Sanofi epilepsy drug linked to over 4,000 child deformities

The epilepsy medication valproate is responsible for “severe malformations” in up to 4,100 children in France since the drug was first marketed in the country in 1967, according to a preliminary study by health authorities.

Women who took the drug during pregnancy to treat epilepsy were four times more likely to give birth to babies with congenital malformations, said the report, jointly issued by the medicines regulator ANSM and the national health insurance administration.

The study confirmed that the drug was “highly teratogenic”, which means it is capable of causing birth defects, said Mahmoud Zureik, scientific director of ANSM and a co-author of the report.

He said the estimated number of babies born with severe malformations, which ranged between 2,150 and 4,100 was “very high”. The types of birth defects attributed to the drug included spina bifida – a condition in which the spinal cord does not form properly, and can protrude through the skin – as well as defects of the heart and genital organs.

The risk of autism and developmental problems was also found to be higher, and will be quantified in a follow-up report later this year.

An earlier estimate suggested that 30-40% of children exposed in the womb could suffer such disorders.

From 1967 to 2016, between 64,100 and 100,000 pregnancies in France were exposed to valproate, resulting in 41,200 to 75,300 live births, according to the report.

The vast majority of the birth defects occurred for women under treatment for epilepsy.

But starting in the late 1970s, valproate – marketed around the world including the US, UK and Australia as Depakine, Depakote, Stavzor and other trade names – was also prescribed in France to treat bi-polar disorder.

Bi-polar women taking the drug were twice as likely to give birth to children with major birth defects, the study found.

The lower risk compared with women treated for epilepsy probably stems from the fact that for pregnant bi-polar women doctors stopped prescribing valproate early in the pregnancy, Zureik said.

“The risk of severe malformation is limited to the first two trimesters of pregnancy,” said Alain Weill, a researcher at the French health insurance administration and a co-author of the report.

The risk of birth defects associated with valproate has been known since the 1980s, especially for spina bifida, which occurs 20 times more frequently in foetuses exposed to the medication.

But the drug can still be prescribed to pregnant women when all other forms of treatment for epilepsy fail. That ruling, however, was put in place only in 2015.

Valproate is sold in France under the brand name Depakine by pharmaceutical company Sanofi, but is also available in generic forms.

Some French families of children with birth defects born to women who took the drug while pregnant – grouped under an umbrella association called APESAC – have sued the company, claiming that it did not adequately warn about the risks.

“The number of victims is potentially huge,” said APESAC president Marine Martin, who says two of her children – a girl and a boy – suffered physical defects brought on by valproate.

“We need to take into account children with malformations and autism, as well as families that lost a baby due to treatment during pregnancy,” she told AFP.

Her association estimates a total of 30,000 to 50,000 victims – children and families included.

In a statement, Sanofi said it had been “totally transparent with health authorities”

“We are aware of the painful situation confronting the families of children showing difficulties that may have a link with the anti-epileptic treatment of their mother during pregnancy,” the drugmaker said.

Sanofi epilepsy drug linked to over 4,000 child deformities

"Gamechanging" cancer drug rejected for use on NHS

A gamechanging immunotherapy drug that can extend the life of patients with advanced head and neck cancer has been turned down for use in the NHS because of its high cost.

Nivolumab is one of a new class of drug that stimulates the patient’s own immune system to fight the cancer. Immunotherapy drugs have had some spectacular successes in some patients with some cancers. But although nivolumab can give people with advanced head and neck cancers an extra three months of life – when survival expectancy at present is around six months – the National Institute for Health and Care Excellence (Nice) has rejected it.

“The committee heard that treatment options for patients in this area are limited, and it’s important to patients that treatment extends their life and improves the quality of life. But the additional costs of nivolumab were considered to be very high in relation to its benefit to be recommended for routine NHS use at present,” said Prof Carole Longson, director of the health technology evaluation centre at Nice.

Nice will not approve drugs that cost more than its threshold of £20,000 to £30,000 per year of quality life, except for an end-of-life treatment – as this drug is – in which case the threshold rises to £50,000. But Nice believes nivolumab would cost between £66,000 to £75,000 per year of quality life.

The Institute of Cancer Research (ICR), which led the UK arm of the final phase of trials before licensing, regretted the decision. “It is disappointing and frustrating that today’s decision means doctors will not be able to offer this gamechanging immunotherapy to patients with advanced head and neck cancer,” said Prof Kevin Harrington, consultant clinical oncologist at the Royal Marsden. “Once it has relapsed or spread, the disease is extremely difficult to treat and options, including surgery and radiotherapy, are very limited.

“Nivolumab is an expensive drug but it is also the only treatment shown in a phase-III trial to improve survival for this group of patients – and it did so without worsening patients’ quality of life, and with fewer side-effects than other options. It’s crucial that talks on the drug’s availability continue and ultimately that this decision is reversed, otherwise patients face missing out on a genuinely effective treatment simply because of cost.”

The ICR’s chief executive, Prof Paul Workman, said the price of cancer drugs was too high. “This decision denies patients a genuine breakthrough treatment that makes a real difference for people with relapsed or metastatic head and neck cancer. It is another example, and a particularly stark one, of an innovative cancer therapy not being made available on the NHS because of cost. I’d urge Nice and the manufacturer to work together to reach an agreement on price so that this decision can be overturned as soon as possible.

“We need pharmaceutical companies to bring down the cost of drug development through smaller, more targeted trials, and to do much more to pass on the savings to patients. Nice for its part must take much greater account of innovation in its appraisal processes to give exciting treatments like nivolumab a better chance of reaching patients.”

"Gamechanging" cancer drug rejected for use on NHS

People at risk of HIV in Scotland to be given PrEP drug on NHS

People at risk of HIV in Scotland are to be given drugs on the NHS that will protect them from infection, it has been announced, in a move that Aids campaigners say will put pressure on the authorities in England to end delays in providing the same medication despite two major court rulings.

In a major victory for campaigners, the Scottish Medicines Consortium (SMC) said pre-exposure prophylaxis (PrEP) would be free on the NHS to those who need it because they are at risk – for instance, if they have a partner with HIV. Access to the drugs could begin within weeks.

NHS England resisted rolling out PrEP due to its cost, although it is a lot cheaper than a lifetime of HIV treatment which could cost £360,000. It lost to campaigners in the high court and then the court of appeal, but those who want to take PrEP have been told they must wait for a big new trial to answer “significant outstanding implementation questions”.

The National Aids Trust (NAT), which funded the court battle in England, said it was delighted PrEP would shortly be available in Scotland. “This game-changing prevention tool has the potential to massively reduce HIV rates and turn Scotland into a model internationally of how to do HIV prevention well. The speed and decisiveness of the Scottish process contrasts starkly with delays in the other three UK nations,” said Deborah Gold, NAT’s chief executive.

“Though we were jubilant when, following our two successful court cases, NHS England agreed steps to fund PrEP, we remain concerned that since that date, progress towards the ultimate goal of PrEP in England has been slow. It is difficult not to think of the possible thousands of HIV diagnoses that could have been prevented had the NHS in England not prevaricated, and we urge them to pursue the promised trial with appropriate urgency.”

The PrEP4Scotland Coalition (made up of HIV Scotland, Terrence Higgins Trust Scotland, Waverley Care and NAT) which has campaigned and negotiated with the Scottish authorities on the introduction of PrEP, said: “We applaud the SMC for taking this bold step to tackling HIV in Scotland. PrEP provides opportunities to reinvigorate how people at higher risk of HIV exposure engage with testing and prevention opportunities, and it is a vital opportunity to make a real reduction in the number of new HIV transmissions.”

Trials in several countries around the world including the UK have shown beyond doubt that PrEP works. The drug used is Truvada, which is one of a cocktail used to treat people infected with the virus and keep them well. PrEP has been hailed as one of the tools that could help end the Aids epidemic and efforts are being made to get it introduced into high-burden countries such as South Africa.

Every year about 5,000 people become infected with HIV in the UK and the rate among men who have sex with men is rising fastest. This was the group in whom the trials in the UK were done and where the evidence is strongest.

But NHS England dragged its feet because of the cost – estimated at potentially £20m a year to treat everyone who could benefit. It attempted to shift the bill to the local authorities, who are now responsible for public health, including HIV, obesity and smoking.

The local authorities said they could not afford to pay either. NAT sued NHS England in the high court and won last August. NHS England took its case to the court of appeal and lost again in November.

In December, NHS England said it would make the drugs available through a trial, which would enrol at least 10,000 people over the next three years.

NHS England said there were issues that needed addressing in a trial – which may include how consistently people take the daily tablets and how long they stay on the regime. But a trial also allows the NHS to obtain cheaper generic versions of the drug, rather than paying the market price to Gilead, the manufacturer and patent holder of Truvada. NHS England has already asked various companies to tender to supply the pills.

Prof Noel Gill, head of Public Health England’s HIV and STI department, said: “All the detailed work underpinning the clinical trial of PrEP is well under way and we expect it will commence by the summer 2017.”

Scotland’s decision to part with the NHS in England follows a huge community effort and years of campaigning, said HIV Scotland. “In 2016 HIV Scotland published a PrEP good practice guide, and administered Scotland’s expert group which produced prescribing criteria, cost assessments, and mapped information and training needs of workers and the community,” said its chief executive George Valiotis.

“Today, Scotland has made history in the fight against the HIV epidemic,” said
Robert McKay, national director for Terrence Higgins Trust Scotland. “PrEP can now be used as a vital tool – alongside condom use, regular testing and early treatment – to help bring an end to HIV transmission in Scotland.

“Not only will this make a life-changing difference to individuals by protecting them from a lifelong and stigmatised condition, but for every person who would have become HIV positive without PrEP, NHS Scotland will save £360,000 in lifetime treatment costs.”

People at risk of HIV in Scotland to be given PrEP drug on NHS

A puke bucket and an ancient drug: is ayahuasca the future of PTSD treatment?

I’m sitting on a blue plastic, wipe-down mattress with my back to a wooden pillar. Within arm’s reach on the floor is a small torch to light my way to the toilet during the night, on the other side an orange plastic bucket to puke into. As the light fades my four companions, each with his or her own plastic mattress and bucket, disappear from view while on every side the barks, croaks, growls and cries of jungle life grow louder. Twenty minutes ago I gulped down a draught of the bitter psychedelic brew known as ayahuasca and I have convinced myself that I can feel its hot, unstoppable progress through my body, from my seething guts into my veins and onwards to my brain.

This is hardly a recreational drug experience, what with the nausea, vomiting and diarrhoea, not to mention the possibility a truly terrifying trip, yet thousands now beat a path to Peru, Ecuador and Brazil every year to drink ayahuasca. Some are just looking for an exotic thrill, but others hope for enlightenment and healing from this ancient plant medicine. In the past few years, many of them have been war veterans desperate to escape the nightmares of post-traumatic stress disorder.

Combat-related PTSD is notoriously difficult to treat and in theory ayahuasca can work as a form of drug-assisted exposure therapy. When traumatised people repeatedly avoid fear-inducing situations this only serves to maintain and reinforce the deeply ingrained conditioning that underlies their illness. The idea is that by dredging up traumatic memories and exposing them to conscious awareness within a safe, controlled environment, ayahuasca allows the brain to reassess and extinguish conditioned fear responses.

Classic psychedelics such as DMT – an active component of ayahuasca – break the control that the prefrontal cortex normally holds over more primitive parts of the brain, triggering vivid hallucinatory memories and emotions. “That lets us go to places in our psyche or internal landscape that we wouldn’t normally allow ourselves to go,” says Gerald Thomas, who researches addiction at the University of Victoria, British Columbia. “In psychotherapy, it’s how we reconcile past events that have traumatised us.”

Thomas has conducted preliminary research suggesting that ayahuasca can reduce dependence on addictive drugs. Part of the explanation may be that it helps ease the pain of traumatic memories that people sometimes “self-medicate” with substances such as alcohol, tobacco and cocaine.

To date, any evidence that ayahuasca can do the same for people with PTSD has been anecdotal. But an ambitious study now under way at the Temple of the Way of Light in the Peruvian Amazon is monitoring its long-term effects on psychological wellbeing and may provide some answers. The research is a collaboration with the International Center for Ethnobotanical Research and Service in Spain and the Beckley Foundation in the UK. Around 580 retreat participants a year – among them combat veterans suffering from PTSD – are being recruited, making it the largest psychedelic study of its kind ever undertaken.

But the study highlights a problem. The Temple of the Way of Light rigorously screens applicants for any history of psychosis or mania, which can be triggered by ayahuasca. Adverse effects like these are rare, says bookings manager Karin Gingras, but the temple is in a remote jungle location far from the nearest hospital. “We have seen how difficult it can be to recover from psychosis for some of these folks and are very aware that we are not equipped with the professional psychological staff to safely support these individuals.”

Most ayahuasca retreat centres in Peru do not go to such lengths to screen applicants, and the cheaper ones advertised on the streets of tourist hotspots such as Iquitos and Cusco will take anyone’s money, no questions asked. They provide little or no psychological support.

“People should pursue using ayahuasca with great care and do thorough research to find reputable retreat centres,” advises Alli Feduccia of MAPS, the Multidisciplinary Association for Psychedelic Studies. “Counselling and support during and after ayahuasca retreats are necessary to integrate the intense experiences that can emerge,” she says. “People have been traumatised by ayahuasca experiences because this very needed support is lacking.”

Then there’s the risk of interactions with prescription drugs. In addition to DMT, ayahuasca contains potent monoamine oxidase inhibitors (MAOIs), which block an enzyme that usually breaks down neurotransmitters in the brain, including serotonin. As a result, taking ayahuasca while on an SSRI or MAOI antidepressant can cause potentially fatal “serotonin syndrome”. Under normal circumstances the enzyme also breaks down tyramine – found in pork, pickles, smoked and fermented foods, chocolate and alcoholic drinks – and excess tyramine can trigger a dangerous spike in blood pressure.

If you’ve got PTSD, it’s unlikely your doctor will send you on an ayahuasca retreat any time soon (you are more likely to be offered psychotherapy under the influence of MDMA). In the US, where DMT is outlawed as a schedule one drug, Feduccia says MAPS has faced major obstacles in its efforts to gain approval for a clinical trial of ayahuasca for PTSD. A perverse effect of the lack of research may be to drive desperate patients into the arms of dubious retreat centres in South America that will fail to screen them adequately, offer advice on potentially dangerous dietary and drug interactions, or provide the necessary psychological support.

My own encounter with ayahuasca was a happy one. I felt nauseous but didn’t vomit. I witnessed no earth-shattering visions, though I did experience perceptual distortions and a temporarily enhanced sense of meaning and beauty. The only long-term effect on my physical and psychological wellbeing appeared to be a persistent bout diarrhoea that lasted several days. Retreats like those at the Temple of the Way of Light usually involve a series of ceremonies over several days, but I attended just this one – at another reputable retreat centre near Iquitos – and requested a very low dose of the medicine. I have a family history of bipolar disorder, which can involve psychosis, so even though I don’t have the condition myself I was unwilling to take a major gamble with my mental health.

This article was edited on 6 April 2017 to make it clear that the author did not participate in a ceremony at the Temple of the Way of Light.

James Kingsland is the author of Siddhartha’s Brain: Unlocking the Ancient Science of Englightenment

A puke bucket and an ancient drug: is ayahuasca the future of PTSD treatment?

Drug scandals and the media – the unresolved case of Primodos

If the history of drug scandals teaches us anything, it is that fair compensation is typically achieved only through lengthy media campaigns and legal battles. Though lacking the direct powers of judges or policymakers, interventions by investigative journalists and broadcasters have sometimes proved decisive.

Take thalidomide: between 1957 and 1961 the widely prescribed morning-sickness treatment caused miscarriages, and many thousands of babies around the world were born with severe limb malformations. In the UK, an adequate settlement was negotiated with the British distributor, Distillers Company (now part of Diageo), only after the Sunday Times took up the cause in 1972.

The thalidomide disaster is the best-known drug scandal involving birth defects, but it is not the only one. In the late 1960s, suspicion fell on Primodos, a hormonal pregnancy-test drug marketed by the German pharmaceutical company Schering (now Bayer). I have previously written on the origins of Primodos and the still unresolved debate over whether the British government should have allowed it to remain on the market until 1978, despite widespread safety concerns and the existence of a highly reliable and perfectly harmless alternative: the laboratory urine test. As with thalidomide, the media played and continues to play a crucial role in the campaign for compensation for those who say they have been harmed by Primodos.

An advertisement for Primodos in The Practitioner from the early 1960s marketing campaign aimed at GPs that aggressively targeted the slower, more expensive toad test. Photograph: Practitioner, vol. 185 July 1960./The Practitioner, Practitioner Medical Publishing Ltd

Blowing the whistle on Schering

The Primodos scandal unfolded in the shadow of the thalidomide disaster and was shaped by it. Primed by thalidomide, the Sunday Times became involved early on. It was in response to a Sunday Times exposé that the British government issued the first official warning of a ‘possible association’ between Primodos and ‘an increased incidence of congenital abnormalities’ in 1975. Despite the warning many doctors continued to prescribe Primodos, and it remained widely available in Britain.

The media campaign took a dramatic turn in 1977 when a concerned employee of Schering’s British subsidiary leaked internal corporate documents to the press. These papers revealed a significant dispute between British and German executives over whether Primodos should continue to be marketed in Britain. Essentially, British executives wanted the drug to be taken off the market, but their hands were tied when the final decision was made in Berlin. The documents eventually reached the light of day by a circuitous route, involving a whistle-blower, a reporter, and a private investigator.

Scandal, leaks and spin

Ian Withers, a private investigator, was enlisted by a law firm to place the documents with one of the national newspapers. Withers was offered a fee of £500 and asked to generate £10,000 for the large folder of correspondence. This sum was intended as an insurance policy for the anonymous ‘whistleblower’, who fully expected to lose his job and forfeit any further career prospects in the industry. Withers spent around ten days hawking sample pages to journalists and media contacts. One was a reporter for the Sunday People, who expressed an interest in the story. But instead of exposing Schering’s internal dispute over drug safety, the reporter decided to portray Withers as a ‘rat’ trying to ‘cash in on a toddler’s suffering’. Withers considered suing, but refrained in order to safeguard his client’s anonymity, which endures to this day. Happily, the incident did not damage the detective’s reputation and he went on to a career as one of Britain’s most successful private eyes.

The leaked folder, meanwhile, soon made its way into the hands of journalist and broadcaster Greg Dyke, who quoted heavily from the documents it contained in his hour-long documentary for London Weekend Television’s The London Programme in 1978. By then, Labour MP Jack Ashley, who had spearheaded the thalidomide campaign in Parliament, had begun calling for a government inquiry into Primodos. Further coverage in The Guardian and The Sunday Times continued to apply pressure and culminated in a first debate, in the House of Commons, on 26 May 1978.

On 6 June 1978, Dyke reported in The Guardian that Health Minister Roland Moyle would that day ‘for the first time, come face to face with a group of parents who believe their children were born deformed because their mothers were prescribed hormone pregnancy testing drugs’. The parents, Dyke explained, wanted ‘just one thing from the Minister – a public inquiry into the way the drugs were used in this country’. But despite Ashley’s efforts, the parents did not receive the inquiry they wanted—until 2014.

The 1970s campaigns relied on just one folder; now there is much more evidence to be considered. Photograph: Alamy Stock Photo

New lines of evidence

The current inquiry, set to report in May, was launched by the Department of Health following a second debate in the Commons, on 23 October 2014. Its brief is to examine the evidence, including new sources that were not available in the 1970s. These include thousands of pages of documents that have resurfaced in London and Berlin and ongoing laboratory research on the effects of Primodos on developing fish embryos. A reenergised campaign, headed by Marie Lyon, now has the support of an all-party parliamentary group formed in 2014 by Labour MP Yasmin Qureshi. And Jason Farrell, a Sky News reporter who became involved in 2011, has made Primodos: The Secret Drug Scandal, an hour-long documentary and the culmination of his investigation.

Back in 1978, Dyke had access to just a single leaked folder of internal correspondence. Today, the evidence base is radically expanded to include new experimental results and masses of previously supressed documents. Farrell makes good use of these, as should the inquiry, which has vastly more material at its disposal than it would have done in the late 1970s. It is possible that legal proceedings, abandoned in 1982, will be started up again. The media, alongside campaigners and MPs, continues to play as vital a role as it did back then. It does so by bringing new evidence to light, using it to form public opinion, and, not least, by applying pressure to government and industry. Will today’s media campaign, armed with new lines of evidence, succeed where that of the 1970s faltered? Will Primodos: The Secret Drug Scandal make history?

Jesse Olszynko-Gryn is a historian of medicine at the University of Cambridge. He acted as historical consultant on Primodos: The Secret Drug Scandal.

Drug scandals and the media – the unresolved case of Primodos

Drug which cuts "bad" cholesterol can help prevent heart attacks and strokes

A new drug can prevent heart attacks and strokes by cutting bad cholesterol levels, scientists have found.

An international trial of 27,000 patients found that those who took the drug evolocumab saw their bad cholesterol levels fall by around 60% on average.

The patients in the trial were already taking statins, which are used to reduce low-density lipoprotein (LDL) cholesterol. Despite this, the patients who took evolocumab saw their bad cholesterol levels fall even further. They were also less likely to suffer from a heart attack or stroke than those who took the placebo.

The study found that for every 74 people who took the drug for two years, one heart attack or stroke would be prevented.

However, the findings, published in the New England Journal of Medicine, found that the drug had no impact on the rate of cardiovascular mortality.

Prof Peter Sever, from Imperial College London – which led the UK branch of the study, said: “This is one of the most important trials of cholesterol-lowering since the first statin trial, published 20 years ago. Our results suggest this new, extremely potent class of drug can cut cholesterol dramatically, which could provide great benefit for a lot of people at risk of heart disease and stroke.”

There are approximately 2.3 million people living with coronary heart disease in the UK, according to the NHS. It is responsible for more than 73,000 deaths a year in the UK, and occurs when fatty substances build up in the arteries, making it harder for blood to get to the heart.

Prof Sir Nilesh Samani, medical director at the British Heart Foundation, said: “Coronary heart disease is the single biggest killer in the UK and worldwide and ‘bad’ LDL-cholesterol is a major cause.

“While statins have had a significant impact in reducing the risk of heart disease for millions of people, they are not tolerated by everyone and only reduce cholesterol by a certain amount.

“A promising new approach is blocking the action of PCSK9, a molecule which reduces the breakdown of LDL-cholesterol in the liver. Creating new treatments which use this approach could prove life-saving for patients with high cholesterol and those who can’t tolerate statins.”

Drug which cuts "bad" cholesterol can help prevent heart attacks and strokes

Drug addiction isn"t going away so why are treatment centres being slashed?

You may not know that your local authority is responsible for funding drug and alcohol treatment. And unless you, a friend or family member have been personally touched by addiction you might not think that these services should be a funding priority for cash-strapped councils.

We’re left to manage a host of intractable problems that we’re not qualified or able to deal with.

I work in a community drug and alcohol treatment centre and my job is to support people to overcome their addiction and support their recovery. When I arrive at work in the morning there is usually a queue of people outside wanting to get help.

They’re vulnerable people with complex needs and demand for our support is increasing. Yet we’ve seen our funding slashed by 42% since 2010. The situation is the same across the country.

I see fewer heroin users nowadays but far more people dependent on alcohol and people getting into problems with so-called party drugs such as methamphetamine and ketamine. The heroin users might be fewer in number, but they require more of our attention as they get older and sicker. They often have hepatitis C and smoke tobacco and succumb to liver and lung diseases as a consequence.

Addiction sits at the centre of a cluster of physical, psychological and social difficulties. Our service users need help and support in all these domains if they are to stand a chance of recovery. Our caseloads have got bigger because we have had to cut posts and as pressures elsewhere in the health and social care system builds, the complexity of the problems we are presented with has increased, too.

Even when there are clear mental health problems, mental health services don’t want to treat people who also use drugs or drink, so they send them to us. The same applies to the general hospitals – with access to liver treatments being rationed. I know they are also under pressure, with ever-expanding waiting lists, but as a consequence we are left to manage a host of intractable problems that we are not qualified or able to deal with.

Some of our clients lead chaotic lives and come to us in desperation with a whole host of difficulties that go far beyond addiction. They might be embroiled in the criminal justice system and need advice, they might have housing problems or be struggling with trauma; it is not uncommon for me to treat clients who used to be in care and have survived institutional abuse. We used to have psychologists in our team who could provide treatment for complex trauma related to sexual abuse but their posts were cut last year.

I have two clients who are so physically unwell that the local residential detox provider does not think they can safely manage them. The NHS-run unit we used to refer to because it had the necessary medical cover has been closed due to the cuts. If they don’t die beforehand, the only hope for my clients is that they will get a detox if they are admitted in an emergency with a physical health crisis to a general hospital.

With diminished resources we have had to prioritise treatments such as opiate substitute medications and needle exchange, which we know can keep people alive. But how are these actually helping people overcome their addiction?

Addictions services are often retendered with contracts being awarded to the cheapest bidder. I work with people who have had their service retendered and employer changed multiple times. This is a massively stressful process and I have friends who have left the sector feeling demoralised and burnt out.

We are judged on figures like the number of people leaving treatment drug-free, and treatment centres know that this can be used against them. The worst-kept secret in our sector is the gaming of this so-called “performance data”.

If a client drops out of treatment it will have a negative impact on our figures. One way to manage this is not to start the most chaotic people in treatment in the first place. People aren’t refused treatment but they are asked to jump through hoops before structured treatment is commenced.

A homeless, mentally unwell heroin user is going to find it difficult to attend a “treatment induction group”, but the consequence is that they never start on the medication that might actually help them.

Some facts are impossible to hide: drug-related deaths are increasing and new drugs and associated problems are causing problems in prisons and emergency departments. Even the shadow health secretary, Jonathan Ashworth, whose father was an alcoholic, has called for greater recognition of the damage done by excessive drinking.

Drug and alcohol use and addiction isn’t going to go away. I try to do the best I can for the people I work with. I try to close my ears to the negative and stigmatising language. Instead I keep my ears open to my clients and I try to find a connection and build a relationship that may help them in their recovery.

This series aims to give a voice to the staff behind the public services that are hit by mounting cuts and rising demand, and so often denigrated by the press, politicians and public. If you would like to write an article for the series, contact kirstie.brewer@theguardian.com

Talk to us on Twitter via @Guardianpublic and sign up for your free weekly Guardian Public Leaders newsletter with news and analysis sent direct to you every Thursday.

Drug addiction isn"t going away so why are treatment centres being slashed?

UK children with cancer could miss out on drug trials after Brexit, doctors warn

Leading doctors are warning that British children with cancer could suffer if they are no longer able to join Europe-wide trials of innovative new medicines as a result of the Brexit deal.

The Institute of Cancer Research (ICR) and the Royal Marsden NHS Foundation Trust say the best hope for some children with cancer is a clinical trial where a new drug is being tested. But because of the small number of children with the same cancers, the trials have to be run in many hospitals, often across Europe.

If the UK leaves the European Union and withdraws from the currently London-based European Medicines Agency which licenses new drugs, as expected, then pharmaceutical companies may choose to trial drugs just for children from countries in the EU. Children in the UK would lose out, and it could take years before they could get access to the newest treatments.

The ICR and the Marsden say EU regulations governing the way medicines are tested in children badly need reform to make companies trial more drugs in children, but the UK would be worse off without them.

“It is imperfect but it is all we have,” said Prof Louis Chesler, a consultant in paediatric oncology at the Marsden.

Children’s cancer is a very small field, he said. “The most effective way to run a clinical trial is to run a big one. If the regulations change and stop us working across European sites, that is a big problem for us,” he said.

The ICR and the Marsden, in their response to a European commission consultation on the future of drug regulation for children, are calling for changes so that drug companies cannot so easily obtain a waiver and duck the obligation to do trials in children once they have shown a drug works in adults.

A new analysis by the ICR shows that over the past five years (2012-2016) pharmaceutical companies were granted waivers from having to trial cancer drugs in children for 33 of 53 approved cancer treatments.

“By allowing pharmaceutical companies to use waivers to avoid trials in children so they can focus on adult treatments, the regulation is stifling progress and could be stopping children receiving a treatment that could save their lives,” said Chesler.

Prof Paul Workman, the chief executive of the ICR, said: “Children with cancer are currently missing out on the kind of innovative cancer treatments that are becoming increasingly common in adults because of outdated European rules that have failed to keep up with advances in science.

“We’ve been urging decision-makers to change the regulation for several years now, so that adult cancer drugs are tested in children whenever their mechanism of action suggests they could be effective.

“This is a real chance for reform to prevent the current out-of-date approach from being cemented for a decade. It could also be the last chance to make meaningful changes that apply across Europe, including the UK, before we leave the EU. It’s vital that whatever deal the UK does preserves access to Europe-wide clinical trials for children with cancer and avoids creating even longer delays in children accessing the latest cancer medicines.”

Dr Lynley Marshall, a consultant in child and adult cancer drug development at the Marsden, said families who are going through the trauma of caring for a child with cancer should not be alarmed. She pointed out that children with cancer in countries outside the EU, as far away as Israel and Australia, participate in some of the big treatment trials because of the difficulties of getting enough children with the same condition in one place.

She did not think fewer children in the UK with cancer would be included in trials. “I think it would be difficult to be categorical about it, but we will all be working very hard to ensure that there wouldn’t be,” she said.

UK children with cancer could miss out on drug trials after Brexit, doctors warn

Cancer drug prices must come down, say leading research institutes

The high price of new cancer drugs is indefensible and unsustainable, say two of the world’s leading cancer research institutions, who propose a different way to develop them that could sideline big pharma.

“There is a clear and urgent necessity to lower cancer drug prices to keep lifesaving drugs available and affordable to patients,” say leading scientists from the Institute of Cancer Research in the UK and the University of Texas MD Anderson Cancer Center, where many important new cancer drugs have been invented, in a paper in the journal Cell.

In the US, cancer bills are the leading cause of personal bankruptcy, while in the UK, drugs that might prolong life are rejected for NHS use because of their price. Many new drugs have to be used in combination, adding to the cost. Treatment with the two new immunotherapy drugs nivolumab and ipilimumab costs $ 252,000, which is more than the median cost of a US home ($ 240,000 in 2016), they write.

Fantastic scientific work is going on – for instance, in sequencing cancer genomes – which should lead to advances in treatment, said Prof Paul Workman, chief executive of the Institute of Cancer Research in London, which is the world’s most successful academic cancer drug discovery organisation. “All this invention is meaningless if patients cannot afford these drugs,” he said.

“It is unsustainable. For those of us involved in research, it is disturbing that the amount of research that goes on and the success that is made is not translated into treatment for patients. And for patients it is a terrible situation.”

Pharmaceutical companies used to justify their prices by pointing to the high cost of clinical trials involving many thousands of patients. But that is no longer always necessary, the scientists say in their paper. The new targeted drugs require a test for a genetic biomarker to see whether patients will respond or not. That means the drug can be trialled on far fewer people. The drug crizotinib, used for advanced lung cancer, was approved following a trial involving only 347 patients, they point out. Trastuzumab (Herceptin) was first approved for advanced breast cancer and later for early breast cancer, increasing the market for the company but with no reduction in price.

“Some drugs are tested on 50 or 100 patients and yet these drugs still go to Nice [the National Institute for Health and Care Excellence, which decides whether the NHS can afford a new drug] at the maximum price,” said Workman.

Workman, together with colleagues from the US and the Netherlands, proposes that academic discovery centres like his should forge relationships with new commercial partners – probably not the major drug companies but smaller biotech or generic drug firms.

Academics should take greater control of the drugs they discover, they argue, and join with small companies that will agree to cap the price when the drug reaches the market. They would not have the expectation of big profit margins, as the major pharmaceutical companies do. But in an era where drugs are tested on smaller populations and genetic testing means they are more likely to be effective, they would not need to “cost in” all the failed attempts at producing blockbusters, as the big companies do.

Workman said the institute was already talking to small companies about the possibility of a new way of developing more affordable cancer drugs. He believes other scientists will support the ideas in the paper. “We’re calling for a more mature and open conversation about how this could be done and offering a solution,” he said.

Cancer drug prices must come down, say leading research institutes

Drug firms ‘put lives of cancer patients at risk’ as out-of-patent prices are inflated

Drug companies have been accused of profiteering by raising the prices of out-of-patent cancer medicines that cost just pence to make, inflating the bills of the cash-strapped NHS by hundreds of millions of pounds.

Academics say the prices of 14 cancer drugs have increased by between 100% and nearly 1,000% over the past five years in the UK. These are all generic drugs where the patent has expired, which means they can be made for little more than the cost of the raw ingredients.

But, say experts who presented their findings at the European Cancer Congress in Amsterdam, generic drug companies have been price-gouging, just as Turing Pharmaceuticals was found to have done in the US with Daraprim, a 70-year-old drug used in Aids treatment. The price rose from $ 13.50 to $ 750 to universal outrage and became an issue in the presidential election. Turing CEO Martin Shkreli was dubbed the most hated man in America.

Andrew Hill from the department of pharmacology and therapeutics at the University of Liverpool, said the price increases in the UK would be costing the NHS a huge amount of money at a time when it has said it may have to ration 20% of the very expensive new cancer drugs coming on the market. “This figure will probably be in the high hundreds of millions of pounds per year, or possibly £1 billion,” he said.

He and co-author Melissa Barber, from the London School of Hygiene and Tropical Medicine, funded by the World Health Organisation and the Open Society Foundation, could not calculate the exact costs to the NHS because they did not have access to data from hospitals, where cancer drugs are usually prescribed. But they also looked at the increase in prices of all types of generic drugs – not just cancer – that are prescribed by GPs and pharmacists and found that the NHS paid £380m more in 2015 than it had five years earlier, as generic companies pushed up their prices. The hospital bill is likely to be much more.

Hill said the rises were going unnoticed because they were “under the radar”. The NHS was not keeping an eye on the steady increases year on year. “They are not negotiating well enough,” he said. “They should be looking at any company that starts raising the price of any drug.”

Among the drugs with price rises is melphalan for ovarian cancer, a drug invented by the UK company GlaxoSmithKline and then passed with a number of other cancer drugs that were going out of patent to Aspen Pharmaceuticals, a South Africa-based generics company, in 2009. GSK took a 16% shareholding in Aspen at the time.

Hill and Barber say the price of melphalan in the UK went up from 55p for 2mg in 2011 to £1.82 in 2016, a rise of 230%. The NHS did not contest it, unlike in Italy. There, Aspen negotiated a deal in 2014 to raise the price of the drug by up to 1,500%, having threatened to stop supplying the ovarian cancer drug to patients altogether. The Italian competitions authority fined the company nearly $ 5.5m last October for its behaviour. “The negotiation strategy adopted by Aspen was so aggressive as to reach the credible threat of interrupting the direct supply of the drugs to the Italian market,” said the authority’s report.

Australia, New Zealand, France and Brazil have also faced shortages of Aspen’s drug, says Hill. Aspen did not return calls asking for the reason for the shortages.

In France, three women died after being given an alternative drug to melphalan. The drug, cyclophosphamide, should have been safe, because it was used before melphalan came on the market. An investigation is now under way into what happened.

The biggest price rise in the UK that the team found was also for an Aspen drug. The cost of busulfan for chronic myeloid leukaemia to the NHS rose from 21p for 2mg in 2011 to £2.61 in 2016, an increase of 1,143%.

Aspen’s share price rose more than 650% from 2009 to last year. GSK sold its shares in Aspen in three tranches – the last one in September – netting about £1.5bn.

The multinational giant Pfizer was fined £84m in the UK in December for conspiring with a generic company, Flynn Pharma, to raise the price paid by the NHS by 2,600% for an anti-epilepsy drug that came off-patent. The Competitions and Markets Authority said it had exploited the fact that the NHS had no alternative, because no other company was making it. In the wake of that case, a bill is going through parliament to give the Department of Health powers to investigate when the price of a drug goes up without clear justification.

Sarah Wollaston, the Tory MP who chairs the health select committee, said the research findings were concerning. “It is unacceptable for drug companies to artificially inflate the cost of drugs. This will inevitably result in less funding being available for other vital treatments,” she said.

Ian Banks, representing the patient organisation the European Men’s Health Forum at the conference, said the price rises were more than outrageous. “Behind these statistics there is a patient not getting treatment that could save their lives or at the very least improve their quality of life. That is unacceptable,” he said.

Mia Rosenblatt from Breast Cancer Now said: “We’d be extremely concerned by any suggestion of companies raising the price of tamoxifen and other generic drugs, which would only add to the current strain on the NHS.

“Part of the understanding – by health bodies – of the high cost of new drugs is based on the knowledge that there is a limited time for drug companies to see return on their investments and that these drugs will be available at a much cheaper rate in due course.

“Significant driving up of the prices of generic drugs would be of real concern and we therefore support the government’s activity to address this possibility through the Health Services Medical Supplies Bill.”

The British Generic Manufacturers Association said that most of the drugs highlighted in the study had no competition, because there was only one supplier. “That is because the total market size is too small to be attractive for generic companies to enter, since they would not recoup the million-pound plus costs of developing, testing and registering a new generic medicine,” it said in a statement.

Hill countered that the cost of developing a new generic drug is not high and that cheaper versions than those sold to the NHS could be imported from India.

Drug firms ‘put lives of cancer patients at risk’ as out-of-patent prices are inflated

Politics and protocol leave Indian teen"s life in the balance pending TB drug ruling | Amrit Dhillon

Shreya Tripathi sleeps most of the day. At night, she lies awake. Only 18, she has been fighting tuberculosis for five years. Her voice on the telephone from her home in Patna, eastern India, is a whisper. If she speaks for more than a few minutes, she becomes breathless.

Though exhausted, Shreya is also fighting another battle – in the Delhi high court – to demand a new TB drug. Every other medication she has tried has failed to beat the disease.

Shreya has a form of TB caused by bacteria resistant to treatment even with the most powerful drugs. She wants the Indian government to give her bedaquiline, the first new TB drug to be registered in more than 50 years. Its use is tightly controlled. Only six government hospitals are allowed to administer it, and even then only as a last resort.

India has one of the highest levels of drug-resistant tuberculosis in the world. To preserve bedaquiline’s effectiveness – if the bacteria mutate to resist it, there is nothing else available – the Indian government is strict on who can have it and how they are monitored. The National Institute of Tuberculosis and Respiratory Diseases in New Delhi, one of the authorised six centres, has refused to give Shreya the drug.

Shreya was diagnosed with TB in 2012, when she was 13. Doctors in Patna started her on a TB regimen but she proved resistant to the first and second lines of treatment. She and her father, Kaushal, a civil servant, are tired of running around hospitals getting nowhere, while Shreya’s condition worsens.

Two years ago, she had to drop out of school because she was so weak. She needs a wheelchair to get around. Swimming and badminton – her favourite sports – have become distant memories.

Shreya is a category five patient, which means she needs treatment for “extreme” drug resistant tuberculosis, or XDR-TB.

The family only became aware of bedaquiline in October, after a visit to Dr Zarir Udwadia, a consultant chest physician at Hinduja hospital in Mumbai. “It gave us hope. I was desperate by then because nothing had worked for my daughter,” says Kaushal.

Udwadia knew the exact combination of drugs that Shreya needed to take with bedaquiline, which does not work on its own. However, government protocol concerning the drug prevents him, as a private doctor, from accessing it. He told the family to get the drugs from the national institute in New Delhi, but they were refused because Shreya was not a resident.

“We argued and fought with them,” says Kaushal. “They agreed to take a sputum sample from Shreya in November for a drug susceptibility test to see which drugs she is resistant to, but they already knew she was drug resistant from earlier such tests. They wasted precious time.”

They kept calling the hospital for the result. Two months later, they were told the sample had been contaminated. On 28 December, Shreya provided a fresh sample and was told to wait four to six weeks for the culture.

“It was then I told Papa to go to court. Even if it’s too late for me, at least other patients will benefit from it. Just imagine how hard it must be for really poor people to get this drug,” says Shreya.

The case has been heard in Delhi high court this week.Saket Sikri, counsel for the national institute, says that the hospital cannot prescribe the other drugs that must be administered with bedaquiline until it gets the culture report.

“A wrong combination can kill and, since this drug is her last hope, we have to get it right. We are being humane, not bureaucratic, and are following World Health Organisation guidelines,” says Sikri. “The institute cannot choose which parts of the WHO protocol to follow and which to ignore.

“I think the judge’s final decision will hinge on whether he thinks my client is following WHO’s guidance on the use of [bedaquiline]. The judge can’t decide which doctor or which line of treatment is correct but he can judge if the guidelines are being followed and, in that respect, the institute is justified in waiting for the drug susceptibility test report to come.”

However, TB experts have said the culture the institute is awaiting is unnecessary, since it is already known that Shreya is drug resistant.

Anand Grover, a senior lawyer with the Lawyers Collective, which represents Shreya, says that the government has failed to update its own protocol to reflect the latest WHO guidance on bedaquiline, under which several XDR-TB patients have been put on drug regimens similar to the one prescribed by Udwadia. “There is evidence from other countries, including South Africa, showing that this combination has been successful in treating XDR-TB,” says Grover.

Grover has told the court that the prospect of Shreya losing her life without access to bedaquiline should outweigh concerns about any possible resistance that might occur. He has also told the court that the government is following the WHO protocol dating from 2013, when there was limited data on the efficacy and safety of the new drug.

Backing Shreya’s team is testimony from Dr Jennifer F Furin, from the Department of Global Health and Social Medicine at Harvard Medical School, who said Shreya should have been started on a bedaquiline-containing regimen in October.

“Additional delays … threaten her life and the effectiveness of this agent. It is unfortunate that there have already been so many significant delays in providing [bedaquiline] to Ms Tripathi,” said Dr Furin in her written testimony.

Dr Furin has previously criticised India for its slow rollout of the drug. In her Delhi high court testimony she said that scientific publications have set a benchmark that between 30% and 45% of patients with multi-drug resistant TB in a country should be able to access bedaquiline.

“In India, this means a minimum of 30,000 persons per year, based on 2016 estimates. As of 1 December 2016, only 164 individuals had been reported … to be receiving [bedaquiline]. This slow rollout … was noted as a problem by the WHO,” said Dr Furin.

There will be a further hearing on 18 January. “I don’t think the court will give it to me,” says Shreya. “But because of Papa’s efforts, at least other patients may get it later.”

Politics and protocol leave Indian teen"s life in the balance pending TB drug ruling | Amrit Dhillon

Laser-activated drug a "leap forward" for prostate cancer treatment

A drug activated by laser light successfully destroys early prostate cancer while avoiding side-effects that commonly occur with surgery, trial results have shown.

The new technique, called vascular-targeted photodynamic therapy (VTP), involves injecting a light-sensitive drug into the bloodstream. The drug is then “switched on” by laser pulses fired through optical fibres inserted into the prostate.

Of 196 men who received the treatment, about half showed no signs of the disease two years later, compared with 13.5% of those given standard care.

Because VTP targets only prostate tumours, it does not cause the long-term problems of impotence and urinary incontinence often associated with “radical” surgery or radiotherapy.

Lead investigator Professor Mark Emberton, consultant urologist at University College London hospital, said: “These results are excellent news for men with early localised prostate cancer, offering a treatment that can kill cancer without removing or destroying the prostate.

“This is truly a huge leap forward for prostate cancer treatment, which has previously lagged decades behind other solid cancers such as breast cancer.

“In 1975, almost everyone with breast cancer was given a radical mastectomy, but since then treatments have steady improved and we now rarely need to remove the whole breast.

“In prostate cancer, we are still commonly removing or irradiating the whole prostate, so the success of this new tissue-preserving treatment is welcome news indeed.”

Currently, men with low-risk localised prostate cancer are put under “active surveillance”, which means monitoring the disease but providing no treatment unless it becomes more severe.

In the trial consisting of 413 men, participants were randomly assigned either to VTP or active surveillance.

Only 6% of the VTP group later needed radical treatment, compared with 30% of active surveillance patients. VTP treatment also doubled the average time of cancer progression from 14 months to 28 months.

The trial, reported in the Lancet Oncology journal, was conducted across 47 treatment sites in 10 European countries, most of which were performing VTP for the first time.

Emberton said: “The fact that the treatment was performed so successfully by non-specialist centres in various health systems is really remarkable.

“New procedures are generally associated with a learning curve, but the lack of complications in the trial suggests that the treatment protocol is safe, efficient and relatively easy to scale up.

“We would also expect the treatment to be far more precise if we repeated it today, as technology has come a long way since the study began in 2011.

“We can now pinpoint prostate cancers using MRI (magnetic resonance imaging) scans and targeted biopsies, allowing a much more targeted approach to diagnosis and treatment.

“This means we could accurately identify men who would benefit from VTP and deliver treatment more precisely to the tumour.

“With such an approach, we should be able to achieve a significantly higher remission rate than in the trial and send nearly all low-risk localised prostate cancers into remission.

“We also hope that VTP will be effective against other types of cancer. The treatment was developed for prostate cancer because of the urgent need for new therapies, but it should be translatable to other solid cancers including breast and liver cancer.”

The drug used, WST11, is derived from bacteria at the bottom of the ocean. To survive with very little sunlight, the bugs have evolved to convert light into energy with high efficiency. This property was exploited to develop the drug, a compound that releases destructive tumour-busting “free radical” molecules when activated by laser light.

Gerald, a man aged in his 60s from Surrey, was one of the first patients to be treated with VTP under the care of Emberton.

He said: “The treatment … changed my life. I’m now cancer-free with no side-effects and don’t have to worry about needing surgery in future. I feel so lucky to be in this position.

“I’ve met other men who had surgery – they had to stay in hospital for days whereas I could go home the next day, and one suffered from terrible incontinence which he found very distressing.

“I had some minor side-effects for a few weeks after the operation, but I’m back to normal now.”

Each year, more than 46,000 men in the UK are diagnosed with prostate cancer and 11,000 die from the disease.

Laser-activated drug a "leap forward" for prostate cancer treatment

Watchdog accuses Actavis of hiking price of lifesaving drug by 12,000%

Pharmaceutical company Actavis is facing accusations from the competition watchdog that it hiked the price of life-saving hydrocortisone tablets by over 12,000%, sending the annual cost to the NHS spiralling from £522,000 before 2008 to £70m by 2015.

The Competition and Markets Authority said the price rises took place after the patent lapsed on the drug. About 943,000 packets of the tablets – prescribed to people whose adrenal glands do not produce enough steroid hormones, such as those suffering from Addison’s disease – were distributed over the last year.

As the CMA set out its reasons why the company had broken competition law, the regulator’s senior responsible officer, Andrew Groves, said: “This is a lifesaving drug relied on by thousands of patients, which the NHS has no choice but to continue purchasing.

“We allege that the company has taken advantage of this situation and the removal of the drug from price regulation, leaving the NHS – and ultimately the taxpayer – footing the bill for the substantial price rises,” said Groves.

The watchdog is continuing other investigations into the pharmaceutical sector. The latest allegations come after last week’s £84.2m fine on Pfizer for increasing the cost of an anti-epilepsy drug. The drugs distributor Flynn Pharma was fined £5.2m for charging excessive and unfair prices in the UK for an epilepsy treatment.

Groves said the current findings against Actavis were provisional and that it was too early to conclude there had been a breach of competition law. “The CMA will carefully consider any representations of the parties under investigation before determining whether the law has been infringed,” said Groves.

Actavis – formerly Auden Mckenzie – is facing accusations about the prices of two forms of the hydrocortisone tablets. The CMA said the increase in price to the NHS for 10mg packs rose from 70p in April 2008 to £88 by March 2016 – a 12,000% rise. For 20mg sizes the price rise was 9,500% – increasing to £102.74 per pack by March 2016, compared with £1.07 when the treatment was still branded.

Once medicine and treatments are no longer branded – known as generic medicine – they are no longer subject to price regulation, a move that is usually expected to reduce the cost rather than inflate it.

Actavis did not immediately comment.

Watchdog accuses Actavis of hiking price of lifesaving drug by 12,000%

The long, difficult search for a drug to treat Alzheimer’s and dementia

Most of us forget names, dates or places from time to time. But Hilary Doxford never did. While the rest of us smile about our common inadequacy, she knew she was experiencing a genuine malfunction of her high-performance brain. “I did have a really good memory and didn’t need to write things down,” she says. “And I used to be able to multiply two four-digit numbers together almost instantaneously.” But one day, she started getting the sums wrong.

The doctors sent her away at first. So you can’t remember names or multiply 3,765 by 1,983 any more? Oh well, that’s middle age for you. But Doxford, who was then in her late 40s, knew differently. “My benchmark is myself,” she says. “Even now, when I tell people, everybody says: ‘I would never have known.’ What I’m doing is no different from what other people are experiencing. But when I compare it to how I used to be … I can’t multiply a two-digit number by a one-digit number. I can do it if I add them – like 27 times three. It’s my short-term memory – when you need to hang on to a number because you need to use it. That’s what’s gone.”

Doxford went to the GP because her partner, Peter, had asked her to marry him. She wanted to be sure she would not be sentencing Peter to looking after a woman whose brain was deteriorating. “My GP said: ‘It’s normal – off you go.’”

Three years later, then a married woman, she went back. “All the symptoms were getting worse,” she says. Doxford is the general manager of a medical research charity.“I forgot the surnames of some of my staff. I started finding it hard to concentrate and focus. Then I started avoiding taking responsibility for things unless I absolutely had to.”

The GP said it was probably stress and sent her home again. But then she had a long, difficult business meeting with somebody, and when he greeted her three weeks later, she had no recollection of having met him before. This time she was sent for tests. She scored incredibly highly on the IQ part and incredibly poorly on memory, and was given an MRI scan. “The diagnosis was Alzheimer’s,” she says, matter-of-factly.

‘My benchmark is myself. When I compare it now to how I used to be’ … Hilary Doxford, who was diagnosed with Alzheimer’s in her early 50s. Photograph: Lydia Goldblatt for the Guardian

Dementia will take hold of one in three people who passes the age of 65, and costs the UK more than £26bn a year. This week the Office for National Statistics announced it is now the commonest cause of death in England and Wales, passing cancer and heart disease. People with dementia lose the ability to care for themselves and can become malnourished, while their immune system weakens. Infections such as pneumonia may be the actual agent of death, but dementia is the underlying cause.

It is a cruel disease, which takes away the person their families love and know, leaving a stranger who looks at them with confusion. And while there are are some drugs that will temporarily alleviate symptoms in some people, there is no cure. But dementia, of which Alzheimer’s is the commonest form, has finally begun to get the attention it deserves. In December 2013, the G8 countries, meeting in London, agreed to set an ambition to cure or come up with a significant treatment for Alzheimer’s by 2025. Last year David Cameron announced that the UK would set up a dedicated dementia research institute, with initial funding of £150m, and a further £100m from Alzheimer’s charities. Although the UK was already spending £300m on dementia research, Cameron believed it should be afforded the same level of resources as Aids and climate change. That was welcome news to campaigners, although, they say, the sum is still much less than that invested in cancer research (£590m in 2010).

Doxford, now 56, was glad to have a diagnosis. “On the one hand, it was a relief, because it explained all the problems I’d been having just doing normal stuff. On the other hand, ‘Oh shit’. The first question I asked the consultant was: ‘How long have I got being normal?’ He was implying that I only had two to three years, but then he said: ‘I do know somebody who is eight years down the line and she is pretty much OK.’” Others Doxford has met have since told her of people managing fine 10 to 15 years after diagnosis. For her, it has now been nine.

Initially she was put on Aricept (the brand name of donepezil), one of the few Alzheimer’s drugs currently available. These drugs help some people by delaying the worsening of symptoms, although they progress faster later on. But they do not work for everyone.

“It was pretty horrendous,” says Doxford. “I seemed to get all the side effects on the packet.” She began falling over. The worst was when she was tying up the boat that she and her husband kept. “We were in the marina and I had the rope in my hand. The next thing I knew, I was in the water.” They sold the boat, fearing for her safety. The consultant gave her another drug, but it was worse. “The first day I took it, I thought I was dying,” she says. She decided drugs were not for her.

There ought to be big money in Alzheimer’s drugs. It is more than a century since abnormal protein deposits in the brain were identified by the German psychiatrist Alois Alzheimer as a likely cause of neurodegeneration. Over the past few decades, drug research has focused largely on attempts to clear the amyloid-beta peptides and tau proteins believed to cause these deposits, which gradually shut down the brain’s normal workings.

But there has been a very high failure rate, often at a late stage of development, when companies have spent a great deal of money – in some cases as much as $ 300m – on research and development. Dr Eric Karran, formerly the director of research at Alzheimer’s Research UK and now leading the efforts of drug company Abbvie to find a cure, says that yes, it’s difficult, but too often in these failed trials, the preparatory work has not been thorough enough.

“Some people in the field would say that [the failure rate] has been a disaster,” he says. They suggest that the underlying concept, that Alzheimer’s is caused by amyloid plaque, may be wrong. “I would not subscribe to that view. There has been some sloppy science. We would not expect such trials to work anyway. In some cases there has been a very strong commercial push. In other cases, there has been a strong desire to get something into [human trials] because we have so little.”

Nobody would say research into Alzheimer’s is easy, says Dr Mike Hutton, chief scientific officer for neuroscientific drug discovery at the pharmaceutical company Lilly. “Amyloid accumulates in the brain for 10 to 15 years before you see clinical symptoms. There is a worry that we’re focusing on populations that are too far advanced.” Ideally, scientists must figure out who is most likely to get Alzheimer’s – then see if they can prevent healthy brains from developing it. But although there are some predictive tests available, it is hardly ethical to offer them to people when medical science cannot offer them treatment.

So the trials must be done in people with the earliest stages of dementia. And this year, for the first time, the results of two separate early trials have suggested that it may be possible to find drugs that might slow down the decay for people with mild dementia. Small wonder there have been wild headlines about a cure – a bit of good news is desperately wanted. One, Lilly’s solanezumab, a monoclonal antibody, failed in a large 2012 trial to slow the deterioration in most people with Alzheimer’s, but there was some improvement in those with the mildest form of the disease. Results from a new trial of more than 2,000 people with early-stage Alzheimer’s are eagerly anticipated, and due before the end of the year.

Another drug, Biogen’s aducanumab, made headlines in September. Trial results showed it almost completely cleared amyloid from the brains of a group of early Alzheimer’s patients,and that the expected deterioration slowed down significantly. The trial was small (166 people), and at the high doses that produced the best results, there were side effects such as headaches, but there was excited talk of “a game-changer”.

There has been such hype before, but David Reynolds, chief scientific officer of Alzheimer’s Research UK, says he is reasonably confident that the solanezumab results in December will also show an improvement in the symptoms of dementia, or at least a slowing of the decline. Sadly, there still seems little hope for those who already have moderate to severe dementia, beyond care and compassion.

In a nursing home in Hertfordshire, surrounded by trees and green spaces, James Gatesman, a former coroner’s officer and enthusiastic allotment gardener, spent the last years of his life in bed, his long legs angled to the wall, under a framed photograph of his Metropolitan police class at Hendon College. A yellow spot attached to the glass identified his own face in the picture.

Deborah Gateman with her father, James, before he died. Photograph: Lydia Goldblatt for the Guardian

You could still see the fine figure of a man that Gatesman had been – 6ft 2in with broad shoulders and a strong, handsome face. But he had ceased to speak, and could no longer move his own limbs. His daughter Deborah and his wife, Doreen, could only communicate with him through touch, or his occasional grunts, moans and howls.

“I always talk to him and treat him as if he understands us,” said Deborah, shortly before her father died. “I think of it as almost like locked-in syndrome – that he is in there. I worry about what we discuss in front of him.” When his wife paid for him to have music therapy, Gatesman responded to the Gilbert and Sullivan tunes he used to love with noises and movement of his hands. That confirmed Deborah’s belief that he was more aware than he seemed.

Gatesman was diagnosed in 2003, but Doreen recalls odd behaviour on a holiday in Australia in 1996, when he wandered away from their group and nobody knew where he was. In later years, his behaviour became unpredictable, and he refused to go to a doctor. “That was probably the worst time,” she said. “Where do you go for help?”

Eventually Gatesman had to see a GP because of an ear infection, and ended up in the memory clinic. He had a brain scan and was diagnosed with Alzheimer’s. The progression was slow but inexorable. He was on Aricept for a year, but that was stopped when he was sent to a residential assessment centre – a care home where people with dementia spend some weeks or months while their condition and needs are determined. Instead, he was put on the drugs that are too often used to control the sometimes bizarre and agitated behaviour of dementia patients – antipsychotics. “He was throwing chairs around and he did pull a handrail off the wall,” Doreen says. When they visited him, he was no longer walking, but sitting in a reclining chair. “He was a bit of a dribbling mess,” said Deborah. “The antipsychotics were turning him into a zombie.” By the time he was moved to the long-term home in Hertfordshire, he was underweight. Doreen insisted the antipsychotics were stopped, and he regained not just his weight, but his spirit. Then “they had quite a lively character on their hands. He would run off down the corridors,” said Deborah. Or he would speak to the Polish nurses in the Flemish and German he had learned while in the RAF, in the war.

The Alzheimer’s Society has campaigned to reduce the use of antipsychotics – often referred to as the “chemical cosh” – in care homes. Dr Doug Brown, the organisation’s research director, says prescriptions have markedly reduced as a result, though “we wouldn’t say the problem has gone away. We still need to keep an eye on it”. The Society has piloted staff training in more than 100 care homes for “person-centred care”, which appears to cut the use of drugs dramatically. It involves taking the trouble to understand the triggers for an individual’s distress – such as the man who gathered all his furniture in the centre of his room every day and became upset when staff put it back. He was a decorator. Given a brush and a bucket of water, he spent contented hours thinking he was painting the walls.

The overuse of antipsychotics is one outcome of the past neglect of dementia, and further evidence of the real need for a drug that will do more than just quieten patients down – one that will slow the progress of a devastating disease and eventually, hopefully, cure it.

The long, difficult search for a drug to treat Alzheimer’s and dementia

Breast cancer drug approved for NHS use after price cut

A drug that can help shrink breast cancer tumours before patients undergo surgery to remove them has been approved for use in the NHS, after the manufacturer agreed a substantial discount on the list price.

Perjeta, the brand name of pertuzumab, could be helpful in the treatment of 1,400 women a year who develop a particularly aggressive form of breast cancer, but it was initially turned down by Nice, the National Institute for Healthcare Excellence, because of the high price set by the manufacturer, Roche.

Nice said it was also uncertain that the drug treatment would help prevent the cancer coming back.

Prof Carole Longson, director of the centre for health technology assessment at Nice, said there was only limited evidence of how well the drug worked because it had been quickly licensed on the back of promising but early trial data.

She said Nice was glad Roche had agreed to drop the price, though she would not reveal by how much.

“The price discount means that, even with the uncertainties in the evidence highlighted by the committee, pertuzumab represents a cost-effective use of NHS money,” Longson said.

The list price of pertuzumab is £2,395 per 420mg vial (excluding VAT). The total cost of four cycles of treatment with pertuzumab – the maximum Nice says should be used – would be £9,580 before the agreed discount.

The drug is used in combination with two others, trastuzumab (Herceptin) and docetaxel (a type of chemotherapy), to shrink tumours prior to surgery. If it works well, it may mean some tumours that were previously inoperable could be surgically removed.

Pertuzumab has been developed to help treat the 10-15% of breast cancers that are HER2-positive, which can be particularly aggressive.

Nice pointed out that the decision was the third green light for a cancer drug in as many weeks. It has been criticised for failing to approve cancer drugs, which often come on to the market at high prices.

Mia Rosenblatt, assistant director of policy and campaigns at Breast Cancer Now, said it was a huge leap forward. “Perjeta is the first addition to primary breast cancer treatment to be approved by Nice since 2006 and marks the introduction of a new type of breast cancer medicine, to be used before surgery,” she said.

“For the small number of women eligible, this drug could mean an enormous amount. It could help shrink their tumours to reduce the extent of the surgery they require or even make inoperable cancers operable.”

Samia al-Qadhi, the chief executive of Breast Cancer Care, said: “This is an exciting turning point. Women with certain aggressive types of breast cancer will have access to an extra drug before surgery that can boost the success of shrinking the tumour. Crucially this may mean people’s long-term survival improves.”

Breast cancer drug approved for NHS use after price cut