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The last few weeks have brought a bevy of exciting stories from press releases and presentations.  AVI BioPharma has been featured prominently due to results from two separate studies.

Study 1:  Safety Data from Ongoing Systemic Trial of AVI-4658 “The open label dose-finding clinical trial is evaluating the systemic delivery of AVI-4658 once per week for 12-weeks by intravenous infusion.  Although the study is primarily a safety trial, it includes measures of drug efficacy and pharmacokinetics…”

– Dose escalating safety study in DMD patients

– 12 week dosing periods showed no adverse side effects

– Data for clinical results will be collected 26 weeks from the first dose

– Final cohort to be dosed at 20 mg/kg

Study 2:  Drug Prevents Muscle Wasting and Maintains Muscle Function in Severely Affected, Dystrophin Deficient Mice

Professor Dame Kay Davies– Results achieved in utrophin/dystrophin deficient mice (dKO)

– Study used mouse-specific PPMO

– Results hailed as “spectacular” (see video here)

Times Online story

The summary of these two studies is that the first generation exon skipping medicine from AVI BioPharma is well-tolerated in boys with DMD up to doses that may be required for prolonged chronic treatment.  We are eagerly anticipating the efficacy data that is being collected in those patients.  Further, the next generation treatment is working extremely well in what is considered the most challenging and most indicative mouse model of DMD.  The double knockout mice exhibit systemic dystrophy and most closely mimic the human disease.  Video results are posted on the AVI website, and they are exciting to say the least.

Dr. Steve WiltonThe final takeaway from all of this exciting news is that there is still a lot of work to be done.  Funding is required to develop these medicines and move them through the regulatory process from IND to Phase I-III and eventual approval.  Charley’s Fund is providing the vital funding that allows companies like AVI BioPharma and investigators at labs like that of Dr. Kay Davies and Dr. Steve Wilton.  It is almost unbelievable how close these folks and others are to finding an effective therapy and even a cure for DMD.  But they need our help to complete their work.  Collectively we can make a difference for this generation of boys.  Boys like Darius and Charley and all of the others.  Please donate to Charley’s Fund or Darius Goes West.  Purchase the Live Inspired app for the iPhone.  Tell your friends and family about the exciting news and about how they can help as well.

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Darius Weems on CNN

I forgot to put this up when it ran last month.  Right before Darius Goes West was broadcast on MTV, Darius was interviewed on CNN about the vehicle and the cause.  CNN videos can’t be embedded into WordPress (at least not by me), so click the image and redirect to the clip on CNN.com.  It’s definitely worth watching.  There is other Darius news to share, so look for that post this weekend.  It has to do with a contest sponsored by DGW and spreading the word through their Play It Forward campaign.  I am constantly inspired in new ways by the crew and especially by Darius.

darius cnn

Charley’s Fund distributed a bulletin about their entry into America’s Giving Challenge.  The overall winner of the challenge receives a prize of $50,000!  This is a great way for all of us to help raise funds and donate to an organization that directs money into the hands of researchers who have the best shot at developing a treatment or cure for Duchenne Muscular Dystrophy.  To donate, please click here.
challenge header
“To win, between now and November 7th we have to get the most donations to our cause (every person can donate once per day and have it count as a unique donation). We can also win daily awards of $1,000 and $500 if we can get the most people to donate in any 24-hour period. The great thing about this Challenge is that it doesn’t matter how much you give, but instead how much you do to encourage friends and family to get involved in our cause. We all need to come together and start promoting the cause if we want to win.

challengeEach of us has tons of friends on Facebook who we can ask to donate to our cause. But let’s think big too—can you put our cause in your email signature, can you throw a party and get people to donate through the cause when they enter, can you organize other people to go out and fundraise from all of their friends? As you reach out to your friends be sure to tell them why this cause matters to you. The possibilities are endless so let’s talk about what we can do to win on the Wall of the cause or by replying to this bulletin.”

To donate to the cause, please click here.

galapagosThere 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.

nash averyTheir 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 cf logosome 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.”

Charley’s Fund Events

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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.

summit photoI’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.

bellevue reporter

daily piolot

news tribune

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.

cure duchenne

BrainPOP Video About DMD

examiner logoI 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 sometimesbrainpopthat’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.