Friday 23rd December 2016- The World Health Organisation: Final trial results confirm Ebola vaccine provides high protection against disease

‘An experimental Ebola vaccine was highly protective against the deadly virus in a major trial in Guinea, according to results published today in The Lancet[*]. The vaccine is the first to prevent infection from one of the most lethal known pathogens, and the findings add weight to early trial results published last year.

The vaccine, called rVSV-ZEBOV, was studied in a trial involving 11 841 people in Guinea during 2015. Among the 5837 people who received the vaccine, no Ebola cases were recorded 10 days or more after vaccination. In comparison, there were 23 cases 10 days or more after vaccination among those who did not receive the vaccine.

The trial was led by WHO, together with Guinea’s Ministry of Health, Medecins sans Frontieres and the Norwegian Institute of Public Health, in collaboration with other international partners.

“While these compelling results come too late for those who lost their lives during West Africa’s Ebola epidemic, they show that when the next Ebola outbreak hits, we will not be defenceless,” said Dr Marie-Paule Kieny, WHO’s Assistant Director-General for Health Systems and Innovation, and the study’s lead author.’

*The final interpretation from the referenced Lancet article:

‘The results add weight to the interim assessment that rVSV-ZEBOV offers substantial protection against Ebola virus disease, with no cases among vaccinated individuals from day 10 after vaccination in both randomised and non-randomised clusters.’

Related Articles:

Final trial results confirm Ebola vaccine provides high protection against disease

Efficacy and effectiveness of an rVSV-vectored vaccine in preventing Ebola virus disease: final results from the Guinea ring vaccination, open-label, cluster-randomised trial (Ebola Ça Suffit!)- The Lancet

Ebola vaccine is safe and effective, scientists declare after trials- The Guardian

Successful Ebola vaccine will be fast-tracked for use- BBC News

The new ‘100% effective’ Ebola vaccine owes a debt to the scientists who beat smallpox- The Independent

Thursday 12th May 2016- The World Health Organisation:Rapid diagnostic test and shorter, cheaper treatment signal new hope for multidrug-resistant tuberculosis patients

‘New WHO recommendations aim to speed up detection and improve treatment outcomes for multidrug resistant tuberculosis (MDR-TB) through use of a novel rapid diagnostic test and a shorter, cheaper treatment regimen.

“This is a critical step forward in tackling the MDR-TB public health crisis,” said Dr Mario Raviglione, Director of WHO’s Global TB Programme. “The new WHO recommendations offer hope to hundreds of thousands of MDR-TB patients who can now benefit from a test that quickly identifies eligibility for the shorter regimen, and then complete treatment in half the time and at nearly half the cost.”’

‘At less than US$ 1000 per patient, the new treatment regimen can be completed in 9–12 months. Not only is it less expensive than current regimens, but it is also expected to improve outcomes and potentially decrease deaths due to better adherence to treatment and reduced loss to follow-up.

The conventional treatment regimens, which take 18–24 months to complete, yield low cure rates: just 50% on average globally. This is largely because patients find it very hard to keep taking second-line drugs, which can be quite toxic, for prolonged periods of time. They therefore often interrupt treatment or are lost to follow-up in health services.’

‘WHO’s recommendations on the shorter regimens are based on initial programmatic studies involving 1200 patients with uncomplicated MDR-TB in 10 countries . WHO is urging researchers to complete ongoing randomised controlled clinical trials in order to strengthen the evidence base for use of this regimen.’

‘The most reliable way to rule out resistance to second-line drugs is a newly recommended diagnostic test for use in national TB reference laboratories. The novel diagnostic test – called MTBDRsl – is a DNA-based test that identifies genetic mutations in MDR-TB strains, making them resistant to fluoroquinolones and injectable second-line TB drugs.’

‘This test yields results in just 24-48 hours, down from the 3 months or longer currently required. The much faster turnaround time means that MDR-TB patients with additional resistance are not only diagnosed more quickly, but can quickly be placed on appropriate second-line regimens. WHO reports that fewer than 20% of the estimated 480 000 MDR-TB patients globally are currently being properly treated.’

See:

Rapid diagnostic test and shorter, cheaper treatment signal new hope for multidrug-resistant tuberculosis patients

Friday 26th February 2016- The Independent: Asthma: Half of children diagnosed with the respiratory disease may not have it, study suggests

‘More than half of the children being treated for asthma might not actually have the condition, new research suggests.

A study, published in the British Journal of General Practice, found 53 per cent of children had no clinical signs of asthma despite being diagnosed at one of four medical centres in the Netherlands, whose healthcare system is widely regarded as one of the best in Europe.

In the UK last year, researchers found that a third of adults diagnosed with asthma did not actually have it.

Dr Ingrid Looijmans-van den Akker, one of the scientists behind the Dutch research, told The Daily Telegraph: “Over-diagnosis of asthma was found in more than half of the children, leading to unnecessary treatment, disease burden, and impact on their quality of life.

“Previous studies have indicated that asthma is over-diagnosed in children. However, the scale of the over-diagnosis has not been quantified.

“Only in a few children was the diagnosis of asthma confirmed using lung function tests, despite this being recommended in international guidelines. Over-diagnosis gives rise to over-prescription and incorrect use of medication, and to anxiety in parents and children.”

The UK’s National Institute of Clinical Excellence (Nice) has warned that doctors have too often tended to diagnose asthma based on a history of wheezing, coughs and other breathing problems, rather than clinical tests.

Professor Mark Baker, director of clinical practice at Nice, said it was developing new advice on how to properly diagnose the condition.

“As part of this work, Nice is inviting GP practices to take part in a project to check the feasibility of some diagnostic tests that Nice proposes to recommend,” he said.’

Related Articles:

Asthma: Half of children diagnosed with the respiratory disease may not have it, study suggests

Half a million children with asthma may not actually have condition- The Telegraph

Friday 26th February 2016- The Telegraph: First drug to reverse Huntington’s disease begins human trials

‘A drug which appears to reverse Huntington’s disease is being trialled in humans after proving successful in monkeys and mice.

The new drug, called IONIS-HTTRx, silences the gene known to be responsible for the production of a protein which causes Huntington’s.

The disease is a hereditary condition which damages nerve cells in the brain and effects around 7,000 people in Britain. It causes uncontrolled movements, loss of intellectual abilities, emotional problems and eventually death.

Now scientists have shown that it is not only possible to halt the disease but to reverse the damage.

“It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease,” said clinical study principal investigator Dr Blair Leavitt, of the University of British Columbia in Vancouver.

“Right now we only have treatments that work on the symptoms of the disease.”

Huntington’s is caused by a mutated HTT gene, and everyone who inherits the genetic defect will eventually develop the disease.

Researchers have been trying to develop a drug which acts like a dimmer switch, turning the gene down so that it can no longer produce the devastating protein which causes brain damage.’

‘When they tested IONIS-HTTRx on mice with the disease their motor function improved within a month and within two months their health was restored to normal. In monkeys the drug was found to decrease the HTT protein throughout the central nervous system by 50 per cent.

The drug is delivered into the cerebral spinal fluid via lumbar injection, as antisense drugs do not cross the blood brain barrier – a protective sheath that prevents toxins entering the brain.

The drug is now being trialled in humans in low doses to check that it is safe for larger trials into its efficacy to begin.

The research was presented at the American Academy of Neurology’s 68th Annual Meeting in Vancouver, Canada, April 15 to 21, 2016.’

See:

First drug to reverse Huntington’s disease begins human trials

Thursday 25th February 2016- The Independent: Pancreatic cancer is four different diseases, study finds

‘A new study which has shown that pancreatic cancer is four separate diseases has been hailed as “incredibly exciting.”

The team at the University of Glasgow said the study was as a “launch pad” for finding new treatments for the disease.

Around 8,000 people in the UK are diagnosed with the cancer which affects the large gland in the digestive system each year. It is particularly difficult to diagnose as it does not show symptoms in the early stages.

The way in which pancreatic cancer is treated has not developed greatly for two decades, said Dr Peter Bailey, one of the study authors, and compared current methods to “hitting the disease with a mallet with your eyes closed.”

Around a fifth of those with the disease survive more than a year after being diagnosed, while less than 5 per cent living after five years, and 1 per cent after a decade.

The team at the University of Glasgow studied around 456 pancreatic cancer tumours for the research published in the journal ‘Nature’.

Scientists were able to categorise the disease into four different sub-types: squamous, pancreatic progenitor, immunogenic and ADEX.

Professor Sean Grimmond, who led the study, said: “This study demonstrates that pancreatic cancer is better considered as four separate diseases, with different survival rates, treatments and underlying genetics.”

“Knowing which sub-type a patient has would allow a doctor to provide a more accurate prognosis and treatment recommendations.

He explained that cancer drugs that doctors use or are in development can target the disease which are similar to other forms of cancer.

For example, some types of pancreatic cancer are associated with mutations normally found in colon cancer or leukaemia, he said.

Pancreatic Cancer UK described the findings as “incredibly exciting”.

Leanne Reynolds, head of research at the charity, said the findings meant that in the future “the right patients can be given the right treatment at the right time”.’

Related Articles:

Pancreatic cancer is four different diseases, study finds

Major insight into killer pancreatic cancer- BBC News

Scientists discover pancreatic cancer is four separate diseases- The Guardian

Thursday 25th February 2016- The Guardian: Lab-created sperm breeds healthy mice, raising hopes for end to male infertility

‘A handful of healthy mice made from sperm cells created in the lab have been hailed as a milestone in research that could ultimately provide new treatments for male infertility.

Scientists in China created the mice by fertilising normal mouse eggs with early-stage sperm cells that were manufactured from embryonic stem cells plucked from the animals.

To make the sperm, the team nudged mouse stem cells through a complex series of steps known as meiosis that must be performed with extreme care to ensure the sperm develop properly.

Despite years of work, scientists have never managed to pull off the same trick with human stem cells, but the latest study could provide fresh impetus to the effort, said Jiahao Sha, director of the Laboratory of Reproductive Medicine at Nanjing Medical University in China.

The creation of sperm and eggs – known as germ cells – for use in IVF raises particular safety issues, because any faults in their genetic material could harm not only the children born from the treatment, but all their future descendants.

“If it works, human germ cells could possibly be produced. However, in the current stage, ethics should be concerned, and any possible risks ruled out,” said Sha, who led the team

The scientists believe that the cautious, stepwise production of the sperm cells was crucial for the mice to be born healthy and fertile. The mice went on to mate and have fertile offspring of their own, Sha said.’

‘Independent scientists welcomed the landmark achievement but cautioned that the creation of human sperm to treat infertile men was a distant prospect fraught with concerns over safety, ethics and legality.’

Related Articles:

Lab-created sperm breeds healthy mice, raising hopes for end to male infertility

Toward Making Sperm in the Lab- The Scientist

Lab-grown sperm makes healthy offspring- BBC News

Sperm grown in lab could allow infertile men to have children- The Telegraph

Wednesday 24th February 2016- The Guardian: Radical cancer treatment seeks to control rather than destroy tumours

‘A radical approach to cancer treatment which keeps tumours under control rather than destroying them completely may be more effective than conventional therapies, scientists say.

The idea draws on Charles Darwin’s 150-year-old theory of evolution and recasts tumours as diverse ecosystems of cells which can be manipulated to prevent them from growing out of control.

The strategy is highly experimental and has only been tested in mice, but successful trials in humans could usher in a transformation in cancer care, where patients live healthy lives with tumours that are constantly kept in check by low doses of medicine.

Routine cancer treatment assumes that patients do best when a therapy kills off the maximum number of malignant cells in their bodies. But tumours are collections of different cells and some are more resistant to drugs than others. A dose of chemotherapy will typically leave drug-resistant cells behind. Unencumbered by their neighbours, they can rapidly grow back when the treatment stops.

Scientists in the US wondered what would happen if anticancer drugs were used to shrink tumours without destroying the diversity of cells inside them. They hoped that the surviving cancer cells would stop more aggressive, drug-resistant ones from taking over, just as grass can prevent moss running wild in a garden.

Writing in the journal Science Translational Medicine, Robert Gatenby at the H. Lee Moffitt Cancer Center in Tampa, Florida, describes how his team tested the idea with the chemotherapy drug paclitaxel (or taxol) in mice with two different forms of breast cancer.

When the mice were given standard chemotherapy, their tumours shrank, but grew back as soon as the treatment ended. For the new therapy, mice were given initially high doses of drugs followed by ever lower doses. The strategy appeared to be more effective than standard treatment. Giannoula Klement, a cancer specialist at Tufts University School of Medicine in Boston, who was not involved in the work, said that in about 60% of the mice, the cancer treatment could be withdrawn completely with no further growth of the tumours.

In an accompanying article, Klement argues that to beat cancer, it must be considered as an ecosystem of different cells. “The likelihood that a ‘magic bullet’ against cancer is going to be found is nil. If we have learned anything from the eco-evolutionary model it is that unless we respect these eco-evolutionary laws, we will continue to play a cat and mouse game with cancer,” she writes.

Instead of eradicating cancer, the new goal for doctors needs to be prevention of cancer disease, she adds. “We need to stabilise tumour growth and enable gradual, controlled regression over time.”’

Related Articles:

Radical cancer treatment seeks to control rather than destroy tumours

Don’t kill cancer, learn to live with it, say scientists- The Telegraph

Thursday 4th February 2016- The Independent: World Cancer Day: Deaths caused by disease fall but number of cases rise

‘Cancer death rates in the UK have fallen by almost 10 per cent in the last decade, although the number of cases is still going up, new figures show.

Analysis by Cancer Research UK found that 284 out of every 100,000 people in the UK died from cancer in 2013 (around 162,000 people), down from 312 in every 100,000 a decade ago.

The slump is largely due to improvements in the detection, diagnosis and treatment of cancer.

A breakdown of the sexes shows men’s death rates have fallen by 12 per cent over the period, while the drop among women is 8 per cent.

This equates to around 85,000 men and 77,000 women dying from cancer each year in the UK.’

‘Cancers of the lung, bowel, breast and prostate account for almost half of all cancer deaths in the UK. These four cancers saw an 11 per cent drop in death rates for the period studied.

But some cancers – such as liver and pancreatic – have seen a rise in the rates of people dying, by 60 per cent over the last 10 years for liver cancer and by 8 per cent for pancreatic cancer.

Experts have predicted that, mostly due to the fact people are living longer, one in two people in the UK will be diagnosed with cancer at some point in their lives.

However, some cases could be prevented, with at least a third of cancer cases each year in the UK linked to unhealthy lifestyles, obesity, smoking and diet.’

‘Figures from Cancer Research UK also show the total number of cancer cases is still going up.

Almost 346,000 people were diagnosed with cancer in the UK in 2012, up from 282,000 in 2002 and 249,000 in 1992.

Around 162,000 people died from cancer in the UK in 2012, up from just over 155,000 in 2002, but a very similar figure to 1992.

Around 80 per cent of cancer deaths occur in people aged 65 and over, and more than half occur in those aged 75 and older.’

Related Articles:

World Cancer Day: Deaths caused by disease fall but number of cases rise

Cancer death rates down by 10% in 10 years- BBC News

UK cancer death rates after diagnosis drop 10% in ten years- The Guardian

 

Monday 1st February 2016- The Guardian: British researchers get green light to genetically modify human embryos

‘Britain’s first genetically modified human embryos could be created within months, after scientists were granted permission to carry out the controversial procedure in a landmark decision by the fertility regulator.

The Human Fertilisation and Embryology Authority (HFEA) regulator approved a licence application by Kathy Niakan, a stem cell scientist at the Francis Crick Institute in London, to perform so-called genome editing on human embryos.

The decision permits Niakan to study the embryos for 14 days for research purposes only. It does not permit them to be implanted into women. Niakan’s research is aimed at finding the genes at play in the early days of human fertilisation.

The decision was hailed by the Francis Crick Institute and British scientists but will be met with disquiet by those concerned that rapid advances in the field of genome editing is precluding proper consideration of the ethical implications.’

‘The work, using embryos donated by couples with a surplus after IVF treatment, will look at the fertilised egg’s development from a single cell to around 250 cells. The basic research could help scientists understand why some women lose their babies before term and provide better clinical treatments for infertility, using conventional medical methods.

Niakan will use a powerful genome editing procedure called Crispr-Cas9 to switch genes on and off in early stage human embryos. She will then look for the effects the modifications have on the development of the cells that go on to form the placenta.

Crispr-Cas9 has revolutionised biomedical research since its invention three years ago. It allows scientists to make precise changes to DNA, and has the potential to transform the treatment of genetic disorders by correcting faulty genes.’

Related Articles:

British researchers get green light to genetically modify human embryos

Scientists get ‘gene editing’ go-ahead- BBC News

Scientists in UK get approval for ‘gene editing’- BBC News (video)

British scientists granted permission to genetically modify human embryos- The Telegraph

 

Tuesday 26th January 2016- The Independent: Diabetes cure may be step closer, scientists say

‘A cure for type 1 diabetes could be a step closer after scientists managed to halt the condition in mice for six months thanks to the use of insulin-producing cells that had been generated from human stem cells.

Experts from US hospitals and institutions including Harvard University managed to transplant cells into mice, which immediately began producing insulin.

The team was also able to show they could prevent the cells being rendered useless by the body’s own immune system, which was effectively “switched off” thanks to scientific work.

It means a cure for type 1 diabetes – which affects 400,000 people in the UK – could be much closer. Scientists are now working to replicate the results in humans with the condition.’

‘The man who led that breakthrough – Harvard Professor Doug Melton, who has been trying to find a cure for the disease since his son Sam was diagnosed with type 1 diabetes as a baby – also worked on the new studies.

The human islet cells used for the new research were generated from human stem cells developed by  Professor Melton.

Following implantation in mice, the cells immediately began producing insulin in response to blood glucose levels, and were able to maintain blood glucose within a healthy range for 174 days – the length of the study.’

Related Articles:

Diabetes cure may be step closer, scientists say

Harvard and MIT close to ‘cure’ for Type 1 diabetes which will end daily injections- The Telegraph