(Reuters) – The Western world’s first gene therapy drug is set to go on sale in Germany with a 1.1 million euro ($1.4 million) price tag, a new record for a medicine to treat a rare disease. The sky-high cost of Glybera, from Dutch biotech firm UniQure (QURE.O) and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model.
(Nature) – The US website ClinicalTrials.gov is the world’s largest repository of clinical-trial information, containing the results of more than 179,000 studies conducted in 187 countries. Yet the database represents only a fraction of the trials that are run. Despite US laws requiring that results be posted to the site, drug companies and academic researchers have found numerous ways to withhold data that show that a drug did not work or had serious side effects.
(Nature) – In the quest to develop personalized cancer therapies, researchers are increasingly examining an individual’s immune response to cancer to find ways to tailor treatments. The shift comes with the emergence of therapies designed to unleash the immune system on cancer cells. Five studies in the 27 November issue of Nature turn to the immune system to investigate which patients are likely to respond to cancer drugs that inhibit the activity of a protein called PD-1, and how tumours trigger immune responses. The approach is in contrast to earlier attempts at personalised therapies that focused on the tumour itself.
(Science) – Researchers have taken several steps toward using stem cells to treat a rare genetic disease that leaves people with skin so fragile it blisters at the slightest touch. A trio of lab and animal studies reported today could help pave the way for a clinical trial for the disorder, called epidermolysis bullosa (EB).
(Nature) – Most journal editors know how much effort it takes to persuade busy researchers to review a paper. That is why the editor of The Journal of Enzyme Inhibition and Medicinal Chemistry was puzzled by the reviews for manuscripts by one author — Hyung-In Moon, a medicinal-plant researcher then at Dongguk University in Gyeongju, South Korea. The reviews themselves were not remarkable: mostly favourable, with some suggestions about how to improve the papers.
(Medical Xpress) – The World Health Organization says 600 new cases of Ebola were reported in the most-affected countries in the past week, more than half of them in Sierra Leone. The U.N. health agency said Wednesday that 15,935 people have been sickened with Ebola as the disease sweeps West Africa and has occasionally popped up elsewhere. Of those, 5,689 have died. It said infections appeared to be stabilizing in Guinea and stabilizing or declining in Liberia, but increasing in Sierra Leone. Officials in Sierra Leone, however, say the outbreak may be on the verge of slowing there.
(Medical Xpress) – Advanced practice clinicians (APCs) use more imaging than primary care physicians (PCPs), according to a study published online Nov. 24 in JAMA Internal Medicine. Danny R. Hughes, Ph.D., from the Harvey L. Neiman Health Policy Institute in Reston, Va., and colleagues compared the use of diagnostic imaging ordered by APCs (specifically, nurse practitioners and physician assistants) versus PCPs following office-based encounters. Data were obtained from 2010 to 2011 Medicare claims for a 5 percent sample of beneficiaries.
(The Guardian) – Metastatic lung cancer is hard to treat. So if there were a treatment for people with the disease that had minimal side effects, could extend not just the quantity of life but also its quality, we’d expect it to be a blockbuster. There is indeed such a treatment, as described in research published in the New England Journal of Medicine in 2010, but it’s not a tablet. It’s palliative care.
(Medical Xpress) – Gene therapy has great potential to treat intractable diseases such as cancer, arterial sclerosis, and Parkinson’s disease. Successful gene therapy requires a gene vector that can deliver the therapeutic gene selectively to the target site. However, the concern is that conventional gene vectors can cause non-selective transfection to normal organs, whereby genetic material infiltrates healthy cells and leads to unfavorable side effects. Now, Nobuhiro Nishiyama and Takahiro Nomoto from Tokyo Institute of Technology, together with Kazunori Kataoka from the University of Tokyo, have developed a novel light-responsive nanomachine as a new type of gene vector.
Synthetic Biology, Genetic Engineering and You: Two-Component Signalling Pathways as Elements in Synthetic Circuit Design
(Phys.org) —Two of the most exciting areas of science and technology, synthetic biology and genetic engineering, have just taken a step towards a brave new future in which large-scale synthetic biological circuits composed of bioengineered logic gates, orthogonal to (that is, independent of) the host in which they operate, will enable a range of applications that include biosensors, gene expression control, cell motility, programmable gene circuits for cell physiology control, and other sophisticated gene circuits.
(BBC) – Burial workers in the Sierra Leonean city of Kenema have dumped bodies in public in protest at non-payment of allowances for handling Ebola victims. The workers, who went on strike over the issue, left 15 bodies abandoned at the city’s main hospital. One of the bodies was reportedly left by the hospital manager’s office and two others by the hospital entrance. The workers have now been sacked for treating the corpses in a “very, very inhumane” way, an official said.
(The Atlantic) – Vancouver, one of North America’s most progressive cities in respect to drug policy, will conduct a groundbreaking experiment: prescription heroin. Following a clinical trial involving 26 subjects, doctors at the city’s Providence Medical Clinic have earned permission to provide doses of the drug to a group of 120 severe addicts. The decision followed a lengthy back and forth with Rona Ambrose, Canada’s federal Health Secretary, who opposes the policy.
(Wired) – “What we’ve learned in the last five years about the underlying genetics is that there are hundreds, if not a thousand or more, different genetic subtypes of autism,” says geneticist David Ledbetter, chief scientific officer at Geisinger Health System in Danville, Pennsylvania. Rather than recruiting people with autism based on outward characteristics, some researchers are turning this flood of genetic information into an advantage: They are classifying children with autism based on their genetics, and thoroughly characterizing each subgroup to map autism’s landscape as a whole.
(Medical Xpress) – With research and development costs for many drugs reaching well into the billions, pharmaceutical companies want more than ever to determine whether their drugs already at market have any hidden therapeutic benefits that could warrant putting additional indications on the label and increase production. Such repurposing of drugs requires evidence of efficacy, and to find candidate drugs for randomized controlled repurposing trials, investigators can use computer simulation and scans of health care billing data, in addition to in vitro and in vivo testing.
(Nanowerk) – Medical researchers from Keele University and Nottingham University have found that magnetic nanoparticles coated with targeting proteins can stimulate stem cells to regenerate bone. Researchers were also able to deliver the cells directly to the injured area, remotely controlling the nanoparticles to generate mechanical forces and maintain the regeneration process through staged releases of a protein growth stimulant.
(Medscape) – Recent offerings from Apple and Facebook to pay for the cost of oocyte cryopreservation as an elective benefit for their female employees are being applauded by some gynecologists and fertility experts as a way to provide women with the ability to choose when they want to have children. Others, however, are more cautious, saying that there isn’t sufficient evidence to support the procedure’s widespread use.
(Slate) – In vitro fertilization was originally intended to help a very specific young, married, and female demographic to conceive. But a lot has changed since Louise Brown, the first test tube baby, was born in 1978. The explosion of single mothers and same-sex parents using the procedure has attracted a lot of attention, but society also needs to grapple with the questions of control, regulation, and access that other emerging reproductive technologies could raise. From pre-implantation genetic screening to exo-wombs, these changes could even evolve our most basic notions of family and society.
(Managed Care Magazine) – Most people don’t lose sleep worrying about health care services provided to inmates in jails or prisons, but maybe they should. Lack of proper coverage is a major cause of recidivism, experts say, but this year the Affordable Care Act (ACA) allows inmates to sign up for Medicaid. This comes with challenges, and health insurance plans involved in managed Medicaid gird themselves to handle an influx of beneficiaries who have historically had little or nothing to do with the health system.
(Medical Xpress) – For many people, talking about end-of-life decisions can be very difficult. Although making choices about health care at the end of life is an important outcome of these conversations, recent research suggests that talking about end-of-life choices with family members in a way that pays attention to how they perceive themselves and maintains your relationship with them may be more important than actually reaching decisions.
(The Times of India) – The much anticipated Assisted Reproductive Technology (ART) Bill, 2014, is said to have specified an upper age-limit for couples wanting to commission surrogacy in India. Likely to be tabled in the winter session of Parliament, the Bill, sources say, specifies that a couple has to be in their early 50s to have a child through a surrogate. The existing Indian Council of Medical Research (ICMR) guidelines do not say anything about an age limit for commissioning couples, leaving it to discretion of individual clinics or doctors.