel wee been working on for a long time. e think SB50 has great potential to stop surgical bleeding, particularly in difficult cases in
#Researchers discover an epilepsy switch A team from the University of Bonn uses a new approach to solve an old mystery Scientists at the University of Bonn
and the Hebrew University of Jerusalem (Israel) have decoded a central signal cascade associated with epileptic seizures.
If the researchers blocked a central switch in epileptic mice, the frequency and severity of the seizures decreased.
it was possible to observe the processes prior to the occurrence of epileptic seizures in living animals.
or her life suffers an epileptic attack, during which the nerve cells get out of their usual rhythm and fire in a very rapid frequency.
Often, a seizure disorder develops after a delay following transient brain damage for example due to injury or inflammation.
these channels act like a doorman to regulate the entry of calcium ions in the nerve cells. t has also been known for a long time that following transient severe brain injury and prior to an initial spontaneous epileptic seizure, the concentration of free zinc ions
Dr. Albert J. Becker from the Institute of Neuropathology of the University of Bonn. The hippocampus, located in the temporal lobe, is a central switching station in the brain.
together with scientists from the departments of Experimental Epileptology and Neuroradiology of the University of Bonn Hospital as well as from the Hebrew University in Jerusalem (Israel), have decoded now a signaling pathway
If the number of zinc ions increases following transient severe brain damage, these ions dock in greater numbers onto a switch, the so-called metal-regulatory transcription factor 1 (MTF1.
This leads to a large increase in the amount of a special calcium ion channel in the nerve cells and overall, this significantly boosts the risk of epileptic seizures.
The scientists demonstrated the fact that the transcription factor MTF1 plays a central role in this connection using an experiment on mice suffering from epilepsy. sing a genetic method,
we inhibited MTF1 in the epileptic mice and as a result, the seizures in the animals were much rarer
This makes it possible to examine the processes which take place during the development of epilepsy in a living animal. f the fluorescence molecules glow,
this indicates that the mouse is developing chronic epileptic seizures, says the molecular biologist Prof. Dr. Susanne Schoch from the department of Neuropathology at the University of Bonn.
The researchers also see a possible potential in this new technology for novel diagnostic approaches in humans.
Hope for new options for diagnosis and treatment The scientists hope that new treatment options will open up for epilepsy patients
therefore increasingly focusing on new therapeutic options that have few side effects, states Prof. Becker. If the zinc ions or the transcription factor MTF1 were inhibited specifically in the brain,
#Researchers discover mechanism that could lead to better ovarian cancer treatment Resistance to chemotherapy is a major problem for those suffering from ovarian cancer problem that prevents a cure from a disease dubbed the ilent killer.
Over the last five years, UGA College of Pharmacy associate professors Mandi Murph and Shelley Hooks have discovered that a type of protein known as RGS10 impacts the effectiveness of ovarian cancer chemotherapy.
Resistance to chemotherapy that was previously very effective is a major roadblock that prevents better outcomes in this disease.
Finding mtor as the mechanism of RGS10 effects could help explain the unique features of chemoresistant cancer cells. hemoresistance to ovarian cancer is what kills women,
Hooks said. t the deadliest gynecologic cancer. Most women with ovarian cancer will have their tumors come back. ithin two years,
85 percent of women will have their cancer come back in a more aggressive form, Murph said. t is during that time that they won respond to the chemotherapy.
Their article ellular deficiency in the RGS10 protein facilitates chemoresistant ovarian cancer, reviews over five years worth of research on RGS10 and was published in Future Medicinal Chemistry.
Their findings on RGS10 have jump-started an interest in the protein as well as created several major research articles on the topic.
GS10 is basically an off switch. It does very little, Murph said. owever, it important because when it gets turned off,
Hooks and Murph tested cells to see how they would react to common chemotherapy medicines.
the chemotherapy for ovarian cancer is more or less effective, Hooks explained. They also found that RGS10 is silenced epigenetically,
she explained. t would mean a better chance of survival for women with ovarian cancer.
it could also be the key to improving treatment of chemotherapy. hemoresistance prevents us from curing the disease,
we can cure ovarian cancer. Currently, platinum chemotherapy drugs, like paclitaxel and carboplatin are used as a one-size-fits-all treatment for ovarian cancer patients.
However, chemoresistance to platinum drugs remains a serious challenge to curing ovarian cancer. Murph recommends more research on mtor inhibitors to see how they can be modified to respond to chemotherapy. ive years ago
this field of RGS10 cancer research didn exist, she said. ut Dr hooks and I have been able to create this area of research
and lead it. Before no one knew or cared about RGS10 effects in cancer cells. Now we have more research that could contribute to improving chemotherapy. h
#Scientists create a new simple method to create stem cells Many new innovative therapies that scientists come up with rely on stem cells,
especially when immune therapy and regenerative medicine are considered. However, these promising research fields are limited by the difficulty of creating stem cells.
since team of scientists from the RIKEN Center for Integrative Medical science (IMS) and other institutions in Japan and Europe have found a relatively simple way to keep immune cells in a multipotent stem cell-like state.
Of course, there had to be experiments with human stem cells to test potential of the method for future therapeutic application.
which could then be used for regenerative medicine or immune therapy. It is hard to overestimate researches like that.
which in turn will most likely spawn many new researches and new therapies for harsh conditions in immune therapy and regenerative medicine.
#Researchers identify a target to stop the spread of ovarian cancer UNSW researchers have discovered a method for disrupting the spread of ovarian cancer, by blocking receptors present on the surface of cancer cells.
and stops the cancer will soon be developed. The findings are published in the journal Oncotarget. With no early detection test and a lack of obvious symptoms in the early stages of ovarian cancer,
women are diagnosed usually late after the cancer has spread to other organs in the body.
Survival rates for ovarian cancer remain at around 40%%with the disease killing 150,000 women worldwide every year.
Very little is known about how and why ovarian cancer spreads and researchers say this is due, in part, to a lack of understanding of the key genes and molecules that initiate and control the progression and growth of ovarian cancer.
That is slowly changing thanks to pioneering work by researchers at UNSW Lowy Cancer Research Centre and their international partners.
In their latest study, the UNSW team found tissue sections taken from ovarian cancer patients had a significantly higher expression of the receptor molecule, Ror2
than tissue sections taken from benign samples. It has previously been shown that its ister receptor Ror1,
is expressed also abnormally in ovarian cancer patients and associated with poor survival. The team also established that concurrently silencing both receptors had a strong inhibitory effect on the proliferation, migration and invasion of the cancer cells.
Lead author, Dr Caroline Ford, said the discovery is exciting because the receptor molecules identified seem to be expressed universally in all epithelial ovarian cancer patients,
not just those with hereditary ovarian cancer. ome drugs are showing promise as a treatment for patients with hereditary ovarian cancer,
however this group makes up only 15%of all ovarian cancer patients, said Dr Ford from UNSW Lowy Cancer Research Centre. or the majority of patients with ovarian cancer,
treatment options have made little progress over the past 30 years and there are currently no targeted therapies available. ersonalised medicine is about targeting treatment to an individual particular genetic profile,
and unlike other cancer types such as breast, this is still very much in its infancy in ovarian cancer treatment.
Researchers believe the Ror amilyof receptor molecules are attractive drug targets for three reasons. First, the receptors are not usually present in normal adult tissues
which means that any drug therapy will likely have few side effects. Second the location of the receptors on the outer surface of cancer cells means they can be accessed easily by drugs.
Third, Ror1 and Ror2 are a specific type of receptor that controls many other genes,
and other members of this specific class of receptors have been targeted successfully for cancer therapies. The cancer drugs Herceptin, Gleevec and Iressa all target receptors of this class.
Separate research has also found that the Ror2 receptor is associated with unfavourable prognosis and tumour progression in cervical cancer.
Dr Ford and her team are currently investigating the role of these receptors in endometrial cancer.
and others suggests that in all gynaecological cancers both Ror1 and Ror2 may be expressed over and is important for disease progression.
Dr Ford believes it is imperative that the relationship of the two receptors is investigated, to further understand their individual and combined roles in the progression
and spread of ovarian cancer. nce we better understand the roles of these receptors we will be in a position to develop a drug to target these receptors and hopefully halt ovarian cancer in its tracks,
The UNSW study focused on epithelial ovarian cancer which is the most common type making up about 90%of tumours of the ovary.
Rarer types of ovarian cancer include germ cell tumours (cancer of the egg making cells of the ovary) and sarcomas t
Those results raised concerns about the use of CRISPR technology in studying human diseases. As a potential solution
Now, a team of researchers from Duke university have shown that these gene-controlling methods are capable of the high degree of precision required for basic science and medical research.
and potentially treating human diseases such as cancer, cardiovascular disease, neurodegenerative conditions and diabetes, which can be driven by mutations in control regions of the genome.
The hope is that overriding one of these switches could uncover and fix the root causes of many diseases.
It could also help researchers understand and change how different people respond to drugs. But only if the CRISPR technique is specific enough.
how those switches differ between individuals and the implications of these insights for human traits and diseases.
Crawford, associate professor of pediatrics, has spent more than a decade developing techniques to identify control regions across the genome
The discovery was published electronically in the journal Ophthalmology. his is the culmination of more than 20 years of work,
first mapped the gene causing North carolina macular dystrophy on chromosome 6 in 1992. The current findings ultimately required an international team of 20 investigators using data from the Human genome Project
and an elaborate computer analysis to identify the actual mutations in INTERGENIC DNA near the PDRM13 gene. ndividuals with this disease have normal eyes except that they fail to form maculas,
Small says. nderstanding how this gene works may help us treat many macular diseases more effectively in the future. cientists already know how to create new retinal cells from a patient skin,
Ltd. s a person who knows firsthand what it is like to lose vision from a rare, inherited eye disease,
and I feel that the prospect of finding a cure is possible and probable in the short term
and certain in the long term. s a public research university working to solve some of society greatest health and medical challenges,
says Jean Robillard, M d.,interim president of the University of Iowa and vice president for medical affairs, University of Iowa Health care s
which is the leading cause of serious long-term disability in adults. The five-year study, performed in an animal model,
and programs in the UCLA department of neurology. he brain has limited a capacity for recovery after stroke,
and to RNA in the brain cells of people with other diseases. They found that GDF10 regulates a unique collection of molecules that improves recovery after stroke.
and Health Conference, being held this week at the University of North carolina School of Global Public health.
The technology transfer company will produce ceramic water disinfection tablets called adidropsfor people in developing countries who have poor access to clean drinking water.
killing pathogens by penetrating cell membranes and disrupting cell division. Extensive testing at UVA labs show that the tablet causes better than a 99.99 percent reduction in such infectious waterborne bacteria as Vibrio cholera
Escherichia coli and other coliform bacteria. The Madidrop also is effective at reducing the infectivity of protozoan pathogens including Giardia lamblia and Cryptosporidium parvum, all of
which cause severe diarrhea, vomiting, dehydration and potential long-term growth and cognition deficiencies. These pathogens are particularly devastating to children and people with AIDS.
Smith said that most of the revenue from sales of Madidrop initially will be fed back into the company to fuel production expansion.
Public service-minded investors will become shareholders and eventually make small profits. Madidrop PBC will provide profit-sharing for employees
Smith said. e are actively raising additional capital to help us bring this innovative public health product to people in need around the world. adidrop PBC administrative office is located on Allied Street in Charlottesville
said Robert Mckenna, Ph d.,a professor of biochemistry and molecular biology in the UF College of Medicine,
San diego, have developed a method that cuts down by half the time needed to make high-tech flexible sensors for medical applications.
Coleman team at UC San diego has been working in medical settings for four years. Their sensors have been used to monitor premature babies, pregnant women,
patients in Intensive care units and patients suffering from sleep disorders. Coleman and colleagues quickly found out that nurses wanted the sensors to come in a peel-and-stick form,
like a medical-grade Band aid. The medium on which the sensors were placed also needed to be approved FDA.
the researchers published their findings in the Proceedings of the National Academy of Sciences. hen you have an infection,
it can be very difficult for a doctor to know which bacteria is infecting you,
A doctor may try one class, and if that doesn work, try another class. We need more broad-spectrum antimicrobial agents.
Such structures have been investigated for various medical applications but because they do not like water, they do not travel well in bodily fluids.
#Nanotechnology could spur new heart treatment for arrthymia A new nanoparticle developed by University of Michigan researchers could be the key to a targeted therapy for cardiac arrhythmia,
and can lead to heart attack and stroke. The disease affects more than four million Americans and causes over 750,000 hospitalizations and 130,000 deaths per year in the United states alone.
The new treatment uses nanotechnology to precisely target and destroy the cells within the heart that cause cardiac arrhythmia.
Their findings are detailed in a new paper published in the journal Science Translational Medicine.
Today, the disease is treated usually with drugs, which can have serious side effects. It can also be treated with a procedure called cardiac ablation that burns away the malfunctioning cells using a high-powered laser that threaded into the heart on a catheter.
The team, led by Jérôme Kalifa, M d.,Ph d.,a cardiologist and U-M Medical school assistant professor at the Center for Arrhythmia Research,
Widely used today to treat cancer, the technique requires doctors to mark unwanted cells with a chemical that makes them sensitive to low-level red light.
The red light then destroys the marked cells while leaving surrounding tissue unharmed. he great thing about this treatment is that it precise down to the level of individual cells,
The major challenge of adapting the therapy to heart cells was developing a nanoparticle small enough to penetrate the tiny pores inside heart capillaries,
yet large enough to carry the chemical payload needed to do the job. n our cancer work,
and a research lab specialist in internal medicine. his cell-selective therapy may represent an innovative concept to overcome some of the current limitations of cardiac ablation,
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