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British firm won't sell us drug that could save our dying 8-year-old
Tim Stewart13.07.09
The parents of a dying eight-year-old girl have criticised a British pharmaceutical firm for refusing to supply her with a drug to combat her rare condition - after they raised almost £1million to buy it.
Sandra Massart is one of only 2,000 sufferers worldwide of the deadly neurological disease metachromatic leukodystrophy. Her parents William, 48, and Olga, 39, say she will be in a vegetative state within months unless she is given an expensive experimental drug which is still being tested.
They claim that a leading British bio-pharmaceutical firm, Shire, is denying them the therapy, and have flown to London to stage a protest.
The drug Metazym is the only therapy which offers any hope for Sandra. Without it, doctors say she will go deaf and lapse into a vegetative state within months and die within a few years.
The Massarts claim Shire promised to sell it to them if they could raise the money. Treatment costs around £44,000 per month and has to be administered intravenously every fortnight for two years.
The couple raised one million euros (£860,000) in just two weeks amid an outpouring of public sympathy in Belgium, with the country's royal family backing their campaign. But they were told the company could not produce enough of the drug and needed their entire stock for clinical trials, which Sandra is unsuitable for, on nine children.
Mr Massart plans to march from the Royal Courts of Justice to the company's headquarters in Basingstoke this week. He will set off today carrying a picture of his daughter and plans to dump one million fake euro notes outside Shire's offices.
Mr Massart, an engineer, said: "At no point was any possible shortage or production problem mentioned. I was never told they only had enough of the drug for nine children in the whole world. They are morally responsible for the promises made by the company they bought the drug from.
"I will never give up fighting for her but my wife and I are having to watch our child dying in front of our eyes. It is the most horrible thing you can imagine."
Metachromatic leukodystrophy is a rare disease in which the lack of a key enzyme causes loss of the protective sheath from the brain. Symptoms include muscle wasting and rigidity, developmental delays, convulsions, paralysis, and dementia. Mr Massart said he was surprised and heartened by the public response in Belgium, but was now being accused of being a crook in his homeland, because he has not bought the drug.
A Shire spokeswoman said the "open-ended" clinical trials began for the nine children in February last year. She admitted the parents of one of the children are paying for their therapy and that further trials for young children will take place later this year.
She said: "We fully understand the seriousness of Mr Massart's situation.But it is a very long, complex process creating the replacement enzyme and our manufacturer is unable to make more than for the nine children currently on the trials. We are looking at all manufacturing possibilities, including bringing it in-house. But that would take two to three years. We are not refusing to sell Mr Massart the drug. We just do not have enough of it."
Reader views (7)
Its an enzyme, why dont they take 1/9th from each dose and give this child a fighting chance.
My prayers for Sandra and for reason to prevail.
- Giles, London, UK
Whilst I feel desperately sorry for these parents, imagine how the parents of the other 9 children will feel if THEIR children cease to receive the treatment they are trialling and are condemned to the same fate because the Massarts raised a million pounds and bought up all the stocks? I regret to say it is more logical to allow the drug to be tested on the 9 children currently being trialed because that may produce a medication to treat the other 2,000 suffers in the world.
- Roz, France
I hope for the family Massart the get the richt medication for their child Sandra from Shire.
- Bousse Danny, Ostend Belgium
Pegkelley, Boston, MA USA - how easy would it be to misinterpret "you couldn't buy it for £1million" when what was said (I am speculating here, of course), "you could buy it for £1million". English is not their first language, it should be noted. What pharmaceutical company ANYWHERE would sell direct to caregivers without proper medical authorization? The legal repercussions would be enormous. It may be, of course, that the family are trying to pressure the pharmaceutical company with this accusation, trying in their desperation to force Shire to bypass regulations, etc.
Desperation DOES affect what you understand to have been said. It comes under the general heading of wishful thinking from those with no way to turn. It is a part of the tragedy that people in this position must endure.
- Rogan, Irving
According to the story, the company told them if they had a million pounds, they could get the treatment. What's now obvious is that they figured the parents could never raise the money. Now the money is available, the company says the rules have changed.
This is not the parents picking and choosing the rules. It's the company changing the rules. Shame on them.
- Pegkelley, Boston, MA USA
I can sympathize with the Massarts and I understand their passion to seek help for their dying daughter. MLD is a very cruel disease. Unfortunately, I am sorry they have misconceptions about the drug. The trial that is referred to was a "safety" trial. The drug is still being investigated and has not been proven to do anything for an affected MLD person. That is the purpose of the next phase, to test for efficacy. We, the MLD Foundation, know hundreds of families, like the Massarts, who are desperate for a treatment or cure for MLD and are watching their children lose abilities ever day. We are hoping the next phase of the trial brings favorable results. At this stage there is no drug to buy. For the latest on the clinical trial for HGT-1111 go to
http://mldfoundation.org/research-shire.html
The MLD Foundation is a US based non-profit dedicated to supporting families dealing with MLD. Our mission of We C.A.R.E. stands for facilitating Compassion for affected families, increasing Awareness, influencing Research, and promoting Education for Metachromatic Leukodystrophy.
My heart goes out to the Massarts and to all the families dealing with MLD.
Teryn Suhr
Executive Director
MLD Foundation
http://mldfoundation.org
- Teryn Suhr, West Linn, OR USA
If the drug is A)experimental and B)not available, then this is a sad story driven simply by desperate parents. Of course they want something - anything - for their child. But experimental treatments are not a right, not ARE they right. The makers would leave themselves open to all kinds of legal and ethical problems if they initiated treatment by supplying the medication and something went wrong. It would have to be prescribed by a doctor, not the parent - and for doctors to prescribe it they would have to know that it was a viable treatment and safe (the whole purpose of controlled experiments) else they would also be open to all kinds of legal and ethical problems if anything "went wrong".
These people can't pick and choose rules, and patients they apply those rules to - and personally, I wouldn't want such people 'taking care of me'. Fickle attention to detail might work sometimes, but at others it can be disastrous.
- Rogan, Irving
