Blog: Carls Blog

Darius Goes West

Mon, 18 Aug 2008 17:20:11 GMT

Gala Charity Film Festival

Première of Darius Goes West!

Location: Bridgewater Hall, Manchester
Event: Family Film Festival
Date: 16th August 2008

Website: http://www.charleysfund.com/ http://www.dariusgoeswest.com/film.html

Introduction

We started off the day by watching on a big projector screen, a few short films made by young disabled people from the Family Friendly Film School which is in Greater Manchester! The films put together was good but was made purely for the younger generation.

The event was put on by the Family Friendly Film Festival and because of there kind generosity they allowed us to raise money for Action Duchenne & Charley’s Fund by putting on a raffle and letting us sell T-Shirts.

After the short films were shown, there was a 20 minute break! Then I and Esther Derber – Action Duchenne Fundraiser manager, made our way onto the stage and we both talked to the audience about the Charity Action Duchenne and what our goal is, which is to raise the awareness (profile) of Duchenne Muscular Dystrophy the severe muscle wasting condition and raise enough money to cure it, or if not a cure at least a treatment to improve quality of life for sufferers all over the world. Also I gave an introduction to the Film Darius Goes West and talked about what it’s like living with this horrific disease. I told the audience that Darius has been in touch with me via the internet just saying that he’s sorry that he couldn’t make an personal appearance in England but thanks everyone for there support and is very happy that Darius Goes West has gone global.

Film – Darius Goes West – Roll of his life!
Dir Logan Smalley, 2007, USA, 92mins

Most suitable for ages 8+

About a boy called Darius Weems from Athens, Georgia, who was born with Duchenne Muscular Dystrophy (DMD). In 1989, Darius watched his beloved older brother, Mario; pass away from the same disease at age 19. Soon after, Darius lost use of the muscles in his legs and began using a wheelchair. A group of Darius’s friends felt there was no need for his quality of life to disintegrate along with his muscles. So, they decided to take Darius, who had never seen a range of mountains, never dipped his toes into an ocean, and never crossed a state line on the adventure of a lifetime. After raising $60,000, this “band of brothers” rented a wheelchair-accessible RV and hit the road in July of 2005. Their three-week cross-country journey had one major goal: to reach Los Angeles and convince MTV’s popular show, “Pimp My Ride,’ to customize Darius’s wheelchair. Along the way, they evaluated wheelchair accessibility at many of America’s major tourist attractions and raised awareness of DMD by holding over a dozen press conferences. They also found joy, brotherhood, and the knowledge that life, even when imperfect, is always worth the ride.

Film review

A very informative documentary which has focused mainly on the youth, a generation of young adults to make awareness of the effects that Duchenne Muscular Dystrophy has on the individuals living with the illness and also the effects it has on the families and friends of that individual. In this film you will experience good feel moments with a mixture of emotions, from shedding a tear to crying with laughter.

An all around awesome documentary and I would recommend it to everybody!

“Certain to stir hearts” – Variety

“A comical and poignant tale… Ulysses and Luke Skywalker have nothing on 15 -year-old Darius Weems“– Los Angeles Times

I urge everyone to buy a copy of the film from the Darius Goes West website, not only is it a film you will enjoy and learn from his experience but in purchasing the DVD you will be making a huge difference to Duchenne suffers like me & Darius all around the world because the money from the DVD goes directly into a cure or treatment!

http://www.dariusgoeswest.com/film.html

Ann seems very Keen

Thu, 17 Jul 2008 19:24:06 GMT

Ann seems very Keen

As you may already know by Nick’s blog I was invited by Action Duchenne to take part in a delegation meeting on Monday 14th July following the debate in the Houses of Parliament that took part on 2nd July between Duchennne champion MP's, Nigel Evans and Dave Anderson with Health minister Dawn Primarolo.

Even though it was hard work getting up early in the morning I thought it would be worth making the effort so me, Esther, my mother & father made our way up to London by the Stockport train station and met with Bernard Mooney, Doron Rosenfeld and Nick Catlin in Westminster, to discuss what was going to be said in the meeting beforehand.

Anyway we then went to Richmond House where we was greeted by MP’s Dave Anderson and Nigel Evans, we all went into the meeting together – the parents, MP’s and Action Duchenne members where we was greeted by the Under Secretary for Health Ann Keen.

When I first met Ann Keen, I was astonished that she was indeed human, very down to earth and so understanding – I thought we would have had to get our hands dirty to at least achieve a hearing but no Ann was very supportive in our quest to save young peoples lives! I made speech about how this muscle wasting illness Duchenne Muscular Dystrophy affects my life and others living with this cruel cold-hearted disease. Also Esther made a lovely photo album showing some of the boys who have the condition and Parent Doron Rosenfeld from the Gavriel Meir trust presented their excellent video, including footage of Mark Styler talking about Duchenne. Bernard Mooney spoke about his son Ben who has Duchenne and showed a recent film of his son. Which all of this made everyone around the table shed a tear or two.

Ann Keen wanted to give us an answer on the same day but she decided to call a meeting that will be arranged in the near future with scientists, researchers and clinicians about what funds we need in place and how far we are in the field of treatment and what route to take to accelerate treatment from the lab to the clinic. I was dismayed by a comment made from the head of the Department of Health research funding team, Peter Sneddon as he recognized that we were exactly in agreement with their policy of funding translational research. Unfortunately he was stuck saying the old broken record of not agreeing to ring fenced funding but did concede that the DoH would look very favorably if our scientists put in bids to the MRC or DoH. Ann Keen asked for a review of the research and clearer guidance on what the DoH and MRC would fund.
Ann Keen was visibly moved by what happened at the meeting, she told me that she had already spoken with Lord Darcy about me and that they wanted to invite me to take part in Lord Darcy's NHS review.
As a whole I think the meeting was a success, not a complete success but another huge step forward to the future that is so bright!

Thank you everyone for the support…

Living on Death-Row

Sun, 27 Apr 2008 20:29:01 GMT

Living on Death-Row

The best way to describe Duchenne Muscular Dystrophy is just exactly that it’s like being innocent but living a death-row sentence – never knowing when time is up! It isn’t a very good feeling living in a disease where it’s not well known. But the time for being silent and keeping it swept under the carpet is over, I know its hard for parents and family to talk about Duchenne but remember its a disease with hope now, to kick this illness in the backside we really need to be shouting on the rooftops and getting big awareness out there!

Together in Action Duchenne we can defeat this condition, treatments are in the pipeline and I think a cure is on the horizon.

MCFC Hall of Fame Dinner

Mon, 10 Mar 2008 11:43:00 GMT

Fred Tilson who played for Manchester City and England in the 1930's was honoured on the 4th March at the MCFC Hall of Fame gala dinner held at The City of Manchester Stadium.

Fred was a centre forward or inside left who also scored FA Cup winning goals in the 1934 Cup!

Carl Tilson below seen holding the award (grandson of Fred Tilson) who has Duchenne Muscular Dystrophy who was very proud said:

"I'm very proud of my grandad and because I have a terminal illness it was my ambition to be alive to celebrate his accomplishments"

The night was a huge success, celebraties including some of the great footballing legends from the past such as Tony Book, Francis Lee, Colin Bell and future raising stars such as Micah Richards, Nedium Onuoha and Danny Sturidge!

The event was hosted by lifelong blues and GM:TV presenter John Stapleton and Susan Bookbinder!

The audience of nearly 400 guests also voted overwhelmingly for Shaun Wright-Phillips' goal in the 2004 derby as the greatest goal scored by an Academy graduate for City.

Renee (Wife of Fred Tilson), Esther & Carl!

Fred (Son of Fred Tilson), Carl & Colin Bell!

Fred (Son of Fred Tilson), Carl & Glyn Pardoe!

Year 2008! A new Start...

Mon, 31 Dec 2007 23:54:11 GMT

Years Resolution.. Determination

A year as come and gone again... And what a year its been! A lot has happened over 2007.

One of the big changes has been the name of the charity - Parent Project United Kingdom! We are now known as Action Duchenne, we didn’t think the name was appropriate anymore because the Duchenne boys thought it was just involving parents so everybody decided a name change would be suitable! Also we decided to have a proper launch and the most appropriate time we felt was at the 5th International conference in London at the Holiday INN between 2nd & 4th November - the conference went very well and was very informative! Professors, researchers and scientists from all over the world working with Duchenne Muscular Dystrophy came and did presentations for the families, explaining the in's and out's of the disease.

Throughout the year Action Duchenne organised fundraising events like love runs, the Manchester Bupa Run, great north run, sky diving and rock climbing! All the events raised very good mounts of money for research, events were also put on where they put on a dinner and had live auctions. Also its not only the adults and Duchenne boys that raised money, even children from schools wanted to help by doing a sponsored walk round the school. the nursery and little ones did 5 laps, the older infants did 10 and the juniors did 20.
Who says the kids of today don't care!

We have had donations from different partners, one organisation called The Professional Footballers Association - donated £500!

Thank you everyone involved with your contribution, every little helps!

Research.. On the front line

Research has come along a lot this year - a treatment called Exon Skipping which is where you get a drug (Morpholino Antisense Oligonucleotides) injected into your muscles which patches over the messed up DNA produces a functional but short protein of Dystrophin - meaning the disease is less severe, alleviating symptoms! Turning Duchenne into a less severe Muscular Dystrophy called Becker with a practical normal life expectancy.

This year on Tuesday 18th December made history when Exon Skipping begun its first human clinical trials - Professor Muntoni and his team injected the first foot muscle of a young person living with Duchenne. This first phase clinical trial aims to test whether Morpholino Antisense Oligonucleotides could become a gene therapy for Duchenne in the near future!

To make this possible, Action Duchenne and our families have lobbied hard and petitioned with the government to win over £2 million pounds to fund this research. Action Duchenne has also funded pre-clinical work to develop this unique approach to fixing the faulty gene. We have also got an online petition going, because funding runs out in 2008 - meaning trials will come to a halt so the petition is created to demand funding from the government, from the lab to the clinic if Exon Skipping shows successful!

Petition website

http://petitions.pm.gov.uk/breakthechain/

We have petitioned about the government funding DMD previously...

The government replied saying “The Government fully accepts that everything possible needs to be done to bring an end to the tragic impact Duchenne Muscular Dystrophy (DMD) has on individuals and their families. There have been significant developments over the last few years, not least in research, and some grounds for hope.” The determination of all those involved - charities, the pharmaceutical industry, and Government - to find a cure for DMD will give us the best possible chance of that hope becoming a reality.

This being said shows that the government is finally listening so it’s our job now to keep the pressure on by lobbying the government and Department of Health to get them to help us fight the good fight! “Time is critical! Money shouldn’t be an option, life is priceless! We deserve to live! We have no other options; I would rather die trying than die without trying!

DMD Registry and new clinical trials

Its time to get Registered. There are two major clinical trials planned in the UK for 2008. The USA drug discovery company PTC are planning a trial for their drug PTC124 in 2008. PTC124 is a drug that hopes to be used to treat young people with Duchenne who have a very specific gene variation. It will target premature stop codons to produce almost a full version of dystrophin in muscle. The MDEX team are also developing another trial that will test to see if the morpholonos can be delivered systemically or throughout the whole body.

Both the MDEX team and PTC have asked to use the DMD Registry to recruit patients to the trials.
We have been working hard to redesign the registration process for the DMD Registry. In partnership with Treat NMD we have now streamlined registration and added new data fields of key clinical information. This information is vital to help to accelerate the process of finding patients for clinical trials. You can now register online for the DMD Registry. Its easy to do and we can give you support.

To register go to: www.dmdregistry.org or call Angela Stringer on 02085569955 for more information.
If you have already registered you need to logon to edit and update your data.

For more information on research please go to the research section of the Action Duchenne website...
Happy New Year

A new years coming! Full of hope, exciting trials will be happening! A change is going to come!

I am very optimistic for the future of Duchenne with all what I’ve heard from the researchers and scientists, all the different ways to treat the condition something must be on the way soon. The main hurdles that we face are funding and regular bodies slowing down research with

the usual paperwork, red tape, Ect. I consider the best way to get around this problem would be to forget the mice/rats and lets get out of the labs and into human and make DMD a special case after all its a Terminal Disease with no hope of survival ... people living with DMD, parents of and of course the Scientists and clinicians must start to adopt the 'can do' attitude to research and trials, I have been hearing 'we must do more work' for years now and nobody getting better! People are dying. Action Duchenne is committed to funding key Duchenne Research projects in 2008. This includes further funding for exon skipping, drug discovery and stem cells. We need £2m to get this research funded and to be able to accelerate clinical trials.

In the meantime clinical trials could be performed in humans by injecting stem cells into individual muscles such as the diaphragm or throat muscles, muscles that will improve quality of life! I for one would take at least being able to eat food properly again a start!

I urge people to keep in regular contact with their MP's and make your voice and opinions heard to the government, in 2008 we need to carry on all the great work we have all done and encourage more people to do the same, together we can all beat the monster that is Duchenne Muscular Dystrophy!

Lets show we mean business...

We would like to wish everyone a very Happy New Year. We would like to thank everyone who has supported Action Duchenne!

Let's hope that 2008 is the year a treatment comes available!!! Here's to 2008!

Dinner Raises £20,000 to fight Disease!

Fri, 07 Dec 2007 13:07:38 GMT

Money in the Bank

A fundraising event at Lydgate's White Hart raised a whopping £20,000 towards finding a cure for a rare muscle-wasting disease. But organisers have taken their campaign a step closer by petitioning the Government to fund treatment for the condition. Duchenne Muscular Dystrophy affects only boys - one in 3,500 are born with it - and there is of yet no cure.

Paul and Heidy Ackroyd's four year old son who is a special happy boy called Edward, was diagnosed with Duchenne this year, described as a ticking timebomb that threatens to take away his independence.

Action Duchenne formerly known as Parent Project United Kingdom (PPUK), are committed to raising money to find a cure.

The Denshaw couple have set themselves the target of raising £150,000 to fund a new research project, Human MDEX Exon Skipping Trial. The 12-month trial takes place at the Oxford University in January and could herald a breakthrough, the quicker they raise the money and the more they collect, the faster the research can take place - meaning we will be able to see the light at the end of the tunnel! The recent dinner they organised at the White Hart was a major boost to the appeal with around £20,000 already collected and money still coming in. It has taken their overall funding to the £100,000 mark.

They also have the backing of myself Carl Tilson, 20 years old of Manchester, Levenshulme who was at the event and has Duchenne. I have launched a petition in an effort to get the funding and backing of the Government that we deserve. Heidy said "The dinner went really well. We were overwhelmed by the support and we say a big thank you to everyone involved. There were 200 people there but if we could have fitted 250 in we would have done because tickets just flew out. We are doing it again next year on 3rd December 2008."

To make a donation or to find out how you can help, log on to www.justgiving.com/tonyackroyd/

Also please sign the petition to demand more funding by the Government, go to http://petitions.pm.gov.uk/breakthechain/

Freemasons Charity Dinner

Tue, 20 Nov 2007 14:25:43 GMT

Heidy and Paul Ackroyd organised a Charity dinner event on Saturday 17th November 2007 at the freemasons Hall, Bridge Street, Manchester!

Here is how they went on...

We held a Charity dinner for Action Duchenne on the 17th November, 160 people attended. Our son Edward Ackroyd aged 4 was diagnosed in May of this year. A champagne reception was had followed by a 5 course meal with entertainment supplied by Shane Hamill, who appeared free of charge. Esther our north west advocate put a very moving power point presentation together, which we presented to the guests. My husband Paul then made a speech thanking everyone for coming and what life had been like since Edwards diagnosis and the way forward for the future for Edward, our family and all the other children & young men with the condition!

We also had Professor Dominic Wells in attendance who was able to speak about the condition, the research and potential cures and treatments. We were also privileged to have Carl Tilson, who is living with Duchenne and is also an advocate for Action Duchenne, and his parents Linda & Fred, Carl made a phenomenal speech on the top floor balcony of the free masons hall, about living with Duchenne. An auction/raffle then took place. So far we have raised £7000.00 an amazing amount of money on a fantastic night. We don’t know the exact total, as money is still coming in watch this space for the final total!

A big thank you goes out to all who contributed to the success of this event.

Heidy Ackroyd

Petition to the Government!

Mon, 19 Nov 2007 18:08:11 GMT

Save us Gordon!

I have created a online petition to the british government because they are not funding us nearly enough, treatment is currently in Clinical trials called Exon skipping which if shows successful will greatly improve life span and improve quality of life for Duchenne Muscular Dystrophy sufferers, thanks to funding from Action Duchenne, Muscular Dystrophy Campaign among all others, plus the government gave £2 million. But in 2008 the funding runs out which means clinical trials will come to a halt!

So we need your help, By signing your name on the website below your demanding to the government to fund DMD and to finally rid the world of this terrible illness!

Please sign the petition!

http://petitions.pm.gov.uk/breakthechain/

Please show your support!

Our aim is to get as many people to sign it as we can!

That would really turn the pressure up on the government... :-) Please pass the petition to your friends & family, also ask your friends to tell their friends. You just need an email address to confirm your signature.

The petition reads!!!

We the undersigned petition the Prime Minister to Fund Duchenne
Muscular Dystrophy if human MDEX Exon Skipping trial works,
from the lab to the Clinic! And help get a treatment for us!

Duchenne Muscular Dystrophy - a boy that is missing the protein Dystrophin, which means his muscles weaken over time and at age 10 are in wheelchairs and without treatment rarely survive past (20 - 25) years! "My condition as got to the point in my life, where it has really started to affect me, I used to be able to eat food, now because my throat muscles have gone weaker that has left me not having a strong swallow, I told myself “I can’t sit here and feel sorry for myself, I need to do something about this” So I want to make more awareness and raise money so we can hurry up and get a treatment as soon as possible, if not a cure, a treatment at least so people can have a better quality of life. I am really passionate about this because I’m tired of suffering and I want them to waste no more time and fix this problem, politics red tape, clinical trial time, Ect… I’d rather die trying, than die without trying!"

All you do is put your name, email address and details - its only to prove your a british citizen no one sees your details only your name, then you'll get an email and you have to confirm it by clicking the link in your email!

Please note - check your junkmail because sometimes it might fall in there.

Carls World

Mon, 12 Nov 2007 22:56:50 GMT

I attended the 5th International conference, I was very impressed wth the way it was set up, very informative and felt very welcome! It was my very first conference so I didn't know what to expect.

I would like to say a big thank you to Action Duchenne for being there for Duchenne Muscular Dystrophy sufferers, without Action Duchenne formerly known as Parent Project I don't think research would of came as far as it has now! 6 years ago the only Charity we had was the Muscular Dystrophy Campaign, which is for all the different types of Muscular Dystrophies, Action Duchenne's mainly focus is Duchenne/Becker Muscular Dystrophy. Also research wasn't going into Clinical trials because the funding for it wasn't there but thanks to Action Duchenne and all the people who have came together, we are getting there!

I am very optimistic for the future of Duchenne with all what i've heard from the conference, all these different ways to treat the condition something must be on the way soon.

I do have real concern though about the cost of treatments such as Exon Skipping, because I've heard that it takes 10 Million for each antisense Oligo to go through toxicology but testing every Oligo would take forever, Also it only takes 10 days to make Oligos. I personally think instead of testing them all, testing a few Oligo would be enough! "Life is priceless, money shouldn't be an option" I think we can overcome this problem if we tell our MPs about this and lobby MHRA and NICE in the UK, also lobby the Government or maybe protest.

We can't stop now, we have got so far!