Friday, March 27, 2009

Stem Cells- Our Hope for the Future

By Rob Waters

March 27 (Bloomberg) -- Jason Burnett and his 10-year-old son Andrew, both born with a genetic defect, have been recruited into an experiment designed to transform bits of their skin into stem cells that may someday hold the key to a cure.

The Burnetts inherited a heart disease that leaves the father exhausted after a short walk. The two are among the first patients working with scientists who are using a new stem-cell technique that may someday revolutionize care for disorders as diverse as diabetes, Alzheimer’s and muscular dystrophy.

Discovered by the Japanese scientist Shinya Yamanaka, the method creates stem cells without using and destroying human embryos. By studying cells created from people with inherited disorders, scientists are observing, in ways never before possible, how diseases progress and react to treatments, said Doug Melton, a Harvard University researcher.

“This is the breakthrough the stem-cell field has been waiting for,” said Beth Seidenberg, a partner at Kleiner Perkins Caufield & Byers, the Menlo Park, California-based venture-capital firm that helped start Google Inc.

Unlike embryonic cells, the cells created using the Yamanaka method opened a path to test drugs for genetic diseases, Seidenberg said.

The approach used in the Burnett family study, being conducted by researchers at the Gladstone Institute in San Francisco, also is being adopted by labs in the U.S., Europe and Asia and helped persuade GlaxoSmithKline Plc to invest $25 million in a joint venture with the Harvard Stem Cell Institute.

Since human embryonic stem cells were first isolated in a laboratory in 1998, they have fired the imagination of doctors, scientists and patients, who envision a day when new tissues or body parts might be grown to replace diseased ones. The cells are pluripotent, meaning they can be turned into any other type of cell, such as those that make up skin, nerves or neurons.

Read complete Bloomberg News article

Sunday, March 22, 2009

Dear Mr. President: Special Olympics & Paralympics - Special Dreams & Amazing People

Dear Mr. President:
Special Olympics & Paralympics - Special Dreams & Amazing People

This is an open letter to President Obama and our governmental officials, this was written by a young woman with FSH Muscular Dystrophy. Although his recent comments which compared his bowling skills to those of someone competing in the Special Olympics was not intended to offend, it does demonstrate our society's regard for people that suffer with handicaps. The letter I am sharing here is an appeal to those in government to support FSH Muscular Dystrophy research.

Supporting FSH research today will provide people with this disabling condition hope that one day their dreams will not be beyond reach. What we do today will impact the lives of those diagnosed with FSH tomorrow.

Dear President Obama, Senator Murray, Senator Cantwell and our many other honorable representatives,

I have a good friend of mine who says, "No one ever dreams of growing up and competing in the Paralympics." This is not meant to be 'glass half empty' spiel. It is simply stating how life is living with FSH. As a kid you dream of becoming an Olympic athlete. The paralympics is amazing at that. It gives us something extremely challenging to strive for, but I think below the surface of the hardworking, 'glass half full' exterior our hearts break because the Olympic dream is out of reach.

I was diagnosed with FSHD shortly after my 20th birthday. I knew for a good 5 years before that that something was wrong, and after being diagnosed looking back all the signs were there. I was unable to whistle, drink through a straw and I slept with my eyes partially open. It wasn't until over the last year that I have come to realize the devastating affect muscular dystrophy has had on my life. Although I try and keep a positive outlook and 'live' my life, thinking about how my disease is going to progress and how it has changed the course of my life leaves me feeling lost and helpless.

My brain is programmed to be an athlete. I competed in athletics since I was very young and loved every minute of it. I marveled over gymnasts, for their absolute dedication to their sport. I loved the fact that they trained everyday for hours on end to perfect their craft. I was not a gymnast, but a volleyball player. But I took the same dedication and determination and lived for the day that I could devote everything I had to my sport. It became clear about my junior year that I was not progressing at the rate of my peers. In fact instead of peaking, I was declining. I was devastated, and for the life of me could not figure out what the problem was. I felt as though I was working twice as hard as everyone else, and yet my skills and endurance were getting worse. I finally convinced myself that my athletic goals were really not that important to me and that my heart just must not be in it anymore. Shortly after I was diagnosed. I continued to live in denial for the next 4 years about how FSH really affected my life.

In the last year I have started competing in triathlon. I have been lucky to be a part of the Challenged Athletes Foundation and actually race against people of similar disabilities. I train every day with a stern voice in my head not to work to my potential. Because if I do, I might permanently loose muscle. It's a double edge sword. I have to compete for my soul, but I am also doing irreversible damage to muscle in order to do what my heart desires. Every day is a constant struggle to find a healthy balance.

I will not have biological children. This has been a heartbreaking decision for me to come to. But with a 50/50 chance of having a child with FSH, I just wont dream of it. Bringing a child into this world, you want them to be able to do whatever they want to do. Not being able to do what my heart desires has been the hardest thing in my life. It haunts me every morning I wake up. And I would not be able to live with myself if my child had to go through the same thing.

It is hard because looking at me it is not always noticeable that I have a disability. I think because of the nature of this form of muscular dystrophy it doesn't always get the attention it deserves. It's physical and emotional effects rival those of other diseases and dystrophy's, and yet it is not researched as other disorders are. I am asking you now to take a hard look into the lives of those who are diagnosed and their friends and family. I know you will find reason for more understanding into this disease.

Thank you.

The funding for research must be restored to pre-Bush era levels, the support of the NIH is critical to advancing FSH Muscular Dystrophy research.

Write to your representative - ask them to support greater research funding for FSH Muscular Dystrophy!

To read more about this inspiring young woman please go to Kalynn's website & read her blog. She is hard at work raising money for FSH research - please support her STP ride in July 2009. Join her team!!

Here is Kalynn's First giving page

Good Luck Kalynn!!

Wednesday, March 18, 2009

Stem Cell Breakthrough Announced - Hope for those with FSHD

MEDIA RELEASE - 19 March 2009

Sydney IVF Stem Cell Breakthrough Gives New Hope To FSHD Cure

Sydney, Australia - Researchers at Sydney IVF and the Australian-based FSHD Global Research Foundation have scored a global break-through, producing the first stem cell line specific to a common form of muscular dystrophy....

This is a great step forward for FSHD research. This work was funded by the Australian FSHD Global founded last year by Mr. Bill Moss. Read more about them at their website

Benefits of a Harsh Economy? Challenging Non-Profits

Words from the founder of "Ladies Who Launch"

Ladies Who Launch founder Victoria Colligan, wrote about doing business in tough economic times. It is not the time to pack up and quit, but a stimulus to rethink what & how you are doing business. As a non-profit we too must think of new ways of doing things, ways to provide a benefit for our sponsors & donors and changes to our events that will attract attendees.

FSHD research is currently being done on a shoestring budget, Friends of FSH Research can't quit and hope that someone else will fill this funding need. So we are in great need of new ideas - new connections - new donors who want to join with us in impacting the future of those with FSH Muscular Dystrophy. According to Victoria Colligan tough economic times can be great times for creative thinking -

Did you know that Fed Ex began during the oil crisis of 1973 and Proctor & Gamble began selling their products during the Panic of 1837? In fact many of the world’s greatest companies were started during economic downturns either out of necessity or creativity. The great thing about a tough economy is that it paves the way for people to think differently, because they have to. Operating “as is” is no longer effective and the world, in financial terms, is suddenly uncharted territory. Here are ways that you can think differently and take advantage of some unexpected benefits of a harsh economy:

1. Structure relationships as partnerships. Since many people have fewer choices available to them right now, they may be willing to take more risk. Instead of paying outright for services, consider creative barter arrangements or revenue share arrangements based on results and actual success of joint sales.

2. Look for talent at lower prices. Due to lay-offs and job scarcity, there is a lot of high level talent and expertise available at less expensive rates. Find and hire these people. Think long term and think big. Seek out the highest level of expertise that your budget allows and find ways to leverage the talent that’s out there through a reward-based structure that has a long-term win-win built in for everyone.

I'll try to "rethink" our events, or "think outside the box" as many say, but I do worry about how we will conduct future fundraising events. I wonder if we will be able to be successful? How can I rearrange & restructure so that will attract more interest & donations?

A celebrity can impact a non-profit organization's success exponentially. Around the time of the Oscars there was alot in the news about Jerry Lewis - both positive and negative. Although Mr. Lewis has been quoted as saying some very inappropriate things, it is hard to deny his incredible impact on the Muscular Dystrophy Association. He is the face of the MDA! Jerry Lewis has been so closely associated with the Muscular Dystrophy Association that many people mistakenly believe he started the organization!! With the few negatives aside, I would do cartwheels if the Friends of FSH Research had a "Jerry Lewis" to help us!

Sunday, March 15, 2009

Shouldn't it be Karaoke for Everyone?

"....I also know the city, have you ever wondered why all the real interesting shops or bars seem to be down or upstairs. Why is this?

This not a complaint, or maybe it should be. I was going to take up a karaoke singing career, but they all downstairs in the city. In hindsight this could be a good thing for the rest of you as I cannot not sing.

Yet what if you are a good singer and use a wheelchair
, shouldn't it be karaoke for everyone?"

Monica Ellis shares with us her view of the world, written with humor & insight. Monica was born in Canada in 1975 and moved to Australia at the age of three, Monica has FSH Muscular Dystrophy.

"The thing I fear most in this life is talking about me. I would rather hide under my bed. But nothing exciting ever resulted from hiding under beds—that’s if you exclude people who like talking to spiders. And as I am so very afraid of spiders, particularly the hairy ones, I think I will talk about me, my art and my dog, Molly. I will also tell you about a girl named Silly who dropped by one afternoon quite unannounced for a cup of tea and began sharing her adventures.

I started drawing at a young age. Like many other little girls I had an obsession with princess’s in fluffy ball gowns who lived in far away castles. I drew thousands of pictures of them and decided that when I grew up I would like that sort of life…and in particular, those sorts of clothes! The other option was to be a ballet dancer (those great tutus). For me any role in life was always about what you got to wear.

To be truthful nothing much has changed except the realisation that I was not born into royalty and so the princess life was out. (In hindsight I am very, very glad) Alas, ballet was out as well. I was diagnosed with muscular dystrophy when I was five and that put an end to a career in dance. It is a condition that eventually led to life in a wheelchair. But through my drawing I could be any of these things I had once dreamed of being, and travel anywhere on the globe.I was only limited by my imagination...."

Read more of Monica's writings and see her artwork at the site. Monica has her artwork available for purchase as well - check out her many fun cards that feature her character Silly.

Also, see her fun website too!

Wednesday, March 11, 2009

Making Up For Lost Time

"The Future Depends on What We Do in the Present"
- Mahatma Gandhi

In their first newsletter, published in October 2008, FSHD Global Research Foundation in Australia clearly states their mission - "to make up for lost time" for those with FSH Muscular Dystrophy. As Bill Moss, the Global Research founder aptly states, "Many people are of the misguided belief that a cure for one of the 30 plus different types of muscular dystrophies is a cure for all dystrophies. Unfortunately this is not the case. As a result of this misconception, very little research has been undertaken into the causes of FSHD."

Due to years of neglect, the missions of both Pacific Northwest Friends of FSH Research and that of the Global Research Foundation are very similar - to promote and support FSH research. The research of FSH Muscular Dystrophy has been ignored, leaving those that suffer with this disabling condition little hope that a treatment or cure was possible.

With research, there is hope!

(October 2008 Volume 1, Issue 1) FSHD Global Research Foundation is an international not-for profit charity dedicated to finding a treatment and cure for Facio-Scapulo-Humeral Dystrophy (FSHD), which is the second most common form of muscular dystrophy seen in adults and the third most common genetic hereditary disease seen in skeletal muscle.

Initially, sufferers experience wasting or weakness of facial muscles, scapula and upper arm muscles which may later progress to other muscle groups in the legs and back that will impact on a person’s ability to walk. The disease may progress slowly over many decades.

While FSHD is one of the most common muscular dystrophy types, research into it is a staggering 20 years behind that of the most common form.....

"The Future Depends on What We Do in the Present" - the future of my son, people living with FSH today and all those which will be diagnosed with FSH tomorrow will be affected by the actions we take today.

Friday, March 6, 2009

Non-Profit Impacts on Society - Why We Started One

Recently there has been much in the news about charities and foundations that have lost incredible amounts of money due to the management of their funds. Billions of dollars raised for charitable causes, now lost. So many people, organizations, hospitals depend upon the non-profit economic sector to help provide services that otherwise would go unmet - they will again go unmet as these non-profits are forced to cut back or close their doors.

In managing the Friends of FSH Research, I fail to understand "gambling" or creative investing with money that has been donated for it is simply not our money. The money we receive we protect and conservatively watched over as it is for the advancement of our mission - this money has been entrusted to us by generous donors specifically for FSH research. So though it isn't millions, every penny we have raised has been placed in conservative investments, guaranteed & insured, so it will be there when needed. It takes an incredible amount of work to raise the money we have so that to risk it would be unthinkable.

Charitable giving is looked upon by many individuals as a luxury, something only the rich are expected to do, a budget item that is the first to go. This is an unfortunate reality - somehow being "non-profit" places the work of these organizations as less than essential.

Non-profit organizations are an integral part of a progressive society; these organizations fill the gaps left by governmental agencies and the "for" profit companies. Non-profits usually emerge out of a need that is recognized in the community. These organizations tend to be advocates for issues which have been ignored or simply not recognized. They tend to speak out for people in need or a cause that needs attention and work to bring it to the attention of the rest of that community.

As I read in Saturday's newspaper about a woman that is now activist for a cause very close to her heart, I began to cry. As I read of her life circumstances and the events that pushed her forward, I was reminded of the events that pushed us to become active and work for FSH research. Following the horrific summer of 2003 with numerous medical appointments, seeking answers, going down wrong pathways, seeking evidence that might lead to an acceptable diagnosis and running away from Muscular Dystrophy. Muscular Dystrophy was not a diagnosis that would fit in our family's plans. We were just an ordinary family, trying to raise 4 normal, healthy children and looking forward to having them successfully launched and on their own. We were busy looking forward to the future, to colleges, marriages, grandchildren and retirement. A straight pathway - without deviations.

But, like the woman that I read about, events stepped in the way - and altered the course to be taken. Brian was diagnosed with FSH Muscular Dystrophy in September 2003. Our life circumstances changed - life events now pushed us forward along a new, very different path. I never ever saw myself as a leader or activist, and still don't, however, as we looked at our son it was clear we were going to have to take some action. The representative from the Muscular Dystrophy Association greeted us at the MDA clinic at Children's Hospital soon after Brian's diagnosis. They invited us to participate in their support groups & made sure we were aware of the adaptive equipment, such as wheelchairs that they could help us obtain. I did not want a wheelchair for Brian.

Our goal was to do all we could to help Brian stay healthy & strong. We started investigating what research was being done in FSH, trying to determine how close were researchers to finding a treatment or cure. It became clear very quickly that the MDA was not actively working on FSH research. Despite that fact that FSH is the 2nd most common form of Muscular Dystrophy in the adult population, affecting more than 25,000 people in the U.S, only 9 of the 385 research studies they supported in 2006 were for FSHD. With only a hand full of FSH research studies being funded by the MDA each year we felt that it was unlikely that something would be developed that would change the course of this condition for Brian.

In the late 1940s, Paul Cohen, a prominent New York businessman helped found the Muscular Dystrophy Association and served as the MDA's first president - ironically, Mr. Cohen had Facioscapulohumeral Muscular Dystrophy. Mr. Cohen & his family and friends founded the MDA because they felt that those with FSH and other neuro-muscular disorders had no organization which represented them. That organization, the MDA, which he helped to organize has failed to fight for those with FSHD. We hope to continue Mr. Cohen's work and fight for those affected by FSH. Friends of FSH Research's mission is to stimulate and fund new FSH research projects with the goal of finding a treatment or cure to this progressively debilitating condition.

Although we could see no other way to help or try to impact Brian's future, not everyone offered their support or positively responded to our decision to form a non-profit organization. Early while we were first getting our organization off the ground, we needed to find people to participate in an event in order to get public awareness for FSH Muscular Dystrophy, it required an afternoon's commitment. When I asked someone quite close to our family to help us, they replied that it "just doesn't interest me." It wasn't the "cause" I needed her to be interested in, it was my son.

Muscular Dystrophy had never been "my cause" before Brian's diagnosis. As a nurse, with a focus on labor & delivery, newborns, etc. I had been a supporter and donor to organizations such as the March of Dimes and Planned Parenthood. With our family outings to the zoo and the out-of-doors, we also took interest in groups such as the World Wildlife Foundation, Seattle zoo and Science Centers. These were the non-profits, the causes that "interested me."

Muscular Dystrophy, specifically Facioscapulohumeral Dystrophy is a cause that chose us. I was never very aware of Muscular Dystrophy, and certainly had never heard of FSHD but, when it hit our family, affecting our son, it became part of me. FSHD is part of my daily life, whether I am preparing for our auction, thinking of next year's event, researching information, communicating with researchers, writing letters or making phone calls - it is part of my existence. It is the battle I have embraced for my son.

"It's just a business Terry." That was a comment made by a long time family acquaintance, someone I had turned to for support and had hoped would support our efforts as we started our fundraising organization. To them, what I was doing was "just a business." From my perspective, this was the only thing I could do for my son, the only thing I could do that might make a difference. I saw no other option.

"Why an Auction?" We tried a letter writing campaign first, that was our first fund raising effort. Through that effort, writing everyone we knew, telling them of our story and asking for their help & support, we raised $4,000. With the cost of research close to $60 per minute according to some sources, $4,000 was not enough to advance this cause. So, in a local paper I read a story of a woman & her family and how she was helping make a difference for the research of a condition she was coping with - she was making a difference by conducting a fund raising auction. I read this article and immediately made some calls to get more information - I decided in the spring of 2004 that we too would have an auction. Our first "FiSHing for a Cure" auction would be in January 2005.

Wednesday, March 4, 2009

Stem Cells Could Regrow Muscles

“What has been the realm of science fiction is looking more and more like the medicine of the future,” said Professor Gunning.

An experimental procedure that dramatically strengthens stem cells’ ability to regenerate damaged tissue could offer new hope to sufferers of muscle-wasting diseases such as myopathy and muscular dystrophy, according to researchers from the University of New South Wales (UNSW).

Read more - Latest report from
March 5, 2009

Photo caption: The researchers have used stem cells to
regrow muscles in a mouse.
Image: iStockphoto

A Funny Thing Happened on my Way to Raise Money - Scam Attempts

I may be naive but, it seems like really hitting a moral bottom when you try to scam people trying to help others, such as a charity organization. Following a recent Craig's list posting, I received the following "purchase" offer. This glass sculpture was donated to our organization for the purpose of raising research money for FSH - I had posted it's sale on Craig's list in Seattle hoping to find a buyer, thus raising additional funds.

Right away I received a reply - "was this item still available?" I had just posted it - so, the answer was "yes." Apparently, just responding entered me into their scam pathway. The following day I received the following offer:

"Am okay with the conditon & price. my mode of payment will be via Certified Check Drawn on Bank Of America,you will probably need a VersaCheck Paper (Business Size) or a Business Check Paper to print and cash or deposit the check today, the VersaCheck Paper or Business Check Paper is sold for $15 at any stationery store,office depot or at your local office supply outlet.i will wait for your bank to verify and clear the check before we proceed with the shipping which i will hire my private shipping company to come for the pickup after the check has been cleared at your bank so do not worry about the shipment fee,I will need your name and address to send the E-check, you can read more on how it works at"
This email provided an address in Portland, a name and partial zip code. Being curious I entered the address into the "reverse directory" and was not surprised to find that the name did not match - no surprise. Later, I did a Google search of iversacheck, or versacheck paper & craig's list - scam appeared, mine was obviously not their first attempt.

Our non-profit has received other scam attempts such as the one from Nigeria that just needs our account numbers in order to get money out of their country, or the story the relative that has left money in a will, but again needs our help (our bank account) to get the money out of the estate, etc. The most original one I had did not want money at all but just wanted me to send them a new stove. They had plenty of wood, but needed a stove in order to burn it and stay warm through the harsh winter. This was a unique approach which came from a family in Eastern Europe.... I never did follow through to find out how this scenario would play out.

One wonders would could be accomplished if these scammers put their energy, computer wizardry & know-how toward something positive.....

Monday, March 2, 2009

Amis FSH Europe News

News Update from Amis FSH Europe -

Summary of 2 FSH research articles published in February 2009.

The D4Z4 Macrosatellite Repeat Acts as a CTCF and A-Type Lamins-Dependent Insulator in Facio-Scapulo-Humeral Dystrophy.

Facioscapulohumeral muscular dystrophy presenting with hypertrophic cardiomyopathy: A case study.

Learn more about this FSH group, visit their website!
Web Site:

Sunday, March 1, 2009

New Partner in the FSH research Quest

The FSHD Global Research Foundation is an Australian not-for-profit organization dedicated to finding a treatment and cure for Facio-Scapulo-Humeral Dystrophy (FSHD).

Founded by Australian former executive Bill Moss, FSHD Global is focused on making up for lost time since FSH research has been so limited, thus restricting our knowledge regarding this condition. Mr. Moss tells of his experience living with the disabling condition of Facioscapulomeral Muscular Dystrophy upon his life as a child and now as an adult in his 50's.

"You get to the point where you are so tired, you just can't do anything," Moss told BusinessDay in an interview in the Collins Street offices of PPB, the insolvency firm he chairs.
"It starts that you can't walk up the stairs without getting really tired, then you can't walk up a lot of stairs, then you can't walk up any stairs, then you can't walk up a hill, then you can't walk. There's all these gradual things that happen."
To read more about Bill Moss, the impact of FSH upon his life, and why he founded this organization please read the article entitled "Foundation's Research to Make Up for Lost Time," written October 30, 2008.

I am very optomistic with the additional interest in FSH research here in the United States and worldwide in Italy, in Australia, the Netherlands and in France, that FSH research will make progress toward finding a treatment or cure.