People say it comes in three’s. I got my three then some this week. The good things that came to me and my community were not a surprise, we have been working tirelessly towards these goals for years. It's just a bit more impactful when it all comes together at once! Clears my plate a bit for all the other projects we have in the works.
JJB and our consortium HANDS have been funding our gene therapy program for 7 years now. About half way through I realized that funding the science wasn’t going to be enough. So with the urging of Sean Ekins I founded Phoenix Nest. Sean partnered with our scientists to write NIH grants we learned quick that the NIH was not keen to see funding sent outside the US. Around that time, a US based scientists Patricia Dickson offered her services for type D. She and Sean wrote a grant and we won, then we won a second and a third. You’d have to be in this space to really have a handle on how hard these grants are to win. The science has to be top notch and the competition is fierce. Now picture a mom with no medical background winning these grants.
The first grant we won, I was grilled hard by my grant manager, she couldn’t grasp the idea that a non-academic person would be sitting at her desk at home, managing NIH grants. I hired a bookkeeper and we made a budget and we follow the budget. I use a payroll company and they cut the checks and pay the scientist's tax’s. I mostly just reconcile invoices, our scientists buy crazy expensive equipment. Sean manages the scientists and keeps them on point. Easy enough right! There is one grant that we put in and then resubmitted 6 more times. lol Sean does not give up. Every time the reviewers came back with suggestions he addressed it and adjusted the grant. This went on for years. I really really really wanted this grant. The other three grants are for subtypes B and D. Jonah has type C. I work for free, managing over two million in grants that won’t benefit Jonah.
Good news
#1, On Monday I got the notice of award, we finally won that damn grant. $225,000, the funding will go to chemistry for our chaperone molecule. Big sigh of relief here! I’m ecstatic. We have won 2.5 million in grants, which doesn't mean much to me, I just manage it. It is funding that will save lives, it pays for the research. I admit I bought a filing cabinet for all the invoices and paperwork.
Managing grants are easy compared to licensing the programs that JJB & HANDS funded through the non-profit donations that many of you contributed to and the research that was funded by the grants that Phoenix Nest wins… Now this is hard! I will refrain from lamenting about the crap I had to do to get here. I learned a lot and this experience will help me in the future. If I keep telling myself that, someday I might believe it.
#2 We finally agreed to and signed off on the licensing terms for the gene therapy research that we have been funding for 7 years. Amen to that! Can I get a little applause over here? You might be asking what does this mean… Without divulging PN’s business plan, it means that we have the control now. I have the legal right to see this thing all the way through clinical trial and onto commercialization. Nobody can take that away from me and my families. Do you want to cry a little bit right now? I do! Dr. Cao is an incredibly humble man with a super cute dog and HUGE heart. https://youtu.be/RL24KsRyjPc
#3 Our NHS (Natural History Study) has received it’s Notification of Approval by the NIH. This is another program that we have been focused on for 7 long years. Why did it take so long to get to this point? FUNDING, politics, red-tape, IRB approval, protocols, experts. Funding is still a huge issue and I loose a bit of my brain when I think about how much this is going to cost. Again… I will refrain from thinking to hard on this. The acid is welling up in my throat.
The NHS is designed to follow the course of Sanfilippo Syndrome, there is not a longitudinal study done for MPS III C and D. We will bring in children across the US to Montefiore Childrens Hospital 1-2 times a year for 5 years. They will stay for two days and have numerous tests performed. 1 day will be dedicated to cognitive and Psych evaluations. The second day will be biological, sedated MRI, where we will scan every inch of the body, while under sedation: fluids will be taken, X-rays given, even dental exams. We need this study to further research and to better understand the disease. Jonah will most likely be the first to enter the study. A huge part of me does not want to know the results of the testing. I don’t want to know how much the disease has attacked Jonah’s brain. I can see that Jonah’s spine is curving, I assume that he has scoliosis. His belly is bloated, is there something going on with his liver? Deep breath… Knowledge is power knowledge is power.
JJBs partners HANDS (Sanfilippo Sud, Sanfilippo Barcelona, Sanfilippo Portugal, Sanfilippo Brasil, JLK, Levi’s Live Love and Laughter, Le Combat Hatiem-Contra-Sanfilippo) and our sister foundations: Sanfilippo Children's Foundation, Cure Sanfilippo Foundation and The Sanfilippo Children’s Research Foundation, whom have also contributed significantly to the HANDS research program’s. These mom and pop foundations help keep our research afloat. By NO means has JJB funded all this research on our own. These associations have carried us. It takes a village.
4-5-6-7....
Other cool stuff: Jonah started 4th grade, I really like his new teacher, Jonah is HAPPY, he’s still learning and he has not cognitively regressed (knock on wood).
Yesterday I received an uplifting text from Patti about our D program, I can’t share it with you, but just know that it was the cherry on my cake yesterday. :)
Alexey is in France for an LSD conference he decided that since he was super close to a mountain that he’s always wanted to climb that he’d climb it in our honor and ask his friends to support him. https://www.crowdrise.com/o/en/team/jilljonahsjustbegunorg/jonahsjustbegunfound
At long last the gene therapy paper that Brian and his post-doc Claire O’leary have been working on, will be published. I’m excited that everyone will see how hard we have been working.
Sean and I were the topic of a recent article in Nature’s Lab Animal journal ( a mention is this journal is coveted by academics.) Sadly you have to be a subscriber to read the Entire article and I promised not to socially repost the entire article. http://www.labanimal.com/laban/journal/v46/n9/pdf/laban.1335.pdf
On Tuesday the 19th, JJB will be issuing a press release about an awareness campaign, designed to help us reach the masses to the signs of Sanfilippo. I have two main goals: earlier diagnosis and correctly diagnosing the Sanfilippo children that have been living under the wrong diagnosis. Jeremy’s college roommate, Steve whom now works in advertising came up with “Mission Hide and Help”. Steve had the help of his work partner and convinced his talented colleagues to pitch in. I absolutely can not wait to share the campaign with you. We will need your involvement to make MHH a HUGE success. Wait for the Announcement, you only have to wait a few days.
Now it’s time for JJB to start earning our keep! Announcing “Monster Mash”, a kid and adult friendly fundraiser October 28th from 2-5:00pm, Brooklyn.
Get your tickets ASAP!
If you can’t make the event, there is still time to help JJB fill our raffle baskets. Purchase JJB something on our Amazon Smile wish list. https://smile.amazon.com/registry/wishlist/3MR2XEXXAI90/ref=cm_sw_r_cp_ep_ws_CvXizbYWC03H6
Did you ever imagine that we’d get this far? I tell people our story and they just sit and stare. I think they think that I’m lying or over exaggerating. I can’t blame them, If I were in your shoes I’d have a very hard time believing my story. How do I prove that I’m not full of it? Brian’s paper will help the world see. People can google me and read articles about our work.
I mention this because I still need a massive amount of help from funding to volunteers. If it wasn’t for my pro-bono lawyers, PR friends, celebs and the academics: Sean, Patti, Brian, Alexey, Elsa, Joel, Geoffrey on and on I would have NEVER made it this far. Next stop clinic trial. This is what we have all been working so hard for. You can help, every dollar helps, every volunteer is greatly appreciated.
For instance I need a volunteer to play Frankenstein at Monster Mash. You'll be required to learn this dance. https://youtu.be/NZ5OJQj7y8M (Free drinks included)
