This is What Charley’s Fund Does, and This is Why We Support Them

There was a press release yesterday from Galapagos NV detailing a new clinical trial for one of their existing drug candidates.  It came as no surprise that Charley’s Fund is helping to fund the trial, along with the Nash Avery Foundation.  This is what Charley’s Fund focuses on.  If money from donors is the fuel and research is the engine, Charley’s Fund is the filling station, pooling money from donors that is then used to drive these research engines with the ultimate goal of finding an effective therapy and cure for Duchenne Muscular Dystrophy.

Their drive and passion is truly inspirational.  Their combination of grace and determination and tireless commitment to their cause has resulted in real progress towards the singular goal.  It is an exciting time and there has been some incredible progress made, but there is more to do.  We can all help.  Please donate to Charley’s Fund or the Nash Avery Foundation.  Buy the Live Inspired app.  Purchase a Darius Goes West DVD.  Our collective donations will fund research that will result in a therapy for DMD for this generation of boys. Below are some quotes from the press release.  The full release is available here.

“Charley’s Fund and the Nash Avery Foundation will support the costs of pre-clinical studies to assess the potential effectiveness of G100192 in treating Duchenne muscular dystrophy.”

“The SARM therapeutic that Galapagos is developing for cachexia could be of potential benefit in treating Duchenne muscular dystrophy,” said Benjamin Seckler, M.D., President of Charley’s Fund.  “If the pre-clinical models show efficacy in treating this disease, G100192 could become one of the first small molecule candidate drugs for Duchenne – complementing the gene therapy and biologic approaches in development.”

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Charley’s Fund Events

Charley’s Fund has some really great opportunities to donate to their cause.  You can donate money directly to the cause, you can buy some Charley’s Fund merchandise, you can support Darius Goes West, you can buy the Live Inspired app for the iPhone.  In addition to all of that, there are fund-raising events that you can attend.  I’ve posted the information below, but it’s also available on the Charley’s Fund website.  If you can help in any way, please do.  This charity does an amazing job funding cutting edge research that has the potential to deliver an effective therapy for this generation of boys with Duchenne Muscular Dystrophy.  We are closer than ever to this goal, but there is still a lot of work to be done.  That work can only be accomplished with adequate funding, and a large part of that funding comes from people like you and me.  We can all make a difference by working collectively towards a goal.  Please help.

Women’s Spa Day in the Berkshires Friday, September 25, 2009 Cranwell Resort & Spa, Lenox, MA This special day will include a spa treatment of your choice, lunch, lectures, fitness classes and all-spa access while benefitting    Charley’s Fund.  Come for the day with a friend (or 2 or 3 or 4).  If you prefer to stay over on either Thursday or  Friday  night, Cranwell has generously offered a reduced room rate of $129. Click Here to learn more and Purchase Tickets

4th Annual Louis J. Capano, Jr. & III Family Foundation Golf Outing Monday, September 21, 2009 Dupont Country Club, Wilmington, DE All proceeds from this golf outing will benefit Charley’s Fund. Please call Alex Walsh at 302.429.8700 to learn more and purchase tickets.

Darius Goes West Television Premiere MTV2 Sunday, September, 27, 2009 at 12 noon In honor of Darius’ birthday, a special one-hour version of Darius Goes West will air on MTV2. Tune in and tell all your friends!

Old Harrison Rye Flag Football Charity Game Sunday, October 11, 2009 Harrison Avenue Football Field A reunion football game sponsored by Benjy’s good friends from his hometown of Harrison, NY to raise money for Charley’s Fund.” For more information, please visithuskieshelpcharleysfund.com.

Live and Let’s Give Friday, November 6 – Sunday, November 8, 2009 Lichtenestein Center for the Arts, Pittsfield, MA link:www.culturalpittsfield.com A group of exceptional artists from the Berkshires and beyond will gather at the Lichtenstein Center for the Arts to create a beautiful and eclectic shopping experience for the community. A portion of the proceeds from the sale, and all of the opening night ticket sales will be donated to Charley’s Fund. This event has been generously organized by Michele O’Hana. More information and invitation to follow.

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Success for 2009 Climb to Cure Duchenne!

I’ve posted a couple of items about the 2009 Climb to Cure Duchenne.  The team summited Mt. Rainier on 9-1 and to date have raised over $130,000 for Cure Duchenne.  This was an amazing effort by all of the people involved, from the climbers to the supports staff to the individuals and companies that donated time and money.  It is inspiring to see such a broad range of people get motivated and get involved to raise funds for such a worthy cause.  My hat is off to all of you.

Here are some follow up articles to the 2009 Climb to Cure Duchenne.  The quotes from parents, participants and others are touching and inspiring.

If you would like to contribute further to the cause, please click on the logo below.  There is an urgent need for funding research and this is a great way to help.  I’m inspired by these efforts and I hope that you are, too.  Please help.

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BrainPOP Video About DMD

I was alerted to this video by the Examiner Miami.  It’s a really informative video that explains Duchenne Muscular Dystrophy.  It was put together by BrainPOP, and although it’s intended for kids I think adults will benefit from watching it as well.  It covers the basics of the disease in an elementary way, but sometimesthat’s what’s needed.  If you’re not familiar with BrainPOP I will take thisopportunity to recommend them.  If you have kids and want to introduce them to scientific concepts and ideas, BrainPOP is a great way to do it.  A free trial is available and the full site is available by subscription.

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A Very Informative Interview from Xconomy Seattle

This interview with Dr. Jeff Chamberlain on xconomy.com is a really informative read.  He is a neuroscientist at the University of Washington in Seattle and has been doing research related to muscular dystrophy for over 20 years.

Jeff Chamberlain, Ph.D. McCaw Chair in Muscular Dystrophy Professor, Depts. of Neurology, Medicine and Biochemistry

The interview covers a lot of ground, but it really lays out how much progress has been made and how much work there is left to do.  He answers questions about the various therapies currently in trial, as well as the possible long term impact of the therapeutic approaches.  It’s a fairly long interview, but I found it inspiring and extremely informative.  These kids need our help, and one of the best ways that we can help is by donating to organizations like Charley’s Fund and Darius Goes West.  Downloading the Live Inspired app for the iPhone gives you a daily reflection and inspiring image and also donates money to a charity working specifically to fund research at labs that are working on these revolutionary therapies.  Please help.

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AVI BioPharma to Present Safety Data

On August 25th, AVI BioPharma issued a press release stating their planned presentation of the full set of data from a completed phase I clinical trial for drug candidate AVI-4658.  As I’ve written before, Charley’s Fund has provided key funding for AVI BioPharma research into finding a therapy via exon-skipping technology, specifically to treat Duchenne Muscular Dystrophy.

Here’s a quote from the release that sums up why so many people are excited about this technology and this type of progress.

“Data from the completed single-blind, placebo-controlled and dose escalationPhase 1 trial showed that AVI-4658 was safe when injected intramuscularly and successfully induced the production of dystrophin protein in patients in a dose-responsive manner. AVI also announced today that these data have been published online in the journal Lancet Neurology and will be featured in the October print edition. This safe and well-tolerated production of new dystrophin is believed to be the key to restoring muscle function and successfully treating patients with DMD – a condition for which there is no currently approved therapy.”

The full press release is available here.

To read the clinical trial abstract, click the Lancet logo.

This is the type of research that Charley’s Fund supports.  This is the type of research that represents real progress.  This is the type of research that can help this generation of boys with DMD.  This is also they type of research that requires significant funding to bring these therapies out of trials and into the clinic.  We can all help by purchasing the Live Inspired app, donating to Charley’s Fund or Darius Goes West.  You can even buy stock in AVI BioPharma (Nasdaq: AVII).  Donate time.  Share the message.  Make sure that your friends and family understand the urgency with which we must approach this cause.  Please help.

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Please Read the Referenced Article – It’s GREAT

I signed up for a Google alert for “Duchenne Muscular Dystrophy” and I get links e-mailed to me every day with stories relating to the disease.  These stories range from press releases from companies doing ground-breaking research (like AVI BioPharma) to community interest pieces about fund raising efforts or a local family that is dealing with the disease.  Sometimes I get one that I feel that I have to share with on the blog.  I posted a little while ago about the 2009 Climb to Cure Duchenne.

This most recent story from the Gainesville Sun (written by Lashonda Stinson Curry) relates to that post, but on a much more personal level.  I’ll share some of the quotes from the story here, but please take the time to read the entire thing.  The article is very well-written.  It’s incredibly poignant, touching and heartbreaking.  Once again, the quotes from the family convey the urgency with which we must approach funding research to find a therapy and a cure.  Bill and Kimberly Procko’s son, Evan, was born with DMD and Bill is training to climb Mt. Rainier as part of the 2009 Climb to Cure Duchenne.

One quote by Bill Procko:

Bill Procko, with his 5-year-old son Evan, climbs a mountain this summer while on vacation in New Foundland, Canada. Procko was training for the climb up Mount Rainier in a few weeks to raise money for Duchenne muscular dystrophy, which Evan was diagnosed with last March. (photo courtesy of Bill Procko from Gainesville.com)

“Birthdays are bittersweet because we don’t want another birthday to come. We want it to slow down. We want time, we don’t want to see him get one year older until this thing is taken care of.”

“In a whole new twisted way you’re told to raise your child. He’s going to be a happy, wonderful little kid. He’s going to learn all these things and he’s going to think he’s fine, but in 15 years prepare, because he’s going to be killed by a car.  Everything he is learning day by day he’s going to lose that ability — his legs, his arms, hands, every use of everything. So every proud little achievement he makes, it’s a twist in your gut.”

Evan’s mother, Kimberly Procko is quoted:

“Evan is moving all around now and having fun with his brother and you see this and you’re knowing that the progression that’s coming is so mind-bending. If you have a child and you know this child has a disability and that’s it, then you learn to work around it. But we can’t work around it because his disability has not fully been realized until he dies.  It’s just going to keep getting worse and worse and worse.”

We can all do something to help fund research to find an effective therapy for this disease.  Buy the Live Inspired application for the iPhone.  Donate to Charley’s Fund.  Donate to a  climber in the 2009 Climb to Cure Duchenne.  Every single dollar helps and collectively, we can make a difference for this generation of boys.  The time is now.  Please help.

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Silver-Lined Heart

It seems that we’re inundated with bad news these days.  Sometimes it feels like I can watch the news for an hour or read the paper and not read or hear a single good or uplifting story.  I suppose it’s possible that doom and gloom bring more viewers to a particular media source, but I know there are inspirational stories out there and uplifting happenings occurring on a daily basis.  In fact, one of the drivers behind Live Inspired was to at least have one point of positive reflection and every day.  When life gets too hectic or too dark, I can look at the daily quote and picture and gain some perspective on life.

Given that we are collectively choosing to be positive and find the upside in a world that is all too often focused on the downside, I would like to share a poem with you today.  It’s by one of my favorite writers, Taylor Mali.  You can learn more about Taylor at his website, read more poems and even see videos of his readings.  He’s been featured in a number of places and one of his most famous pieces is titled “What Teachers Make” and he rarely gets the credit he deserves for this and his other works.  If you like this piece, visit his site and do what you can to support your local artists (like ordering his book, for example).  We all need inspiration in all of its many forms, now more than ever.  If you’re inspired by the blog and the app and seek out the positive, show your support by buying the app and supporting Charley’s Fund.  Charley and is family and the other families dealing with DMD are forced to find the silver lining when facing this disease.  We all have a chance to help them and fund research that would very well result in an effective therapy for this generation of boys.

Silver-Lined Heart
By Taylor Mali (www.taylormali.com)

I’m for reckless abandon
and spontaneous celebrations of nothing at all,
like the twin flutes I kept in the trunk of my car
in a box labeled Emergency Champagne Glasses!

Raise an unexpected glass to long, cold winters
and sweet hot summers and the beautiful confusion of the times in between.
To the unexpected drenching rain that leaves you soaking
wet and smiling breathless;
“We danced in the garden in torn sheets in the rain,”
we were christened in the sanctity of the sprinkler,
can’t you hear it singing out its Hallelujah?

Here’s to the soul-expanding power
of the simply beautiful.

See, things you hate, things you despise,
multinational corporations and lies that politicians tell,
injustices that make you mad as hell,
that’s all well and good.
And as far as writing poems goes,
I guess you should.
It just might be a poem that gets Mumia released,
brings an end to terrorism or peace in the middle east.

But as far as what soothes me, what inspires and moves me,
honesty behooves me to tell you your rage doesn’t move me.
See, like the darkest of clouds my heart has a silver lining,
which does not harken to the loudest whining,
but beats and stirs and grows ever more
when I learn of the things you’re actually for.

That’s why I’m for best friends, long drives, and smiles,
nothing but the sound of thinking for miles.
For the unconditional love of dogs:
may we learn the lessons of their love by heart.
For therapy when you need it,
and poetry when you need it.
And the wisdom to know the difference.

The solution to every problem usually involves some kind of liquid,
even if it’s only Emergency Champagne
or running through the sprinkler.
Can’t you hear it calling you?

I’m for crushes not acted upon, for admiration from afar,
for the delicate and the resilient and the fragile human heart,
may it always heal stronger than it was before.
For walks in the woods, and for the woods themselves,
by which I mean the trees. Definitely for the trees.
Window seats, and locally brewed beer,
and love letters written by hand with fountain pens:
I’m for all of these.

I’m for evolution more than revolution
unless you’re offering some kind of solution.

I’m for the courage it takes to volunteer, to say “yes,” “I believe,” and “I will.”
For the bright side, the glass half full, the silver lining,
and the optimists who consider darkness just a different kind of shining.

So don’t waste my time and your curses on verses
about what you are against, despise, and abhor.
Tell me what inspires you, what fulfills and fires you,
put your precious pen to paper and tell me what you’re for!

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Helena Bonham Carter Speaks for Action Duchenne

Action Duchenne is the only UK charity exclusively fighting for a cure for Duchenne.  They have done some really incredible work raising funds and awareness in the UK and worldwide.  They work grass roots all the way to the government.  Their mission is similar to Charley’s Fund in that they focus heavily on funding research to find effective therapies for DMD.  They have also supported research at AVI BioPharma.  Their board of Directors are all parents or grandparents of people living with Duchenne.  One of their patrons is Helena Bonham Carter.  She is a talented actress and mother of two, and she recently spoke out to urge the government in the UK to adopt certain practices that would better equip their National Health Service in serving patients who require treatment with genetic medicines in the near future.  A recent press release by PR Artistry outlined some of the concerns and also how Action Duchenne is pressing the government to adopt best practices in the way that NHS administers care to DMD patients.

Bonham Carter’s direct quote is “Recent developments in genomic medicine are very exciting, but are still thwarted by lack of funds. It is incredibly important that single-gene disorders like Duchenne Muscular Dystrophy are not left behind in the rush to treat more widespread diseases like cancer or diabetes. Scientists are very close to significant breakthroughs with treatments such as exon skipping, and it is vital that they received adequate funding and support from the Government, rather than relying on charities to provide funds. We also need to make sure that the health service is able to deliver treatments for boys and young men with Duchenne, as soon as they are developed.”

CEO of Action Duchenne, Nick Catlin, added “The recognition that genomic medicine is a significant step forward is very exciting, as Duchenne is a disorder that looks very likely to benefit. However, we are very concerned that single-gene disorders like Duchenne Muscular Dystrophy that are relatively rare will now become the poor relation as funds are diverted to more complex conditions. This must never be allowed to happen. We would argue that Government should concentrate on building a delivery model for genomic medicine for single-gene diseases, like Duchenne, where the cause is already identified, and therefore easier to deal with. This should be based on ring-fenced funding for a programme of research and clinical trials, and roll out of Centres of Excellence where working with experts means that more clinicians become trained in the delivery and monitoring of these new genomic treatments and drugs.”

These are just more examples of people who are working very hard to make sure that research is funded and awareness is raised.  The sense of urgency among the DMD community is really inspiring.  The science, the outreach, the hope and the possibilities are all extremely exciting.  We all have an opportunity to make a difference and see a breakthrough therapy for this heartbreaking disease.  Donate to Charley’s Fund, buy a Darius Goes West DVD, buy the Live Inspired app for the iPhone.  Now is the time.

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2009 Climb to Cure Duchenne

I just came across this story and I thought it was worth sharing.  We have chose to support Charley’s Fund but there are very many other worthy charities and creative efforts aimed at fundraising to support research aimed at finding an effective therapy and even a cure for DMD.  Traditional fund-raising efforts are ongoing, but sometimes the disease and those suffering from it get more publicity and recognition from some of the less traditional efforts.  Darius Goes West is a great example of this.

Another is Climb to Cure Duchenne.  Several climbers from all over the U.S. will climb Mt. Rainier in Washington to raise awareness and, more importanly, funds for Cure Duchenne.  They hope to raise over $170,000 for the cause by having each of the 11 climbers collect $1 for each of the 14,410 feet of mountain that they will climb.  The story referenced above is specifically about one of the climbers, Thomas Seoh.  I wanted to share some of the quotes from the story because they express the hope and the sense of urgency that is swirling around DMD therapy at the moment.  He has experience in biotech and specifically in searching for treatments for DMD, so his thoughts certainly carry some weight.

“This is a particularly anxious and hopeful time because several experimental medicines are working their way through clinical trials and financial support now can help this generation of boys.”

“Some of them will be on the market in a few years.”

“We’re not looking for something that could be a cure someday.  We are seeking funds to apply to research that can help this generation of boys.”

If you can, please help.  Donate to Charley’s Fund or buy some merchandise.  Buy a Darius Goes West DVD.  Buy the Live Inspired App.  Every bit counts and every bit helps, and our collective efforts can make a difference for boys like Charley and Darius and all of the rest.  The time is now.

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