An Overview of the Charity in 2013 - and a Thanks for all of your Support
Since Joining Jack was established in 2012, we have gone from strength to strength. 2013 has seen our cause becoming better known, our army of supporters growing, and this year we have found ourselves in the amazing position of being able to make our first major decisions on funding. We could not have done this without you. 2013 has been a year of both struggles and successes for us, and we would all like to offer a resounding thank you to everyone who has joined us in our fight to find a cure for Duchenne Muscular Dystrophy.
This year has seen a huge number of fundraising events taking place for the charity. One of the many highlights of the last twelve months was the very first Wigan 10k, which saw over 2500 people taking to the streets of Joining Jack's hometown of Wigan to raise money in September. The event, which we organised with the help of the local council, was sponsored by a number of local companies and started by none other than Olympic winner Sir Bradley Wiggins.
September also saw a team of over 500 cyclists raising £100,000 by taking part in the Manchester 100 bike ride, thanks to the huge efforts of Joining Jack supporter Paul Holden.
In July 2013, Andy and a number of rugby superstars saddled up to tackle the mountains of France in the L'Etape du Tour, one of the stages of the Tour De France. The gruelling cycle ride was tough going, but gained great publicity for the cause.
And we took our fight to end Duchenne to the UAE - we attended the Dubai Sevens rugby tournament for the second time with a team of rugby legends (captained by Andy himself, and including Andy Farrell and Jason Robinson) that reached the semi-finals of the competition. Sponsored by award winning airline Emirates among other businesses, and with two local Dubai teams playing in the competition for the cause too, the event was a massive success.
In addition to these events, we organised a Royal Ascot event in June that raised thousands of pounds, and two hugely successful celebrity golf days added to our funds. And there have been many, many other fundraising opportunities organised this year by people who have simply discovered the charity and have taken it upon themselves to help in any way that they can.
From those that have found the determination to run a marathon, to climb a mountain, to jump out of a plane for our cause to those who have organised school fete's, golf days, dinners, sports matches and sponsored swims (and everyone in between!) we would like to send you an enormous THANK YOU. Everyone who has donated their money or time this year, no matter how much, has helped us get that step closer to finding a treatment for Duchenne Muscular Dystrophy.
You have joined our fight with passion and we would not be where we are now without you.
So, where are we?
Well, we have recently found ourselves to be in a position to begin the fight against DMD in earnest. Working closely with our trusted team of scientific advisors, we have made our first major decisions on how to spend the funds that we have raised.
The projects that Joining Jack has decided to contribute to have been judged by our advisors to be the most promising in the field - the ones that, should the research continue, will have the best chance of finding a cure for the thousands of sufferers out there with DMD.
The first of these projects is a drug that's being developed by Halo Therapeutics, a US based company that specialises in DMD and works in strong partnership with a number of muscular dystrophy charities. The drug is a derivative of a natural compound found in a species of plant. It acts as an anti-fibrotic, meaning that it prevents scarring and build up in muscle tissue when cells are lost, something that often occurs in those who suffer from DMD, and worsens the effects of the condition.
"We are extremely pleased that Joining Jack has chosen to provide critical funds to Halo for clinical testing of HT-100 in boys with DMD," said Marc Blaustein, Halo Therapeutics CEO. "We are in a race against time to conquer DMD, and Joining Jack's financial support allows us to continue development of this promising therapeutic candidate as rapidly as possible."
For more info on Halo's project, please click here: https://www.alumni.hbs.edu/stories/Pages/story-bulletin.aspx?num=3032
The second recipient of the charity's funding is an Oxford based drug development company, Summit PLC, who is developing a utrophin drug called SMT C1100 that could potentially slow or even stop progression of the disease in all DMD boys. The funding will support development of bio-markers that will help measure the effectiveness of this drug as it now starts to be tested in patient clinical trials.
"The DMD community is playing a key role in the development of promising therapies for DMD such as our drug SMT C1100,"commented Glyn Edwards, Chief Executive Officer of Summit. "Summit is delighted to receive this funding from Joining Jack which will help advance a vital part of our research towards generating potential life-changing treatments for this devastating disease."
For further information on Summit, please click here: http://www.summitplc.com/
We have also recently announced that Joining Jack has decided on another breakthrough research project to receive funding from the charity - ISOFEN 3 is a phase II clinical trial to investigate whether the combination of two drugs, ibuprofen, (a non-steroidal anti-inflammatory agent) and isosorbide dinitrate, (a drug that releases nitric oxide) is capable of slowing the progression of Duchenne Muscular Dystrophy. The study will be carried out in non-ambulant patients affected by DMD, and it is hoped that if successful, a drug will be available in early 2016.
The decision to spend some of the charity funds in supporting these projects was not easy, but we are confident that it was the right one.
In addition to our funding decisions, we have also begun working closely with a number of other Duchenne charities in the hope that we can all reach our common goal that bit sooner.
The collaboration established between Joining Jack and The Muscular Dystrophy Campaign, Alex's Wish, The Duchenne Children's Trust, The Duchenne Research Fund, and Harrison's Fund - called the Duchenne Forum - will accelerate progress in the search for effective treatments and eventually cures. The partnership is supporting seven pioneering Duchenne research projects, to which it will commit £840,000 over the next four years.
Chief Executive at the Muscular Dystrophy Campaign, Robert Meadowcroft, commented: "The Duchenne Forum is a great example of effective charity collaboration. By funding together we have been able to make a larger investment to move promising therapies forward with greater speed, avoid duplication and keep costs to a minimum. The Duchenne Forum is a really positive step forward in charitable funding for Duchenne muscular dystrophy research, and should be encouraging news for the thousands of families who loyally support our six charities in the hope of beating this devastating condition."
Despite all of the highs of 2013, the last 12 months hasn't has been without its lows.
The recent position change of the FDA (the board that makes decisions on approving new drugs in the US) against accelerated approval for Eteplirsen (a promising DMD treatment using exon skipping technology) was very difficult, and resulted in Alex's recent trip to Washington for PPMD's Duchenne Policy meeting with a number of other DMD charities and parents. But the outcome from this meeting is looking promising, as it seems that the FDA are taking note. (For more information on Alex's trip to Washington, please take a look at her blog post in the 'Parents Diaries section of this site!)
Things are, in leaps and bounds, moving in the right direction for the Duchenne community. Yes, we have encountered obstacles, but these have only served to strengthen our resolve and direct us more surely on our way to find a cure. The added support of Andy Burnham, MP for Leigh and the Shadow Secretary of State for Health, has also been absolutely invaluable to us, and we'd like to thanks him for his continued help.
With your continued support, we look forwards to the New Year with more than hope - we look forwards with the belief that we are on the brink of a real breakthrough in treating this condition.
So again, we'd like to take this opportunity to thank all of you for getting involved. We will cure DMD, and each one of our supporters who has helped us raise funds or awareness will be a part of that.
From all at Joining Jack, we hope you have a fantastic Christmas and happy New Year, and we look forward to working with you in 2014!