15-Year-Old Boy with Dense Deposit Disease Looks to NHS for Help


The family of a teenager with an extremely rare condition that may kill him within five years is begging the NHS to fund a drug which could save his life.

Matty Jones, 15, from Chester, suffers from a rare condition called Dense Deposit Disease (DDD,) which is slowly destroying his kidneys.

The disease, which affects just two in every million people, means large proteins gather in excessive quantities in the organs, causing the filters to become blocked and die.

The only drug which his family claim can stop his inevitable deterioration is currently not available – as NHS England say there is no evidence it helps DDD patients.

 

He applied to have the drug funded by his local hospital, but his parents, Nick and Karen, were left heartbroken when the application was rejected.

The drug is prescribed in Scotland and other European countries and insurance companies pay for it in the US.

Now, the family are writing to health secretary Jeremy Hunt to raise awareness to Matty’s case in the hope the NHS will fund the drug before it is too late.

But NHS England said there is no evidence the drug would benefit Matty or anyone else with his condition and it would be offering ‘false hope’ to DDD patients.

Mr Jones, 53, said: ‘There is virtually no chance that he will spontaneously recover from this and he’ll then be on a permanent cycle of dialysis, transplant and temporary remission.

‘It’s impossible to put a number on it but his life expectancy in these circumstances – without the drug – will be significantly affected.

‘He’s relatively lucky at the moment, with his main symptoms being managed through diet and medication – but this won’t always be the case and it definitely won’t cure it.

‘Already he doesn’t have enough energy and stamina to participate properly in sport and do the things a 15-year-old boy should be doing – though he normally tries.

‘He’s also been severely limited in what he can eat.’

The family do not know when Matty’s kidneys will fail – but after it happens he will go through dialysis and eventually need a transplant.

WHAT IS DENSE DEPOSIT DISEASE?

The rare but aggressive dense deposit disease (DDD) is a condition that prevents the kidneys from correctly filtering waste from the blood.

Symptoms of the disease include blood in the urine, dark foamy urine and cloudiness, swelling of any part of the body and high blood pressure.

The condition can affect people of all ages, though it is more aggressive in younger girls and more commonly appear with people aged from about five to 15.

About half of all those suffering DDD eventually experience kidney failure, meaning they usually require dialysis.

However, even a transplant is unlikely to save him.

Even aside from the obvious risks of major surgery and organ rejection – the DDD is likely to return and destroy his new kidneys too.

The disease is caused by a malfunctioning part of the immune system, causing the formation of extra proteins.

The drug Eculizumab blocks the production of a protein called C5b9 – which is accumulating in his kidneys.

The patent for the drug expires in 2020 so even though it is expensive now, it’s likely to be a lot cheaper in five or so years.

However, for Matty, that is too late, as his kidneys will almost certainly have failed by then, his parents claim.

They have already almost lost their son twice to the disease.

Mrs Jones, 45, said: ‘Matty contracted pneumonia on a school trip to Austria at Easter and we were moments away from losing him as he was transferred between three hospitals over one night.

Read more: http://www.dailymail.co.uk/health/article-3322073/Family-teenager-rare-condition-causing-kidneys-blocked-fail-make-desperate-plea-NHS-life-saving-drug.html#ixzz3ro5yZ31I
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Global Genes Comments

  1. Anyone have any advice or any doctor referral for Dense Deposit Disease? Need information for a 10 year old child ASAP.

  2. My name is Chris Okert and I suffer from DDD. I’ve been on the drug Kyprolis for over a year now and it has brought my mgus down from .8 to .1 which brought my gfr from 16 to 33. It took a little over six months before I saw positive results but as soon as my mgus got below .4 it shot up like ol faithful. I’ve also had very little in the side effects and actually feel better on the chemo drug than off of it. Also I’m on state assistance and social security disability for another issue but Dr Coffee at the Seattle Cancer Care Alliance, Dr Minn, where able to get the drug for free from the drug company since the stupid insurance won’t pay for it because I don’t have multiple myeloma. Still can’t believe that one. Oh I live in Washington State if your wanting to check out the docs. Its been a year since I started the drug and now it looks like I will be able to live a long semi normal life without dialysis or needing a transplant. Oh almost forgot if you can get the mgus down to 0.0 you can get a new kidney. Dr Coffee assured me of this and has apparently done it before. The UW also has some good docs that are knowledgeable on DDD. Its a tuff disease to have and the more of us that survive it the better chances other people have. God bless and keep fighting.

  3. Richard Delmolino says:

    Praying for your family. Also praying that a permenate treatment is found as my daughter is approaching 8 years being diagnosed with DDD.

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