Drug trial for Hayley

By Camilla Lake

She flies out to Boston tomorrow and on Monday faces a battery of tests and assessment before beginning the groundbreaking treatment on Wednesday.

If doctors give her the go-ahead, Hayley will start on Farnesyltransferase Inhibitors (FTI); this medication has already been given to children with cancer, but this is the first clinical trial for victims of Progeria, a rare genetic ageing disease.

Hayley's parents Kerry and Mark are "excited but apprehensive".

Kerry said this week: "When Hayley was first diagnosed there was no hope. They didn't even know what caused Progeria. So, within seven years she has been diagnosed, they have identified the gene that causes it, and now a possible treatment, too. So that is reassuring.

"At the end of the day we have got nothing to lose - and everything to lose.

"But the prognosis is not good without it, so we have no choice really."

Progeria mimics the characteristics of normal ageing but up to eight times faster, which means a child faces problems associated with old age, such as arthritis and heart problems, as well as baldness and restricted growth.

Hayley, now nine years old, is a pupil at Sidley CP school and lives in Meadow Crescent - she has a younger brother Louis, who is five, and little sister, Ruby, almost two.

Hayley was around the same age when doctors at Guy's Hospital identified her condition.

Kerry said: "For the first year, she was pretty normal. She was a normal healthy baby, apart from very slow weight gain, but everything seemed to normal up until she was a year old, when they started investigating.

"She was referred to Guy's Hospital, but even then I didn't know what the outcome was going to be. It took a year to discover what it was.

"We were a small family anyway - I thought she was going to be a small baby. I thought that whatever was wrong with her could be treated, if it was something serious. Never in a million years did I think it would be this - I had never even heard of it.

Having been given the diagnosis, Kerry was "devastated".

"She was my first baby. For almost two years she was a healthy baby, and I remember the day they told me, it was like I had lost her already.

"They were very straight with it. They told us what the prognosis was - I think they told us she might live to 13 ... but then you think of the worst case scenarios and that she might never go to school."

Hayley has been the subject of Channel 5 documentaries, has appeared on ITV's This Morning, and has won many fans because of the way she lives with her condition.

She has a cheeky smile and feisty personality, an enduring love of the colour pink, and it is her deep wish that she might grow some hair. She often wears a pretty bandanna round her head but is currently hankering after a wig, and her mum is thinking of finding her one.

Kerry believes Hayley realised more of the meaning and impact of Progeria when she lost a friend last August.

"When her friend passed away, we thought it was a bit too close to home, and it was a big shock. It was at that time Hayley found out what Progeria was, and what it was all about.

"A couple of months before that I was talking to her palliative nurse, and I said I thought Hayley knew more that we thought she did, just by putting the pieces together.

"She was going to Progeria reunions, and children wouldn't be there who were before, and Hayley was putting two and two together. But it was when her friend passed away that we were quite straight with her, and it obviously came together for her.

"It was hard because you want to protect them as long as you can, but that was an instance when it was taken out of my hands. It was devastating. It was a big shock for all of us.

"I was a bit worried about her in the beginning - but kids are very resilient. I think she has dealt with it better than we have."

The family first heard about the FTI trial last June at the Progeria reunion, which brings together children and their families once a year - there are just 40 victims worldwide.

"Mark never had any doubts - I think I did in the beginning, but I just want to get out there now, and get on with it, and see the results."

Hayley will start the week with "base line testing" so that doctors know the exact state of her health before she starts new medication.

She is well used to the routine, having attended the children's hospital in Washington DC for tests over the last two years.

"She will be checked inside out, head to foot, every inch.. Blood pressure, weight, height, bone scans, two lots of blood tests which she is not overly happy about, and she might have to have MRI as well, which she did last year. I think she was the first Progeria child to have full results in an MRI, because they have to literally lie there for 45 minutes.

"They tried to do it the previous year, but she was too scared, so they took her out, but she was really brave last year - she knew how important it was, so she was really brave. I was very proud of her."

Hayley already takes tablets as a matter of routine - she has aspirin and synthetic statin to stop the arteries in her heart furring up. The new drug has possible side-effects, including diarrohea or constipation, so her health and tolerance must be monitored constantly, and she will return to Boston every four months to see if the dose can be increased.

"It is all positive," said Kerry, "We have been positive with her. And they have told her she could possibly grow hair if she has the follicles, which is her one wish.

"We will see how the treatment goes."

Kerry is glad that Hayley has strength to draw on and thinks she gets it from her dad.

"It is down to Mark's way of bringing her up. When she was first diagnosed I wanted to wrap her up and keep her safe, which is what any mother wants to do for her children.

"Then she started school and was away for hours, so I couldn't be behind her 100 per cent, and I had to let her go, and that is where Mark's toughness came in."

It's been a busy time on all fronts - Kerry and Mark were married last year, and Hayley and Ruby were both bridesmaids in pink dresses, with Louis as pageboy.

The couple spent their wedding night with 45,000 other people at Global Gathering, a dance festival in Warwickshire, having travelled up in a limo with friends. Last year also saw their first ever break from the children, when they went away for a long weekend.

"Hayley is still into the pink," Kerry added, "but one of her wishes at the moment is she wants to go to America to meet Hannah Montana, Billy Ray Cyrus's daughter, who is on the Disney Channel. Hayley wants to sing a song with her."