In only four months and with fundraisers organized by over twenty families and their friends across the globe (literally), “Unite For The Cure” (Unite) met and surpassed their goal of raising $100,000 for Dr. Keirstead’s promising SMA research. In fact, over $145,000 — yes, one-hundred-forty-five-thousand-dollars — was donated to Families of SMA (FSMA) through Unite efforts earmarked specifically to accelerate Dr. Keirstead’s stem cell research to human clinical trial. This is an enormous accomplishment in and of itself in such a short period of time, but when you take into account that every single one of these remarkable families is either caring for a terminally ill child or grieving the loss of their precious baby — it is nothing short of miraculous!

Each of these inspiring Unite families has poured their heart and soul into fundraising, has shown tenacity and dedication, has asked friends and family to be involved, and has campaigned tirelessly to get Dr. Keirstead the money he needs. In November 2009, Dr. Keirstead’s SMA stem cell program was named by Esquire magazine as a “Project That Will Change Science Forever”. We truly hope that this will prove to be the case and that each of us will have had a material impact on accelerating this groundbreaking, game changing science into the history books.

A message from Kenneth Hobby, President, Families of SMA:

Thank you all for the tireless efforts involved here to support this program and our mission to develop a cure. Thanks to you we will be one step closer.

Reaching $100,000 raised is a tremendous milestone to be able to hit. I think we all have that additional bit of extra hope now in the community with the funds you have all raised to help move us forward.

The initial investment from Families of SMA in stem cell research was in 2000 and this funded efficacy studies using motor neurons from mouse stem cells. Over the following years additional funding lead to the first, highly-pure therapeutic population of human motor neurons for cellular replacement therapy for SMA. Click here for full details on the program.

The team involved in this collaboration includes the laboratory of Dr. Keirstead at The University of California, Irvine, the laboratory of Dr. Kerr at Johns Hopkins University, California Stem Cell, Inc., and FSMA.

In October of this year we completed a pre-Investigational New Drug (IND) Meeting with the FDA. Click here for details of this recent meeting. This meeting gave us guidance from the FDA on the clinical and regulatory pathway and requirements for submission of an IND application to be able to initiate human clinical trials.

The stage of the program we are now beginning, and the work and studies that all of your efforts are helping to support, is the preparation of the final IND application. This involves a lot of regulatory work and applications, and several experiments and tests showing that manufacturing of the motor neurons to be used in the clinical trials is reliable and scalable. The final IND application that is given to the FDA is meant to comprise all of the information that has been generated for the program to date. This can easily run into many thousands of pages in length. This application is submitted to the FDA, which then has 30 days to review the information and then give a final say on whether we are clear to start the first clinical trial.

At this final stage just before clinical trials nothing is yet guaranteed. Programs can and often do get put on hold by the FDA at this stage. This particular program looks to not only be the first new IND application put into the FDA for SMA, but also one of the first for a stem cell therapy for any disease! So, the unknowns and risks are still high, but so are our hopes that we will be successful in getting approval and then move forward into clinical trials with patients.

Thank you all so much for all the amazing energy you have put in to support this program and to help all in the community.

Very best,

Kenneth Hobby, President
Families of SMA

And a message from Dr. Hans Keirstead:

I am excited to let you know that we have finished all of the animal work that is required by the FDA, covering both efficacy and safety. We are currently writing up all of the results in a manner that the FDA requires for submission of an IND. We are also completing work to document the cell preparation and shipping procedures, and conducting experiments to ensure that those procedures do not adversely affect the cells. Lastly, we are preparing the IND.

FSMA, and many families, have generously contributed to the funding of many of our research needs. However, many non-research tasks must be preformed before we can submit our IND. These studies include the cell preparation and shipping procedures mentioned above, and the IND ‘paperwork’ preparation costs.

Thank you so very much Unite families for all of your support. We simply could not have done this without you, and your ongoing support is nothing short of enabling this research to move forward into the clinic.

Hans S. Keirstead, Ph.D.
University of California, Irvine – Reeve-Irvine Research Center
Sue and Bill Gross Stem Cell Research Center

At this point, we can all know and be proud of the fact that, as one united community, we’ve helped accelerate this program. We now simply have to wait for the IND process to play itself out, the science to prove itself in humans, and — hopefully — pave the way for getting this treatment to children as safely and efficiently as possible.

From the bottom of our hearts, thank you Unite families. It has been an incredibly inspiring experience working with you and we hope that each of you found it to be motivating and uplifting. This has been a wonderfully successful campaign and we are so proud of every single effort that has helped make this such a huge success — a $145,000+ collective success!

As we intended Unite to be a one-off campaign, we will be closing Unite down (although this site will live on in perpetuity in its current form). However, we believe this type of earmarked, directed fundraising serves an enormous benefit to both fundraisers and researchers and in the coming months we plan to launch a similar program. After all, it is your money — you should decide where it goes. If you are interested in joining this effort, please contact us or stay tuned for the big launch in the coming months. Together we will see SMA cured!

The McClay Family
Raised over $16,700!

The Span Family
Raised over $10,700!

The Potter/Reilly Families
Raised over $10,000!

The Schmid Family
Raised over $9,500!

The Marshall Family
Raised $9,000!

The Strong Family
Raised over $8,300!

The Gooden Family
Raised over $7,700!

The Pringle Family
Raised over $7,700!

The Butler/Trakas Families
Raised over $6,900!

The Gustafson Family
Raised over $6,700!

The Lunt Family
Raised over $6,600!

The Lucas Family
Raised over $6,000!

The Zahn Family
Raised $6,000!

The Coleman Family
Raised over $5,200!

The Wright-Beasley Family
Raised over $5,000!

The Ragland Family
Raised over $5000!

The Howell Family
Raised $5,000!

The Calafiore Family
Raised over $4,500!

The Felix Family
Raised over $4,000!

The Greene Family
Raised over $1,400!

The Lovelace Family
Raised nearly $1,000!

An additional $2,000 was raised in private donations sent directly to Unite from individuals, ranging from $25.00 to $500.

Our daughter Lucy was born July 9, 2009. She was developing normally, it seemed, until 4 weeks, when she stopped lifting her head. Everyone reassured us that all babies developed at their own pace, so we tried to push our concerns to the back of our minds. Lucy was never a big leg mover, either, but we thought she’d catch up. At her 2 month check-up, her pediatrician was immediately concerned with the lack of muscles in her legs and neck. She made us an appointment to go to the Children’s Hospital in Milwaukee, and we went down there right away that night. We were there for 3 days while they drew vial after vial of blood, took urine samples, did x-rays and an MRI. Then they told us it would be 6 weeks for results and sent us home to wait. Four weeks later, on October 14, we met with the geneticist, who told us it was what she had suspected, SMA Type I, and that our beautiful baby would most likely not live to see her second birthday. The genetics counselor, bless her, gave us info on FSMA and a clinical trial in Madison. We found strong, faithful people fighting for their children, and learned about all the hope there was for treatment and a cure. We knew that we were in the right place, and that we wouldn’t take Lucy’s diagnosis without fighting alongside these inspiring people. There IS hope for our children; a cure CAN be reached in their lifetime!  LucysHopeForACure.homestead.com

Why are the Trakas’ uniting for the cure?

When the Trakas’ second daughter, Andrea, was born, she  seemed to be perfectly healthy. When she was 7-weeks-old, they began to realize that she wasn’t holding up her head and moving around the way her older sister did at that age. After several months of tests, the dreaded diagnosis was confirmed: Type I SMA. SMA was not new to the Trakas’. Their best friends’ 15-year-old daughter has Type I SMA as well, and she has defied the odds with her tremendous strength, courage, and faith. It is still beyond their understanding how two lifelong friends could each have daughters born with the same genetic disease. Andrea was so happy and loved her 7  months of life and had an extraordinary impact on so many. There would be no greater joy for her family than for a cure to be found so that other parents would not have to endure the same difficult journey. The Trakas’ have been involved in many fundraising activities in support of SMA programs — long before Andrea was even born. One of their biggest and proudest accomplishments is the building of the “Andrea Trakas Memorial Playground” in the Tampa Bay area. This handicap accessible playground built in Andrea’s memory is a playground built “for all children”, and is state of the art. They know how many children will enjoy this facility for many years to come. The Trakas’ strongly believe in the potential that stem cell research has to treat and cure SMA, and they are honored to part of Unite for the Cure and partners with the Butler Family.

Why are the Colemans uniting for the cure?

Whit and Lindsey Coleman are thrilled to be the parents of Jonas Mathie Coleman. Jonas joined their family perfectly healthy on December 2, 2007. Whit and Lindsey were so excited to start the adventure of raising their first baby boy. Six weeks later, they were heartbroken to learn that Jonas was diagnosed with SMA Type I. They had never heard of this horrible disease and neither of their families have any history of SMA. Coming to terms with their new life and changing their expectations of what they wanted was difficult. However, Jonas wasn’t going to give up and so they couldn’t either. Since then, the Colemans decided not to let this disease run their lives but would do all they could to give Jonas every opportunity to have a full and fun life. Jonas is now 22-months-old and each day they have with him is a gift. Due to the progressive nature of SMA, they know that their time with him is limited. Unite for the cure gives them the hope that they will have a lifetime with him and the security that they can have a healthy family in the future.

You can follow Jonas’ adventures on their blog at Coleman2Family.blogspot.com.

Why are the Felix’ uniting for the cure?

Mike, Jaci, Amber (15), Jacob (3), and Alexa (22-months-old with SMA Type I) — Alexa came into this world on November 16th, 2007. A beautiful perfect baby girl. But from day one, Alexa has been fighting for her life. Not even 24-hours-old, she was taken to the NICU with severe jaundice and anemia. Doctors told Mike and Jaci Felix three days later that her organs were shutting down. No matter what they did, her bilirubin would not come down, and her anemia kept getting worse. They gave permission for her to have a total body blood exchange, a very risky procedure. The procedure was scheduled for the morning and they just prayed. The thought of losing their daughter was overwhelming. The prayers must have worked because one last blood check showed that her numbers were finally getting better. After several blood transfusions and lots of medicine, Alexa was finally ready to go home. The Felix’ thanked God for being so blessed.

Then just shy of turning 6-months-old, the Felix’ were told their daughter had SMA Type I, and the NICU stay at the beginning of her life was nothing compared to the fight she was facing now. They almost lost her once, and they are determined not to let that happen again! Since Alexa’s diagnosis, she has been hospitalized numerous times. The Felix’ feel so strongly that this stem cell program could one day help her, and so many others. Their daughter has had many obstacles in her life and she keeps overcoming them. She keeps showing them what a fighter she is, that she wants to be here.  The Felix Family is going to fight right along side her. That is why it is so important to them to raise money for this wonderful cause.

Why are the Spans uniting for the cure?

Zachary Louis Span was born on April 18th, 2009. Parents Kellie and Dave were overjoyed at the birth of their son and had many great things planned for their family’s life. After a short time, they became concerned with his lack of muscle tone and movement of his legs. At his 2 month appointment Zachary’s pediatrician was also concerned, but more so about his tummy breathing, and he was admitted to the hospital. Within a couple of days a preliminary diagnosis of SMA Type I devastated the family (the diagnosis was later confirmed through a genetic test). Determined to make the most of his potentially limited time, the Span family packed up their SUV, took leaves of absences from work, and set out on a journey across the country less than a week after the preliminary diagnosis. Their journey is chronicled as “Zachary’s Great Adventure” on their website – TheSpanFamily.com. Their journey took them over 9,000 miles in 22 states over six weeks. Did we mention they took their dog Lucy as well?

Currently, Zachary is happy and healthy as the Spans plan out his next adventure – see their website for recent updates. The Spans are excited about Dr. Kierstead’s research and believe that it will lead to a cure for SMA.

“Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has.” — Margaret Mead

Why are the McClays uniting for the cure?

The McClay’s seven year old daughter, Kiley, was diagnosed with SMA Type II at 16-months-old. First and foremost, they see Kiley as a beautiful, smart, funny and kind little girl who just started the second grade. Secondly, the McClays know the heartbreak she has endured not ever having stood or crawled and the knowledge she has of being different from most of her peers. They want to join this fight because no one should ever have to hear their 3-year-old child cry because her mom and dad have not “taught” her how to walk.

The McClays have been active in Families of SMA, fundraising and spreading the word about the devastation of SMA for the last six years. They have heard Dr. Keirstead speak at several FSMA conferences and it has given them much hope, not only for themselves, but for all the families that are suffering. They do not want the clinical trials to be postponed or prolonged one day longer because of lack of funding.

They thank everyone who is already involved and encourage those who aren’t to help in any way they can.  “If we all work together we can truly make a difference in the progress to cure SMA.”

“Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has.” — Margaret Mead

Why are TJ and Jaime Gooden uniting for the cure?

Most people fail to grasp exactly what occurs when you hear your child will likely die. It’s the little things that seem to hurt the most. You spend 9 months daydreaming about her life: how you will handle that first boyfriend she brings home or how she will look when she walks across the stage at her graduation. Once you realize your child will die early, there is an empty void left in the place your daydreams used to go. That’s the part that hurts the most. I already miss the future my daughter will never have.

Nora Madison Gooden was born January 23, 2009. At 6-months-old, Nora was diagnosed with SMA Type I. We started a website – GoodenTree.com – for Nora before she was even born. We began documenting everything starting from pregnancy so that one day we could show the entire story to Nora. It’s ironic that Nora may die before she has a chance to read any of it. Nora is an incredibly beautiful red-haired little girl who spends most of her day happy and smiling. It’s often too much to bear to see her so happy and think “how many days does Nora have left?”. Today, minimal movement in her arms is the only thing keeping her from being completely paralyzed. Her nursery, once beautifully decorated, now looks more like a hospital room packed with medical equipment and devices.

Unite For The Cure is the single best opportunity SMA children will have in their lifetime. This is your chance to do something to help Nora and all the other devastated parents of SMA children. We are joining Unite For The Cure along with our family and friends to give our daughter and possibly your next son or daughter a chance for a future.

Why are Jennifer and Tim New uniting for the cure?

Chance New was born on June 25, 2008 in Riverdale, Georgia. As most babies, he was the light of the New’s world. He was the 3rd of three children given to “hold” by GOD by Jennifer and Tim New. At 4-months-old the doctors noticed Chance was unable to roll over, hold his head up, even move his hands off the floor. Although his mental skills were exceeding, his motor skills lacked. This quickly led to physical therapy, feeding therapy, neurologists, and then ultimately geneticists. At 9-months-old, Chance was diagnosed with Spinal Muscular Atrophy Type I. Unsure of any treatment/cure, cause, and what could be done, the News sought help from doctors and mainly the Internet. During a visit to the geneticist they discovered that Chance had already made it past average life expectancy for children with SMA Type I and that there was absolutely nothing they could do. They would not accept that as the answer. They would not sit back and watch their youngest child be taken away! Well, GOD had other plans! Just two weeks after his diagnosis, while visiting with his grandparents, Chance woke up very lethargic and pale. After not eating and a breathing treatment, his symptoms became worse. Jennifer and Chance’s Grandpa rushed him to the emergency room, watching Chance take very little breaths, and he even stopped breathing for a minute in Jennifer’s arms. When they got to the emergency room the doctors and nurses took Chance in and immediately hooked him to a nasal canula and oxygen mask. Chance was then rushed to Children’s Healthcare of Atlanta where the News found an amazing team. Exactly one week after this incident, Chance went home to be with his Heavenly Father, April 17, 2009, at just 9-and-a-half-months-old. Jennifer and Tim have started a foundation in Georgia, Second Chance 4 SMA, in hopes of finding a cure for this disease. They want nothing more than to watch their children grow up and live healthy, “normal” lives. They feel that Unite For The Cure is a wonderful way to not only help funding for Type I research, but will also help bring much needed awareness of SMA. Jennifer and Tim are very pleased to be part of Unite For The Cure and are going to do all they can to make sure that their children and families living with SMA will soon be able to sleep at night knowing that they will not wake up with their child forever “asleep” in their arms!

Why are the Lunts uniting for the cure?

Chad and Cherisse Lunt live in Menlo Park, CA with their three children: Dallin, Jocelyn, and Lucy. Dallin (5) and Jocey (3) are both perfectly healthy and strong while Lucy (10 months) was diagnosed with SMA Type I in March of 2009. The Lunt’s are keenly interested in the research to cure SMA and up to this point have felt like there was little they could do, directly, to help in this great cause. Helping raise awareness, while providing critical funding for this treatment, has brought increased hope and peace to this family.

Like many other families who are experiencing this illness, the Lunt’s had no family history of the illness and, after having two perfectly healthy children, took it for granted that they would have children born into their family without any complications. Although Lucy suffers from this disease, she doesn’t let it affect her disposition. She is constantly smiling and bringing happiness and hope to all who come in contact with her.

The Lunt’s are incredibly hopeful that creating additional awareness of SMA, as well as providing critical funding for this potential cure, will garner the support and funding needed to bring an end to this disease. Unite For The Cure is a critical tool for the Lunt’s as they strive to achieve their goal of increased awareness and funding focused on this specific disease.