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.

Way to go Calafiore family!!! Thank you so much for being part of UFTC.

My husband works for UPS and he gave out letters to his co-workers asking for donations for UFTC.  One of his co-workers is married to a realtor (Tammy Henderson) at Century 21 Northwest Realty in Glendale, AZ.  The woman who owns that Century 21 (Rodica Bartels) also happens to own the house that we rent.  Tammy and her husband donated $100 to the UFTC already, but Tammy also gave the letter to Rodica.  In the very wonderful season of giving, the entire office decided instead of buying gifts for each other that they would chip in for Unite For The Cure. They collected an amazing $1350 for UFTC!!  I was in shock and humbled. My husband and I are blown away by their generosity and so very appreciative.

This is truly wonderful. Thank you Felix family for sharing this act of kindness and thank you Rodica Bartels and Tammy Henderson for organizing such a thoughtful Christmas gift. Funding research to change the future of SMA is a true gift indeed.

Below is an article for Parachutist magazine by Jennifer Domenico about the skydiving fundraiser:

On November 28, 2009, the Perris Valley skydiving community, in association with numerous sponsors from the skydiving industry, united together to raise money to fund a cure for Spinal Muscular Atrophy (SMA).  SMA is a terminal motor neuron disease and is currently labeled the number one genetic killer of toddlers worldwide.  The fundraising event was coordinated by Dave and Kellie Span, longtime family of Perris Valley Skydiving and Square One Parachute Sales, whose son Zachary was unfortunately diagnosed with this disease in June at two months old. At the time of the fundraiser, Zachary was hospitalized and fighting for his life, up against a disease for which there is currently no cure or treatment.

Through a comprehensive fundraising effort which included a silent auction, Chinese raffle, classic raffle, bake sale, and poker tournament, Perris Valley skydivers, various commercial sponsors and friends of the community raised in excess of $10,000.  Mirage Systems donated a new container for the centerpiece raffle which was won by skydiver Shawn Foley.  Other primary sponsors included Perris Valley Skydiving, Icarus Canopies, United Parachute Technologies, Square One Parachute Sales, Aerodyne Research, and Perris Fury.  Secondary gifts and prizes were donated by Skydive Hawaii, Bonehead Composites, Sky Systems, Bev Suit, Alti-2, Gatorz Sunglasses, and numerous other individuals and businesses whose generosity made this event such a huge success.  Thanks to everyone who gathered on that cold, rainy November day in Perris, a significant step was made to give little Zachary and his family hope that there is a fighting chance for other SMA children in generations to come.

The support structure within the skydiving community, and particularly Perris Valley Skydiving, is truly remarkable.  In times of need it is often that our family is called upon to unite for emotional support, financial support, and this time, a cure.  Having raised a significant sum of money for Families of SMA, once again, we can say we were there for our skydiving brothers and sisters; once again we can say we made a difference in the lives of others.

Unfortunately, on December 12th, only 2 weeks after the fundraiser, Zachary Span lost his battle with SMA and was taken from this world all too soon.  But with the assistance of his loving family and such a supportive community, his legacy leaves behind a contribution to the fight against this disease to better the lives of other SMA families like the Spans.  If you would like to make a contribution to this effort, donations can be made in the name of Zachary Span at FSMA.org.

Zachary Span, you will be missed…

In Jaci Felix’s words:

This donation is in memory of Alexa’s great-grandmother, Edna Dahlstrom (1/18/1920-12/1/2009). Edna passed away while Alexa was in the PICU a few weeks ago. My father’s (Roger Dahlstrom) co-workers from Banner Health all chipped in and wanted the money to be donated in memory of Edna to benefit SMA. Alexa never got to meet her great-grandmother due to both of them having health complications. Alexa has another Angel watching over her and all of her SMA friends. May Edna Rest In Peace.

Here is what Barbara Zahn had to say about their great event:

Lucy’s grandparents Jim and Jeanne Zahn collaborated with her great-uncle Dave to start a pizza sale to raise funds for Lucy and 5-year-old Lily, who has neuroblastoma, an aggressive brain cancer. The youth bowling league of Eldorado got involved, selling over 1,000 pizzas as a group. The total amount of pizzas sold by the end of the fundraiser was 4,500! They had to order 1,000 pounds of cheese alone! Over 100 people showed up to help make and deliver the pizzas, which were put together in Charlie Becker’s shop. Thank you to everyone who helped get the word out about Brenner’s delicious pizzas and to everyone who volunteered their time to make and distribute them. Another thank you to those who decided not to buy pizzas but offered generous donations anyway. We raised over $10,000 to go towards treatment for Lily and finding a treatment for Lucy! We are happy to be able to offer $6,000 to go to Unite for the Cure, and we still have fundraisers in the works! Onwards to the CURE!!

Way to go Zahn family!!! This is such an uplifting story to see your community joining you in fighting for a cure!!!

Click here or on the image below to read the full press release…

Beauty Temptations hosted the raffle drawing to benefit Unite for the Cure! The raffle raised $933 through generous donations of family, friends, and community members. I was told that one gentleman drove 45 minutes to buy raffle tickets! Katie let Nicholas pick a couple of winners…Then her son, Grant, picked one…and her daughter, Gwendolyn, picked a couple, too…All the excitement tuckered Nicholas out so he cuddled with Grant on the couch while Katie called the winners.

Thank you to everyone who participated in this fundraiser and a special thanks to Katie Lewis…you are an amazing friend! We love you so much and appreciate everything you’ve done for us!

And then for their final fundraiser, the Gustafson’s gathered friends and family for a fun night of poker.

We raised $600 total and couldn’t be happier! It was a fun night for everyone. Thank you to all of the people who came out to support this worthy cause and a special thanks to my parents who let us host the event at their home.