Australian Schoolboys - Create Justice

The man who sparked outrage last year by hiking the price of a life-saving drug may have met his match in some Australian schoolboys.

US executive Martin Shkreli became a symbol of greed, when he raised the price of a tablet of Daraprim from $13.50 (£11) to $750.
Now, Sydney school students have recreated the drug's key ingredient for just $20.
Daraprim is an anti-parasitic drug used by malaria and Aids patients.
Martin Shkreli: 'The most hated man in America'

The Sydney Grammar boys, all 17, synthesised the active ingredient, pyrimethamine, in their school science laboratory.
"It wasn't terribly hard but that's really the point, I think, because we're high school students," one boy, Charles Jameson, told the BBC.
The students produced 3.7 grams of pyrimethamine for $20. In the US, the same quantity would cost up to $110,000.
In most countries, including Australia and Britain, the drug retails for less than $1.50 per pill.

                           

The Sydney students at work

in their high school laboratory

The boys said they conducted the year-long experiment to highlight the drug's inflated cost in the US.
"It seems totally unjustified and ethically wrong," student James Wood said. "It's a life-saving drug and so many people can't afford it."
Supervising teacher Dr Malcolm Binns said: "Everyone is very happy and pleased with the result. All the boys think it's the most amazing thing."
Developed in the 1950s, Daraprim is the best treatment for a relatively rare parasitic infection called toxoplasmosis.
Mr Shkreli, also known as "Pharma Bro", was chief executive of Turing Pharmaceuticals when it acquired exclusive rights to Daraprim.
Its decision to increase the cost by more than 5,000% in August last year drew international condemnation. Mr Shkreli has argued the Daraprim price increase was warranted because the drug is highly specialised.
But the firm eventually agreed to lower the price to something more affordable.

'Real monetary value'

Dr Alice Williamson, a University of Sydney research chemist, supported the boys' project through online platform - Open Source Malaria.
"They've transformed starter material that's worth pennies into something that has a real monetary value in the States," she told the BBC.

"If you can obtain it cheaply in schools, then there's no excuse for charging that much money for a drug. Especially from people that really need it and probably can't afford to pay for it."
Dr Williamson called the pricing in the US "ludicrous".
Mr Shkreli was arrested in December on allegations of securities fraud. He subsequently stepped down as the head of Turing. His trial is set for 26 June, 2017.

Alzheimer's - New Development

A pioneering new drug has been shown to clear away toxic clumps of protein material in the brain, thought to be a primary cause of Alzheimer's.

The drug, verubecestat, is one of a number being trialled by pharmaceutical companies in a race for the first treatment that can halt or reverse the disease.
Results from a small Phase 1 study confirm that verubecestat can reduce levels of beta-amyloid, a protein fragment that accumulates in sticky deposits or "plaques" in the brains of Alzheimer's sufferers.
Most importantly, it appeared to produce no severe side effects.
Earlier experiments with drugs that work the same way, by targeting an enzyme linked to beta-amyloid build-up, were abandoned when they were found to cause liver and nerve damage.
Publication of the trial findings comes as two much larger international trials testing the effectiveness of verubecestat in more than 3,000 patients nears completion.

Depending on the outcome of these Phase III studies, the drug could be licensed in the next two to three years.
Verubecestat, made by drug giants Merck, is not alone.
Two other medicines with the potential to modify Alzheimer's progression, solanezumab and aducanumab, are also undergoing Phase III trials.
However, both differ from the Merck drug in that they are antibodies designed to mount an immunological-style attack on beta-amyloid.
Dr Rosa Sancho, from the charity Alzheimer's Research UK, said: "It has been over a decade since a new drug was licensed for the treatment of dementia, so we urgently need new medicines that can provide real benefit for people living with dementia.
"There is a wave of potential new treatments currently being tested for dementia, with the results of these studies hotly anticipated over the course of the coming months and years.
"In this small Phase I clinical trial, verubecestat was able to reduce the levels of a key Alzheimer's protein, but it remains to be seen whether this translates into slower memory decline for people with the disease."
A history of past failures mean that scientists tread carefully when announcing apparently promising results from early studies of new Alzheimer's treatments.
Currently there are medicines available that can alleviate some of the symptoms of the disease, but nothing capable of halting or slowing its progress.
That situation may be about to change if drugs such as verubecestat pass the stiff tests of safety and effectiveness imposed by medicine regulators.
The Phase I trial reported in the journal Science Translational Medicine recruited just 32 patients and was chiefly concerned with dosage and safety.
Participants had mild or moderate Alzheimer's disease but were otherwise healthy.
The study, led by Dr Matthew Kennedy, from Merck's US research laboratories in New Jersey, looked for markers of beta-amyloid in cerebrospinal fluid.
It showed that single and multiple doses of verubecestat could lower levels of the harmful peptide without serious side effects.
Other research by the same team found that a single dose of the drug markedly reduced levels of beta-amyloid in rats and monkeys. The animals showed no sign of toxicity even after extended treatment.

Verubecestat is known as a BACE1 inhibitor. It targets an enzyme called beta-secretase 1 that slices up a precursor protein to produce beta-amyloid fragments.
Dr Sancho added: "As verubecestat works differently to other drugs currently being tested for Alzheimer's, if it proves successful, it could be an important weapon in the arsenal for doctors treating the disease in future.
"Alzheimer's is a complex and devastating disease and so a multi-pronged approach may give us the best chance of tackling it in the coming years. As the first BACE1 inhibitor to reach the final stages of testing in people, the ultimate test will be whether these ongoing trials show verubecestat can provide tangible benefits for people with Alzheimer's.
"Scientists have reflected on past lessons in Alzheimer's drug development and it is promising to see an increasing number of drugs now entering Phase III clinical testing in the disease, giving us renewed hope in the hunt for new treatments."

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'Golden age' of antibiotics 'set to end'

Far fewer pharmaceutical companies are working on new antibiotics than in the past.  Prof Jeremy Farrar, the new head of Britain's biggest medical research charity the Wellcome Trust said it was a "truly global issue".

Prof Farrar said that the golden age of antibiotics could come to an end unless action is taken.
Previous chief medical officers have also warned about the threat from pathogens - bacteria, viruses and parasites.

Multi-drug resistant (MDR) tuberculosis is rising steadily worldwide.  Doctors in London have reported a sharp rise in cases of MDR TB, which can take two years to treat successfully.  Another example is the rise in infections from enterobacteriaceae - bugs that live in the gut like E. coli and Klebsiella.  They are not the commonest cause of hospital acquired infection and some are becoming resistant to carbapenems, a powerful last resort group of antibiotics.

Prof Farrar said we could have used our antibiotics better and we should have invested more in research on infectious diseases.

He said there are just four pharmaceutical companies working on antibiotics now compared to 20 years ago.

And Prof Farrar called for industry to be given incentives to work on antibiotics and greater restrictions placed on access to these medicines

Read full article here on BBC News

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