A father's fight for a drug to treat his kids

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This man has spent 17 years battling to save his children from an incredibly rare genetic disease and find a reliable and approved treatment.

And eventually he did in the form of an unusual product: weed killer.

Nick Sireau's sons have what's called "black bone disease" - formally known as Alkaptonuria, or AKU for short.

"So AKU is an ultra-rare genetic disease. It affects roughly one person in half a million and it's due to a mutation in a gene which means that patients can't break down a certain acid. That accumulates in cartilage and bone and goes black which is why it's called black bone disease and over the years that leads to severe joint deterioration, spinal problems, heart problems and intense pain."

Sireau's eldest son Julien was diagnosed as a baby, but symptoms can be delayed until the patient is an adult. He's now 20.

As a patient at the National Alkaptonuria Centre in Liverpool, he has had early access to a weedkiller-derived drug called Nitisinone.

Scientists knew Nitisinone should help prevent the damage - but needed to prove it using large clinical trials.

But with only 1,000 known sufferers worldwide, there was no help from big drug companies.

So 10 years ago Sireau gave up his job to concentrate on the campaign. He's raised millions of pounds.

The research did prove Nitisinone works, not only stopping symptoms developing but even reversing some of the damage done.

It has now been licensed by the European Medicines Agency.

And for the last three years, while taking Nitisinone, Julien has been symptom-free.

"...if I wasn't on the drug it could be that within the next ten years or likely within the next ten years I would definitely feel the effects in my joints, possibly in my heart valves, darkening, like dark spots in the eyes, ears, just everywhere there's cartilage or anywhere that something called ochronosis can form."

The AKU Society now plan to raise funding for a gene therapy treatment - which could eventually mean just one injection would cure the condition for good.