Congratulations to California Stem Cell, an Irvine, California based privately held company developing the stem cell-derived motor neuron replacement for Dr. Keirstead’s groundbreaking SMA stem cell program, on closing this critical Series B round of funding. Another hurdle overcome and another step closer to getting this promising research to human clinical trials as safely and efficiently as possible.
Click here or on the image below to read the full press release…
Another important milestone was achieved by the promising Spinal Muscular Atrophy (SMA) stem cell program today: California Stem Cell (CSC) received orphan drug designation from the FDA to its stem cell-derived motor neuron product aimed at treating SMA. This provides several incentives to companies in the private sector, such as CSC, developing novel drugs or biologics to treat diseases with relatively small market potential. This is also extremely important as reaching this milestone is a positive step forward for the SMA community and shows significant progress being made to develop an effective treatment for our children.
Click here or on the image below to read the full press release…
Congratulations to Dr. Hans Keirstead and his team for being featured in Esquire as one of 2009′s “Six Radical Projects That Will Change Science Forever“. There is no way for you to begin to understand how much pure hope you have given this vibrant SMA community and we are confident that your research will have dramatic impacts on SMA and many other life-altering conditions in the future. Keep up the inspiring work!
Click on the image below or click here to read the article on Esquire.com.
Have you signed the Stem Cell Charter? Watch the video below, THINK!, then go to StemCellCharter.org and click on Charter to sign on in support of the possibilities this important research brings.
Check out the great article in The Chronicle, “A Joyful Life, a Race for the Cure”, about the McClay family of Washington and their Unite For The Cure event. Click here or on the image below to read the article.
Significant Progress made Towards Moving Novel Therapy into Human Clinical Trials.
California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that they have completed a formal pre-Investigational New Drug (Pre-IND) meeting with the Food and Drug Administration (FDA) for guidance on the clinical and regulatory pathway and requirements for submission of an IND to initiate human trials for a stem cell-derived motor neuron replacement therapy for Spinal Muscular Atrophy (SMA) Type I.
CSC, a leading stem cell therapeutics company, has developed a stem cell-derived motor neuron replacement product, for the treatment of SMA Type I. Pre-clinical studies, completed in collaboration with professor Hans Keirstead of the University of California, Irvine, have shown clinical proof of concept through the demonstration of functional benefit in animal models treated with CSC’s motor neuron replacement product, MotorGraftTM.
MotorGraftTM has the potential to provide benefit for SMA by two mechanisms: 1) direct replacement of the motor neurons lost during the disease course, resulting in new muscle innervation, and 2) providing a nursing support function to remaining motor neurons.
“We are greatly encouraged by the feedback we received from the FDA,” said CSC COO Chris Airriess. “It will greatly assist us in preparation of our final IND application. Completion of this key milestone is an important step towards moving our SMA program to the clinic.
“We are very pleased with the outcome of the Pre-IND interaction with the FDA,” said Kenneth Hobby, President FSMA. “FSMA has invested almost $2 Million over the last 8 years to develop this motor neuron replacement therapy for SMA. This meeting was a significant accomplishment towards the filing of the IND and ultimately bringing this therapy to patients.”
Click here to read the entire press release on the FSMA website…
Congratulations Dr. Hans Keirstead, Families of SMA, and California Stem Cell on this important and momentous milestone! We are all behind you and are so encouraged that this groundbreaking and promising research is now one step closer to human clinical trials.
What a touching segment on CBS3 Philadelphia featuring the Schmids, Uniter family from Pennsylvania. The Schmids are hosting the First Annual Zane’s Walk/Run on September 20, 2009.
Click here to see the news segment…
Click here or on the article below to read the article…
Way to go Schmid family!!!
Check out the Marshalls, Uniter family of California. Their event that is to take place this weekend, Micah’s March 2009, was featured on the front page of the Ojai Valley News this week…
Click on the article below or here to read the story…
Way to go Lovelaces, Uniter Family of Texas! What a great article and news segment!
KLTV-7 (ABC: Tyler, TX): The most deadly genetic killer of infants
Last week, Families of SMA congratulated the Gaynor Family of New York on their amazing fundraising and SMA awareness success. Families of SMA and the Gaynors have partnered up to raise funds for the final stages needed to ask the FDA for approval to begin clinical trials with the SMA stem cell program and to try and accelerate the program. The research team is now preparing for the final steps before meeting with the FDA to request approval to move forward toward the critical clinical trials in patients. The Gaynors, through Sophia’s Cure, will be attempting to raise a total of $200,000 specifically for the SMA Motor Neuron Replacement Program. Families of SMA will then attempt to meet this sum by raising additional donations for the program. Our efforts through Unite For The Cure will be going to help Families of SMA raise these additional funds.
Click here or on the article below to read more about this on the Families of SMA website…
