Old Before My Time Part 7

After the Was.h.i.+ngton tests, we returned home and waited and waited. Winter turned to spring, and there was still no news. Summer came and we flew to America for another reunion, but there was still no start date. With every week that pa.s.sed we became more frustrated. We were told that the treatment had been tested on mice and the early results were promising. The mice had put on weight and lived longer. I understood that the US Government's Drug Agency had to give its approval before they could be allowed to test the drug on children, but a clock in my head was ticking. Time was not on our side. Every month we waited was almost a year off Hayley's short life. The wheels of government might move slowly, but progeria doesn't.

In the meantime Mark registered a charity in Hayley's name to raise money to support children and their families living with progeria. With Mark as chairman, me as vice-chairman and a family friend as trustee, Hayley's Hope gathered the numerous offers of help with fundraising and donations from well-wishers into a central charity, which helped to pay for extra expenses for our trips to Boston, which weren't being covered by the Progeria Research Foundation. Through the charity we were also able to donate money to the foundation for their on-going research.

On April 28 2007, we finally had the official call we had been waiting for. Hayley had been accepted on to the drug trial. I remember the day well as our Belgian friends, the Vandeweerts, who had been frequent visitors to our home since our first meeting at the Progeria Reunion, were on the final day of a holiday with us in England. Hayley and Michiel were playing in the garden, I was in the kitchen cooking pasta for our dinner when the phone rang. It was Leslie.

'Great news. We've had the go-ahead from the Drug Agency. The trials are starting next week,' she said. Sixteen months of antic.i.p.ation were over, 28 children from 16 countries would soon be flown out to Boston Children's Hospital, two at a time, to be given what we hoped would be the wonder drug. They would start with some of the American children. Hayley was being paired up with her friend Michiel for her treatment, which would start on May 26. It meant that for the next two years we were committing ourselves to fly out to Boston every four months, for a total of seven visits.

Leslie explained that arrangements were being made for Hayley and Michiel to spend five days at the children's hospital. For the first four days she would have every test and scan available to ensure her ageing body was capable of taking the new drug. Then on the last day she would be given the pill, making history as the first child from the UK to test it. In the meantime we had already spoken to our own doctors in the UK, who consented to monitor Hayley on a weekly basis checking for growth and improvement and potential side-effects. Their health reports would then be fed back to the research team in Boston.

'Are we doing the right thing, taking part in this trial?' I asked Mark. 'This drug has never been tested on humans before. We don't know if it will work or if it will cause more damage.' We had been warned about the possible side-effects which ranged from mild nausea on one end of the scale to possible death at the other end.

'Without it the prognosis is not good, so we have no choice, really,' Mark said.

He was right. Without the drug we were four years away from the dreaded life expectancy age of thirteen. For every year after that we would be living on borrowed time, worrying that at any moment Hayley could suddenly drop and pa.s.s away in our arms, just like her friend Maddie.

'There are side-effects for everything. Even paracetamol can cause breathing difficulties in some extreme cases if you read the small print. The drug companies are just covering themselves. We have to look on the positive side. We can't let Hayley sense we are worried,' Mark said. I knew he was right, but it wasn't easy to follow his advice. My mum had also been putting a dampener on it ever since we first mentioned the possible trials a year earlier.

'What if it goes wrong?' she asked.

'I know it's a risk because the drugs have never been used on progeria children before,' I replied. 'But we know what the best outcome could be a longer and healthier life for Hayley. We just don't know what the side-effects will be, if any. She's getting older, Mum. It's a risk I'm willing to take. We have no choice.' I rea.s.sured her that we were in safe hands as the drug company was one of the biggest in the world. 'They are not going to play G.o.d with our children,' I continued. But I understood Mum's concerns, I had already spent many nights lying awake worrying about the consequences if it went wrong.

In the days leading up to our departure for Boston, I began to sense that Hayley might be getting nervous. One night as I tucked her into bed and kissed her goodnight, she asked, 'Can we delay the trial, Mummy?' Waiting was no longer an option, but how could I tell a nine-year-old child that her life is so fragile we may not have the opportunity next year?

Instead I replied, 'We've got our plane tickets and hotel all booked, we can't change the dates now. And Michiel will be disappointed if you don't go, he's looking forward to seeing you again.' I was aware that Hayley was afraid of sharing worries with Mark and me. She worried that she might upset us. I was apprehensive too, but I had to put on an optimistic face for everyone's sake.

The night before our first trip to Boston we threw a 'good luck' party with all of our family and Hayley's friends at a local pub, the White Rock Hotel. 'Can I wear make-up, Mum?' Hayley asked as she picked out her outfit for the night a white flowery dress and matching headscarf. I let her choose the colour of her eyeshadow. 'Pink to match the flowers on my scarf,' she said. She was growing up into such a girlie girl, she had her own sense of style and fas.h.i.+on. At eleven some people might have said she was too young to be dressing up in make-up when she was pretty enough without it. But my att.i.tude was always to let her be. She might never reach an age when she can go out for a night on the town independently, so I wanted her to enjoy her life.

A long, black stretch limo pulled up outside our house to chauffeur Hayley round the corner to her party where all of her aunties, uncles, cousins, friends from school and their parents were waiting. She danced the Locomotion and the Macarena, she ate sandwiches and crisps, and she spent the night telling everyone how excited she was to be going to America. When the party ended, Mark took over the DJ's microphone and thanked everyone for coming.

'Keep your fingers crossed when we're out in America and we will do our best to come back with some good news.'

All our friends raised their crossed fingers in the air in unison and cheered. 'Good luck, Hayley!' We didn't know what lay ahead. We had nothing to lose, but, in a way, we had everything to lose.

Chapter 21.

Hayley

I'm Gonna Grow Hair

ONE DAY MUM SAID to me that they had found some tablets that might cure progeria and she asked if I wanted some. I was really excited and said to myself, 'You're gonna grow hair. You're gonna grow tall.'

'Will I have to have needles?' I asked. I hate needles. Even if the doctors put stuff on my skin to make it go numb first, I still don't like them. I always have to have needles for blood tests and stuff. But Mum said it would just be tablets, so I said that would be all right. Here we go again, that means there will probably be needles, I thought.

Mum explained that we would have to go to a hospital in America if I wanted to have the tablets. I thought that was a long way to go just to get tablets. We usually get them from the chemist in town. I had been to America for the Progeria Reunions and when we went on holiday to Disney World and stuff but going to America to go to the hospital was different. Mum said that the tablets were very special and hadn't been given to many children with progeria before and I would be one of the first. That made me feel really special.

Before I could have the drug I had to have an MRI scan to take pictures of the inside of my whole body. It was really scary, like going into a big tunnel. I couldn't lift my arm or move or anything and I had to lie still for 45 minutes. When I went into the tunnel only my foot was sticking out of the end. Mum held on to it and I said, 'If I shake my foot, I want to come out.' Inside the scanner there were little things on my chest to hold me down. At first it was like being in a dark tunnel and the loud bangs sounded like someone trying to get in. It was scary, but exciting. I thought to myself, at least I'm not having needles and if I don't do it I will be ill and I could possibly die. So I just thought of all the positive stuff and tried to be still. I was so still I almost fell asleep. The doctors said it was the first time a child had stayed still for 45 minutes. Other children were moving too much. I told them I had fallen asleep and they laughed. If I could I would make it so that there were no noisy machines and no needles. But I can't, and that's OK.

At first I didn't really think too much about what the trial meant because I was still quite little. I just thought, let's go for it. Mum and Dad were worrying about it and making all the decisions. But I was too little to understand. Some days I could tell they were really worried. They didn't have to say anything, but I could tell. When I saw they were scared I wondered why they were so worried. What's the worst thing it could do? I could get really poorly. The best thing that could happen would be if I was just like a normal kid. One day I overheard Mum on the phone to Nanna and she was crying. I thought it must be something really bad. Later that night when Mum was tucking me up in bed I said, 'Can we go to America another time?' But she said we had to go because everything was booked and the doctors would be waiting to see us. Then she said I would get to see Michiel and his mum and dad and sister, Amber, and that cheered me up. I was looking forward to seeing them. Michiel is just crazy.

Whenever we used to see him at the reunions he was always hyper and running around, and he speaks good English.

Before we went to Boston for the first time Mum and Dad threw a party for all my friends to say goodbye. It was good fun and I ran around and played. Everyone was giving me presents and saying good luck, which was nice of them. But when we left everyone was crying. I thought that maybe they were crying because they weren't sure what was going to happen and whether the drugs would work. Then I thought to myself it must be all right, Mum and Dad would not let me go on the trial if it was going to be dangerous. But when everyone was crying I started to get a bit nervous, and I thought, what if something went wrong?

The night before we left I had a dream. In my dream I took the drug and woke up and had really long hair and was really tall. I looked like my sister Ruby. It was a cool time. When I woke up I remember thinking, that's what's going to happen, your hair is going to grow. But I knew it wouldn't happen overnight.

Chapter 22.

Kerry

Boston Drug Trials

MAY 19 2007: ALMOST 400 years after the first English settlers founded Boston, Ma.s.sachusetts, Mark, Hayley, Louis and I arrived in the city to create our own little piece of medical history. It was amazing to think that in seven years scientific progress had advanced so quickly on progeria and Hayley would soon be the first child from England to try out the new miracle drug farnesyltransferase inhibitor, or FTI for short. We were well aware that Hayley was being used as a human guinea pig but to be given the chance to prolong her life was beyond anything we had ever wished for.

After a seven-hour flight we were all exhausted and couldn't wait to check into our hotel. I carried Hayley through Boston's Logan Airport while Louis, who was only four at the time, slept in his daddy's arms. We had arrived two days early to give us all the weekend to recover from the jetlag and calm our nerves before the trial started. Travelling with the Vandeweert family meant we had been able to take our minds off the uncertainty that lay ahead. For two days we were European tourists on vacation. We rode the green and orange Old Town trolley bus around the city, taking in all the sights, from the aquarium to the famous Boston Red Sox baseball stadium.

On the Monday morning we arrived at Boston Children's Hospital, which would be our home for the next five days. We checked into our family room at a nearby hotel and took the shuttle bus to the hospital to meet Dr Mark Kieran, director of paediatric medical neurooncology at the hospital. He had extensive experience with the FTI drug in children and was leading the team of 28 medics and scientists working on the progeria drug trial. It was crazy to think that in just four years, through their exhaustive efforts, the Progeria Research Foundation's research team had not only discovered the gene that caused progeria but had found a drug that might work to slow down the ageing process. They had also raised enough money to fund the costs of the trials including the travel, food and lodging expenses for the 28 families taking part.

Before we could start any tests we had to sit down with the doctors and scientists leading the trial to make sure that we understood everything that the trial involved. There were risks and benefits. By being given the drugs, which could save Hayley's life, we were also agreeing to follow their strictly confidential guidelines and sign a legal doc.u.ment showing that we were agreeing to take part in the trial with our eyes open. At that point, when the doctors handed us a thirty-page consent form to sign, we realised the seriousness of the process we were entering. Once again we were given the list of the possible side-effects, which ranged from mild nausea to heart attack and possibly death. The trials were not something we had entered blindly, but seeing everything spelled out in black and white and our signatures on the bottom meant that there was no going back. It was scary but also strangely uplifting to think that this could be the cure we had prayed for. We were putting all our hopes in the hands of these doctors; who admitted that they didn't know with any certainty what would happen.

The doctors tried their best to explain to us how they expected the new drugs would work. Progeria was caused when a 'bad' molecule called a 'farnesyl group' attached itself to the progerin protein and stopped the cell from working properly. The FTIs would act as a barrier, stopping the progerin from damaging the cells and slowing down the ageing characteristics of progeria. It wasn't only children like Hayley who could benefit from this group of drugs. We were told that drug companies had spent the last 10 years and millions of pounds developing FTIs that they hoped one day would cure cancer. Although cancers were caused by very different proteins they were using the same principle that the drug could block the path of the cancer-causing protein.

Before Hayley could take the drug she had to have a full MOT to make sure her 'seventy-five-year-old' body was strong enough to cope. We presented the doctors with our daily weight and food diary, which we had been asked to keep during the month before the trial date. They noted her weight and height against the chart. She weighed 12kgs and was 97cm tall. The average nine-year-old girl should weigh double that and be at least 30cm taller. As she lay on a bed looking helpless, naked except for the hospital regulation paper pants, the nurses tested her heart with an ECG then fitted a clip on her finger tip to check the oxygen levels in her blood.

Once the tests came back positive, it was time for Hayley to take this 'wonder drug'. It was the final day of our visit and we were taken into a room. Doctors and nurses were there, waiting to give Hayley her first shot at a longer life. It was an historic occasion; she was the first child in Europe, and only the third child in the world, to test the drug. We sat her down beside a table, where the white capsule was laid out ready. Beside it was a paper cup filled with water. Mark had his video camera running, recording every magic moment, and one of the hospital nurses had her camera ready too. Hayley smiled, holding the pill bottle in her hand. Then, with no hesitation, she picked up the pill and popped it in her mouth. Against the frailness of her tiny, bony fingers the pill looked enormous. With both hands she picked up the paper cup and took a big gulp of water. We stood nervously watching and holding our breath. Hayley's little cheeks puffed out with the water and her eyes grew wide. 'Big mouthfuls of drink,' I shouted in encouragement, thinking she was about to choke.

Just as I felt the panic rising inside me, she opened her mouth wide and grinned for the flas.h.i.+ng cameras. History had been made. We now had to wait to see what effect it would have.

We flew home two days later with a suitcase full of pill bottles, enough to cover her daily dose of one pill in the morning and one pill at night for the next four months. But as soon as we arrived home the side-effects started. Hayley got up the next morning and said she didn't want any breakfast as she was feeling sick. I looked at Mark, terror was written all over his face. Was this the start of something really serious? Panic set in and I got on the phone to Boston Children's Hospital to find out what to do. They prescribed Zofran, a drug which is used for patients undergoing chemotherapy. Mark rushed down to our pharmacist to collect the drugs. The sooner we could give her them, the quicker they would make her feel better. For the rest of the day she lay in bed, too weak to be bothered with anything or anyone. Mark and I asked ourselves, had we made the biggest mistake of our lives?

I wasn't aware of it at the time, but we must have gone into nostalgia mode. Mark and I found ourselves watching old home videos of Hayley just after her diagnosis. In the film she was splas.h.i.+ng around in her little paddling pool in the garden, her hair was swept back over her head in a big brown quiff. She seemed so happy and carefree, unlike the frail and sickly child we had brought back from Boston.

'Are we doing the right thing for her?' Mark asked. I looked at her happy smiling face on the TV screen and thought of the frightened nine-year-old girl who lay in the room above me, too ill to move, and I couldn't be certain.

'We have to remember our daughter is a little fighter. That's what makes her unique. We have to hope that she will get through this and be stronger and live longer,' Mark said.

The next day Hayley seemed a bit perkier, it seemed that the anti-sickness drugs had taken effect and by day three she was back to her old self and we could breathe a sigh of relief.

We began monthly appointments with Dr Whincup who was monitoring Hayley's progress and reporting back to the doctors in Boston in between visits. For the first month her weight actually dipped due to the early bouts of sickness and diarrhoea. But once that was under control, and her appet.i.te was back to normal, she started to pick up and we noticed her weight crept up month by month. Several months into the trial, Dr Whincup noticed that her blood pressure was creeping up to a rate that was above normal for a young child. Hayley was put on a 24-hour blood pressure monitor which meant that she had to wear a tight cuff on her arm day and night to measure her blood pressure when she was awake and sleeping. After that, Dr Whincup prescribed the beta-blocker Atenanol to bring her blood pressure down and reduce the risk of heart disease or a stroke. This new drug brought additional worries. The side-effects were fainting and dizziness and I was concerned that she might suffer from dizzy spells or sickness at school. In order to eliminate any problems we gave her the tablets when she came home from school and we could watch out for any problems.

For the rest of the year, and throughout 2008, we continued our trips to Boston for the drug trial. Every 16 weeks Hayley would get another dose of FTIs and she would be measured and have her body tested. On our first return visit there was not much to see. The doctors didn't give too much away, but they seemed pleased with the way it was progressing. The side-effects that had scared us so much in the early days had receded and she was improving. In between each visit we would notice something new in Hayley's development. She had grown a couple of centimetres. Her eyebrows had started to grow and she had a layer of light downy hair on her arms. It gave us the glimmer of hope that we needed to help us to carry on.

Chapter 23.

Hayley

Hope Is when Mum Stops Crying

WHEN WE GOT TO BOSTON we met with Michiel and his family and it was like being on holiday, so I wasn't too worried. We had left Ruby at home with a family friend so it was just me, Mum, Dad, and Louis who went. In Boston we all went on the trolley bus with Michiel and his mum and dad and sister, Amber. We stopped at the aquarium and saw giant sharks and sting rays, which were really cool.

But after a couple of days we had to go to the hospital and they started doing lots of tests again.

When we got there the nurses weighed and measured me and did an ECG on my heart. Then they wanted to do a skin biopsy which is like sc.r.a.ping off a bit of the skin from my arm. They also wanted to take one from Mum and Dad. First they put a numbing cream on my arm and I had to wait for one hour. When the cream made my arm numb the nurse got out a needle. But when the nurse got closer with the needle I said I didn't want to do it. The needle was like a corkscrew and they wanted to put it in under my arm and take out the skin. When someone said I would only need one st.i.tch, I thought, no way. The nurses gave me a lolly but I still wouldn't do it. I felt really bad after because I made Mum and Dad wait an hour for nothing. Mum tried to bribe me with sweets and said she would even buy me a new phone. It almost worked but I started to cry and she said 'Fine you don't have to do it.' But I think she was peed off. Afterwards I wished I had done it On the day I got to take the tablet everyone was making a big deal about it. I had to go in a room with Mum, Dad, and the doctors. I felt a little bit scared but I kept telling myself, 'You're gonna get hair. You're gonna grow tall.' First I had to swallow a Tic Tac to show them I could swallow it. At first I was afraid it would get stuck in my throat and make me choke. Then they gave me the real tablet and I put it on my tongue took a big mouthful of water. Mum looked scared. I think she thought I was going to choke or something. But I gulped it down easily. Then I poked my tongue out to show it was gone. Everyone in the room went 'yay!' and they were all taking pictures. It was scary but really exciting at the same time.

When we got home I felt really sick. When I told Mum and Dad they were worried and that made me scared. It was really horrible. I felt sick, and then I felt tired so I slept. Because I was sleeping I didn't eat much and that made me feel sick again. It kept going round for ages and I thought, why am I doing this if it makes me feel worse? Dad went out and got some more tablets. After I started taking them I felt better and Mum and Dad stopped worrying. Mum tried to make me feel better by saying that if I jumped up and down they could hear me rattle because I was so full of pills.

I started having the tablets twice a day one in the morning before school and one at tea-time. To see if the tablets were doing any good and making me grow Mum made a height chart on the kitchen door and some days I would stand and measure myself against it. I remember once being 3cm taller and I was really pleased. I thought it must be working. Mum and Dad started taking me to see Dr Whincup more often and he was pleased with the way I was growing too. I started to feel like I was getting stronger. I could run about in school and do star jumps with my friends. But before the drugs I was too weak to jump very much at all.

Sometimes, when Mum and Dad weren't looking, I would look in the mirror to see if I had more hair, and I did. I started to grow eyebrows like the other kids in school, and some hair. I said to myself, 'You're turning into a monkey.' Mum used to tease me that I was growing a moustache like Dad, but she was only joking. I hope I don't get a moustache, but lately I have noticed some more hairs on my arms and my chin. I think I might be getting a beard. I hope I don't grow a moustache and beard because I would have to start shaving and that would be embarra.s.sing.

When I started the trials my biggest dream was to have long hair that I could tie back in a pink hair band. Every time we go back to Boston the doctors check if my hair has grown. They draw a little square on the back of my head and count the hairs in it. The first time there were no hairs and now there are seven hairs. I say 'Wow! Seven hairs!' I know that's not a lot, but it is to me.

The drug hasn't worked out quite like I had wished. When I realised I wasn't going to get long hair, I was quite sad. But now it's OK. Just the other day I looked in the mirror and I found a few more little black eyebrows. They are really tiny, you can barely see them, but it's good I have some. I don't want to get really thick eyebrows. I would like to grow more hair and get taller. If I get eyelashes I can put mascara on them. Since I've been taking the drugs I do feel taller.

I am definitely getting taller. At first I noticed it when I stood up in the bath. It used to be that I couldn't see myself in the mirror unless I stood on a bowl. I know I am definitely taller because when I open the fridge I bang my head. It used to just skim my head but now I have to duck when I open it. I know now that the drugs won't ever make me completely better, and I won't look like a normal person, but it still makes me feel really happy that they are working.

One day when I was eleven and I was bored with nothing else to do, I sat down in my bedroom and thought about the drugs. Usually I try not to think about it too often, it's not something that's always on my mind, but this day I remember thinking how cool it was and I was glad I had decided to do it. I thought progeria was so weird but it's kind of cool because I have to go to Boston and I don't have to do much work. I don't know any people who have been away from England, yet I get to go on cool holidays all the time.

It's like the drugs are making my mum stronger too. She used to be so sad and sometimes she used to cry when she was talking about me to her friends and Nanna. But now I have been taking the tablets she has stopped crying, and that's good. I think she knows there's hope.

Chapter 24.

Kerry

High School Days

WE HAD ALWAYS LIVED one year at a time and never daring to plan ahead when it came to Hayley's education. When she was younger I had thought that there was no point in wasting six hours a day in school, learning things that she might never get to use, when she could be travelling the world and trying new experiences. But the older she became I realised going to high school was a normal progression. In July 2009 it was time for Hayley to leave the comfortable surroundings of her primary school, where all the teachers and pupils knew and cared for her, and prepare to make the step to secondary education.