Latest advances in medical research thread

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'Pocket' diagnosis for Parkinson’s
9 September 2014 ~ Smartphone technology revealed at the British Science Festival could help diagnose and treat Parkinson's disease.
Symptoms of Parkinson's are currently difficult to measure objectively after the patient leaves the doctor's clinic. New smartphone software developed at Aston University will bring the doctor into the patient's pocket to assess their movements and speech at home. Trials are now recruiting online, seeking people with and without the disease. Parkinson's is one of the commonest neurodegenerative diseases, affecting around 127,000 people in the UK.

Diagnosis is based on symptoms including tremor, stiffness and difficulty with movements and speech. However studies have shown that up to 20% of people diagnosed with Parkinson's show no evidence of the disease in post-mortem examinations. "Most people who have the disease will never be objectively measured," explained Dr Max Little, a mathematical researcher with Aston's Nonlinearity and Complexity Research Group. Dr Little's team has developed software that uses the microphone and motion detector of a standard smartphone to provide data to supplement traditional clinical assessment.

Machine learning

Voice change can be an early indicator of Parkinson's. Patients or their family may notice their voice becoming quieter, drifting in pitch and showing vocal tremors. Over the past eight years, Dr Little and colleagues have been developing tools to capture and quantify these changes in the lab and in the home. Using machine learning they are now able to "very accurately separate those who have Parkinson's from those who don't" - with up to 99% agreement with the diagnosis made by the neurologist in clinic. Their most recent study, the Parkinson's Voice Initiative, included 17,000 participants providing voice samples via telephone. Smartphones use accelerometers to measure force in three dimensions. These sensors can be used to collect data on Parkinson's with the phone stowed in the pocket - detecting "freezing of gait" when walking and other characteristic signs of the disease. By integrating this with GPS and other smartphone data, Dr Little's software can perform complex analyses of behaviours including "how many phone calls you make, what's your socialisation behaviour, are you spending a lot of time outside of the house, are you predominantly sitting or walking, how much do you explore your environment" - all of which can contribute to a diagnostic algorithm for Parkinson's.

Parkinson's is one of the commonest neuro-degenerative diseases

Personal diagnostic software raises new issues for diseases like Parkinson's which currently lack disease-modifying treatments. "For the first time, we could do population screening for Parkinson's," explained Dr Michele Hu, consultant neurologist at the Oxford Parkinson's Disease Centre. "But preclinical testing is a massive ethical can of worms - the issues must be carefully pre-empted and thought out. We don't know yet how accurate this could be as a predictor and the only way we will know is by very carefully following at-risk individuals over time." "The ethics clearly has to be worked out - what are we going to use these tools for? What would you like to know? What would you not like to know?" asked Dr Little. "We have made this tool and it's up to the community to decide what to do with it."

Ongoing trials

 

[ScienceRocks]

Activating gene in key organ systems slows aging process throughout the body
By Darren Quick
September 9, 2014

With a typical lifespan of around six weeks, the common fruit fly is one animal that could benefit from a slowing of the aging process. And that's just what a team of biologists at UCLA have achieved by activating a gene called AMPK. Possibly of more interest to us higher life forms is the researchers' belief that the discovery could help delay aging and age-related diseases in humans.

Read More

 

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Milestone reached in building replacement kidneys in the lab

Regenerative medicine researchers at Wake Forest Baptist Medical Center in North Carolina have developed what they say is the most successful method to date to keep blood vessels in new human-sized pig kidney organs open and flowing with blood — a major challenge in the quest to build replacement kidneys in the lab.

http://www.kurzweila...neys-in-the-lab

 

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http://machprinciple...r-than-thought/

A study has linked a type 2 diabetes drug to small increases in people’s lifespans. And a separate study has found greater lifespan boosts in fruit flies, from a treatment thought to work in a similar way.

Both are believed to boost the activity of a gene called AMPK, which in turn facilitates what biologists describe as a tiny “garbage disposal” process within cells of the body.

 

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HOW 2 GENES SYNCH YOUR BODY’S CIRCADIAN CLOCK

Researchers have figured out how two genes keep the circadian clocks in all human cells in time and in proper rhythm with the 24-hour day, as well as with the seasons.

The discovery has been a “long time coming,” says Aziz Sancar, a professor of biochemistry and biophysics at the UNC School of Medicine.

“We’ve known for a while that four proteins were involved in generating daily rhythmicity but not exactly what they did. Now we know how the clock is reset in all cells. So we have a better idea of what to expect if we target these proteins with therapeutics.”

 

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Stanford researchers have developed a "decoy" protein that disrupts metastasis, the process that makes cancer cells spread to other sites in the body:


http://engineering.s...ancer-spreading

 

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Breast cancer drug Perjeta could extend patients’ lives by 15 months – study

A new but very expensive breast cancer drug has shown “unprecedented” benefits in extending the lives of women with an aggressive form of the disease.

Data released by the pharmaceutical company Roche suggests Perjeta could, in combination with other drugs, increase the survival of women with advanced breast cancer by 15 months. The results will raise the stakes in the battle in the UK over the funding of cancer drugs.

http://www.theguardi...end-lives-study

 

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Scientists Coax Human Embryonic Stem Cells Into Making Insulin
Scientists Coax Human Embryonic Stem Cells Into Making Insulin Shots - Health News NPR

A team of Harvard scientists said Thursday that they had finally found a way to turn human embryonic stem cells into cells that produce insulin. The long-sought advance could eventually lead to new ways to help millions of people with diabetes.

Right now, many people with diabetes have to regularly check the level of sugar in their blood and inject themselves with insulin to keep the sugar in their blood in check. It's an imperfect treatment.

"This is kind of a life-support for diabetics," says Doug Melton, a stem-cell researcher at Harvard Medical School. "It doesn't cure the disease and leads to devastating complications of the disease."

People with poorly controlled diabetes can suffer complications such as blindness, amputations and heart attacks.

 

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Broccoli Sprouts Chemical May Treat Autism

Results of a small clinical trial suggest that a chemical derived from broccoli sprouts — and best known for claims that it can help prevent certain cancers — may ease classic behavioral symptoms in those with autism spectrum disorders (ASDs).



The study, a joint effort by scientists at MassGeneral Hospital for Children and the Johns Hopkins Univ. School of Medicine, involved 40 teenage boys and young men, ages 13 to 27, with moderate to severe autism.



In a report published online in PNAS this week, the researchers say that many of those who received a daily dose of the chemical sulforaphane experienced substantial improvements in their social interaction and verbal communication, along with decreases in repetitive, ritualistic behaviors, compared to those who received a placebo.

 

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Creating e-noses using fruit flies
"Surprisingly capable of distinguishing chemicals that they have not evolved to process"

The “nose” (sensors on the antennas) of the common (drosophila melanogaster) could soon be used to detect illegal drugs and explosives, based on new research that has revealed the fly’s impressive ability to detect a wide range of smells, as described in an open-access paper published today (October 15) in the journal Bioinspiration and Biomimetics.

The researchers, from the University of Sussex, Monash University, and CSIRO Australia, hope to integrate the fly sensors into future electronic noses (e-noses), making the devices much faster and more sensitive to a host of chemicals.

The e-noses could be used in applications such as law enforcement, health monitoring, food-quality testing, environmental management, and even geological monitoring (volcanoes) and agriculture (detecting pests).

 

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Melton Lab Cells Could Help Cure Type 1 Diabetes

A team of Harvard researchers led by natural sciences professor and Eliot House Co-Master Douglas A. Melton developed a scalable technique for creating human insulin-producing beta cells in vitro, a major stride towards an effective treatment for type 1 diabetes.

According to a report published in the journal Cell on Oct. 9, this development is the first to yield beta cells that closely resemble normal functioning beta cells, in the quantities necessary for cell transfer and pharmaceutical use.

“This is an important venture for the field of stem cell research, with real potential to transform treatment for diabetics,” said Daniel G. Anderson, an MIT professor working with Melton’s laboratory on preparations for clinical implementation.

Type I diabetes, colloquially referred to as juvenile diabetes, results from an autoimmune destruction of beta cells in the pancreas which normally produce insulin—a hormone which regulates glucose levels in the blood.

 

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Stem cells improve vision (well) enough for horse riding

Seeing is definitely believing when it comes to stem cell therapy. A blind man has recovered enough sight to ride his horse. A woman who could see no letters at all on a standard eye test chart can now read the letters on the top four lines. Others have recovered the ability to see colour. All have had injections of specialised retinal cells in their eyes to replace ones lost through age or disease.

A trial in 18 people with degenerative eye conditions is being hailed as the most promising yet for a treatment based on human embryonic stem cells.

"We've been hearing about their potential for more than a decade, but the results have always been in mice and rats, and no one has shown they're safe or effective in humans long term," says Robert Lanza of Advanced Cell Technology in Marlborough, Massachusetts, the company that carried out the stem cell intervention. "Now, we've shown both that they're safe and that there's a real chance these cells can help people."

Ten years ago, the team at Advanced Cell Technology announced that it had successfully converted human embryonic stem cells into retinal pigment epithelial cells. These cells help keep the eyes' light-detecting rods and cones healthy. But when retinal pigment epithelial cells deteriorate, blindness can occur. This happens in age-related macular degeneration and Stargardt's macular dystrophy.

 

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Surgeons transplant heart that had stopped beating

BBC News - Surgeons transplant heart that had stopped beating

Surgeons in Australia say they have performed the first heart transplant using a "dead heart".

Donor hearts from adults usually come from people who are confirmed as brain dead but with a heart still beating.

A team at St Vincent's Hospital in Sydney revived and then transplanted hearts that had stopped beating for up to 20 minutes.

The first patient who received a heart said she felt a decade younger and was now a "different person".

Hearts are the only organ that is not used after the heart has stopped beating - known as donation after circulatory death.

Beating hearts are normally taken from brain-dead people, kept on ice for around four hours and then transplanted to patients.

'Significant development'
The novel technique used in Sydney involved taking a heart that had stopped beating and reviving it in a machine known as a "heart-in-a-box".

The heart is kept warm, the heartbeat is restored and a nourishing fluid helps reduce damage to the heart muscle.

The first person to have the surgery was Michelle Gribilas, 57, who was suffering from congenital heart failure. She had the surgery more than two months ago.

 

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New treatment approved for rare form of hemophilia
(HealthDay)—Obizur (antihemophilic factor recombinant) has been approved to treat a rare, non-inherited form of hemophilia in adults.
New treatment approved for rare form of hemophilia

Unlike the more common form of hemophilia that's inherited and affects males, acquired hemophilia affects both males and females. The rarer form of the blood disorder occurs when the body's immune system attacks a protein that's necessary for normal blood clotting.

About half the cases of acquired hemophilia are related to other medical conditions, such as pregnancy, cancer or use of certain medications. But about half of cases have no known cause, the FDA said Friday in a news release.

Obizur was evaluated in clinical studies involving 29 adults with acquired hemophilia, who were given Obizur to treat a serious bleeding episode. The trial didn't identify any safety concerns, the FDA said.

 

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Scientists restore hearing in noise-deafened mice, pointing way to new therapies

Scientists have restored the hearing of mice partly deafened by noise, using advanced tools to boost the production of a key protein in their ears.
By demonstrating the importance of the protein, called NT3, in maintaining communication between the ears and brain, these new findings pave the way for research in humans that could improve treatment of hearing loss caused by noise exposure and normal aging.

In a new paper in the online journal eLife, the team from the University of Michigan Medical School's Kresge Hearing Research Institute and Harvard University report the results of their work to understand NT3's role in the inner ear, and the impact of increased NT3 production on hearing after a noise exposure.

Their work also illustrates the key role of cells that have traditionally been seen as the "supporting actors" of the ear-brain connection. Called supporting cells, they form a physical base for the hearing system's "stars": the hair cells in the ear that interact directly with the nerves that carry sound signals to the brain. This new research identifies the critical role of these supporting cells along with the NT3 molecules that they produce.

Scientists restore hearing in noise-deafened mice pointing way to new therapies -- ScienceDaily

 

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Paralysed man walks again after cell transplant
BBC News - Paralysed man walks again after cell transplant

A paralysed man has been able to walk again after a pioneering therapy that involved transplanting cells from his nasal cavity into his spinal cord.

Darek Fidyka, who was paralysed from the chest down in a knife attack in 2010, can now walk using a frame.

The treatment, a world first, was carried out by surgeons in Poland in collaboration with scientists in London.
Details of the research are published in the journal Cell Transplantation.

BBC One's Panorama programme had unique access to the project and spent a year charting the patient's rehabilitation.

Darek Fidyka, 40, from Poland, was paralysed after being stabbed repeatedly in the back in the 2010 attack.

He said walking again - with the support of a frame - was "an incredible feeling", adding: "When you can't feel almost half your body, you are helpless, but when it starts coming back it's like you were born again."

 

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Highly effective new anti-cancer drug shows few side effects in mice

A new drug, known as OTS964, can eradicate aggressive human lung cancers transplanted into mice, according to a report in Science Translational Medicine. The drug, given as a pill or by injection, inhibits the action of a protein that is overproduced by several tumor types, including lung and breast, but is rarely expressed in healthy adult tissues. Without this protein, cancer cells fail to complete the cell-division process and die.

When taken by mouth, the drug was well tolerated with limited toxicity. An intravenous form, delivered within a liposome, was just as effective with fewer side effects. Both approaches -- described in the October 22, 2014 issue of Science Translational Medicine -- led to complete regression of transplanted tumors.

"We identified the molecular target for this drug ten years ago, but it took us nearly a decade to find an effective way to inhibit it," said study author Yusuke Nakamura, MD, PhD, professor of medicine at the University of Chicago and deputy director of the University's Center for Personalized Therapeutics. "We initially screened 300,000 compounds and then synthesized more than 1,000 of them, and found a few that were likely to work in humans. We focused on the most effective. We think we now have something very promising."

OTS964 targets TOPK (T -- lymphokine-activated killer cell -- originated protein kinase), a protein that is produced by a wide range of human cancers and is believed to promote tumor growth. High TOPK expression correlates with poor prognosis in patients with breast and lung cancer.

Highly effective new anti-cancer drug shows few side effects in mice -- ScienceDaily

 

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Toxin-secreting stem cells to treat brain tumors

Scientists from Harvard Medical School have discovered a way of turning stem cells into killing machines to fight brain cancer.

In experiments on mice, the stem cells were genetically engineered to produce and secrete toxins which kill brain tumours, without killing normal cells or themselves.

 

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The genetics of epilepsy: bringing hope to families

Twenty years ago doctors tended to regard most forms of epilepsy as acquired rather than inherited.

In other words, they believed epilepsy was mostly due to injury: the result of things like a crack on the head in a car accident, a bad fall in the playground, a tumour or something having gone wrong in labour. Parents felt responsible, and the resulting guilt was enormous.

 

[Sarah G]

Anything about Pulminary Hypertension in your travels, Matthew?