Tag Archives: Drug

Cannabis drug cuts seizures in children with severe epilepsy in trial

A new drug derived from cannabis has been shown to reduce the convulsive seizures experienced by children with a severe form of epilepsy by nearly a half – and in a small number, stop them altogether.

Doctors involved in the trials say the drug could change the lives of thousands of children for whom there is little treatment, and might also help children and adults with more common forms of epilepsy.

Dravet syndrome, which affects one in 40,000 children in the UK, can cause life-threatening convulsions several times a day. The trial at Great Ormond Street children’s hospital in London and centres in the US and Europe was launched because some parents desperate to help their children told of improvements after giving them cannabis derivatives bought on the internet.

“There was a lot of interest on the internet three to four years ago,” said Prof Helen Cross, a consultant in paediatric neurology at Great Ormond Street. That led to the trial of a carefully formulated pharmaceutical form of cannabidiol with virtually no THC (tetrahydrocannabinol), which is responsible for psychoactive effects.

“This is cannabidiol. It is not the oils that are available over the internet and the results cannot be ascribed to that,” she said. “Families should not be feeling this is something they should be able to get [for themselves]. This is a pharmaceutical product.”

The trial involved 120 children, aged two to 18, with an average age of nine. They were randomly assigned to take either cannabidiol in liquid form twice a day or a placebo. Neither the families nor the doctors knew which children were getting the active drug.

On average, the seizures experienced by the children were reduced by nearly 40% and 43% of those taking cannabidiol saw their seizures cut by half. Three children – 5% – stopped having seizures altogether. There were side-effects, which included drowsiness, fatigue, diarrhoea and reduced appetite – but these are similar to those caused by other epilepsy drugs.

The drug is not a cure, however. Cross said seizures returned in those who had stopped the drug. Children would probably be on the medication for life.

There is a need for more and better epilepsy drugs. A third of people with epilepsy do not respond to those that exist. Doctors think cannabidiol may work in at least some of those cases too, although the reason it works in the case of Dravet syndrome is unclear. “I have to say we don’t know,” said Cross. But asked whether it could be effective in other children and adults, she said, “Probably, yes.”

In young women, there has been concern over the drug sodium valproate, which can cause birth defects. Women and girls who may get pregnant are faced with deciding whether to stop taking a drug that may successfully keep their epilepsy under control.

Cross said cannabidiol may also prove to be an option for them, although trials would need to be done.

The results of the trial are published in the New England Journal of Medicine. In a commentary in the journal, Samuel Berkovic, from the Epilepsy Research Centre of the University of Melbourne, called medicinal cannabis “a hot-button issue in the treatment of epilepsy”, after anecdotal reports in the media of “spectacular results, coupled with the allure of using a ‘natural’ compound and long-held beliefs surrounding its recreational use”.

The trial was the beginning of solid evidence for the use of cannabinoids in epilepsy, but more research was needed, he said.

GW Pharmaceuticals, which makes the drug, will apply for a licence to the authorities in the US and Europe. If it is approved, the National Institute for Health and Care Excellence will have to assess the drug for cost-effectiveness before it can be used in the NHS.

Cannabis drug cuts seizures in children with severe epilepsy in trial

A new drug derived from cannabis has been shown to reduce the convulsive seizures experienced by children with a severe form of epilepsy by nearly a half – and in a small number, stop them altogether.

Doctors involved in the trials say the drug could change the lives of thousands of children for whom there is little treatment, and might also help children and adults with more common forms of epilepsy.

Dravet syndrome, which affects one in 40,000 children in the UK, can cause life-threatening convulsions several times a day. The trial at Great Ormond Street children’s hospital in London and centres in the US and Europe was launched because some parents desperate to help their children told of improvements after giving them cannabis derivatives bought on the internet.

“There was a lot of interest on the internet three to four years ago,” said Prof Helen Cross, a consultant in paediatric neurology at Great Ormond Street. That led to the trial of a carefully formulated pharmaceutical form of cannabidiol with virtually no THC (tetrahydrocannabinol), which is responsible for psychoactive effects.

“This is cannabidiol. It is not the oils that are available over the internet and the results cannot be ascribed to that,” she said. “Families should not be feeling this is something they should be able to get [for themselves]. This is a pharmaceutical product.”

The trial involved 120 children, aged two to 18, with an average age of nine. They were randomly assigned to take either cannabidiol in liquid form twice a day or a placebo. Neither the families nor the doctors knew which children were getting the active drug.

On average, the seizures experienced by the children were reduced by nearly 40% and 43% of those taking cannabidiol saw their seizures cut by half. Three children – 5% – stopped having seizures altogether. There were side-effects, which included drowsiness, fatigue, diarrhoea and reduced appetite – but these are similar to those caused by other epilepsy drugs.

The drug is not a cure, however. Cross said seizures returned in those who had stopped the drug. Children would probably be on the medication for life.

There is a need for more and better epilepsy drugs. A third of people with epilepsy do not respond to those that exist. Doctors think cannabidiol may work in at least some of those cases too, although the reason it works in the case of Dravet syndrome is unclear. “I have to say we don’t know,” said Cross. But asked whether it could be effective in other children and adults, she said, “Probably, yes.”

In young women, there has been concern over the drug sodium valproate, which can cause birth defects. Women and girls who may get pregnant are faced with deciding whether to stop taking a drug that may successfully keep their epilepsy under control.

Cross said cannabidiol may also prove to be an option for them, although trials would need to be done.

The results of the trial are published in the New England Journal of Medicine. In a commentary in the journal, Samuel Berkovic, from the Epilepsy Research Centre of the University of Melbourne, called medicinal cannabis “a hot-button issue in the treatment of epilepsy”, after anecdotal reports in the media of “spectacular results, coupled with the allure of using a ‘natural’ compound and long-held beliefs surrounding its recreational use”.

The trial was the beginning of solid evidence for the use of cannabinoids in epilepsy, but more research was needed, he said.

GW Pharmaceuticals, which makes the drug, will apply for a licence to the authorities in the US and Europe. If it is approved, the National Institute for Health and Care Excellence will have to assess the drug for cost-effectiveness before it can be used in the NHS.

Cannabis drug cuts seizures in children with severe epilepsy in trial

A new drug derived from cannabis has been shown to reduce the convulsive seizures experienced by children with a severe form of epilepsy by nearly a half – and in a small number, stop them altogether.

Doctors involved in the trials say the drug could change the lives of thousands of children for whom there is little treatment, and might also help children and adults with more common forms of epilepsy.

Dravet syndrome, which affects one in 40,000 children in the UK, can cause life-threatening convulsions several times a day. The trial at Great Ormond Street children’s hospital in London and centres in the US and Europe was launched because some parents desperate to help their children told of improvements after giving them cannabis derivatives bought on the internet.

“There was a lot of interest on the internet three to four years ago,” said Prof Helen Cross, a consultant in paediatric neurology at Great Ormond Street. That led to the trial of a carefully formulated pharmaceutical form of cannabidiol with virtually no THC (tetrahydrocannabinol), which is responsible for psychoactive effects.

“This is cannabidiol. It is not the oils that are available over the internet and the results cannot be ascribed to that,” she said. “Families should not be feeling this is something they should be able to get [for themselves]. This is a pharmaceutical product.”

The trial involved 120 children, aged two to 18, with an average age of nine. They were randomly assigned to take either cannabidiol in liquid form twice a day or a placebo. Neither the families nor the doctors knew which children were getting the active drug.

On average, the seizures experienced by the children were reduced by nearly 40% and 43% of those taking cannabidiol saw their seizures cut by half. Three children – 5% – stopped having seizures altogether. There were side-effects, which included drowsiness, fatigue, diarrhoea and reduced appetite – but these are similar to those caused by other epilepsy drugs.

The drug is not a cure, however. Cross said seizures returned in those who had stopped the drug. Children would probably be on the medication for life.

There is a need for more and better epilepsy drugs. A third of people with epilepsy do not respond to those that exist. Doctors think cannabidiol may work in at least some of those cases too, although the reason it works in the case of Dravet syndrome is unclear. “I have to say we don’t know,” said Cross. But asked whether it could be effective in other children and adults, she said, “Probably, yes.”

In young women, there has been concern over the drug sodium valproate, which can cause birth defects. Women and girls who may get pregnant are faced with deciding whether to stop taking a drug that may successfully keep their epilepsy under control.

Cross said cannabidiol may also prove to be an option for them, although trials would need to be done.

The results of the trial are published in the New England Journal of Medicine. In a commentary in the journal, Samuel Berkovic, from the Epilepsy Research Centre of the University of Melbourne, called medicinal cannabis “a hot-button issue in the treatment of epilepsy”, after anecdotal reports in the media of “spectacular results, coupled with the allure of using a ‘natural’ compound and long-held beliefs surrounding its recreational use”.

The trial was the beginning of solid evidence for the use of cannabinoids in epilepsy, but more research was needed, he said.

GW Pharmaceuticals, which makes the drug, will apply for a licence to the authorities in the US and Europe. If it is approved, the National Institute for Health and Care Excellence will have to assess the drug for cost-effectiveness before it can be used in the NHS.

Cannabis drug cuts seizures in children with severe epilepsy in trial

A new drug derived from cannabis has been shown to reduce the convulsive seizures experienced by children with a severe form of epilepsy by nearly a half – and in a small number, stop them altogether.

Doctors involved in the trials say the drug could change the lives of thousands of children for whom there is little treatment, and might also help children and adults with more common forms of epilepsy.

Dravet syndrome, which affects one in 40,000 children in the UK, can cause life-threatening convulsions several times a day. The trial at Great Ormond Street children’s hospital in London and centres in the US and Europe was launched because some parents desperate to help their children told of improvements after giving them cannabis derivatives bought on the internet.

“There was a lot of interest on the internet three to four years ago,” said Prof Helen Cross, a consultant in paediatric neurology at Great Ormond Street. That led to the trial of a carefully formulated pharmaceutical form of cannabidiol with virtually no THC (tetrahydrocannabinol), which is responsible for psychoactive effects.

“This is cannabidiol. It is not the oils that are available over the internet and the results cannot be ascribed to that,” she said. “Families should not be feeling this is something they should be able to get [for themselves]. This is a pharmaceutical product.”

The trial involved 120 children, aged two to 18, with an average age of nine. They were randomly assigned to take either cannabidiol in liquid form twice a day or a placebo. Neither the families nor the doctors knew which children were getting the active drug.

On average, the seizures experienced by the children were reduced by nearly 40% and 43% of those taking cannabidiol saw their seizures cut by half. Three children – 5% – stopped having seizures altogether. There were side-effects, which included drowsiness, fatigue, diarrhoea and reduced appetite – but these are similar to those caused by other epilepsy drugs.

The drug is not a cure, however. Cross said seizures returned in those who had stopped the drug. Children would probably be on the medication for life.

There is a need for more and better epilepsy drugs. A third of people with epilepsy do not respond to those that exist. Doctors think cannabidiol may work in at least some of those cases too, although the reason it works in the case of Dravet syndrome is unclear. “I have to say we don’t know,” said Cross. But asked whether it could be effective in other children and adults, she said, “Probably, yes.”

In young women, there has been concern over the drug sodium valproate, which can cause birth defects. Women and girls who may get pregnant are faced with deciding whether to stop taking a drug that may successfully keep their epilepsy under control.

Cross said cannabidiol may also prove to be an option for them, although trials would need to be done.

The results of the trial are published in the New England Journal of Medicine. In a commentary in the journal, Samuel Berkovic, from the Epilepsy Research Centre of the University of Melbourne, called medicinal cannabis “a hot-button issue in the treatment of epilepsy”, after anecdotal reports in the media of “spectacular results, coupled with the allure of using a ‘natural’ compound and long-held beliefs surrounding its recreational use”.

The trial was the beginning of solid evidence for the use of cannabinoids in epilepsy, but more research was needed, he said.

GW Pharmaceuticals, which makes the drug, will apply for a licence to the authorities in the US and Europe. If it is approved, the National Institute for Health and Care Excellence will have to assess the drug for cost-effectiveness before it can be used in the NHS.

Cannabis drug cuts seizures in children with severe epilepsy in trial

A new drug derived from cannabis has been shown to reduce the convulsive seizures experienced by children with a severe form of epilepsy by nearly a half – and in a small number, stop them altogether.

Doctors involved in the trials say the drug could change the lives of thousands of children for whom there is little treatment, and might also help children and adults with more common forms of epilepsy.

Dravet syndrome, which affects one in 40,000 children in the UK, can cause life-threatening convulsions several times a day. The trial at Great Ormond Street children’s hospital in London and centres in the US and Europe was launched because some parents desperate to help their children told of improvements after giving them cannabis derivatives bought on the internet.

“There was a lot of interest on the internet three to four years ago,” said Prof Helen Cross, a consultant in paediatric neurology at Great Ormond Street. That led to the trial of a carefully formulated pharmaceutical form of cannabidiol with virtually no THC (tetrahydrocannabinol), which is responsible for psychoactive effects.

“This is cannabidiol. It is not the oils that are available over the internet and the results cannot be ascribed to that,” she said. “Families should not be feeling this is something they should be able to get [for themselves]. This is a pharmaceutical product.”

The trial involved 120 children, aged two to 18, with an average age of nine. They were randomly assigned to take either cannabidiol in liquid form twice a day or a placebo. Neither the families nor the doctors knew which children were getting the active drug.

On average, the seizures experienced by the children were reduced by nearly 40% and 43% of those taking cannabidiol saw their seizures cut by half. Three children – 5% – stopped having seizures altogether. There were side-effects, which included drowsiness, fatigue, diarrhoea and reduced appetite – but these are similar to those caused by other epilepsy drugs.

The drug is not a cure, however. Cross said seizures returned in those who had stopped the drug. Children would probably be on the medication for life.

There is a need for more and better epilepsy drugs. A third of people with epilepsy do not respond to those that exist. Doctors think cannabidiol may work in at least some of those cases too, although the reason it works in the case of Dravet syndrome is unclear. “I have to say we don’t know,” said Cross. But asked whether it could be effective in other children and adults, she said, “Probably, yes.”

In young women, there has been concern over the drug sodium valproate, which can cause birth defects. Women and girls who may get pregnant are faced with deciding whether to stop taking a drug that may successfully keep their epilepsy under control.

Cross said cannabidiol may also prove to be an option for them, although trials would need to be done.

The results of the trial are published in the New England Journal of Medicine. In a commentary in the journal, Samuel Berkovic, from the Epilepsy Research Centre of the University of Melbourne, called medicinal cannabis “a hot-button issue in the treatment of epilepsy”, after anecdotal reports in the media of “spectacular results, coupled with the allure of using a ‘natural’ compound and long-held beliefs surrounding its recreational use”.

The trial was the beginning of solid evidence for the use of cannabinoids in epilepsy, but more research was needed, he said.

GW Pharmaceuticals, which makes the drug, will apply for a licence to the authorities in the US and Europe. If it is approved, the National Institute for Health and Care Excellence will have to assess the drug for cost-effectiveness before it can be used in the NHS.

Cannabis drug cuts seizures in children with severe epilepsy in trial

A new drug derived from cannabis has been shown to reduce the convulsive seizures experienced by children with a severe form of epilepsy by nearly a half – and in a small number, stop them altogether.

Doctors involved in the trials say the drug could change the lives of thousands of children for whom there is little treatment, and might also help children and adults with more common forms of epilepsy.

Dravet syndrome, which affects one in 40,000 children in the UK, can cause life-threatening convulsions several times a day. The trial at Great Ormond Street children’s hospital in London and centres in the US and Europe was launched because some parents desperate to help their children told of improvements after giving them cannabis derivatives bought on the internet.

“There was a lot of interest on the internet three to four years ago,” said Prof Helen Cross, a consultant in paediatric neurology at Great Ormond Street. That led to the trial of a carefully formulated pharmaceutical form of cannabidiol with virtually no THC (tetrahydrocannabinol), which is responsible for psychoactive effects.

“This is cannabidiol. It is not the oils that are available over the internet and the results cannot be ascribed to that,” she said. “Families should not be feeling this is something they should be able to get [for themselves]. This is a pharmaceutical product.”

The trial involved 120 children, aged two to 18, with an average age of nine. They were randomly assigned to take either cannabidiol in liquid form twice a day or a placebo. Neither the families nor the doctors knew which children were getting the active drug.

On average, the seizures experienced by the children were reduced by nearly 40% and 43% of those taking cannabidiol saw their seizures cut by half. Three children – 5% – stopped having seizures altogether. There were side-effects, which included drowsiness, fatigue, diarrhoea and reduced appetite – but these are similar to those caused by other epilepsy drugs.

The drug is not a cure, however. Cross said seizures returned in those who had stopped the drug. Children would probably be on the medication for life.

There is a need for more and better epilepsy drugs. A third of people with epilepsy do not respond to those that exist. Doctors think cannabidiol may work in at least some of those cases too, although the reason it works in the case of Dravet syndrome is unclear. “I have to say we don’t know,” said Cross. But asked whether it could be effective in other children and adults, she said, “Probably, yes.”

In young women, there has been concern over the drug sodium valproate, which can cause birth defects. Women and girls who may get pregnant are faced with deciding whether to stop taking a drug that may successfully keep their epilepsy under control.

Cross said cannabidiol may also prove to be an option for them, although trials would need to be done.

The results of the trial are published in the New England Journal of Medicine. In a commentary in the journal, Samuel Berkovic, from the Epilepsy Research Centre of the University of Melbourne, called medicinal cannabis “a hot-button issue in the treatment of epilepsy”, after anecdotal reports in the media of “spectacular results, coupled with the allure of using a ‘natural’ compound and long-held beliefs surrounding its recreational use”.

The trial was the beginning of solid evidence for the use of cannabinoids in epilepsy, but more research was needed, he said.

GW Pharmaceuticals, which makes the drug, will apply for a licence to the authorities in the US and Europe. If it is approved, the National Institute for Health and Care Excellence will have to assess the drug for cost-effectiveness before it can be used in the NHS.

Cannabis drug cuts seizures in children with severe epilepsy in trial

A new drug derived from cannabis has been shown to reduce the convulsive seizures experienced by children with a severe form of epilepsy by nearly a half – and in a small number, stop them altogether.

Doctors involved in the trials say the drug could change the lives of thousands of children for whom there is little treatment, and might also help children and adults with more common forms of epilepsy.

Dravet syndrome, which affects one in 40,000 children in the UK, can cause life-threatening convulsions several times a day. The trial at Great Ormond Street children’s hospital in London and centres in the US and Europe was launched because some parents desperate to help their children told of improvements after giving them cannabis derivatives bought on the internet.

“There was a lot of interest on the internet three to four years ago,” said Prof Helen Cross, a consultant in paediatric neurology at Great Ormond Street. That led to the trial of a carefully formulated pharmaceutical form of cannabidiol with virtually no THC (tetrahydrocannabinol), which is responsible for psychoactive effects.

“This is cannabidiol. It is not the oils that are available over the internet and the results cannot be ascribed to that,” she said. “Families should not be feeling this is something they should be able to get [for themselves]. This is a pharmaceutical product.”

The trial involved 120 children, aged two to 18, with an average age of nine. They were randomly assigned to take either cannabidiol in liquid form twice a day or a placebo. Neither the families nor the doctors knew which children were getting the active drug.

On average, the seizures experienced by the children were reduced by nearly 40% and 43% of those taking cannabidiol saw their seizures cut by half. Three children – 5% – stopped having seizures altogether. There were side-effects, which included drowsiness, fatigue, diarrhoea and reduced appetite – but these are similar to those caused by other epilepsy drugs.

The drug is not a cure, however. Cross said seizures returned in those who had stopped the drug. Children would probably be on the medication for life.

There is a need for more and better epilepsy drugs. A third of people with epilepsy do not respond to those that exist. Doctors think cannabidiol may work in at least some of those cases too, although the reason it works in the case of Dravet syndrome is unclear. “I have to say we don’t know,” said Cross. But asked whether it could be effective in other children and adults, she said, “Probably, yes.”

In young women, there has been concern over the drug sodium valproate, which can cause birth defects. Women and girls who may get pregnant are faced with deciding whether to stop taking a drug that may successfully keep their epilepsy under control.

Cross said cannabidiol may also prove to be an option for them, although trials would need to be done.

The results of the trial are published in the New England Journal of Medicine. In a commentary in the journal, Samuel Berkovic, from the Epilepsy Research Centre of the University of Melbourne, called medicinal cannabis “a hot-button issue in the treatment of epilepsy”, after anecdotal reports in the media of “spectacular results, coupled with the allure of using a ‘natural’ compound and long-held beliefs surrounding its recreational use”.

The trial was the beginning of solid evidence for the use of cannabinoids in epilepsy, but more research was needed, he said.

GW Pharmaceuticals, which makes the drug, will apply for a licence to the authorities in the US and Europe. If it is approved, the National Institute for Health and Care Excellence will have to assess the drug for cost-effectiveness before it can be used in the NHS.

Drug and alcohol charity Lifeline Project collapses

One of the UK’s leading drug and alcohol treatment charities has collapsed days after the Charity Commission launched an investigation into claims that it had critically weak financial controls.

Frantic efforts are being made to save the jobs of 1,300 employees of the charity, Manchester-based Lifeline Project, and the services it provides for 80,000 people a year, including prisoners in 22 jails and young offender institutions.

Staff were told on Thursday that the charity was seeking to transfer services to other providers and warned that not all the work the charity does would continue.

The shock failure of Lifeline follows the collapse of the charities Kids Company in 2015 and 4Children in 2016 and is likely to reignite the debate about the running of essential public services by charities.

Lifeline was set up in 1971 and grew particularly rapidly in recent years. Its annual income soared from £26m in 2012-13 to almost £62m in 2015-16, when it reported annual growth of 45%.

It was formerly chaired by Paul Flowers, the disgraced former Methodist minister and Co-op Bank chair whose drug use was exposed in 2014. He was asked to resign from Lifeline in 2004 over allegedly excessive expenses claims.

Allegations of mismanagement were made to the commission last month by Roger Howard, a former trustee of Lifeline’s board, who is a leading figure in the drug and alcohol treatment field and served as chief executive of the UK Drug Policy Commission.

Howard said on Thursday: “Those of us who work in the charity sector and have a long history of serving on boards or as executives know that good governance is absolutely crucial. We have seen examples of other bodies, like Kids Company, where governance and leadership has been questioned.

“It’s really critically important to ensure that organisations delivering large volumes of public services have the right assurances in place.”

Howard served on Lifeline’s board from December 2015 to November last year, when he was asked to resign after seeking to raise concerns. These included the sudden rundown of the charity’s working reserves.

The Charity Commission said on Thursday that Lifeline’s collapse highlighted the need for tight financial controls and oversight by charity trustees.

Paul Holdsworth, the commission’s chief operating officer, said: “We are sad to hear of the charity’s planned closure, though note that the trustees have worked to ensure that the majority of its services will continue and that the impact on beneficiaries and staff is managed and minimised.

“In the meantime we are engaged with the charity and its trustees, both to assess the events that have led up to this outcome and to ensure that trustees fulfil their duties and responsibilities in winding the charity up and passing its services to another charity.”

The charity runs drug and alcohol services across much of England and in Scotland. Its last annual report said it employed almost 1,500 people, but it is believed that many have been made redundant in recent weeks in efforts to stay afloat.

The biggest not-for-profit provider of treatment services, CGL, confirmed that it had been asked by Lifeline “four or five weeks ago” to step in to help and that it was working to take over “a significant proportion” of its projects.

David Biddle, CGL’s chief executive, said: “We are moving to ensure the security and stability of projects that deliver services to vulnerable people across the country. We are providing the resources to ensure that they are able to continue.”

CGL, which stands for Change, Grow, Live, has annual income of £158m and employs 2,800 people. Biddle said it would “initially” make no job cuts among the Lifeline staff it was taking on, including its head office team, and he praised the role being played by Lifeline’s long-serving chief executive, Ian Wardle.

Lifeline did not respond to requests for comment.

Pfizer to give out breast cancer drug free while awaiting NHS decision

A drug described as one of the most important advances in treating breast cancer in the past 20 years is to be given to women in the UK for free while the medicines regulator decides whether it should be available on the NHS.

The National Institute for Health and Care Excellence’s provisional decision in February that palbociclib should not be routinely funded on the NHS in England was decried by patients but its final appraisal has been paused for the drug manufacturer Pfizer to present further clinical data.

In the meantime, Pfizer has said it will make palbociclib available free of charge. The drug in combination with another can stall the growth of the cancer for about 10 months in comparison with existing treatments.

Fiona Hazell, director of policy and engagement at the charity Breast Cancer Now, said: “This is an unexpected lifeline for thousands of women living with this type of breast cancer. We are delighted that Pfizer have listened to our campaigners and have found a way to enable patients to access this first-in-class drug in the short term.

“While only an interim measure, more than 16 women every day could have their lives changed by this drug during this window. Palbociclib can offer a large proportion of patients with incurable metastatic breast cancer significant extra time before their disease progresses – time that can be truly invaluable to them and their loved ones.”

She said anyone who thought they might be eligible should speak to their doctor, but also urged Nice to reach agreement with Pfizer on making it available on the NHS to anyone who needed it.

At the time of the draft guidance that recommended palbociclib be rejected, Dr Nicholas Turner, team leader in molecular oncology at the Institute of Cancer Research and consultant medical oncologist at the Royal Marsden in London, described the drug as “one of the most important advances in treating the most common type of breast cancer in 20 years”.

The ICR and Breast Cancer Now both urged Pfizer to reduce the price to allow it to be offered on the NHS. Nice said at the time that a full course of treatment would cost £79,560 and the benefits were “still not enough to make palbociclib cost effective at its current price”.

It estimated that about 5,500 people in England – out of 45,000 new diagnoses of breast cancer each year – would potentially be eligible for treatment with the drug.

Results of a trial in 666 women with advanced breast cancer, presented at the American Society of Clinical Oncology meeting in Chicago last June, showed that taking palbociclib in combination with letrozole increased progression-free survival for a median 24.8 months compared with 14.5 months for letrozole alone.

The free programme is expected to be open for a maximum of five months – closing six weeks after Nice issues final guidance on the drug, or on 30 September, whichever is earlier – with access being dependent on individual NHS trusts signing up to the scheme.

Pfizer said women who took up the offer would receive the full duration of treatment, regardless of Nice’s final decision.

A spokeswoman for the company said: “Pfizer believes women with metastatic breast cancer deserve access as soon as possible to this innovative medicine that has been shown to significantly increase progression-free survival.

“Acknowledging calls from physicians and patient groups across the UK for timely access to palbociclib, Pfizer has made the decision, in this instance, to provide palbociclib free of charge whilst the appraisal process continues.”

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.