Follow by Email

Monday, September 18, 2017

BEST WEEK EVER!

Best week ever.  Not to be greedy but I hope this coming week tops last week.

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)

Tuesday, August 8, 2017

Bad energy

We’re between trips. Just home from the National MPS society family conference in Minneapolis. Before that Jonah and I were in Canada at the Canadian MPS society family conference.

I have a love hate relationship with the family conferences. It was several years into our diagnosis before I attended one of the meetings. I had been warned that it would be very difficult to see the later stages of Sanfilippo. Jonah was so young and in great health, I sheltered myself by not going. The first conference I went alone and attended only a couple of breakout sessions, just the ones that discussed the research. I saw in real life what was to come for Jonah and I wasn’t courageous enough to embrace the Sanfilippo children. I hid in my room and worked until it was time to go home. The second conference I went to was a bit easier. This year I had a blast at the National MPS Society meeting. I can embrace all the children now.




Go figure…  It’s the newly diagnosed parents that I find harder to look at. Seven years I have been in this space, it feels like such a long time. The first three years, I was out of my mind, inconsolable. A seasoned parent’s advice only pissed me off. I didn’t want their words of wisdom I wanted them to help me fight our children's fate.

People grieve in many different ways. You never know how one parent is going to respond to the diagnosis.  I joined the Mothers breakout session, we went around the room and introduced ourselves. There must have been 50 of us from all over the US in the room. I noticed that most of the newly dx’d parents were sitting side by side. They were the last to introduce themselves. They had to go all the way around the room listening to the seasoned mom’s lightheartedly introduce themselves. After the session I found myself standing next to one of the new mom’s, her face was long and contorted in pain. I asked her if I could give her a hug, she looked at me, lips quivering and embraced me. But I didn’t know what to say to her, to ease the pain. There is nothing that can do that. The only thing that came to mind was: “Have you joined our patient registry yet?”

I didn’t say it. Instead I found myself saying: “It get’s easier”.

I went on to stumble through the conversation shoving my foot in my mouth further and further.
Later that evening, mingling at the bar, with parents and kids. I tried to engage a typical child who was pulling on her dad . Her dad was trying to have a meaningful conversation with another newly dx’d family. I bribed her with a cookie to come sit and read with Jonah. I didn’t hear what she said to Jonah, but the father was scolding her. He introduced himself and told me that his daughter was type A. I told him that Jonah was type C. He was taken aback and commented to me that he looked SO normal. His daughter thought otherwise and her dad turned to her and said: “Look Jonah is like your sister, he has special needs too and you’re not being nice to him.”

He said it right in front of Jonah. Oblivious, Jonah sat grinning at the little girl, anxious for her to come sit with him and read books together. I have this rule, nobody tells Jonah that he’s different. Here was another Sanfilippo parent having to tell his typical child to be nice to Jonah, because he was different. My feelings were hurt. I quickly brushed it off, remembering that I have said very similar comments to Jonah in front of other Sanfilippo children and their parents.  I sat down with Jonah and the little girl and read to both of them.

I was asked to speak on a panel with Mark Dant and Elizabeth Linton, at the Canadian MPS family meeting. Alexey, offered to have us stay at his house and his wife offered to help me out with Jonah.  Jonah is on good terms with Alexey and his wife, I felt like it would be a good trip and it was. The conference was fun, the Canadians are the NICEST people in the world. Why is that?


 Alexey gave Jonah and I a tour of the brand new medical building on campus. His new lab is state-of the art, beautiful and spacious. I didn’t realize that Alexey’s students would be at work in the lab. It felt like a Saturday but it was still before 5:00 on a Friday. I first saw Carla, the student that Raquel recruited from Portugal. As we chatted all the other students started coming out from behind their hoods and desks. They were wide-eyed looking at Jonah, nervous about interacting but wanting to get a good glimpse at him. I wasn’t prepared to see all of them and have them see Jonah. How do you respond to the team of young people who are working on a treatment to save your child and all of your friends children.  Alexey had a BBQ for all of us and we got another chance to get to know each other. A brilliant, passionate group of young scientists. Thinking about them now and having a face to go with names is comforting.


The week after the Canadian conference and the week before the National MPS society conference we attended Danielle and Kevin’s BBQ in L.I., NY. That was a lot of fun too, a smaller intimate group, I like the off shoot family get togethers, where you can just chill out with friends. Most of the MPS families at Daniel’s had already met Jonah in person. They are accustomed to Jonah and not shocked by his skills.

Two more reasons why I never went to the MPS society meetings until recently. One, I didn’t want Jonah to make the connection that he was sick. Secondly, I didn’t want the other families to be resentful that Jonah is doing so well. I know that there is some underlying resentment for many families. They can’t help it, I would be hurt too, to see my child wheelchair bound and unable to talk or do anything unassisted. While a boy the same age is running around playing tag or sitting down reading a book while waiting for dinner.  That would be hard to bare, it actually makes me feel ashamed that Jonah is doing so well. I feel like an outsider at the big conferences, even though the disease ends the same way,  for Jonah it’s slower to progress. During the slower progression, Jeremy and I get to see more of who Jonah would be if he didn’t have this disease. Parents I don’t take this for granted, I appreciate how ‘lucky’ I am. I saw the sting in the eyes of parents, when they saw and talked to Jonah. I know that you know that we can’t help it. Do people realize that I’m jealous that they have other healthy children to live for? I think of the many families that are watching ALL their children dying from Sanfilippo. No matter what, there is always somebody that has it worse then you.


At the moment I’m white knuckled fighting every urge to not jump on social media and read for myself what people are saying. I had hoped that I could sit here and reflect on the family meetings in a positive and productive light. Alas there is major strife going on between our community over an overt action taken by a parent against pharma that has gone viral in our space. It’s hard to ignore. On one hand I don’t even want to engage in the conversation on the other I feel that my hand is being forced to comment.




Back story for the layman, during the last 12 mo our MPSIII community has seen three treatments for MPS III dropped, 1 for  type A and 2 for type B (MPSIII C and D, don’t yet have any trials). Shire and Alexion being the most controversy. UniQure never got off the ground before they pulled the MPS III B program from their pipeline, there is still hope for this program, they are looking for  different investors. Shire had an ERT trial going on for MPSIII A, they abruptly stopped the trial and called the families in to have the ports removed from their children. Alexion announced this past winter that they were dropping their III B program but would finish out the trial for the families that had committed their time to the trial. Last month they went against their word and pulled the plug. Both companies stated that the results from the study were not meeting their endpoints. The families argue that they are happy with the changes that they are seeing in their children.  I have sat in on conversations and been a part of privileged meetings and my take is that the closing of the trials are for financial reasons. That’s my opinion, with that said I don’t know if the drug was really working. What is the definition of ‘working’ for pharma and the parents are different. The families are looking for quality of life, pharma is looking for a “cure”. I have heard from several families that they believed that the drug was working. But I have not personally talked to every family, the thing is not every patient will respond to a drug, for numerous reasons, this is one of the many reasons why we have trials. For ultra-rare diseases we have only a handful of patients to choose from for a trial, which makes trials less expensive for us to sponsor but also makes it harder to prove to the FDA that the drug is working, because we don’t have enough patients in the trial. The PI’s overseeing the Shire trial have stated that they don’t believe that Shire gave the trial enough time to come to a conclusive decision. I don’t know what the PI’s of the Alexion trial are saying.

I have already blogged about the trial closures, I did not start out writing thinking I’d blog about it again today. This is what our fellow mom did at the conference. She wore this shirt then (among other things) posed herself in front of the Shire table at the MPS society meeting. I don’t know this mom personally and I can’t condone nor applaud her actions. I can sympathize, when Jonah was dx’d I went out of my mind and for those of you that know me well would not be surprised if I did something like this. With that said I would have sought counsel before I made such a bold statement. I reckon my council would have advised me otherwise. Sean and Mari, I have considered your council and you’re right I I shouldn’t make the analogy of beating a dead horse with a stick reference in my next presentation.

For the record I DON’T like the company Shire, I fully agree that upper management is disinclined to listen to what the patients want. I have personally been insulted to my face by upper management at Shire. But I have also gotten to know a lot of great people employed by Shire.

I DO stand by the National MPS society. I know that the society is on our side and not in the pocket of big pharma. Families, you would be remiss to think otherwise. I know for a fact that Mark Dant has stood up to pharma and fought for these trials time and time again. I have witnessed these calls and meetings first hand. I’m hurt and disappointed in my community. We must work together to get things done.

Pointing the finger and blaming each other is the best way to NOT get what you want.
My agenda was to discuss the relevance of our patient registry ConnectMPS.org. I feel defeated today and I don’t have the energy to try and convince people why it’s so important to work together for our common goal. Treatment. I feel like I’m beating a dead horse with a stick…. oops, it just slipped out.

For those of you that have registered, but have not finished the diagnosis survey please do so by September first and you will be entered into a drawing to win a $1,000 Visa gift card. Log in and win.

I also wanted to thank all those that supported Jonah’s B.day wish. THANK YOU to all those that donated and I hope your kids have a fantastic new school year.

Sincerely, JIll

















Sunday, July 16, 2017

Bring it on!


Bring it on... I'm waiting.

I have a wall of inspiration. It’s aggressive, powerful and motivating. It used to get me moving in the morning. Today I stood and stared at my to-do list; scribbled on the dry erase board.  Bored of it all. I just want my ultimate desire to come to fruition.

I’m waiting.  When you have something that is so heavily weighing on you, It’s hard to do anything else but wait. What do you do when you’re waiting? I feel like I'm sitting at the DMV office.  I can't wait to get the fuck out of here, but I need my license.

What do you do when you're waiting? I have already binge watched Breaking Bad and just finished King of Thrones. After several recommendations I tried watching Handmaids Tale, I hated the book.  I found the show to be even more annoying. Once an avid reader, I can’t even fathom picking up a book now. I’m getting my Phoenix tattoo colored in tonight, that’s another three hours killed. I got a tattoo of a chainsaw on my forearm last month. It makes me smile. For downtime these days, I chase Pokemon at the cemetery. 

I have been working... but it all feels like busy work.  I have a dozen or two irons in a ROARING fire, many others have been stoking it and I feel the heat.  It's good that  people keep probing me on and in return I probe the powers that be. I explain: "It's not just me!" 

I attempt to get excited about all the ‘small’ accomplishments that we have won.
We're here now, our accomplishments have added up.  A pharma employee commented on my last blog he said: "Wow! Congrats Jill! What amazing efforts - large companies would be hard-pressed to accomplish a fraction of this. You rock"

Once our drug goes to trial it will take a few years from trial-approval-commercialization. They call this 'bench to bedside' onto jumping the 'valley of death.' Sometimes your drug gets picked up immediately after FDA approval, other times it just sits there. No takers to bring your drug to commercialization, this is the valley of death.

Today I cleaned out all three of our AC units.... DISGUSTING! Meanwhile, Sean wrote me an abstract for a poster presentation for a conference in Brazil... Hey, at least I made a couple of edits and submitted it. I also had a couple of conference calls today and I followed up with my action items. I keep plugging my way along. Waiting for something awesome to happen. The enthusiasm for my B.day wish was a turn on and a break from the norm. Thank you all for donating!  Monster Mash will for sure be a blast and should turn my focus to this gig as it is uplifting. 

We just got home from the west coast visiting family.  The only overlap that Jeremy and Jonah both had off. I offered to go anywhere with them for vacation, Jeremy just stared at me. He responded calmly: “We’re not spending any money on an exotic vacation, because I know you won’t be able to stop yourself from working.”

This summer Jonah and I will be landing, taxing home, repacking and heading out again. 

I did well in Oregon, just a few breaks here and there on the computer and phone. We hiked the pacific trail, went to Seattle to visit the cousins, made smores, celebrated July 4th  with friends from NY doing the root 66 thing. Jeremy’s major goal was to canoe, my aunt granted that wish and brought down her canoe…  we all paddled up and down our river. Jonah did an awesome job and fully enjoyed himself. Jeremy found himself using some major clich├ęs: “Jonah, don’t rock the boat.” “Jonah don’t drop the paddle, we’ll be up shit creek, without a paddle if you do.”

Jennifer and I leisurely paddled alongside them on our floaties. Scaring the schools of fish, freaking out the crawdads and annoying the cranes.  A red tail hawk made sure we knew he was there. 

 I’m back in Brooklyn working, Jeremy and Jonah are now upstate at the-in-laws. Elementary starts back up next week.  Jonah and I will be working the Canadian MPS family conference next week. We have a booth representing the ConnectMPS.org patient registry, I have a panel talk along with the esteemed Mark Dant and Elisabeth Linton. These are the folks that inspired Jeremy and I to follow the same path.

It’s an immense honor to be sitting side by side with my heroes and I’m really looking forward to it.  The Linton foundation SCRF, http://www.alifeforelisa.org/ is one of the three founding foundations for MPSIII.  SCRF gave funding to Alexey Pshezhetsky, before Jonah was dx’d wich allowed Alexey’s lab to find the affected spot on chromosome 8 that causes type C. We are forever in their debt. Sadly Elisa (pronounced Alyssa) passed away last November. The Abeona and BioMarin trials for MPSIII B had just started, Elisa was not eligible for the clinical trials. Trials that were inspired by Elisa and in part funded by SCRF foundation.  Mark Dant's son Ryan lived to see his parents crusade for a treatment come to fruition. Ryan’s treatment is not perfect and we still work for the silver bullet. In the mean time Ryan has graduated from college! Please watch this powerful episode and know that this could be Jonah’s future too. http://www.cnn.com/2017/05/12/health/ryan-dant-college-graduation-mps-rare-disease-profile/index.html 

It’s hard for me to call this a miracle… Because I know how hard Ryan’s parents worked to make this treatment a reality.  The miracle is that Ryan lived to see it.  Another powerful message from my friends the VanHoutan’s, they lost one child to a similar syndrome as Jonah, Batten disease. The VanHoutan family helped make treatment happen for those that follow in Noah and Layne’s footsteps. http://www.chicagotribune.com/news/ct-batten-disease-drug-met-20170707-story.html

What is in Jonah’s future?  I can’t tolerate thinking about it. I’d rather sit through three hours of a tattoo, rather then mentally take myself down that road. I had an emergency root canal this past month that hurt and after 5 visits it was finally resolved yesterday. I’d take a root canal and a tattoo everyday for the rest of my life over losing Jonah. Picture that. I’d have to tattoo over tattoos and replace crowns for the fun of it. So maybe that’s not a good comparison. But that’s the patients and pain that I would endure to see a treatment through to the end to save Jonah and or the next generation.

July, 30th is Jonah’s 9 Birthday, while in Oregon Grandma Wood had a surprise B.day party for him . We were having dinner out on the patio, Grandma and Aunt Jennifer whipped out a banquet table, dressed it and overloaded it with presents. Jonah watched everything come out. He backed up into my lap and whispered: “Mom, who’s B.day is it?”

He sat on my lap spell bound not moving an inch, as if he flinched the mirage would disappear. Once everything was in place Jonah moved into position with no hesitation or prodding. Loving every present that he opened for a moment.

He has asked several times since then if he’s 9 now?  He’ll be getting another party at Jeremy’s parents this week. I still need to plan his party with friends in Brooklyn. I wonder if he'd notice if he didn't get a friend B.day party on his B.day? I hope next year he automatically anticipates three B.days.

I applied for a new school for Jonah. Have no idea if we will get in. Back to School will be here in a flash. Fingers crossed he gets in.
Public school vs Private school

WE NEED YOUR HELP!

It's super easy, don't be lazy or tell yourself: "I won't be able to make a difference."

Listen to me and watch Jonah thrive, because people helped.

Everyone and their dog shops at Macy’s.  Even more so since Trump black listed them for not carrying Trump products, lol.  Politics aside.

Back-to-school will be here before you know it. Please act now to help JJB earn cash for our clinical trial.  For every $5 donation people will earn a savings pass up to 25% off at Macy’s. I take that back, NYC parents make it a point to not go to Macy’s, it’s an insane tourist trap in the heart of the city. For my NY friends please shop Macy’s online.

I have two asks, take your pick or pick both and earn extra credit.
1.)  Create a profile and join our team, share through social media to All your friends emploring them to donate at minimum five dollars and earn a 25% off Macy’s shopping pass.
2.)   Or you can just donate to our team
3.)  Extra Credit- Do both and help us win up to $100,000 in cash prizes.

·      Create your profile
·      SHARE SHARE SHARE. Tell everyone about JJB’s plight. Tell them that orphan bats have raised more $$ then terminal children.

Thursday, June 1, 2017

It's My Bday and I'll celebrate if I want to


Buy me something so I can re-gift it. http://bit.ly/JJBWishlist
I’m actually caught up! Knock on wood, I haven’t had a breather like this since I can remember, albeit it will only last for a couple of days. But for these few days I’m NOT going to create any extra work for myself. Tomorrow I’m going to go to Yoga class and then binge watch Game of Thrones. Today I walked through Greenwood cemetery, Brooklyn’s best kept secret. I met with a tattoo artist, the date is set for my new tattoo. Had lunch with a dear friend then bought some new sneakers for my hikes through Greenwood. Furthermore, I didn’t call anyone back or respond to any emails. I’m so proud of myself!
Red Tail Hawk at Greenwood Cemetery, just chilling.

Jonah is doing really well, he’s super funny and charming, even when he doesn’t mean to be, which is most of the time. I’m not living in denial, my entire week is consumed with the logistics of how we’ll bring a treatment to trial and then to market. I know exactly what will happen if we don’t. It’s just not worth obsessing over when he’s going to die or when will the treatment come. Live Life!

Happy Mother's Day, Anniversary and MPS awareness day to me.


Recently we have had some pretty big negotiations and projects come to fruition. Anticlimactic to say the least. It took over two years to sort out the terms and conditions on our exclusive licensing rights for our ERT type D program with LaBiomed. There was a lot of arguing involved, more back and forth then you could ever imagine. The fact that I have to go through these legal negotiations in the first place is a put off.  Phoenix Nest, our virtual biotech funded all the research to date. JJB won the creation of the knock out MPSIII D mouse model years ago, which put the science in motion. There are 2 people, twins, in the United States. Yes, I'm sure more children (young adults) will come out of the woodwork when we go to trial. Can you imagine trying to convince pharma to help you commercialize a treatment for two patients, in the US, possibly a 100 world wide?  Yet, I had to fight for the terms we got. The IP departments at University’s can be  greedy and tenacious.  Phoenix Nest has won over a million in grants to support this research. Phoenix Nest funds post docs that I have not met in person, they're great to work with though and I purchase equipment that I can’t even pronounce. Add grant manager to the list of my qualifications and I do the work for free.
That 'thing' cost $80,000.


I would not have been able to get this far if it wasn’t for Sean, my grant writer and CEO of PN,  Peg our  pro-bono lawyer from King&Spalding, Mary & Amy at McConville, Considine et al and my bookkeeper Deb. Mari, well she goes without saying.

Meanwhile I’m working on the same type of deal with an intellectual property department in the UK for the exclusive licensing rights for our MPS III C gene therapy program. Negotiations have gone a little more smoothly, but only after I contacted the Dean of the University and flew out to talk face to face with the manager of the IP department. The terms have finally been agreed upon, they can’t go back now. I’m just waiting on one little signature from an outside collaborator, then PN will have the exclusive licensing rights to our gene therapy program. These guys were smart enough to add a clause in our contract stating that I couldn’t disparage their name in public. LOL Not that I would, I think they just got nervous after I called the Dean.  Sometimes a little threat is what you need to get the ball rolling. The signing of our Gene Therapy agreement won’t be as anticlimactic. Our MPSIII C GT is nearly ready for trial. There is one MAJOR hurdle that we have to address, licensing and manufacturing the vector. I have to depend on my collaborators to move this portion along, this one is a bit out of my hands. I will lean hard on these people to move it along.

Years ago PN received the exclusive licensing rights for a chaperone molecule for MPSIII C and D. Working with the entity that holds the original patent was seamless. Every institution is different.  We have been modifying the compound for a couple of years now. Every 4mo we get several new versions of the original compound and it keeps getting better and better. The original version rescued enzyme but it was too toxic. We work with a chemistry group to help us design the new compounds, which are then sent to Alexey to test. The research lulled last winter when JJB couldn’t afford to replace the postdoc who left. Jeremy and I sold our parking spot and granted the money to Alexey, our new postdoc is now testing the backlog of compounds with great success. Our chemist is already working on additional designs from the new leads. This is the kind of stuff I get excited about.

Our chaperone program gets the least funding attention from our consortium HANDS, because we have placed Gene Therapy as our top priority. A chaperone for Jonah or anyone with a responding mutation could be our silver bullet, but it won’t work for all our kids. Gene Therapy is for all of our kids, the late staged young adults might opt to not have the drug. We continue with the chaperones, for several reasons. For late staged patients this is a more favorable approach then GT. Also the chaperone that we’re creating will most likely have implications for other lysosomal storage diseases. Gene Therapy (GT) is a one time surgery, a chaperone treatment is forever and can enhance GT. Brining us as close to a cure as possible.

Alexey also has two other MPSIII C programs in his lab. But that’s enough for the science for now.

Our Natural History Study has FINALLY been approved by the Internal Review Board (IRB) at Montefiore Children’s Hospital. I swear I thought I was going to have to beat someone up, I called the boss too, but I think you have to be scared of your boss to pickup the pace.The IRB is now with the National Institute of Health and waiting for approval. Dr. Levy, Paul is a very calm thoughtful man. He reminds me of Jeremy, he can’t be rushed and sometimes I just want to wring his neck. He is passionate about the kids and prioritizes them over the science. A lot of PI’s conducting trials of this magnitude put pharma first, Paul can’t be bought. His ability to connect with children is priceless, he can put children at ease, which makes for a productive visit. I’m anxious to meet our Neuropsychiatrist, I realize now the crucial role that she will play.

When Jonah was first diagnosed we drove out to North Carolina to meet with a geneticist, neuropsych etc..  We were at the clinic for a few days, the first day Jonah threw up all day.  We waited out a day at the hotel hoping he’d get over the bug before we did the cognitive testing. The next day Jonah was still irritable, not vomiting anymore but not happy. We met with the neuropsychologist and she immediately got down to business, ushering us all into a little room. The back of the room had a few book shelves, the other walls were lightly decorated and kid friendly the room was taken up by short round table, surrounded by furniture. The furniture overpowered the room, there were no windows and it was claustrophobic. Jonah was anxious he wanted to be free to move around. The doctor was commanding, she didn’t try to engage Jonah and win over his affection.  I was surprised by her rigid borderline militant demeanor. She knew that Jonah had a profound neurological disorder that caused caused cognitive delays, brain damage and sever behavioral issues. Her job was to administer a few cognitive tests, to do so she needed Jonah to be comfortable. She didn't bother to get to know Jonah and disarm him. I didn’t get it, I thought maybe for our sake she wanted to get us in and out. Later I learned that the evaluator was an outside contractor and her original appointment with us was cancelled because Jonah was sick, so she had to reschedule, I’m thinking she was pissed about having to rearrange her schedule. I’ll never know the truth.

On top of her abrasive personality, she had two different colored eyes. Not something you see everyday and not something Jonah had ever seen. Her job is to administer cognitive tests to children with neurological and behavioral disorders, kids like Jonah have irrational fears. Animals and anyone in costume freak him out (he’s almost over this now).  Probably not the wisest career choice for her.  Jonah lost it, he was inconsolable, he wanted to leave charging at the door, screaming bloody murder.  The evaluator was incensed, she kicked me out of the room, insinuating that I was the reason for Jonah’s ‘bad’ behavior. I was ready to leave, the whole experience was cruel, Jonah was sick, scared and tired. Jeremy was willing to try and hold down the fort, out of spite I agreed to leave.  Knowing full well that things would only get worse if I left.  I sat down the hall in the waiting room. Jonah didn’t stop screaming for one second, he was inconsolable, I don’t know how long I let him scream for, it felt like 20 minutes. Enough was enough I stood up and walked down the hall, just as I was about to knock the doctor opened the door.  She was harried, sweating her eyes bulging.

She attempted to apologize, then switched gears stating that a child such as Jonah needed strong discipline, if we didn’t do it now we’d never regain control of him. I took Jonah in my arms and we left. Never to go back again. She was right that Jonah and our family would need extra help to negotiate his behavioral issues. But that day was not the time nor the place, Jonah was upset with just cause.  Instead of setting Jonah up for success, she brought out the worst in him. Jonah didn’t know any better, but she should have. He was 2, sick and scared.

The silver lining…. I learned an invaluable lesson. Five years ago I didn’t know that I’d be lining up the team of doctors for our Natural History Study. I know first hand what it’s like for a parent and what works and doesn’t work for our Sanfilippo children. I will make sure that our families do not meet with the same arrogant belligerence. The NHS, is a five year longitudinal study that follows the course of the disease. The kids will come in once or possibly twice a year, they will have one day of clinical testing and another day of cognitive testing. If the children are upset during the cognitive tests it will affect the results of what they’re really capable of doing.
For parents starting down this road, take note and choose your team very very wisely. Ask yourself if this is the doctor, scientists or institution that you want to work with side by side for the next ten years….

What’s next…. We’re waiting for lambing season to begin again, which won’t happen again until next season…. Who knew.

We injected 12 healthy sheep last Winter. We’re looking for the best distribution avenue into the brain for our gene therapy. Explanation… the gene therapy drug can be broken down to three parts (layman terms) The missing enzyme, a vector and the capsid. The enzyme that Jonah is missing is called HGSNAT, the enzyme can be man made, the vector is a virus and is the delivery method (like a flu virus) and the capsid is the package around the enzyme and the virus. The virus delivers the enzyme to the cell. The enzyme then does it’s job of breaking down the storage i.e. Hepran Sulfate, the substance that should have been recycled by HGSNAT.  Sounds simple right, totally doable.
Once you have designed the drug to perfection, then you have to figure out the very best way to get it into the body. In the case of Sanfilippo syndrome which primarily affects the CNS, getting a virus into the CNS and past the blood brain barrier (BBB) is an extraordinary challenge.  The brain protects itself from viruses getting it past the wall i.e. the BBB is a major challenge.

The virus has to be minuscule, but still large enough to carry the drug. In the case of MPSIII C we have an added problem, in that our enzyme can't leave the cell. Most enzymes move around, but HGSNAT is a homebody and attaches itself to the lysosomal of the cell. So we’re planning to go straight into the brain. For the surgery several teeny tiny holes will be drilled into the skull and the serum will be injected in.  Sadly I can’t say anymore. One day it will all be published and available to the world. More importantly we will have a TREATMENT for our kids!

Claire, Brian and Ian, the neurosurgeon went out to New Zealand and with a team of vets; performed the surgery’s. That fancy brain software that you all helped us buy, guided Ian in the surgery. Today the sheep brains are back at Manchester, where Brian, Claire and their team are analyzing the vector distribution. Claire, below has the job of slicing the brains of 12 sheep and categorizing the sections then analyzing them. She's the best thing since sliced bread!
Claire O'Leary, our postdoc at Manchester, meets P.J. Brooks, NCATS, NIH. ASGCT conference 2017

I can’t apologize if this makes you squeamish, we don’t have any other options. This is my life and I want to share it with you.  The animals are all free range they are loved by vets and during surgery are given the same respect as a person. They were raised for this single purpose and everyone involved in this process is deeply respectful to the sacrifice that these animals have given, albeit unknowingly made for science; i.e. my son and maybe even yours. FYI I’m pretty sure that Brian likes his dogs more then he does people.

The sheep studies will determine how we will proceed with the clinical trial. We’re almost there, manufacturing the gene therapy will be our next biggest hurdle.

We’re actively seeking partnership now. For the first time we’re meeting with a tepid response from pharma and investors. Back in the day people looked at me with pity and politely implied: "You're screwed"

We have done EVERYTHING we set out to do. My ducks are in a row now and it feels good. I’m stretching out and preparing to jump over the “valley of death.”

In a few months we will be launching a few awareness campaigns focused on finding and identifying people with MPSIII.  I WILL NEED YOUR HELP!
Behind the scenes pic during the filming of Mission: Hide and Help
In the meantime you can buy your favorite Gemini a birthday present, I hope you choose me.  Trump is also a Gemini... yeah yeah, I know we both talk too much, it's a Gemini thing. At least I also LISTEN to people, especially to my advisors.  Go here,  Jill's B.Day wish list
The gifts will go to our next benefit  Monster Mash 

This is the best idea Mari has ever had... Well close to it. http://bit.ly/JJBWishlist



Thursday, December 8, 2016

OUR story began in the South of France.







OUR story began in the South of France. 

In January, five years ago Jeremy, Jonah, Elvis and I traveled to France to meet with Ghulian & Francine, Belen, Raquel & Antonio.  We had to go, besides the Burke girls and Levi these were the only MPSIIIC families that we knew of. Jeremy and I were desperate to find more families like ours, we aspired to build our patient population and hoped to work together to find a treatment for our children.  Our friends in France speak next to no English and Jeremy and I don’t speak any French or Spanish. Belen only speaks Spanish, but fortunately Raquel (Portuguese) speaks a little of everything.  Our friend Elvis came along as our French translator and camera guy. The parents embraced Elvis as family.  I hadn’t seen him for 5 years.

After Thanksgiving Jonah and I went to Paris to meet up with our families again.  Our network of French families has grown, expecting to meet with 4 French families this time; too much for Raquel to translate for.  I texted Elvis out of the blue. “We need you Elvis… can you go to Paris and London with Jonah and I next month?”  He responded a few minutes later: “I’ll make it work.”

Jeremy couldn’t get out of work. No matter who you are in the network television industry, taking the week after Thanksgiving off is gravely frowned upon.

Before I go any further I want to mention to those wondering if we travel to exotic places to hang out with our friends on JJB’s dime?  To that I answer NO, it comes out of our own pocket.

The French families made the arrangements and found us an awesome three story house with a finished basement.  Perfect for our crew.  I wish we were still altogether.  How we look out for each other, nobody has to apologize for their child’s behavior.  They are better then some blood family members,  they would never judge you for having a ‘naughty’ child. No matter how bad your kid is, it doesn’t phase them, they just help. The odd thing about Elvis is how he responds to our kids, it’s as if he has a beloved sibling with Sanfilippo. There is no reason for how awesome he is, he just is. I mention this because Elvis deserves recognition, like our kids, he’s 1 in a million. I have never met a calmer person that has reflexes as fast as Elvis, he doesn’t skip a beat. Even if he wasn’t in the room he knows what just happened and he translates the scene before I even ask him to. If he could respond right now, he’d remind me of how I yelled at him from the ground floor up two flights of stairs to come down.  When he came down, I told him it was time for him to get to work. The look on his face put me in my place and I think I stopped bossing him around after that.

Besides just being with each other our meetings are a chance to catch our families up on the science. This year we welcomed a recently diagnosed family, the parents of 4 year old Jewels.  Devastation clouded their faces, Jewels’s dad could hardly make eye contact with me.  Out of respect I tried not to catch his eye. His wife… she braced herself for the answers to every one of her questions.  They found Ghuilain and Francine after diagnosis, they did what we all did when our doctors had no answers, they found other parents. We were just planning our get together when they were diagnosed. So they found themselves sitting at a dinning room table, with strangers, their heads down afraid to ask the questions that they drove 6 hours to ask.  Four seasoned families starred at them waiting for them to gather the strength to talk.

Stopping for a moment now to remind you that we still need your help!  Our holiday giving campaign has been awesome so far, but I see that momentum has slowed down.  We really really really really really need to raise another $70,000 dollars. I was blown away by the $18,000 that we raised in one day, we exceeded our Giving Tuesday goal of $10,000 and an awesome person doubled our goal with $20,000.00!!  "Save Me from MPSIII" runs until the 4th of January.  Join our team, help promote or donate. https://www.crowdrise.com/save-me-from-mpsiii/fundraiser/jonahsjustbegunfound


They arrived the afternoon that we took the kids to Disneyland.  Francine and Ghulian waited for them at the house.  When we got home our motley crew burst in exhausted and exited from our day at the park.  I can imagine what they must be thinking… “How can they all be so happy, when their kids are dying.”


Our day at Disneyland had been a blast and I didn’t think to dial it down before I walked in the door.  I wish I had been more respectful of their state of mind.

Disneyland was so successful because we got the special need passes for Haitem and Jonah, the pass gave us the ability to cut to the front of the line. It was a huge hassle to get them, I was ready to give up, but Elvis wouldn’t let it go.  He told me he was going to lay it on thick.  I didn’t have documentation that Jonah was ill.  We had to prove it to them. The first line that we cut through was at least an hour long and made up for the process that it took to get us the pass. Plus I got in for free, Jonah was half price and three other people got to be on our pass!  Disney Paris was decked out in lights and Christmas scenes and special surprises were everywhere.  Watching out for Jonah was a bit scary at times.  He always has to be up front and despite the freezing weather the crowds were big.  I don’t know how we managed to keep us all together.


Obviously I live a very frazzled life, I have my fingers in a million pies, my brain is always preoccupied with something.  When we’re in a new and exciting public environment with Jonah we have to pay extra attention to him. Jonah gets over stimulated easily and when that happens all hell breaks loose.  When I know I’m going to be in one of these situations, I wear my fanny pack.  I can’t spend time digging in a book bag or purse trying to find my wallet.  I have to be ready to pay and bolt. Our families went to check out this huge Christmas fair, in the heart of Paris, up and down the street for a half mile on each side vendors lined the streets.  There were rides for the kids, an enchanted forrest delighted Jonah.  It was a very merry time. We walked and walked checking everything out.  Jonah really wanted to go on the little Christmas train ride. I promised him we would go back.  We stopped to buy some Christmas presents, there was a really cool soap stall.  I stopped to buy some things. Raquel was looking after Jonah by my side.  I just pointed and said I want that and that and that.  I noticed a man next to me and I apologized for cutting. He didn’t care, he started to chat me up, asking lots of questions.  He told me how much my order was and I thought that was weird. He was really tall, he towered over me, he watched my every move. I reached into my fanny pack and opened my wallet to pay, he did not take his eyes off of me.  He made me nervous but I shrugged it off, he wasn’t going to rip the wallet out of my hand in front of everyone.  I paid and we moved on. 

At the end of the boardwalk the families wanted to cross the street for something else.  Jonah was freaking out; he demanded that I take him back to the train. It was dark, we needed to be going home. We compromised and made a place and time to meet back.  Alone, Jonah and I ran all the way back to the train, weaving in and out of the crowds.  I grabbed some change out of my wallet and zipped my pack back up and covered it with my coat. Jonah got his ride everything was fine.  Then he announced that he really had to poop. Like a four year old he waits until the very last second to go.  So we started running again, Jonah kept stopping to look at things. I was stressed; afraid that he was going to poop his pants, afraid that our friends were standing waiting for us.  We finally found a bathroom, FYI you have to pay to use the toilets. Jonah rushed in while I went to pay.  Yup you guessed it, my wallet was gone. There was nothing I could do. The bathroom attendant didn’t care that I didn’t have the money or that I had just been ripped off. I knew immediately that I had been targeted. These guys work as a team, that man was hanging out at one of the most popular vendors waiting for someone like me.  I swear I couldn’t have made it any easier for them. 

As it all sunk in I started to cry, so many cards to cancel, I hadn’t paid for my train tickets or hotel room yet.  Then Jonah yelled: “Mom I have poop in my pants.” I went to check on him.  Not only did he poop in his pants but it was all over the floor.  Of course there wasn’t any toilet paper, I tried to pretend like everything was fine as I took toilet paper from the other stall. Trying to clean him up as best as I could.  Then he stepped in it and started yelling at me to clean his shoe. I hadn’t paid for him to use the bathroom and here he had shit all over the place. I laughed and cried all the way back to my friends. I had a good cry then let it go. I didn’t have our passports in my wallet and I had my friends to help me out.

That same morning, I had locked the entire group out of the house. Ghuilian left the key on the table along with his phone that had the landlords # in it. So what… We found a ladder, Raquel had left the window open upstairs. Antonio climbed up and let us back in. No harm done. What’s that saying… the last one out locks the door?

Three days with the families in Paris then off to London. We planned the trip around a conference that I was invited to attend.  But that’s enough for now. I tired myself out just thinking about our adventures in Paris. You won’t believe what happened in London. lol 

I'm thinking maybe Elvis should write the blog about London.  He's the one who spent three days alone with Jonah...

Monday, November 21, 2016

Woohoooo it's the 22nd of November, no really Woohooo!

How much time do you think you spend: thinking, reading or talking about what Donald Trump just said? It happens to me every morning and I want to kick myself when I realize that I just lost 20 minutes of the first hour of my workday.  Here I am wasting more time again aarg. Please tune everything out and help us out for a second. It will be the best 20 minutes you spent today.

It’s the 22nd, of November our Holiday Campaign: “Save me From MPSIII" has begun.

Haven’t heard of our campaign yet?  Go here. https://www.crowdrise.com/save-me-from-mpsiii

Crowdrise is a fundraising platform that JJB use’s every holiday to raise funds for our research programs. In particular we are fundraising for the last leg of research for our gene therapy program. On March 1st we owe a payment of 77,768.50 GBP.  This past October JJB and our consortium HANDS paid the first payment of 77,768.50GBP.  With that funding our scientist was able to purchase a very expensive piece of software that they will use in this machine (pictured below.)  The software will help guide the needles through little holes drilled into the skull of our children.  We are also using this funding to secure the salary of our most valued postdoc, Claire. Claire will be making the GMP vector which is the drug that will be administered into the brain.  We have practiced on mice and gotten very good distribution results in the brain.  But we want the best distribution that we can possibly get, our children only have one chance at this and it has got to be perfect.  Failure is not an option.  So we will now be testing in sheep, I’m sorry if that makes you queazy and sad, it makes me sad too and I don’t take it lightly.  But remember we’re going to be doing this same operation on Jonah and our friends’s children. If it was your child receiving this experimental drug and invasive operation wouldn’t you want to take every precaution that you could before putting your child through it?  I know it sounds horrific, but for these children the only other option is death.


Last month I went to England to meet with the lead scientist, Brian and his postdoc Claire, they took us through the lab and we got to see everything.  I try to imagine what Brian’s office looks like all the time, what the lab is like, where do our mice live.  Now I know.  We walked over to the hospital and met with the neurosurgeon that will be using the software and preforming the surgeries. Raquel, from Sanfilippo Portugal met me there.  Now that we are so close to trial, we wanted to see for ourselves where our kids would be staying, what they would be going through and who will be taking care of them.  We wanted to meet the doctors and nurses. Put yourself in our shoes, what we’re doing is terrifying, monumental and groundbreaking. Can you imagine our fear and excitement?   We talked to Claire and Brian again about our plans for administration and of course the funding and the next step… trial.



This is it.  This is what we have all been working so hard for.  I hope you can continue to help us get there. I ask those that can use a computer to set up a profile and join our crowdrise team.  Mari will be adding a step by step powerpoint on our website tomorrow. Our campaign will run from November 22nd-January 5th.  There will be several Bonus Challenges throughout the time frame.  We will keep you updated on those dates.  The first one is November 29th, #GivingTuesday, the team to raise the most money on the 29th will win $25,000.  I hope it’s us.

Please share our story with everyone, post your link everywhere, send to all your colleges, clubs, friends and family. You’ll be receiving your JJB 7th annual newsletter in the mail shortly, so don’t feel bad if you don’t use the computer or social media.  We love to receive checks in the mail.  BTW ask your HR department at work if they match donations?! We love two checks in the mail even more.

I hope my fellow Americans enjoy a wonderful Thanksgiving feast.  We sure will, Jeremy is the best cook ever. On Saturday Jonah and I will be joining 6 Sanfilippo families in Paris, for a patient population meeting!  We rented a huge house for all of our families.  I hope they have good insurance.  It’s going to be hysterical!  We will have a french translator to help us explain our research progress, otherwise it would be me, drawing pictures and playing charades. That would be funny too. We always have the best time together.  Ce Bon.
Raquel, the brain guiding machine and Dr. Kamaly

Raquel, Claire, me and Brian

Over the weekend we took Jonah and Sebastion on the Polar Express

Saturday, November 12, 2016

She's got grit

So how about that election…  I kind of had a sinking feeling. I take solace in the fact that Hillary won in the popular vote. I didn’t vote for Hillary because I’m a feminist or because I think Donald is a piece of sh*& but because Hillary is the better person for the job.

Up until now I have never given much thought to being a feminist.  You can call me a feminist and I won’t consider it a label but just one of my attributes. 

I admit I’m a sell out. I haven’t stood up for equality in my mission to create a treatment for Jonah’s syndrome.  When I formed Phoenix Nest, my first thought was, who’s going to be the CEO?  It can’t be me.  Nobody will take me seriously, I’m just a mom, a women.  Asking Sean to stand in front of me as CEO was not a hard decision- he’s a man with a Ph.D even, he’ll be accepted.   My only thought was for Jonah and doing what it took to get a treatment for our kids. Someday maybe I’ll go back and get my MBA and take the helm of Phoenix Nest.

Call me a feminist but don't call me a‘fighter mom’.  People call me a fighter mom all the time, as if it’s a compliment.  For me, as a feminist it’s a derogatory remark.  As if because I’m a women I’ll have to fight the system.  While a man can just make a few phone calls and it’s all taken care of.  You never hear anybody call Mark Dant a fighter dad.

Somebody once told me about a time that they had to try and explain my personality to another person.  This was an awkward conversation for me.   Listening to someone try to sum me up in a word. I stopped her and told her next time you find yourself talking about me, just say: “The girl has got grit.” It has a nice ring to it.

When I found out that Hillary didn’t win office, I was in denial, like watching the trade towers falling- this just can’t be happening.  I couldn’t wrap my brain around it. When it sunk in- my heart  sank.  I thought if Hillary couldn’t convince millions of people that she was the better person for the job.  Then how can I ever expect people to donate to our campaign?  I felt defeated on many levels.

JJB is getting ready to launch our Holiday Giving campaign.  Mari and I have been working very hard on how it is that we’re going to convince people to give this Holiday Season. We talk about what it takes to convince people all the time.  We’re a non-profit we survive off of donations. Yet, it seems impossible to convince people to donate.  Almost a thousand people read my blogs.  Over the last two weeks I have been posting to: Twitter, Instagram and FB asking people to join our: “Save Me From MPS III” campaign.  Mari sent out an email blast. I personally emailed friends and family asking for help. This message hit thousands of people.  In the last two weeks, guess how many people took action and joined our team? 2 people.

I had a little pity party for myself and Hillary earlier this week.  I did things that made me happy- retail therapy helped. I made a collage frame of Jonah and Jeremy’s selfies.  I stare at it constantly, their smiling faces make me so happy. During my down time I realized that I had convinced 100’s of people to do things that they never saw themselves doing.  Case in point, Sean gave up his day job to help me. Our lawyers work pro-bono and discounted rates.  I convinced  the scientists to work on an ultra-rare disease, when they could have been focusing on cancer. I have brilliant advisors around the country that take time out of their busy schedules to help me whenever I ask. I convinced Jonny Lee Miller to help, he ran a 100 miles in one day to help JJB.  I have given talks that have inspired others to follow my lead and who are now creating their own biotech’s and winning grants!  Friends and family from coast to coast (but nobody in the middle…) have given their blood, sweat, and tears to put on fundraisers for JJB.  I have spoken to our congress people and they have been convinced to help us.  It occurred to me that I am good at convincing people to help.  But maybe it’s not completely about convincing people, but reminding them to help.  Reminding them over and over and over again.

So here is your reminder.  Giving Tuesday starts November 22nd, on that day, Crowdrise opens up it’s donation portal.  JJB has set up a campaign on Crowdrise called “Save me from MPS III”
https://www.crowdrise.com/save-me-from-mpsiii/fundraiser/jonahsjustbegunfound
JJB is asking you to donate or join our team. All you have to do is create a little profile and hit the tab on our page that says “fundraise for this campaign” We're asking that you join the team NOW so that we can hit the ground running on November 22nd.  The campaign will run all the way through December. There will be contests and cash prizes that we need to be ready for.

When you link your profile to “Save me From MPS III” you can go in and edit your fundraiser.  Make it personal, upload your own relevant pictures and story of why helping JJB is important to you. For inspiration, read what Jennifer Wood-Mercier wrote.  Then blast the link out to everyone you know and even some that you don’t.

Can you do it?  I’ll be watching.

Wait a second.  It just occurred to me it's INSPIRATION that you all need not convincing!  Duh.  I don't have to convince you that Jonah and the children suffering from MPS are worth saving.  I need to inspire you.  Hm what can I say or do to inspire you?   I need to think on that.  In the meantime look into these engaging, brilliant and happy blue eyes. Jonah doesn't know that he's dying, lets get him a treatment before he does.