National Center for Advancing Translational Sciences (NCATS) Announced By National Institutes of Health (NIH) Director Francis Collins
March 9, 2011 by Chris Hempel
Filed under Featured Stories
Dr. Francis Collins wearing Denim Genes Ribbon designed by Global Genes Project Advocates: Rare Disease Day at NIH: February 28, 2011
The Global Genes Project, a rare disease patient advocacy organization, was invited to present a giant handmade denim genes ribbon to Dr. Francis Collins on February 28 — Rare Disease Day 2011.
This year the NIH Office of Rare Disease Research (ORDR) encouraged everyone to wear jeans to show support for the campaign of using a genes and jeans concept to educate the public on the prevelance of rare genetic diseases and the lack of available drug treatments.
I was invited to speak at Pfizer in Boston about Niemann Pick Type C disease and our medical odyssey over the past three years getting FDA approval to treat Addi and Cassi with a potentially life-saving sugar compound called cyclodextrin.
I wish I could have been in two places at the same time. I would have liked to meet Dr. Collins to thank him for supporting our campaign and to find out more about the NIHs plans for the National Center for Advancing Translational Sciences (NCATS).
Apparently, the National Institutes of Health’s Scientific Management Review Board (SMRB) recommended that the NIH open a new center this fall in an effort to spur creation of medications for rare diseases. Read the SMRB report here. Collins says that the NIH is not going to turn into a “drug-development company” but will move research forward so it will be more attractive to commercial investments.
With fewer than 200 of 7,000 rare diseases have any available therapies creating a center like this at the NIH is long overdue.
It’s encouraging that the Director of the NIH is taking action and recognizes the seriousness of the problem we have with drug development for rare diseases.
I hope the strategy works because it’s the only chance for millions of rare disease patients to have treatments developed to save their lives.
Dr. Stephen Groft of NIH Visits Global Genes Project In Advance of Rare Disease Day 2011
February 24, 2011 by Chris Hempel
Filed under Featured Stories, Uncategorized
Monday, Feb. 28, is Rare Disease Day 2011. Here is a great story from the Orange County Register, which is helping to raise awareness for rare diseases and our efforts to spread awareness through our blue denim jeans ribbon and “Wear That You Care” campaign.
Local advocates looking to kick-start research to help patients with rare diseases got a boost Wednesday afternoon when an official with the National Institutes of Health stopped by to talk.
Dr. Stephen Groft, director of the NIH’s Office of Rare Diseases Research, visited with a Dana Point nonprofit group aimed at building a community for people with rare diseases. A rare disease is defined as one that affects fewer than 200,000 Americans.
Groft informally talked with people who went to meet him at an office in La Plaza shopping center off Pacific Coast Highway that houses the RARE Project (Rare Disease Advocacy Research Education). As he did, children created blue denim ribbons similar to the red ones for AIDS awareness or the pink ones for breast-cancer awareness.
“It’s symbolic that the NIH is coming to speak to this organization. It shows they recognize the power it has,” said Beth Anne Baber, a San Diego cancer researcher and mother of a child with a rare pediatric cancer.
Baber spent hours talking to Groft about what can be done to make advances in rare-disease research.
Monday is Rare Disease Day, and the RARE Project plans to present a 3-foot denim ribbon to NIH Director Francis Collins at a ceremony at NIH headquarters outside Washington, D.C.
The NIH Office of Rare Diseases Research, which stimulates research and acts as an information center, has been around since 1993, but rare-disease research has taken a step into a faster lane in the past five years. Groft attributes that to a greater sense of collaboration among patients groups, researchers, pharmaceutical companies and government. In 2009, NIH dedicated $24 million to pave the way for new treatments for rare diseases. Soon after, the U.S. Food and Drug Administration began providing more grants to encourage researchers to produce more rare-disease treatments.
But Groft’s office is one of many NIH branches. Of the NIH’s $31 billion budget, the Office of Rare Diseases Research gets about $18 million a year. Patients advocates say more money is needed for research, but it can be tough to persuade the government or private pharmaceutical companies to spend money on products when a small number of people have a particular disease. It costs about a billion dollars for a company to develop a new drug.
Groft said he often plays the role of myth-breaker when he talks to the pharmaceutical and medical communities. Many think there is little information about rare diseases and that research is scarce. Groft said that’s not true.
About 1,100 advocacy groups worldwide, including the one in Dana Point, are working to increase awareness of diseases most people have either never heard of or wouldn’t think of as rare. Childhood cancers, for example, are considered rare. So, too, are developmental disorders with names such as campomelic dysplasia and pachygyria.
The RARE Project began in 2008 as an umbrella organization to connect multiple rare-disease groups. Though each disease affects a small number of people, almost 7,000 rare diseases affect 30 million people in the United States, 75 percent of them children, advocacy groups say. Most rare diseases don’t have a drug designed to treat them.
Nicole Boice began the RARE Project after a friend had a child with a rare disease. She saw how isolated the family felt and how worried they were. After some research, she learned how many people have rare diseases.
“I saw how large the community was and was just absolutely blown away,” Boice said. “I thought with so many people, why are they not talking to each other?”
Advocacy groups for patients of rare diseases often focus on a specific disease, making their numbers small. If the groups had an organization to connect them, Boice thought, they would have more power in numbers.
Numbers are often what it takes to get research done. Baber, who started an advocacy group for childhood cancer research called the Nicholas Conor Institute (after her 7-year-old son), joined forces with Boice. They figured they could play a role in showing scientists a large number of patients willing to be tested.
“We have to make it easy. That’s how this community will win,” Boice said.
Advocates aren’t expecting cures, she said. “We’re just looking for treatments, something to help these kids feel better for a while, maybe even live normal lives.”
This year her group launched the Global Genes Project, a campaign to increase awareness of children’s rare diseases. The group is encouraging supporters to wear denim ribbons or jeans on Rare Disease Day.
The group plans to start its first round of fundraising for research by this summer. The dream is to get as big as the Susan G. Komen for the Cure breast-cancer foundation.
Global Genes Project Logos To Support Rare Disease Day 2011 Now Available
January 31, 2011 by Chris Hempel
Filed under Featured Stories
When I opened my in-box today, I received the Global Genes logos to support Rare Disease Day 2011 from the Global Genes Project. The logos are so cool — the work is being done by an amazing strategic communications consultancy and reputation management firm called Charleston | Orwig.
The rare disease community is incredibly lucky to have such great people trying to help educate the world on rare diseases that affect millions of people worldwide. The majority of the 7,000 different rare diseases we know of are genetic in origin — that’s why the Global Genes Project is using jeans and denim as their awareness theme!
My entire family and a bunch of friends will be involved in the “Wear That You Care” denim campaign and will wear jeans and jean ribbons on Rare Disease Day on February 28, 2011. I have been asked to speak on Rare Disease Day at Pfizer’s event in Boston and you’ll find me in my jeans and wearing my denim blue ribbon!
Please show your support by displaying this Global Genes logo for Rare Disease Day on your website and raise awareness for all people with rare diseases.
National Institutes Of Health to Host Rare Disease Day Event; Dr. Francis Collins To Speak; Wear Jeans!
January 9, 2011 by Chris Hempel
Filed under Featured Stories
The National Institutes of Health (NIH) will celebrate the 4rd Rare Disease Day on February 28, 2011, with a day-long celebration and recognition of the various rare diseases research activities. The event will be held in the Lipsett Amphitheater from 8:30am to 5:15pm. Dr. Francis Collins is expected to speak. A tentative agenda has been posted.
In association with the Global Genes Project, the NIH Office of Rare Diseases Research is encouraging all attendees to wear their favorite pair of jeans to the event to support the Global Genes Project awareness campaign. The Global Genes Project awareness campaign is designed around a denim blue jeans theme and a blue denim ribbon which has become the unifying symbol of hope for the rare disease community worldwide. Check out our kid volunteers who make ribbons!
Denim blue ribbons will be distributed at the event and the Global Genes Project will be bringing some of the 7000 Bracelets of Hope which have also been made by volunteers around the world to represent the 7000 rare diseases that afflict an estimated 250 million people globally.
Supporters from the NIH Clinical Center, the NIH Institutes and Centers, the Health Resources and Services Administration (HRSA), the Food & Drug Administration’s Office of Orphan Product Development (OOPD), the National Organization for Rare Disorders (NORD), and the Genetic Alliance will also be at the event.
Attendance is free and open to the public. Unfortunately, I will not be able to attend as Pfizer is holding a Rare Disease Day 2011 event the same day and I have been invited to speak along with other rare disease advocates.
Pepsi Refresh Links For Vote4Hope, Vote 4 Hope Rare Disease Campaign
September 2, 2010 by Chris Hempel
Filed under Featured Stories
If you are looking for information on the Pepsi Refresh Vote4Hope Rare Disease campaign being run by the Children’s Rare Disease Network and Global Genes Project, here are the links and information you need to help fund hope for sick kids.
Click here for Pepsi Refresh Site Link – actual URL if you need it is: http://www.refresheverything.com/fundhopeforsickkids
Text To Vote: Text to 73774 (Pepsi), followed by our voting code (102614) in the text message. You will then receive a confirmation text.
Click here for Facebook Vote4Hope Team: actual URL for Facebook is http://www.facebook.com/ivote4hope
Click here for Vote4Hope.org and meet our Rare Disease Kids Who Are ‘Ambassadors of Hope’ – URL if you need it is: http://www.vote4hope.org/
Click here for Press Release about Pepsi Refresh Vote 4 Hope Rare Disease campaign. Actual press release link if you need it is: http://www.prnewswire.com/news-releases/the-global-genes-project-aims-to-battle-rare-disease-through-the-vote4hope-campaign-and-pepsi-refresh-competition-101956358.html
Click here for Vote4Hope Ambassador or Angel badges – link if you need it is: http://204.42.150.181/vote-4-hope-widgets-and-badges/
