John Bell, or “JB”, as he’s known to his fans, is the lead singer of jamband Widespread Panic. He’s also godfather to 11-year-old Hannah Elliott of Tampa Fight SMA chapter Hannah’s Buddies, and a full-fledged member of the fight against spinal muscular atrophy (SMA). JB was profiled in a recent article in the film, culture, and music magazine “Paste”. The article, which discussed his role in raising money for Hannah’s Buddies and SMA, was part of the cover story profiling musicians making a difference in the world.

Here’s an excerpt:

Like any concerned relative, Bell was moved to do what he could to ameliorate his niece’s difficult situation. “I had this idea of a silent auction, music and golf all balled into one,” says sports enthusiast Bell. “All I had to do was put it into action and, at the very least, I knew I was going to be there, if that’s all there was to it.”

Bell wanted the event to be one where those with the disease and those who wanted to support the cause could come together and have fun. “I’ve seen charity events turn into an obligation, and though people are happy to write a check, they’re ready to go home,” he says. “[But] this is a giving thing in both directions.”

The singular effort turned into a full-fledged annual golf tournament named Hannah’s Buddies Charity Classic, followed later in the evening by the John Bell and Friends Benefit Bash, which includes dinner, a silent auction and, of course, plenty of live music from Bell and his cohorts. Now in its eighth year, the show has attracted musical guests including Col. Bruce Hampton, Kevn Kinney, Galactic and the Dirty Dozen Brass Band.

“Well over half of the kids with this disease don’t make it past their second birthday, so they’re never really acknowledged as part of their community,” says Elliott. “When John came forward to help, it was the loudest voice this cause had ever had. It penetrated the fanbase pretty deeply, making it a 365-day-a-year cause for a lot of them.”

Read the entire article on the Paste website.

Note: There is an error in the article. It refers to John Bell as Hannah Elliott’s uncle. He is her godfather.

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From today’s press release:

June 22, 2007, Libertyville, IL–Families of Spinal Muscular Atrophy (FSMA) is pleased to announce the selection of a Clinical Candidate for Spinal Muscular Atrophy through its program being conducted at deCODE chemistry. At the same time FSMA is now extending its contract with deCODE to continue work towards an Investigational New Drug (IND) application with the Food and Drug Administration. If successful, this would be the first novel drug designed specifically to treat Spinal Muscular Atrophy (SMA).

SMA is a genetic disorder with no current treatment that is the leading killer of children under two years of age. SMA is typically marked by the degeneration of voluntary muscle movement including the muscles that control crawling, walking, swallowing or breathing. This is an important step in the development of a small molecule therapeutic for this debilitating and normally fatal disease.

The lead compounds have shown the ability to extend survival in a mouse model of SMA. Due to this successful result the organizations have now been able to select a Clinical Candidate. This is the first time a novel compound specifically designed to treat SMA has reached this stage in drug development. Families of SMA has been funding and directing this particular program for the last 7 years. The total investment to date in this program alone is over $10M.

“Selecting a Clinical Candidate means that we are now preparing to run the safety tests required to apply to the FDA to begin clinical trials. This work will require funding of $2.5M and take almost 12 months to complete,” said Kenneth Hobby, Executive Director of Families of SMA. “If we are successful in this stage we would then look to start phase I clinical trials.”

“Excitingly, the program has identified drug-like compounds that act on SMA patient cells in culture to increase SMN2 gene activity and thereby the amount of functional SMN protein. The same compounds also have therapeutic benefit in a mouse model of a severe form of SMA. We are very pleased to see the project come this far,” said Mark Gurney, Senior Vice President, deCODE genetics and deCODE chemistry.

To read the full press release, click here.

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 Fighting Back #6 - Jennifer Sutton [10:00m]: Download

One thing that’s universally known by parents fighting against diseases affecting their children is that improvements to the home are usually necessary. Unfortunately, those improvements are sometimes so expensive that it’s difficult if not impossible without some assistance. Jennifer Sutton found herself in that situation, and fought back.

In episode six of Fighting Back, a podcast that tells inspirational stories about people and families fighting serious diseases, we talk to Jennifer Sutton from New York. Her 13-year-old daughter, Shannon, has spinal muscular atrophy type 2. As Shannon grew older and became less mobile, it became apparent that improvements would need to be made to her home. Ms. Sutton fought to get assistance with those improvements and won, with her state helping with 15 to 20% of the bill.

Ms. Sutton says different states have different rules about what money is available. For more information, start with your local health department. A list of websites for health departments in individual states is available here.

To hear Episode 6 of the Fighting Back Podcast, you have several options. You can listen using the Flash mp3 player above. Or, if you’re a fan of podcast RSS feeds, you can use the Fighting Back Podcast Feed in your favorite feed reader. If you’re an iTunes user you can subscribe and download via our iTunes store. Finally, you can, of course, download this episode using the link above.

If you have feedback, a comment or question, or are interested in having Fighting Back tell your story, please contact us via the Fight SMA contact page or leave us a voice mail message at 206-984-3669. You can also leave a message in the form of a comment on this blog as well. Finally, we’re also excited to now be featured on iTunes, and you can leave feedback there!

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We are very pleased to announce that Spinal Muscular Atrophy legislation, “The SMA Treatment Acceleration Act”, will be introduced in both the U.S. House of Representatives and the U.S. Senate in the next few weeks.We wanted to provide each of you, SMA families, researchers and friends, with a preview of this exciting step forward for all of our individual and collective efforts to raise awareness and secure more federal resources dedicated to finding treatments and a cure for SMA.

Once this legislation is introduced in the House and Senate, we also wanted you to know that we will be issuing a “Nationwide Call to Action” for all SMA families, researchers and friends, to help engage every Member of Congress in support of this bill.

The proposed legislation, “The SMA Treatment Acceleration Act”, has been developed through a collaborative effort of the SMA Foundation, Families of SMA and FightSMA and is modeled on other disease-specific legislation currently before Congress or that has been previously considered and became law.

Specifically, “The SMA Treatment Acceleration Act” would provide for the following:

• Federal support for a national clinical trials network for SMA
• Federal support to enhance the existing SMA patient registry and for expanded research on the epidemiology of SMA
• Establishes an SMA Coordinating Committee to include federal agencies, SMA researchers, and SMA families
• Establishes a trans-Institute research collaboration at NIH under the Director of NIH to ensure all relevant Institutes at NIH are contributing and collaborating on SMA research
• Requires the Secretary of the U.S. Department of Health and Human Services (HHS) to study and report to Congress on ways to improve existing incentives, as well as to recommend additional incentives, necessary to promote SMA drug development among private industry
• The bill provides for the Secretary of HHS to establish a program to provide information and education on SMA to health professionals and the general public

The launch of this exciting initiative would not be possible without the active support of key Members of the House and Senate who have agreed to lead the effort as sponsors of this legislation. We look forward to joining with these Members in the official public announcement of the bill’s introduction, which we hope will occur no later than July. Some of these Members will be familiar to you as they have been long time friends of the SMA community and others are more recent and most welcome champions of SMA!

Lastly, let us again note the important role that we hope each of you can play to help ensure the success of this legislation. Getting a bill introduced in Congress is just the very first step and we will need your help to encourage each and every one of your Members and Senators to join the cause. It will take time, effort, patience, determination, and most importantly, your help!

Just as soon as the bill is formally introduced in the House and Senate, we will be issuing a “National Call to Action” on the SMA Treatment Acceleration Act where we will provide all the necessary materials for you to be able to call, write or email your Members of Congress to ask them to join the fight for treatments and a cure for SMA by cosponsoring the SMA Treatment Acceleration Act.

Sincerely,

Cynthia Joyce
SMA Foundation

Kenneth Hobby
Families of SMA

Martha Slay
Fight SMA

NOTE: If you have any questions, please feel free to contact any one of our Government Affairs staff:

Laura Breiteneicher, SMA Foundation: laurab@wswdc.com
Spencer Perlman, Families of SMA: spencer@fsma.org
Caroline Gibson, Fight SMA: carolinegibson@fightsma.com

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From the SMA Project email service (June 8, 2007):

Dear Colleague,

I am pleased to announce the following funding opportunity for researchers in the field of spinal muscular atrophy (SMA):

The SMA Project (http://www.smaproject.org) expects to release a Request for Proposals (RFP) within the next 14 days to identify and fund facilities capable of providing the necessary research and development support for an Investigational New Drug (IND) submission in a format acceptable to the U.S. Food and Drug Administration (FDA).

The intention is to award a subcontract to a facility capable of conducting safety, toxicology, pharmacokinetic/dynamic, and biodistribution studies with candidate small molecule compounds that act as therapeutic agents for SMA. These studies will be done in partial fulfillment of the requirements for submitting an IND application to the FDA. Science Applications International Corporation (SAIC), on behalf of the Government, shall supply the Offeror with a small amount of each candidate therapeutic compound.

The Offeror will be required to prepare and deliver, for each selected therapeutic compound provided by SAIC for testing, a specific Test Implementation Plan including a proposed budget for the specific testing activities. Activities within the facility shall be highly coordinated with efficacy testing activities outside the facility by SAIC. SAIC shall require from the Offeror preclinical development data that are required by the FDA for filing successful IND applications.

Offers will be due approximately 28 days from the release of the RFP. Relevant documents will be available through the Solicitations page of the SMA Project website at http://www.smaproject.org/solicitations.html.

The National Institute for Neurological Disorders and Stroke (NINDS) (http://www.ninds.nih.gov/) launched The SMA Project: A Collaborative Program to Accelerate Therapeutics Development for Spinal Muscular Atrophy in September 2003. This program funds milestone-driven research aimed at identifying and rapidly developing a treatment for SMA, a paralyzing neurodegenerative disease of childhood.

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We’ve gotten a bit of feedback since launching Fighting Back almost two months ago, and we figure it’s time to share some of it. The podcast features inspirational stories about people and families fighting serious or incurable diseases. It’s produced for Fight SMA and FighterMom.

First up is a voice mail from a fellow podcaster. Dale from Tech Talk for Families called our feedback hotline at 206-984-3669 and left a message, which is loaded into the flash mp3 player below so everyone can hear it.

Thanks for listening, Dale!

Next up are some comments received via iTunes:

• (Rating: 5 stars) This is a well-produced cast about living with and beating diseases. Whether you suffer from such a thing or are completely healthy, there’s always something inspiring to be had from listening to the triumphs of others. Worth your Time.
• (Rating: 5 stars) Great Show! Tough topic and very inspirational! Well done!
• (Rating: 5 stars) Dealing with these topics is important, yet incredibly difficult. What an inspiration for those who struggle or have families who struggle with incurable diseases.

Thanks to all of those who have left feedback for us. If YOU have a question or comment, or would like to have your story told on Fighting Back, please email or (even better) leave a message on our feedback hotline at 206-984-3669!

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A big double belated “Happy Birthday” goes out to Sam and Alex Bolton.  They’re the inspiration for the British organization and website Twins With SMA.  They turned two years old at the end of May.

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Assistive technology has advanced by leaps and bounds, allowing those with seriously debilitating illnesses to do more.  A company in the Netherlands called AssistiveWare recently released a video showing how Mchael Phillips, a teen with SMA, can play video games, instant message, and even write a book … all using only his thumb.  It’s amazing technology.

Learn more about Michael on the AssistiveWare website, and be sure to check out the video.

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