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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

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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.

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Here’s the latest newsletter from Darius Goes West.  They were kind enough to mention Live Inspired and they’re doing a bunch of fun stuff to raise funds and awareness.  I think Darius and his crew are inspiring beyond words.  Please do what you can to help fund research for DMD.  We can all make a difference.

REMINDER…

STILL SPINNIN’

FIFTH ANNUAL

DARIUS GOES WEST DAY

(Now a weekend-long celebration)

July 17-18, 2009

Athens, Georgia

It’s all happening in just a few days, and we hope you’ll join the fun…

FRIDAY, JULY 17: FUN FOR ALL AGES

PRESBYTERIAN STUDENT CENTER

1250 S. LUMPKIN – 6:00 -10:00 PM

FREE – NO TICKET REQUIRED

Challenge Darius in his favorite video games! Check out the RV that’s traveled nearly 50,000 miles this year! (We apologize for any and all bad smells and/or general filthiness on the RV.) Dunk a crew-member in a tank of water! Toss a pie in a crew member’s face. Win a chance to ride shotgun in the 1969 Dodge Charger (pictured below) featured in Darius Goes West. Or help fight DMD by smashing a car with a sledgehammer! And much, much more! Entertainment will be provided by The Robert Prior Band and The Associates.

Major props to DGW 13th crew members Robert Sewell and Margaret Gaby  (with assistance from George Gaby and Anna Sewell) for taking charge of this event!

SATURDAY, JULY 18: MOVIE & “EXTRAS”

CINE THEATRE

234 W. HANCOCK STREET

3:00, 5:00 and 7:30 PM

FREE (But we recommend e-mailing or calling to reserve your seat)

At 3:00 and 5:00 PM, Darius and the crew will show new footage from this year on the road. Witness the insanity! At 7:30 PM, Darius Goes West will be screened in its entirety. To reserve a free ticket, please email dariusgoeswest@gmail.com or call DGW Headquarters at 706-613-7237.

SATURDAY, JULY 18: CONCERT

THE CLASSIC CENTER

300 N. THOMAS ST.

10:00 PM

PASTOR TROY, KILLER MIKE, AND THE NAPPY ROOTS

TICKETS: $5 per person (We encourage you to throw in an extra $1 when you buy your ticket to help fund the rebuilding of the Georgia Theatre)

Tickets, available at the Classic Center box office, online, and at the door. Doors open at 9:30 PM, and Pastor Troy starts promptly at 10:00.

ALL WEEKEND: HUGE SALE!

At all three events, DGW shirts and DVDs will be available for $10—that’s half off the normal price! (Proceeds still go to the fight against DMD.) Also, be the first to collect the brand new, $25 Darius Bobblehead, available starting DGW weekend!

THANK YOU DGW WEEKEND SPONSORS: ABC Printing, The AdSmith, Athens Banner-Herald, Athens Classic Center, Athens First Bank and Trust, Barberitos, Barron’s Rental, Budweiser, Cine, Coca-Cola, Family Connection Communities in the Schools Program, Gray Man Creative Enterprises, Harry’s Pig Shop, Tom Henson DDS, Jittery’ Joe’s, Mama’s Boy, Marti’s at Midday, Presbyterian Student Center, Neal Pylant, D.M.D., P.C., Raymond James Financial Services, REM, Shirtworks, Stickman Design, Winder Insurance Center and Zaxby’s.

ALSO, special shout-outs to Kitty Wilson (grandmother of DGW crew member Dylan Wilson) and  DGW 13th crew members Lucy Rowland and Anita Will for their generosity in making DGW Weekend possible.

Hope to see you there!

DARIUS AND LOGAN NAMED “CHANGE IT CHAMPIONS”

On June 27, Darius and Logan traveled to Atlanta to receive a “Change It Champion” Award at the Parent Project Muscular Dystrophy’s Annual Connect Conference. PPMD is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. The organization’s mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, advocacy, education and compassion.

Darius had a blast at the conference banquet, where he was able to meet many fans who had seen Darius Goes West. He and Logan also lingered with attendees for over an hour, posing for pictures and signing DVDs. Thanks, PPMD, for this huge honor!

HELP FUND DMD RESEARCH (FOR LESS THAN $1)

If you have an iPhone or iPod Touch, please consider downloading the Live Inspired application. By doing so, you’ll help fund DMD research.  Live Inspired provides a daily inspirational quote and photo that prompts personal growth and reflection. The app is priced at only 99 cents, and 10% of your purchase price will be donated to Charley’s Fund.


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Bee-Purple-Flower-MacroWe ran into a bug with our previous update and the release of version 3.0 software for the phone.  Our developers worked very hard to address this and get it submitted and approved, and as of today the update is available.  This update addresses the connection issue that was causing the error.

Thank you all for your patience and feedback.  I hope that we can all work together to improve our offering and make a difference in our own lives as well as the lives of others.  Even your comments and involvement are inspiring.  Thanks again.

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Only Watch is a charity auction of unique luxury watches held every two years.  The event coincides with the Monaco Yacht Show.  The auction will be held on September 24 and prior to the auction the timepieces will be taken on a preview tour by the auctioneers.  This event is amcmrelevant to this blog because the auction’s proceeds directly support ongoing research for Duchenne Muscular Dystrophy (DMD).  All funds raised at auction will be contributed to the Monegasque Association Against Duchenne Muscular Dystrophy and support its related international research efforts focused on combating the degenerative genetic disorder.

In particular, I’d like to focus on the piece being donated by MB&F (Maximilian Büsser &mbf sage vaughn hm2Friends).  This particular piece was created in collaboration with artist Sage Vaughn and stands as his interpretation of their Horological Machine No. 2.  I find the story of the watchmaker and the artist inspiring.  MB&F refers to their timepieces as “horological machines” and after visiting their site and taking a peek at the engineering they certainly seem to deserve a higher title.

The same can be said of Sage Vaughn.  The butterfly and barbed wire theme is indicative of his artistic style and it’s pretty impressive to see his vision move effectively from canvas to chrome (or sapphire crystal in this case).  In 2007, the auction of 35 unique timepieces raised the exceptional sum of $3,878,946.

"Ruby Throats" Sage Vaughn 2007

"Ruby Throats" Sage Vaughn 2007

Obviously these are not watches that most of us can afford.  It’s just great to see an event on the other side of the world at a yacht show auctioning watches for hundreds of thousands of dollars that benefits the same cause.  Every bit helps and we can all do something.  We can download the Live Inspired app for 99¢.  We can buy a “Darius Goes West” DVD for $20.  We can buy a small gift from the Charley’s Fund online store or just make a donation.  If we can’t give money we can donate time or expertise or effort.

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I received a few links and some information from Charley’s Fund that I’d like to share.  The fund supports a broad array of research and some of that is outlined on the “Charley’s Fund” page of this blog.  This post will focus on AVI BioPharma and the work of this company towards finding an effective therapy for Duchenne Muscular Dystrophy.  AVI BioPharma holds the

Human chromosomes highlighted by fluorescent
Human chromosomes highlighted by fluorescent

predominant patent estate for exon skipping technology.  The relationship between Charley’s Fund and AVI BioPharma is an excellent example of how a not for profit can spur development at a for profit.  I really admire the way that Charley’s Fund approaches these types of contracts.  They are aggressively funding research into therapies that can make a difference in treating Duchenne Muscular Dystrophy in the very near future.  The work is urgent and important and I find it very inspiring.  We can all make a difference by downloading the Live Inspired application, donating money, spreading the word etc. 

Below is an excerpt from a press release issued on June 4 that demonstrates the commitment of both parties to bring this therapy from the lab to patients. 

Avibio

AVI BioPharma Signs an Additional Drug Development Contract with Charley’s Fund Inc. for Duchenne Muscular Dystrophy

Fund has committed total of $5Million to AVI-5038

For Immediate Release

PORTLAND, Ore., and SOUTH EGREMONT, Mass. — June 4, 2009 — AVI BioPharma, Inc. (Nasdaq: AVII) and Charley’s Fund Inc. (CFI), a not for profit organization, today announced that AVI and CFI have amended their existing sponsored research agreement to provide for an additional $3 million in sponsored research funds, for a total of $5 million in support of the development of AVI-5038 through to IND. The drug candidate is based on proprietary PPMO chemistry and has the potential to skip exon 50 in certain patients with Duchenne muscular dystrophy (DMD). AVI’s first contract with the Fund was initiated in October 2007 and partly supported the research that identified AVI-5038.

“We are excited that the research supported by our Fund allowed AVI to identify this drug candidate for further development. We are pleased to extend additional support to AVI to help the company reach its clinical goals in this devastating disease so that more children with DMD can be treated” said Benjamin Seckler, M.D., president of Charley’s Fund Inc.

“Our new drug candidate — AVI-5038 — is based upon novel PPMO chemistry, which will potentially enhance the bioavailability and potency of exon-skipping drugs,” said Leslie Hudson, Ph.D., President and Chief Executive Officer of AVI BioPharma. “We appreciate the support and commitment of Charley’s Fund Inc. to help advance this promising drug candidate towards the clinic.”

AVI has selected and begun preclinical development on a lead molecule, based on AVI’s proprietary PPMO chemistry that has the potential to skip dystrophin exon 50 and so not only restore the proper RNA reading frame but also produce functional dystrophin in patients with certain types of mutation. This therapeutic approach is similar to that of AVI-4658, which AVI has in more advanced development to potentially treat DMD patients with mutations that could benefit from skipping exon 51 of the dystrophin gene.

 Below is an excerpt from AVI’s about us page.

Company Overview

AVI BioPharma is a biopharmaceutical Company specializing in the discovery and development of novel, RNA–based drugs targeting a wide range of important diseases.

A pioneer in the fast growing field of RNA therapeutics, AVI has developed and optimized derivatives of its proprietary antisense chemistry (phosphorodiamidate morpholino oligomers or PMOs) that can be designed to target the diseases through several distinct mechanisms of action.

 

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