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Why I'm Asking Jeremy Hunt Not to Shut the NHS Door on My Son Matty

07/06/2016 08:52 | Updated 09 June 2016

My son, Matty is an exceptional 15-year-old boy. He is intelligent, confident, loving, caring, sociable and loves to participate in life to the maximum.

Unfortunately, he has a very rare and life-threatening condition called Dense Deposit Disease (DDD) which is killing his kidneys. Conventional therapies have no effect on the condition and there is no recommended treatment available on the NHS. There is a treatment in other countries called Eculizumab but the NHS have denied treatment to DDD sufferers, historically blaming insufficient evidence and excessive cost.

Recently the NHS has accepted the evidence that the treatment works but still refused to treat on grounds of cost. Expensive though this treatment is, it is already provided to almost 200 patients in England for other conditions and as the patent expires in 2020 that high cost is relatively short term.

I started a petition on Change.org six months ago to press for change in government policy as there is undeniable and mounting research evidence that Eculizumab is effective for DDD patients. We have been overwhelmed by the support we have received with over 353,000 signatures. There has been some real progress as the NHS have finally accepted the truth that this treatment is effective for DDD patients and have drafted a limited policy for treatment. However, the policy process is very slow and the policy itself very limited.

Matty is reaching a crossroads in his life because if we cannot get treatment for his condition very soon then his kidneys will go beyond saving and his life will change forever. Even if the current draft policy is approved in the coming months it will not help Matty, as he will be excluded for the bizarre reason that he has not yet had a kidney transplant.

In the film Sliding Doors, Gwyneth Paltrow plays out two very different journeys based on one simple change in her life - either catching or missing a train. Matty is now waiting on the platform and Jeremy Hunt is operating the door - Matty's future is in his hands.

So what do Matty's different journeys look like? The first could be that, Jeremy Hunt closes the door and stands by current policy. Matty goes home with monitoring and supportive therapy from the NHS and without additional treatment his condition deteriorates in a predictable and inevitable way; with dialysis likely to be required in 18 months. Dialysis is great compared to not having dialysis - it keeps you alive - but compared to normality it sucks.

If Matty passes his A-levels and goes to university, it will probably have to be close to home so his family can support him through what will be a Herculean effort. Suppose all goes well on dialysis (one in six people on dialysis die every year) then typically he might get a transplant three or four years later. Let's assume again that Matty is lucky and the operation goes well then remember that transplant is not a cure. Matty will be on immune-suppression drugs for life with a weak immune system and susceptible to more and longer lasting infections.

Life will be very much harder and much shorter. Matty will underachieve educationally and will not contribute to society in the way he is capable of. Instead of driving the country forward as an entrepreneur, of saving lives as a doctor, or finding new forms of energy as a scientist, he will be surviving and much more dependent on the state.

The second journey could be that Jeremy Hunt holds the door to let Matty in. Matty goes home and gets a call the next day from his Renal unit. He starts treatment with Eculizumab - a routine two-weekly treatment in his local hospital which has little or no effect on his education.

Within a few weeks his condition improves as the drug blocks the overactive part of his immune system. Some parts of Matty's kidneys have already died and these will not be regenerated; but many of the filters are just 'clogged up', and many of those will clear over time and return to normal function. Because Jeremy Hunt holds the door open for Matty, allowing his treatment at an appropriate time, Matty retains significant function within his own kidneys.

As his kidney function improves, Matty's life more closely resembles that of a normal 15-year-old with dreams and goals. Because Matty has been supported when he most needed it, he retains his own Kidneys and a normal life expectancy. He also completes his education and contributes to society as a taxpayer for the next 50 years generating tax income to the Treasury instead of being a drain on resources through benefits and endless dialysis and transplant procedures.

Other desperate patients will now get kidney treatments and transplants that might otherwise have gone to Matty. More healthy people leading fulfilling lives because Jeremy held the door open for Matty in 2016.

The train is ready to leave the station and Matty's not on it. There is draft NHS Policy in place to allow Eculizumab for post-transplant patients with severe forms of the disease but that will not save Matty's kidneys. Quite simply we need Matty to be given Eculizumab now.

A new or amended national policy is no doubt the correct formal answer, but time is absolutely essential to Matty so we are asking Jeremy Hunt in conjunction with NHS England to approve Matty's treatment without delay. This family, and I believe this nation, will hold Mr Hunt and NHS England for ever accountable if they close the door on Matty.

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