For example researchers could make synthetic strains of yeast to produce rare medicines such as the malarial drug artemisinin or vaccines like the Hepatitis b vaccine.
Nature News In a milestone for a politically charged field, the US Food and Drug Administration (FDA) has approved the world's first clinical trial of a therapy generated by human embryonic stem cells.
Nature News The US Food and Drug Administration (FDA) has adopted a policy that will govern approval of the use of genetically engineered animals.
to be safe an important step towards the eventual approval of the drug for sale in US markets.
just as it regulates drugs, under the Federal Food, Drug, and Cosmetic Act. The agency argued that the RECOMBINANT DNA used to engineer the animals was in effect an animal'drug'.
'As such, the agency will investigate the safety of the'drug'as well as possible environmental impacts if, for example,
because the decision-making process to approve a new drug is carried largely out behind closed doors.
a fearful memory can be erased by a drug that is usually used to control blood pressure. When memories are laid first down,
and a preliminary study has hinted that the drug might do the same in humans2. But Kindt and her colleagues wanted to know
In addition, the drug treatment didn't affect how well the participants remembered the link between the spiders and the shock.
What's more, the response of a second control group who took the drug but did not go through the reactivation on day 2 was similar to that of the group given the placebo,
suggesting that it is the reconsolidation of the memory the second time around that is affected by the drug.
"says Roger Pitman at Harvard Medical school, who led the original study on the effects of the drug on fear memories in patients with PTSD2.
Those who had taken the drug showed smaller responses to remembering the traumatic event. Rubbing out fear
In addition, the drug treatment didn't affect how well the participants remembered the link between the spiders and the shock.
even though the patient no longer takes antiretroviral drugs. Nature News takes a look at the promises and limitations of the experimental treatment.
The risks involved with a bone-marrow transplant far outweigh those that come with years of antiretroviral drug therapy
even considering the troublesome side effects of these drugs. Before receiving the transplant, recipients are conditioned with drugs
and radiation to destroy their own blood-producing stem cells. The procedure leaves them vulnerable to infection,
Other companies are busy developing additional CCR5-targeting drugs. Unfortunately, maraviroc does not completely prevent the virus from binding to CCR5,
Basically HIV can find its way around the drug and still use CCR5 says Riley,
or may be able to bind to a different region of CCR5 than the drug. Others are trying gene therapy approaches to prevent CCR5 from being made at all.
Children diagnosed with ADHD are treated frequently with drugs such as Ritalin that affect the dopamine system.
"You would identify to the patient that the drug is not working when in reality it is doing well,
if it survives the U s. Food and Drug Administration s grueling approval process. He says it is a precursor to
and Drug Administration this March about the safety studies required to test platelets derived from ips cells in humans,
deliver drugs to specific sites4; and even switch a chemical catalyst on or off5. Molecular machines inspired by biology could eventually enable chemists to build materials with a specific sequence of molecules#a strand of polystyrene in which each component bears one of a range of extra chemical groups, for example.
-gene could be deleted at will by giving them a specific drug. This allowed the researchers to deplete the enzyme during adulthood,
he discovered the short#comings of cholesterol-lowering drugs #and of the clinical advice guiding their use.
Francis, the director of clinical analysis and reporting at the Veterans Health Administration (VA) in WASHINGTON DC, started taking Lipitor (atorvastatin), a cholesterol-lowering statin and the best-selling drug in pharmaceutical history.
In 2011, US doctors wrote nearly 250#million prescriptions for cholesterol-lowering drugs, creating a US$18. 5-billion market, according to IMS Health,
"The drug industry in particular is very much in favour of target-based measures, says Joseph Drozda, a cardiologist and director of outcomes research at Mercy Health in Chesterfield, Missouri."
the big pharmaceutical companies are racing to bring the next LDL-lowering drug to market. In particular, millions of dollars have been poured into drugs that inhibit a protein called PCSK9,
#Stealth nanoparticles sneak past immune system s defences Small man-made peptides can help to sneak drug-bearing nanobeads past the ever-vigilant immune system,
To work effectively, drugs and imaging agents need to get to the diseased cells or tumours where they re needed most.
Although scientists are developing nanoparticles that help to deliver drugs to the right place, all therapeutic molecules face a deadly foe#the immune system.
Researchers at the University of Pennsylvania in Philadelphia have now found a way to stop macrophages from destroying drug-bearing nanoparticles.
Discher and his team also tagged their nanobeads with the anticancer drug paclitaxel. They saw that their peptide-carrying system shrank tumours just as well as the standard paclitaxel carrier, Cremophor,
"It s a new way of trying to get the immune system to prevent phagocytosis of drugs or particles.
The Food and Drug Administration (FDA) Thursday approved the first retinal implant for use in the United states. The FDA s green light for Second sight s Argus II Retinal Prosthesis
which key genes are turned off until activated by a signal compound, permanently turning on production of a drug, for example,
Mice with glioblastomas that were treated with TIC10 in combination with bevacizumab#a drug used against diseases including brain tumours
because it is much smaller than proteins that have previously been tested as TRAIL-based drugs.
but can also thwart anticancer drugs by keeping them out.""We didn t actually anticipate that this molecule would be able to treat brain tumours#that was a pleasant surprise,
In particular, cancer researchers have been developing a number of drugs, including TRAIL-based therapeutics, that work by activating the cellular messenger tumour protein 53 (p53).
Many large biomedical research groups have shelved their TRAIL-based drugs L
#Europe bets on drug discovery Two sites shuttered by the pharmaceutical giant Merck, one in Scotland and one in The netherlands, will soon be humming again with the work of drug discovery.
But the hum will not be business as usual. It will be the sound of a public-private consortium placing a high-stakes wager:
a nearly##200-million (US$271-million) bet that it can boost a languishing pharmaceutical sector by fusing academic innovation with industrial-scale screening,
and aims to fill company pipelines with promising drug candidates. The current dearth of candidates
the pharmaceutical partners will be able to use the library#including molecules from their competitors#in their own drug screens.
such as tumour shrinkage, that the compounds may work as drugs. These molecules can then be licensed back to companies for further development.
The scheme hopes to become self-sustaining by requiring milestone payments as drugs move from laboratory to clinic and from additional partnerships and screening services."
but its goal was not to find potential drugs but to identify biological pathways that might make good drug targets.
The European initiative, by contrast, aims to propel drug development. Both the chemicals in the screening library and results from the assays will be proprietary.
Factory partners will get first right of refusal in licensing deals. Such restrictions are essential
if a compound is ever going to make the long journey from a screening hit to a viable drug candidate,
"To justify the subsequent investments you have to make in hit-to-drug lead programmes,
and focusing on specific drug mechanisms makes his consortium s approach much simpler.""Intellectual-property deals, assays coming from everywhere, multi-institutional agreements.
That s a promising proof of concept for creating a MATE-blocking drug because the inhibitory peptide can do its job without having to enter cells,
appetite and mood#and could provide targets for future drugs to combat depression, migraines or obesity.#"
they might now be able to make breakthroughs in drug discovery and in understanding how the physical structures of the brain produce consciousness,
Christoph Anacker, a neuropharmacologist at King's college London, agrees that the findings are important for drug discovery."
when drugs or neurotransmitters lock into the receptors from outside the cell, they trigger the release of other chemicals inside the cell.
Some drugs bind at more than one receptor, setting off not-fully-understood reactions that can produce unwanted side effects.
To avoid this, researchers want to fine-tune drugs so that they activate only the desired signalling pathway.
some drugs that activate the 2b receptor have been thought to cause heart problems3, and have been withdrawn as unsafe.
They triggered both 1b and 2b with the powerful psychedelic drug LSD and one of its precursors, a migraine drug called ergotamine.
The drugs produced two different chemical cascades#G-protein and ß-arrestin#from the 1b receptor,
Rosenberg points to a collaboration formed in August last year between June's group and the drug giant Novartis,
With further development, the authors say the technology might one day be used for visualisation of the shapes of molecules used in drug development,
or to test the effects of drugs on individual cells. A transistor acts like a switch in an electrical circuit:
He also hopes to test the effects of candidate drugs on the metabolism of human cells, by monitoring ph changes, for example."
because the path from a genetic-disease marker to a profitable drug has not been straight#forward.
or precisely deliver drugs to a particular area when they are needed. No one device can do all of these things simultaneously,
to have an implant with electrodes paired with drug delivery pumps that could sense an oncoming epileptic seizure
which could revolutionize drug discovery and personalized medicine. In a laboratory first, Duke researchers have grown human skeletal muscle that contracts
The lab-grown tissue should soon allow researchers to test new drugs and study diseases in functioning human muscle outside of the human body.
Bursac and Madden studied its response to a variety of drugs, including statins used to lower cholesterol and clenbuterol,
a drug known to be used off-label as a performance enhancer for athletes. The effects of the drugs matched those seen in human patients.
The statins had a dose-dependent response, causing abnormal fat accumulation at high concentrations. Clenbuterol showed a narrow beneficial window for increased contraction.
and experiment to see which drugs would work best for each person. This goal may not be far away;
Bursac is already working on a study with clinicians at Duke Medicinencluding Dwight Koeberl, associate professor of pediatricso try to correlate efficacy of drugs in patients with the effects on lab-grown muscles.
Bioengineered human myobundles mimic clinical responses of skeletal muscle to drugs Existing in vitro models of human skeletal muscle cannot recapitulate the organization and function of native muscle
In response to diversely acting drugs myobundles undergo dose-dependent hypertrophy or toxic myopathy similar to clinical outcomes.
Human myobundles provide an enabling platform for predictive drug and toxicology screening and development of novel therapeutics for muscle-related disorders. ioengineered human myobundles mimic clinical responses of skeletal muscle to drugsby Lauran Madden, Mark Juhas, William
might instead arise from ongoing alterations in synaptic signaling that can be corrected by drugs. Current research indicates that well over 100 distinct gene mutations can manifest as intellectual disability and autism.
as they indicate not only that drug therapies might be effective to improve cognition and behavior in affected individuals,
The findings may one day lead to drugs or other interventions that would lessen the effects of the mutations,
According to the UCSF scientists, their unexpected new findings could reshape basic research on feeding behavior as well as strategies for the development of new anti-obesity drugs.
what we might be manipulating with drugs targeting Agrp pathways. We might be manipulating the decision to go to the grocery store,
but that they can help pinpoint key biological pathways relevant to ALS that then become the focus of targeted drug development efforts,
and mouse models with mutations in TBK1 or OPTN to study ALS disease mechanisms and to screen for drug candidates.
extending the range of options for future drug development. The research was carried out by an international team comprising academics from the Department of chemistry at the University of Cambridge, the Karolinska Institute in Stockholm, Lund University, the Swedish University of Agricultural Sciences,
Their discovery suggests that drugs designed to target NHE9 could help to successfully fight the deadly disease.
Drugs targeting EGFR in these patients are sometimes effective. As they suspected, the team found that alkaline endosomes slow down the removal of EGFR from cell surfaces.
when treated with both a drug countering NHE proteins and a drug against EGFR than when treated by the EGFR-targeting drug alone.
Quinones-Hinojosa says: e are still five to 10 years away from testing this idea in patients,
but feared the discovery could one day be a bonanza for drug lords. Other experts agreed, saying anyone with basic skills could use such a yeast to churn out morphine,
codeine and drugs using a simple home-brew beer kit. The discovery, published in the scientific journal Nature Chemical Biology, comes on the heels of a study published last month in the journal PLOS ONE.
and exude opiates and other therapeutic drugs. The goal is to provide cheaper and possibly less addictive painkillers from a dependable source as compared to the poppy.
as well as antispasmodic drugs like papaverine. The team did not go on to make these drugs, but the process of going from reticuline to codeine
and morphine in yeast is known already. What had been missing in the knowledge chain was getting from tyrosine to reticuline.
when home-brewing drugs could become a reality, "the researchers cautioned.""We're likely looking at a timeline of a couple of years, not a decade or more,
the trio said the way was now open for engineering a yeast strain that would do the whole drug-making trick.
"In addition to tighter lab security and tougher laws, the trio called for yeast strains to be engineered to produce drugs with limited street value,
It could be, for all intents and purposes, the world first digital drug. Will Shanklin of Gizmag first tried Thync at the Consumer electronics Show in January 2015
the electric field can directly couple to the electric circuitry of the neural network. he nanoparticles could be used to deliver drugs to specific parts of the brain.
and anticancer drugs could be delivered and released in this way. Wearable Device Changes Your Moodthe technique could also be used to create a new kind of brain-computer interface.
#'Edible Barcodes'Help Fight Counterfeit Drugs Who knew that the answer to fighting the trillion-dollar global counterfeit drug problem rested in a particle the size of a speck of dust?
Essentially, a drug that has one of these microscopic ignaturescould easily be identified as genuine and not a fake.
they consume 40 percent of all prescription drugs and 35 percent of all over-the-counter drugs.
what the drug interaction would be. With that information you are back in control think how powerful that is. or Wuh
Wuh said the idea of tiny microscopic particles containing data about a drug is farfetched no more than someone 20 years ago saying that a person would have a upercomputer the size of his palm. ut,
whether or not a drug is a counterfeit is eally quite tedious. For example, say law enforcement seizes
what is perceived to be a counterfeit of a drug like Sovaldi, which is used to treat hepatitis.
The law enforcement has to ship it by express mail to a lab that then has to process the drug and run a series of tests.
and can aid in evaluating heart drugs for safety, particularly for pregnant women. The tiny hearts could also serve as models to treat damaged hearts.
but if the drug makes it through clinical trials, it would be needed a much weapon against several increasingly hard-to-treat infections.
a drug long relied upon to treat the obstinate methicillin-resistant Staphylococcus aureus (MRSA), by a factor of 100,
increasingly feared in hospitals for their resistance to existing drugs. But the authors suggest it could be of great value to people fighting MRSA, tuberculosis,
and infections with rare-but-nasty Enterococcus bacterial strains that aren responding to available drugs.
when exposed to drugs. A demonstration of the approach, published online today in the Proceedings of the National Academy of Sciences
The dance of levitating cells can also carry information about how well a drug works.
Researchers could identify which individual cellsrom a tumor or a strain of bacteriaurvive a drug treatment and study them further, something that's not possible with current culture-and-stain tests,
or antiretroviral (ARV) drugs. eservoirsof infected cells that hold latent provirus are a key reason why powerful combinations of ARVS cannot eliminate infections and cure people.
or determine whether drugs have reached successfully their targets. In a paper appearing in the Nov 18 issue of Nature Communications the researchers demonstrate the use of the particles which carry distinct sensors for fluorescence
They have created also nanoparticles carrying the fluorescent agent plus up to three different drugs. This allows them to track
and based on natural or engineered microorganisms that have higher efficacy and fewer side effects than conventional drug treatments.
when we deliver the drug locally. The paper s other senior authors are Robert Langer the David H. Koch Institute Professor at MIT
Targeted deliverychemotherapy drugs are delivered usually via intravenous injection. To make sure that enough reaches a tumor very large quantities must be given often producing side effects.
when drugs are delivered directly into the abdominal cavity. However this is not widely done because it requires implanting a catheter in the patient for 12 weeks
To overcome these delivery issues Cima s lab is working on small implantable devices to deliver drugs for ovarian cancer and bladder disease as well as brain cancer.
The capsules are small cylinders with a 1. 5-milliliter drug capacity; the drug diffuses out through a small hole.
These are experimental devices the researchers selected to test whether the concept of local delivery would work
The properties of the drug molecule have to be taken into account in the design of local therapy that s effective says Cima.
The researchers are also working on using this approach to precisely deliver drugs to very small regions of the brain in hopes of developing better treatments for psychiatric and neurodegenerative disorders.
They envision using this technique to create mice with tumors carrying the same genetic profile as a patient then testing different drugs on them to see which have the best effect.
which causes painful and frequent urination that can interrupt daily life currently requires infusing the drug lidocaine into a patient bladder through a catheter.
But Taris now plans to tailor the platform device to carry other drugs into the bladder to treat various diseases,
the researchers developed a prototype device by using a laser to cut a hole in a silicone tube to add drugs. ight
Moreover, the researchers proved that drugs injected slowly into the bladder for so long could actually be absorbed.
Surprise findings Tarisfirst trial involved implanting an empty device (with no drugs) inside volunteers to test comfort levels.
With the Allergan acquisition funds, Taris will further develop the device to deliver drugs for other bladder diseases,
to difficulties delivering drugs in a sustained way. Last year, Taris entered a research collaboration with Astrazeneca to develop novel treatments for bladder cancer. his device is a platform
overactive or underactive bladder any of these indications where you might want to deliver drugs right to the bladder it can do that.
A member of the MIT Koch Institute, Cima is also working on other drug-delivery projects,
#High-speed biologics screen MIT engineers have devised a way to rapidly test hundreds of different drug-delivery vehicles in living animals making it easier to discover promising new ways to deliver a class of drugs called biologics
because they are significantly larger and more complex than conventional drugs. By combining this work with our previously published high-throughput screening system we are able to create a drug-discovery pipeline with efficiency we had imagined never before adds Tsung-Yao Chang a recent MIT Phd recipient and one of the paper s lead authors.
Peng Shi a former MIT postdoc who is now an assistant professor at the University of Hong kong is the paper s other lead author.#
because their larvae are transparent making it easy to see the effects of genetic mutations or drugs.#
and in zebrafish was 97 percent suggesting that zebrafish are a good model for predicting drug-delivery success in mammals.#
#The ability to identify useful drug delivery nanoparticles using this miniaturized system holds great potential for accelerating our discovery process Anderson says.
Yanik s lab is currently using this technology to find delivery vehicles that can carry biologics across the blood-brain barrier a very selective barrier that makes it difficult for drugs
or chemical agents to drug interdiction discovery of stress fractures in submerged structures and hulls or even faster processing
Each year these superbugs including drug-resistant forms of tuberculosis and staphylococcus infect more than 2 million people nationwide
Lu hopes that both technologies will lead to new drugs to help fight the growing crisis posed by drug-resistant bacteria.
and CRE (carbapenem-resistant Enterobacteriaceae) organisms have evolved to become virtually untreatable with existing drugs. In the new Nature Biotechnology study graduate students Robert Citorik and Mark Mimee worked with Lu to target specific genes that allow bacteria to survive antibiotic treatment.
Both of these carriers successfully spread the CRISPR genes through the population of drug-resistant bacteria.
The researchers then delivered the gene pairs into drug-resistant bacteria and treated them with different antibiotics.
Once scientists understand how these genes influence antibiotic resistance they could try to design new drugs that mimic the effects Lu says.
#An end to drug errors? MIT alumni entrepreneurs Gauti Reynisson MBA 10 and var Helgason HS 08 spent the early 2000s working for companies that implemented medication-safety technologies
Indeed a 2006 report from the Institute of Medicine found that 1. 5 million hospitalized patients in the United states experience medication errors every year due in part to drug-administration mistakes.
and check them against medication records to ensure that a patient gets the right drug and dosage.
This circuit could offer a target for new drugs to help treat conditions such as posttraumatic stress disorder the researchers say In the future one may be able to develop methods that help people to remember positive memories more strongly than negative ones says Susumu Tonegawa the Picower
whether reactivating pleasant memories has any effect on depression in hopes of identifying new targets for drugs to treat depression and posttraumatic stress disorder.
Furthermore these RNA therapies could be combined with more traditional drug therapies for an enhanced effect.
and how it will respond to different drugs. For example patients with glioblastoma a type of brain tumor respond well to a certain class of drugs known as alkylating agents
if the DNA-repair gene MGMT is silenced by epigenetic modification. A team of MIT chemical engineers has developed now a fast reliable method to detect this type of modification known as methylation which could offer a new way to choose the best treatment for individual patients.
It can take up to a year to determine the function of a single gene which has slowed efforts to develop new more targeted drugs and vaccines.
and boost drug-development efforts says Jacquin Niles an associate professor of biological engineering at MIT. Even though we ve sequenced the entire genome of Plasmodium falciparum half of it still remains functionally uncharacterized.
whether it s drugs or vaccines says Niles the senior author of a paper describing the technique in the Aug 10 online edition of Nature Methods.
and artemisin but the parasite is becoming more resistant to these drugs. There is an urgent need to develop new drugs
but potential genetic targets are hard to identify. In animals such as mice it is fairly routine to study gene functions by deleting a target gene or replacing it with an artificial piece of DNA.
which could generate new drug and vaccine targets. I think the impact could be quite huge Niles says.
Using CRISPR to generate tumors should allow scientists to more rapidly study how different genetic mutations interact to produce cancers as well as the effects of potential drugs on tumors with a specific genetic profile.
No fundamentally new drugs have been introduced since the 1950s. All but a handful of pharmaceutical companies have abandoned the pursuit of new treatments
As president of Merck Research Laboratories Scolnick led the development of the first drugs to effectively combat HIV;
the first drugs to effectively treat high cholesterol statins; the first vaccine against cervical cancer; and many other breakthroughs.
and Crohn s disease and translated that knowledge into descriptions of the underlying biological processes a critical step in the development of rationally designed drugs.
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