RARE Project » FasterCures http://rareproject.org RARE disease awareness, support, advocacy, & research Thu, 17 May 2012 21:31:56 +0000 en hourly 1 http://wordpress.org/?v=3.3.2 Wall Street Journal Reports – Do It Yourself Drug Development, With Some Help! http://rareproject.org/2011/12/27/wall-street-journal-reports-do-it-yourself-drug-development-with-some-help/?utm_source=rss&utm_medium=rss&utm_campaign=wall-street-journal-reports-do-it-yourself-drug-development-with-some-help http://rareproject.org/2011/12/27/wall-street-journal-reports-do-it-yourself-drug-development-with-some-help/#comments Tue, 27 Dec 2011 23:45:38 +0000 nboice http://RAREproject.org/blog/?p=5547
The effort by two families to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy — described today in the WSJ — is the result of an innovative new model set up to support the burgeoning phenomenon of do-it-yourself drug development.

Before the Seckler and Wicka families bought halofuginone, a drug that showed promise in experiments done with Duchenne mice, they set up Dart Therapeutics. Dart is funded by foundations set up by the two families, and run by Eugene Williams, a drug industry veteran with 25 years’ experience, including seven years at Genzyme.

Mutual acquaintances led the Secklers and Wickas to Williams in 2010, when the families were discussing how to advance drug development in Duchenne muscular dystrophy. The disease is rare, with only around 20,000 new cases a year, and both families were increasingly frustrated that despite raising many millions to help fund research and experimental drug development programs, promising compounds frequently went nowhere.

They wondered if, with expert advice, patients and advocates in some cases might be able to develop drugs themselves.

That idea led to the formation of Dart in June, 2010. Williams, who is the chairman, put together a “virtual company” — with no big in-house staff — comprised of former senior executives and consultants expert in developing drugs and getting them through the FDA approval process. The idea, says Tracy Seckler, whose son Charley has Duchenne and whose Charley’s Fund foundation is co-owner of Dart, was to create a vehicle to identify potential compounds, make deals, and develop the drugs.

Williams says that in developing drugs for rare diseases, patients and advocates have to take a variety of approaches. Dart’s job is to assess how to best move a drug forward. In some cases, that may mean putting up some money to get other companies to focus on a compound or to pay for testing or other studies to move the program forward. In other cases, Dart will provide advisers and consultants to oversee pre-clinical work that a traditional pharma or biotech company might not want or be able to take on. “We want to help reduce the risk so drugs don’t get stalled,’’ says Williams.

By Amy Dockser-Marcus

In cases where Dart decides it’s worth it to buy the drug — which is what happened when it took a look at halofuginone — a separate company will be formed. (In the case of halofuginone, the company is called Halo Therapeutics.)

Williams  presented the model at a FasterCures conference last year in an effort to spread the word to other patient-driven foundations.

Ultimately, some of those foundations may become customers of Dart. Seckler tells the Health Blog that the goal is not only to help do-it-yourself drug development for Duchenne but also other orphan diseases that can use outside experts to negotiate deals, oversee experiments, and work with the FDA.

http://blogs.wsj.com/health/2011/12/27/do-it-yourself-drug-development-with-some-help/

“We hope to generate revenue to continue what we’re doing in Duchenne muscular dystrophy by providing these services,” she says.

]]> http://rareproject.org/2011/12/27/wall-street-journal-reports-do-it-yourself-drug-development-with-some-help/feed/ 1 'Denim Jean' Ribbon Unveiled To Promote Rare Disease Awareness http://rareproject.org/2010/02/01/global-genes-project-to-raise-awareness-for-millions-of-children-living-with-rare-disease/?utm_source=rss&utm_medium=rss&utm_campaign=global-genes-project-to-raise-awareness-for-millions-of-children-living-with-rare-disease http://rareproject.org/2010/02/01/global-genes-project-to-raise-awareness-for-millions-of-children-living-with-rare-disease/#comments Mon, 01 Feb 2010 23:06:47 +0000 nboice http://RAREproject.org/blog/?p=1097 Campaign Brings Together Support from over 90 Corporations, Non-Profit Foundations, Research Institutions, Hospitals and Advocacy Groups; New Blue Jeans Denim Ribbon Unveiled To Promote Rare Disease Awareness

DANA POINT, CA – February 1, 2010 – Hope – It’s In Our Genes.™ The Children’s Rare Disease Network today announced the Global Genes Project, an initiative designed to raise awareness about the prevalence of rare diseases that afflict millions of children worldwide. Rare disease advocates organizing the campaign selected a blue jeans denim ribbon as the international symbol of hope for children suffering from life threatening and chronically debilitating rare diseases.

More than 90 corporations, non-profit foundations, research institutions and children’s hospitals have signed on to support the Global Genes Project including: 23andme, Amicus Therapeutics, Better Health, BioMarin, CheckOrphan, CollabRx, Counsyl, Coriell Institute, DonateGames.org, FasterCures, FreeForm, Genetic Alliance, Geni.com, Genzyme, Hide and Seek Foundation, Jackson Laboratory, Kakkis EveryLife Foundation, Knome, Lili Claire Foundation, Life Technologies, Mayo Clinic and Shire.

Numerous rare disease foundations from countries including Australia, Argentina, Belgium, Canada, China, New Zealand, Philippines, the United Kingdom and United States are helping support the global movement by organizing activities throughout February and leading up to World Rare Disease Day 2010.

“The Global Genes Project is providing a new platform to help unite people under a common goal and to make a difference for all children suffering from rare disease,” said Nicole Boice, Founder, Global Genes Project. “We want to create a world where children suffering from chronic disease have equal access to life saving drug treatments.”

Growing Public Health Concern; Inequity Exists

Rare disease is an important and growing global public health concern that can no longer be ignored. While each disease may be rare when considered by itself, rare disease collectively impacts the lives of millions. According to estimates from the National Institutes of Health (NIH), approximately 6,800 rare diseases exist, many of which are genetic in origin, translating to an affected population of 25-30 million Americans or approximately 1 in 10 people. In Europe, the number affected is almost identical. Approximately 75% of those affected by rare diseases are children, making the rare disease category one of the most deadly and debilitating for children worldwide.

Despite the success of the Orphan Drug Act of 1983, pharmacologic treatments exist for only a small fraction of rare diseases, or approximately 200 of the 6,800 rare diseases. After 25 years of orphan drug regulations and legislation, the development path to approve new drugs has not stimulated orphan drug development for small populations of patients facing rare disease.
Current studies also suggest that it takes more than a dozen years and hundreds of millions of dollars to take a potential drug from discovery to the marketplace. As a result, severe inequity exists in access to care and treatment for millions of rare disease patients.

Denim Blue Jeans Ribbon Campaign

Leading up to the annual World Rare Disease Day on February 28, 2010, the Global Genes Project will help facilitate a series of denim focused awareness events, charitable activities and engage corporate sponsors, government officials, celebrities and the media to help spread its lifesaving message of hope.

“Blue is the color of strength and our blue jeans denim ribbon is an ideal symbol that expresses solidarity,” added Boice. “Genes and jeans are a natural fit – both are universal, come in pairs and are unique to the individual. It’s a simple concept that anyone can embrace and a powerful way to raise awareness for rare disease.”

The Global Genes Project is asking rare disease advocates and supporters across the world to wear denim jeans and blue jeans denim ribbons the week of February 22 and leading up to World Rare Disease Day 2010. The organization is also asking supporters to display the blue jeans denim ribbon symbol on their websites and to actively invite co-workers, friends and family and their community to join in spreading this global message.

Blue jeans denim ribbons can be obtained by visiting www.globalgenesproject.org. Denim jeans and ribbon wearing supporters can submit digital photos on Facebook. To join the growing list of supporters, visit http://www.globalgenesproject.org/sponsors.php.

About The Global Genes Project

The Global Genes Project is a campaign of the Children’s Rare Disease Network and a grassroots effort with the goal to increase awareness about the prevalence of rare diseases worldwide. The Children’s Rare Disease Network is a registered 501c3 non profit organization. For more information, visit www.crdnetwork.org or www.globalgenesproject.org.

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