Posts tagged treatment
Posts tagged treatment
Researchers from the Universitat Autònoma de Barcelona (UAB), led by Fàtima Bosch, have shown for the first time that it is possible to cure diabetes in large animals with a single session of gene therapy. As published this week in Diabetes, the principal journal for research on the disease, after a single gene therapy session, the dogs recover their health and no longer show symptoms of the disease. In some cases, monitoring continued for over four years, with no recurrence of symptoms.
The therapy is minimally invasive. It consists of a single session of various injections in the animal’s rear legs using simple needles that are commonly used in cosmetic treatments. These injections introduce gene therapy vectors, with a dual objective: to express the insulin gene, on the one hand, and that of glucokinase, on the other. Glucokinase is an enzyme that regulates the uptake of glucose from the blood. When both genes act simultaneously they function as a “glucose sensor”, which automatically regulates the uptake of glucose from the blood, thus reducing diabetic hyperglycemia (the excess of blood sugar associated with the disease).
As Fàtima Bosch, the head researcher, points out, “this study is the first to demonstrate a long-term cure for diabetes in a large animal model using gene therapy.”
This same research group had already tested this type of therapy on mice, but the excellent results obtained for the first time with large animals lays the foundations for the clinical translation of this gene therapy approach to veterinary medicine and eventually to diabetic patients.
The study was led by the head of the UAB’s Centre for Animal Biotechnology and Gene Therapy (CBATEG) Fàtima Bosch, and involved the Department of Biochemistry and Molecular Biology of the UAB, the Department of Medicine and Animal Surgery of the UAB, the Faculty of Veterinary Science of the UAB, the Department of Animal Health and Anatomy of the UAB, the Spanish Biomedical Research Centre in Diabetes and Associated Metabolic Disorders (CIBERDEM), the Children’s Hospital of Philadelphia (USA) and the Howard Hughes Medical Institute of Philadelphia (USA).
A new survey of stroke survivors has shown that those with post-traumatic stress disorder (PTSD) are less likely to adhere to treatment regimens that reduce the risk of an additional stroke. Researchers found that 65 percent of stroke survivors with PTSD failed to adhere to treatment, compared with 33 percent of those without PTSD. The survey also suggests that nonadherence in PTSD patients is partly explained by increased ambivalence toward medication. Among stroke survivors with PTSD, approximately one in three (38 percent) had concerns about their medications. Results of the study, led by Columbia University Medical Center researchers, are published today in the British Journal of Health Psychology.
According to data from the American Stroke Association, nearly 795,000 Americans each year suffer a new or recurrent stroke. Stroke is the fourth-leading cause of death and the top cause of disability in the United States. Survivors of strokes are often prescribed treatment regiments, including antiplatelet agents, antihypertensive agents, and statins, which help reduce the risk of subsequent strokes. Previous research has shown that PTSD triggered by medical events—which affects 18 percent of stroke survivors—may impair recovery.
“Unfortunately, too many stroke survivors are not compliant with these regimens, even though we know that adherence to post-stroke treatment regimens is one of the most important components of reducing the risk of a future stroke,” said Ian M. Kronish, MD, MPH, assistant professor of medicine (Center for Behavioral Cardiovascular Health) and one of the study’s authors.
“For those with PTSD, this study shows that concerns about medications are a significant barrier to treatment adherence. Stroke survivors should be assessed for concerns about medications and PTSD symptoms, so that interventions may be introduced as early as possible to get patients back on track to avoid future stroke events.”
A drug used for decades to treat high blood pressure and other conditions has shown promise in a small clinical trial for autism. The drug, bumetanide, reduced the overall severity of behavioral symptoms after 3 months of daily treatment. The researchers say that many parents of children who received the drug reported that their children were more “present” and engaged in social interactions after taking it. The new findings are among several recent signs that treatments to address the social deficits at the core of autism may be on the horizon.
Several lines of evidence suggest that autism interferes with the neurotransmitter GABA, which typically puts a damper on neural activity. Bumetanide may enhance the inhibitory effects of GABA, and the drug has been used safely as a diuretic to treat a wide range of heart, lung, and kidney conditions. In the new study, researchers led by Yehezkel Ben-Ari at the Mediterranean Institute of Neurobiology in Marseille, France, recruited 60 autistic children between the ages of 3 and 11 and randomly assigned them to receive either a daily pill of bumetanide or a placebo. (Neither the children’s parents nor the researchers who assessed the children knew who received the actual drug).
As a group, those who got bumetanide improved by 5.6 points on a 60-point scale that’s often used to assess behaviors related to autism, the researchers report today in Translational Psychiatry. That was enough to nudge the group average just under the cutoff for severe autism and into the mild to medium category. The study did not look directly at whether the drug improved all symptoms equally or some more than others. “We have some indications that the symptoms particularly ameliorated with bumetanide are the genuine core symptoms of autism, namely communication and social interactions,” Ben-Ari says. More work will be needed to verify that impression. Ben-Ari says his team is now preparing for a larger, multicenter trial in Europe.
New research suggests that the molecular mechanism leading to schizophrenia may be different in patients who fail to respond to anti-psychotic medication compared to patients who do respond.
The research, from King’s College London’s Institute of Psychiatry may help explain why up to one third of patients with schizophrenia do not respond to traditional anti-psychotic medication.
Schizophrenia is known to be associated with an overactive dopamine system, meaning that the brain processes abnormally high levels of dopamine. Traditional dopamine-blocking anti-psychotic medication attempts to normalise this process. However, approximately one third of patients with schizophrenia do not respond to this treatment, and until now, no study has examined whether dopamine abnormality is present in patients resistant to antipsychotic treatment.
The study was led by Dr Arsime Demjaha, Dr Oliver Howes, Professor Shitij Kapur, Professor Sir Robin Murray and Professor Philip McGuire from King’s Institute of Psychiatry and published in the American Journal of Psychiatry.
Dr Arsime Demjaha and co-authors, say: ‘Despite considerable scientific and therapeutic progress over the last 50 years, we still do not know why some patients with schizophrenia respond to treatment whilst others do not. Treatment resistance in such a disabling condition is one of the greatest clinical and therapeutic challenges to psychiatry, significantly affecting patients, their families and society in general.’
The authors conclude: ‘Our findings suggest that there may be a different molecular mechanism leading to schizophrenia in patients who do not respond to anti-psychotic medication. Identifying the precise molecular pathway particularly in these patients is of utmost importance and will help inform the development of much-needed novel treatments.’
Researchers used PET scan imaging to investigate dopamine synthesis capacity in 12 patients with schizophrenia who did not respond to treatment, 12 who did, and 12 healthy controls. They found that schizophrenia patients whose illness was resistant to antipsychotic treatment have relatively normal levels of dopamine synthesis capacity which would explain why the dopamine blocking anti-psychotic medication was not effective in this group.
However, the authors add that the findings need to be replicated in larger samples before the research can affect clinical practice. They add that future research will need to focus on long-term prospective studies of patients who have never taken anti-psychotics to determine whether presynaptic dopamine synthesis capacity was normal in patients in the treatment-resistant group at the onset of their illness, and predates antipsychotic exposure.
A new drug may bring help for people with insomnia, according to a study published in the November 28, 2012, online issue of Neurology®, the medical journal of the American Academy of Neurology.
The drug, suvorexant, blocks the chemical messengers in the brain called orexins, which regulate wakefulness. Other drugs for insomnia affect different brain receptors.
Taking the drug suvorexant increased the amount of time people spent asleep during the night, according to the study. The study involved 254 people ages 18 to 64 who were in good physical and mental health but had insomnia that was not due to another medical condition.
The participants took either the drug or a placebo for four weeks, then switched to the other treatment for another four weeks. The participants spent the night in a sleep laboratory with their sleep monitored on the first night with each treatment and then again in the fourth week of each treatment.
While taking the drug, participants’ “sleep efficiency,” which reflects the total amount of time they slept during a fixed, eight hour time in bed, improved by 5 to 13 percent compared to those taking the placebo. They also experienced 21 to 37 fewer minutes awake during the night after they had fallen asleep than those who took the placebo. “This study provides evidence that suvorexant may offer a successful alternative strategy for treating insomnia,” said study author W. Joseph Herring, MD, PhD, of North Wales, Penn., Executive Director of Clinical Research with Merck, the maker of suvorexant, and a member of the American Academy of Neurology. “Suvorexant was generally well-tolerated, and there were no serious side effects.”
Herring said larger, longer studies have recently been conducted on suvorexant, along with studies to determine whether the drug could be safe and effective for elderly people, who make up a large percentage of those suffering from insomnia.
Combination of two pharmaceuticals proves effective in the treatment of multiple sclerosis
A new substance class for the treatment of multiple sclerosis and other neurodegenerative diseases now promises increased efficacy paired with fewer side effects. To achieve this, a team of scientists under the leadership of Prof. Gunter Fischer (Max Planck Research Unit for Enzymology of Protein Folding, Halle/Saale, Germany) and Dr. Frank Striggow (German Center for Neurodegenerative Diseases (DZNE)) have combined two already approved pharmaceutical substances with each other using a chemical linker structure. The objectives of this combination are to ensure maximum brain cell protection on the one hand and the suppression of unwanted side effects on the other. The new class of substances has now been registered with the European Patent Office as the DZNE’s first patent in the form of a joint patent application with the Max Planck Research Unit. “The patent approval process can take several years. During this phase we are planning to conclude the pre-clinical development. It is our aim to start with clinical research and development at the earliest possible time. Overall, we have identified substantial therapeutic potential as far as chronic and age-related neurodegenerative diseases are concerned,” comments Dr. Frank Striggow.
Johns Hopkins researchers report the successful use of a form of MRI to identify what appears to be a key biochemical marker for cognitive impairment in the brains of people with multiple sclerosis (MS). In follow-up experiments on mice with a rodent form of MS, researchers were able to use an experimental compound to manipulate that same marker and dramatically improve learning and memory.
Half of people with MS experience learning and memory problems, for which there is no approved treatment, along with movement abnormalities that characterize the debilitating autoimmune disorder.
“We have a potentially novel treatment for cognitive impairment in MS, a devastating condition on the rise that affects at least 400,000 people in the United States,” says study leader Adam I. Kaplin, M.D., Ph.D., an assistant professor of psychiatry and behavioral sciences and neurology at the Johns Hopkins University School of Medicine.
Kaplin cautions that the treatment has so far been used only in mouse models of MS and is years away from clinical trials in people.
Nevertheless, he says, the research, described in the Proceedings of the National Academy of Sciences published online on Nov. 19, has the potential to speed development of new drugs to treat cognitive impairment not only in MS patients, but also in patients with Alzheimer’s disease and other neurological conditions.
Mayo Clinic Researchers Develop New Tools to Better Treat ADHD Patients in Early Stages
Mayo Clinic researchers are presenting new findings on the early treatment of child and adolescent attention deficit hyperactivity disorder this week at the American Academy of Childhood and Adolescent Psychiatry annual meeting in San Francisco. They include a method to get better input from parents and teachers of children who are being diagnosed with ADHD for the first time — allowing for more effective treatment upon the first consultation. Researchers also showed how a tool can help clinicians better diagnose and treat children who have both ADHD and oppositional defiance disorder.
(Image credit: Psyc3330 w11, Wikimedia Commons)
In an Australian first, researchers are studying Magnetic Seizure Therapy (MST) as an alternative treatment for the 30 per cent of patients suffering from depression who don’t respond to traditional treatment.
The treating team; Anne Maree Clinton, Dr Kate Hoy and Professor Paul Fitzgerald with the MST machine
The study, led by researchers from the Monash Alfred Psychiatry Research Centre (MAPrc) and funded by beyondblue and the National Health and Medical Research Council (NHMRC), has been published in two leading journals: Psychiatry Research: Neuroimaging and Depression and Anxiety. Both papers are a result of the same study.
MAPrc Deputy Director Professor Paul Fitzgerald, who led the study, said depression was a common and disabling disorder, affecting up to one in five Australians during their lifetime.
“Electroconvulsive Therapy (ECT) is one of the only established interventions for treatment resistant depression,” Professor Fitzgerald said.
“But use of ECT is limited due to the presence of memory-related side effects and associated stigma.”
For this reason, the MAPrc researchers began exploring new treatment options. MST is a brain-stimulation technique that may have similar clinical effects to ECT without the unwanted side effects.
“In MST, a seizure is induced through the use of magnetic stimulation rather than a direct electrical current like ECT. Magnetic fields are able to pass freely into the brain, making it possible to more precisely focus stimulation,” Professor Fitzgerald said.
“By avoiding the use of direct electrical currents and inducing a more focal stimulation, it is thought that MST will result in an improvement of depressive symptoms without the memory difficulties seen with ECT.”
Research is still at an early stage and MST is only available in a handful of locations worldwide. The MAPrc is the only centre in Australia conducting trials with this therapy.
The study found that MST resulted in an overall significant reduction in depression symptoms; 40 per cent showed overall improvement and 30 per cent showed some improvement. None of the trial participants complained of cognitive side effects.
“MST shows antidepressant efficacy without apparent cognitive side effects. However, substantial research is required to understand the optimal conditions for stimulation and to compare MST to established treatments, including ECT,” Professor Fitzgerald said.
“In order to accurately assess the comparable efficacy of MST to ECT, large-scale randomised controlled trials are required. There remains considerable work to be done before statements of the relative efficacy of these treatments can be made.”
Could Stem Cells Treat Autism? Newly Approved Study May Tell
Autism researchers have been given the go-ahead by the U.S. Food and Drug Administration to launch a small study in children with autism that evaluates whether a child’s own umbilical cord blood may be an effective treatment.
Thirty children with the disorder, aged 2 to 7, will receive injections of their own stem cells from umbilical cord blood banked by their parents after their births. All of the cord blood comes from the Cord Blood Registry, the world’s largest stem cell bank.
Scientists at Sutter Neuroscience Institute, in Sacramento, Calif., said the placebo-controlled study will evaluate whether the stem cell therapy helps improve language and behavior in the youngsters.
There is anecdotal evidence that stem cell infusions may have a benefit in other conditions such as cerebral palsy, said lead study investigator Dr. Michael Chez, director of pediatric neurology at the institute.
“We’re hoping we’ll see in the autism population a group of patients that also responds,” Chez said. Other autism and stem cell research is going on abroad, but this study is the first to use a child’s own cord blood stem cells.
Chez said the study will involve only patients whose autism is not linked to a genetic syndrome or brain injury, and all of the children will eventually receive the stem cells.