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.’

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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

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

Friday 22nd January 2016- The Guardian: Breast milk protein could be used in fight against antibiotic resistance

‘An antibiotic developed from human breast milk could combat certain drug-resistant bacteria, British scientists have found.

Tackling antibiotic-resistant bacteria – known as superbugs – is a priority for the government. A panel set up by David Cameron forecast that superbugs would cost the world ten million lives and £700bn a year by 2050 if the problem went unchecked.

The breakthrough, developed by the National Physical Laboratory (NPL) and University College London, found that the minuscule fragment, less than a nanometre in width, is responsible for giving the protein its anti-microbial properties.

This is what makes breast milk so important in protecting infants from diseases in their first months of life. The protein, called lactoferrin, effectively kills bacteria, fungi and even viruses on contact.

After identifying the fragment, scientists re-engineered it into a virus-like capsule that can recognise and target specific bacteria and damage them on contact, but without affecting any surrounding human cells.

Hasan Alkassem, a student who worked on the project, said: “The capsules acted as projectiles … with bullet speed and efficiency.”

The team suggested this could help the fight against antibiotic resistance by serving as “delivery vehicles” for cures. The capsules could even pave the way for treatments for previously incurable diseases such as sickle-cell disease, cystic fibrosis or Duchenne muscular dystrophy.’

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Breast milk protein could be used in fight against antibiotic resistance

Scientists discover new antibiotic from breast milk- The Telegraph

Monday 18th January 2016- The Independent: France clinical trial: Man left brain-dead following drug test dies

‘A patient in who was left brain dead following a botched drugs trial in France has died, AFP has reported.

Six people became unwell following experimental trials at a hospital in Brittany.

The patients, all men aged 28 to 49, had been treated in Rennes University Hospital, some in intensive care.

The head of the hospital’s neurology department said that based on evidence from magnetic resonance imaging scans, three of the patients may be suffering from irreversible brain damage, the New York Times reported.

Originally, 90 people were given the unknown drug in the trial, out of a total of 128 participants.  The rest were given placebos.

The drug that was being trialled is not known, despite some reports that it was a new cannabinoid-based painkiller.

Marisol Touraine, the French health minister, later confirmed that the pill did not contain cannabis or its derivatives but acted on the body’s endocannabinoid system.

Taken orally, the drug was undergoing a Phase 1 clinical trial at a licensed private European laboratory, Biotrial.  The company specialises in clinical trials and are based in Rennes.

In a statement Biotrial said: “The trial has been conducted in full compliance with the international regulations and Biotrial’s procedures were followed at every stage throughout the trial, in particular the emergency procedures for the transfer of subjects to the hospital. We are in close and regular contact with the Health Authorities and Ministry in France. The priority at Biotrial remains the safety of our subjects.”

Biotrial were conducting the tests on behalf of Bial, a Portuguese drug manufacturer. ‘

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France clinical trial: Man left brain-dead following drug test dies

France drug trial: Brain-dead man dies in hospital- BBC News

Friday 15th January 2016- The Guardian: French drug trial leaves one brain dead and five critically ill

‘One person is brain dead and five others are seriously ill after taking part in a drug trial for Portuguese pharmaceutical firm Bial at a clinic in north-west France.

The French health ministry said the six male patients aged 28 to 49 had been in good health until taking the oral medication. They started taking the drug on 7 January. One person started feeling ill on Sunday and the other five afterwards. The brain dead volunteer was admitted to hospital in Rennes on Monday. Other patients went in on Wednesday and Thursday.

Pierre-Gilles Edan, head of the hospital’s neurology department, said one man was brain dead, three others were suffering a “handicap that could be irreversible” and another had neurological problems. The sixth volunteer had no symptoms but was being monitored.’

‘The French health minister, Marisol Touraine, said 90 people in total had taken part in the trial and received some dosage of the drug; others had taken a placebo. All trials on the drug have been suspended and all volunteers who have taken part in the trial are being called back.

The ministry said the test was carried out by the Biotrial clinic for Bial, which “specialised in carrying out clinical trials”.

The trial was intended to test for side-effects of the new drug but all trials at the clinic have been suspended and the French state prosecutor has opened an inquiry.

Touraine said the drug was a so-called FAAH inhibitor meant to act on the body’s endocannabinoid system, which deals with pain. Earlier reports suggested that the drug contained cannabinoids, an active ingredient found in cannabis plants, but the minister said it did not contain the drug or any derivatives of it.

Touraine said the study was a phase one clinical trial, in which healthy volunteers take the medication to “evaluate the safety of its use, tolerance and pharmacological profile of the molecule”.’

‘Testing had already been carried out on animals, including chimpanzees, starting in July, Touraine said.

Bial said it was committed to ensuring the wellbeing of test participants and was working with authorities to discover the cause of the injuries, adding that the clinical trial had been approved by French regulators.’

Related Articles:

French drug trial leaves one brain dead and five critically ill

Multiple investigations launched into France drug trial that left man brain dead- The Telegraph

Saturday 14th November 2015- The Telegraph: Diabetic blindness could be reversed with eye injection

‘Diabetics blinded by the disease have been offered new hope afters scientists unveiled the first new treatment in 40 years.

Researchers said that injections of the drug ranibizumab improved sight when compared to traditional treatments for people with proliferative diabetic retinopathy (PDR).

The disorder occurs when high blood sugar levels damage the cells at the back of the eye. If it isn’t treated, it can cause blindness.

PDR is a leading cause of vision loss in patients with diabetes and the standard treatment has been laser surgery which can result in loss of peripheral vision and difficult seeing at night.

However the new treatment allowed patients to keep their peripheral vision while improving central sight so they could see eye charts more accurately and read half a line more, on average.’

‘”This important study represents a major step forward for patients with PDR by providing the ophthalmologists who manage their retinal disease with new options,” said Dr Timothy Olsen, of Emory University, Atlanta.

The injections would need to take place once a month for three months.

The number of adults with diabetes has risen more than 65 per cent since 2005, with more people than ever at risk of blindness.’

‘The new research was presented at the American Academy of Ophthalmology annual meeting and published in the journal JAMA.’

See:

Diabetic blindness could be reversed with eye injection

Friday 13th November 2015- The Guardian: AstraZeneca lung cancer drug given green light in US

‘FDA approval of Tagrisso offers major boost for British company, seeking to release six new cancer medicines by 2020’.

‘A new lung cancer pill from AstraZeneca has been approved by US regulators, in a major boost for the British drugmaker.

AZD9291, which will be sold as Tagrisso, is for advanced non-small-cell lung cancer, the most common form of lung cancer. Tagrisso targets a genetic mutation, known as T790M, that helps tumours evade current lung cancer pills. The drug will be made available to patients in the US as soon as possible and its price will be “comparable to other oral cancer therapies,” a spokeswoman said. AstraZeneca will reveal the price early next week.

Lung cancer is the leading cause of cancer death among men and women, accounting for a third of cancer deaths, more than breast, prostate and colorectal cancers combined.

The treatment, developed in Cheshire, is  ione of several highlighted by AstraZeneca chief executive Pascal Soriot in his defence against a £69bn takeover approach from American rival Pfizer, the maker of Viagra, last year. AstraZeneca estimates that Tagrisso could bring in sales of $3bn (£2bn) a year but analysts are more cautious, forecasting sales of $1.1bn in 2020. The company needs new blockbuster medicines to make up for sales losses on older drugs that are losing patent protection.

The once-daily Tagrisso tablet had a “significant effect on reducing tumour size in over half of patients who were treated,” said Richard Pazdur of the FDA’s centre for drug evaluation and research.’

See:

AstraZeneca lung cancer drug given green light in US

Tuesday 10th November 2015- The Telegraph: Cholesterol vaccine could end need for daily statins

‘A vaccine which significantly lowers cholesterol could end the need for daily statins.

Health regulators currently recommend that around 17.5 million people in Britain should take the cholesterol busting drugs but they are known to cause debilitating side-effects such as muscle cramps.

Now, researchers at the University of New Mexico have found that a jab can lower cholesterol better than statins. And it is likely to be cheaper than the daily pills.’

‘Dr Alan Remaley, one of the authors of the study from the National Heart, Lung and Blood Institute, National Institutes of Health added: “Statins are still the most commonly prescribed medication for cholesterol.

“Although they are effective in many people, do have side effects and don’t work for everyone. The results of our vaccine were very striking, and suggest it could be a powerful new treatment for high cholesterol.”

So far the vaccine has only been tested on mice and monkeys but tests have shown it can reduce bad cholesterol by up to 55 per cent compared with the 30 per cent managed by statins. It can also be used to boost the effectiveness of statins by a further 40 per cent. The report authors are hoping to move to human trials soon.’

See:

Cholesterol vaccine could end need for daily statins

Thursday 29th October 2015- BBC News: ‘Milestone’ prostate cancer drug

‘The first drug that targets precise genetic mutations in prostate cancer has been shown to be effective in a “milestone” trial by UK scientists.

The study, at the Institute of Cancer Research in London, took place on 49 men with untreatable cancer.

The drug, olaparib, had low overall success, but slowed tumour growth in 88% of patients with specific DNA mutations.

Cancer Research UK said the trial was exciting.

The future of cancer medicine is treating cancers by their mutated DNA rather than what part of the body they are in.

The breast cancer drug Herceptin is already used only in patients with specific mutations. Olaparib targets mutations that change the way DNA is repaired.’

‘The trial results, published in the New England Journal of Medicine, showed the drug worked in 14 out of 16 men with such mutations.’

See:

‘Milestone’ prostate cancer drug

Monday 21st September 2015- The Independent: Arthritis drug could soon reverse Alzheimer’s symptoms after successful tests on mice, say scientists

‘A painkiller widely used to treat rheumatoid arthritis has been shown to reverse the symptoms of dementia in the brains of laboratory mice, raising hope that there may soon be an effective treatment for Alzheimer’s disease, scientists have said.

The drug, salsalate, is a licensed pain killer but in mice with a form of dementia similar to Alzheimer’s it reversed the changes to a key protein in the brain that builds up in patients with the debilitating neurological disease, they found.

The researchers said it is the first time any drug has been shown to have an effect on the “tau” protein that accumulates in the brain of people with Alzheimer’s and a range of similar dementias known as “tauopathies”. It could lead to an effective therapy even for patients in the later stages of disease, the researchers said.

“We identified for the first time a pharmacological approach that reverses all aspects of tau toxicity,” said Li Gan, PhD of the Gladstone Institutes, a non-profit research organisation affiliated with the University of California, San Francisco.’

See:

Arthritis drug could soon reverse Alzheimer’s symptoms after successful tests on mice, say scientists