On April 22, a conference call was held with many families affected by MPS II along with two special guests, Dr. Haiyan Fu and Dr. Doug McCarty from Nationwide Children’s Hospital (NCH) in Columbus, OH. These two doctors are working together along with NCH on a cure for MPS II, Gene Therapy. It truly was a pleasure to hear the voices of the SuperHeroes that are on a mission to save many children’s lives across the country!
The purpose for this conference call was to accomplish several aspects concerning Gene Therapy, along with more detailed information as to where we currently are in the process for the cure and the clinical trials that are soon to come. As they did go into detail to account for the total $2.5m for the cure, the part that stuck out the most is that we need $450k raised by November 2017. Then an additional $450k by February 2018. The total sum of roughly $900k will cover expenses such as acquiring the facility where the clinical trial will be held, along with the building of the vectors that will be used to introduce the cure our little SuperHeroes.
As the facility is currently the main topic of conversation, we learned it’s imperative to attain the $450k by November in order to secure the facility. If we fail to raise the funds, we may lose our spot in line to use the facility and will have to wait a longer period of time to save our little SuperHeroes.
The remaining balance of the $2.5m will be used for medical costs, room and boarding costs, and miscellaneous costs for the patients who will be joining the clinical trial.
It will take roughly 6-9 months for the completion of the vectors that will undergo production shortly after November 2017. If all deadlines are met, we hope to undergo clinical trials by end of Summer 2018.
Melissa Hogan, Founder of Saving Case and Friends and producer of the very touching ProjectAlive video, also gave us a moment of her time to speak with the families on the line. As a Dragon Fighter in the world of MPS, she has been fighting for some years now for her son Case. She has become really involved in our MPS community and has accomplished many objectives including raising huge funds for the research team at NCH. Melissa mentioned a quick synopsis of a plan that can potentially cover the entire $450k by November and the additional $450k by February. This year marks the 100th year of the first diagnosis of MPS II and through 100 families nationwide, we hope to raise all the money in efforts to make this the last year for a disease with no cure; The 100 Families Campaign, is soon to come.
We are “one team, one goal”!
Dr. Fu closed out the conference call with beautiful words. As the MPS II families collaborate in efforts to acquire the funds for our cure quicker, it was duly noted of how proud she was of all us parents regarding the fight to save our children’s’ lives. Along with all the parents, both Doctors have no doubt that, with your help, we can save the lives of many children across the nation, including ours!