Purple Lemonade - A Stand for Sanfilippo Syndrome
If a glass of lemonade could save a child—would you buy one? Of course, you would!
That is what 6 year old Grey, Purple Lemonade A Stand for Courage founder, knew when she decided to host her first lemonade stand. Determined to fund a cure for the rare and terminal disorder her older sister suffers from, she delivered flyers, hung posters, baked goodies and made PURPLE lemonade. She chose purple to represent the courage and strength of the kids who, like her sister, bravely face the declines of Sanfilippo Syndrome each day.
Her stand was a huge success & today Purple Lemonade is a worldwide effort. Families & foundations have joined together to raise funds for breakthrough gene therapy research. Once only a possibility, gene therapy for Sanfilippo Syndrome is NOW on the horizon. A clinical trial is slated for 2015 ...
but it needs funding—it needs YOU to buy a glass of lemonade!
Buy a glass, buy a pitcher, host a stand and take a STAND in helping to find a cure for Sanfilippo Syndrome! Thank you.
About Sanfilippo Syndrome
Sanfilippo Syndrome, or MPS III, is a deadly genetic disease resulting from the body’s inability to produce an enzyme and properly break down certain sugars. The disease causes progressive muscular and cognitive decline. Children slowly lose their ability to walk, to swallow and to communicate. The average lifespan of a child with Sanfilippo is 14 – 20 years. Sanfilippo breaks down into 4 types; A, B, C, and D; each differentiated by the missing enzyme.There is no cure and currently no approved treatments for Sanfilippo Syndrome.
About the Research
All monies raised through Purple Lemonade for Courage will be used to support two critical, groundbreaking research projects.
The Research Institute at Nationwide Children’s Hospital - AAV9 Gene Therapy in Patients with Sanfilippo Type A and B
Investigators: Dr. Kevin Flanigan, Dr. Haiyan Fu, Dr. Douglas McCarty
Research at NCH is poised to begin a phase I/II gene therapy clinical trial for children with types A and B as early as December 2014. An AAV9 vector containing a functioning copy of the gene is injected into the patient intravenously. In animal models, the therapy resulted in widespread expression of the gene, a clearance of the stored material, improved cognitive function and increased lifespan. An investment of approximately $4 million dollars is needed to bring this research to trial.
Manchester University Institute of Human Development - Intracerebral AAV-hHGSNAT Gene Therapy in Patients with Sanfilippo Type C
Investigator: Dr. Brian Bigger
The strategies that are applicable for types A,B&D are less apt to work for MPSIIIC. Therefore, Dr Bigger and his team are developing a gene therapy that delivers the missing enzyme directly into affected cells in the brain using an AAV10 vector introduced directly into the brain. They aim is to prove efficacy in the mouse model by Fall 2014 in preparation for a phase I/II clinical trial. The HANDS consortium (JJB, JLK, Sanfilippo Sud and Sanfilippo Barcelona) have already funded a 1/2 a million dollars into the preclinical research. The large animal longevity studies will begin in Fall 2014 with an estimated budget of $2.5 million and a Phase I/II trial would then start in 2016, with an estimated to cost of $4 million.