Double edged sword.

After Jeremy and Jonah sang me Happy Birthday, Jonah proclaimed "my turn!"

Just happy to oblige.

So annoying! My yahoo account was hacked. I changed the PW and that seemed to stop it.  Sorry for the spam.  It’s bad enough that I naturally spam my friends, only to have someone else hack in and do it for me, well that’s just overkill.

My life is a double edged sword, catch 22, damn if you do damn if you don’t.  The future is terrifying.  I’m glad to be a year older and closer to the finish line.  Time means that Jonah is getting older too and closer to brain damage.  His 4th Birthday is just 6 weeks away.  Two years ago, when we started this campaign we assumed he’d have brain damage by now.  He doesn’t!  Now we "assume" that we will have another 2 years before he starts to change (based on observance of other MPSIIIC kids.)

For the most part our business plan and executive summary for Phoenix Nest is done and I’m ready to hit Boston with it.  I feel like it’s finals week, Ive done all the cramming I can do I’m just waiting for the test.  Monday can’t get here fast enough!

While researching for PN, I found a few articles discussing the most expensive drugs in the World.  I was relieved to find that out of the 12 most expensive drugs in the world, five of them are for Lysosomal Storage Diseases (LSD’s) which Sanfilippo is.   Sanfilippo is also part of the MPS’s (Mucopolysaccharidosis.)  2 of the 5 LSD treatments are for MPSI and MPSII.  I was relieved because... I’m scared.

I’m scared that I won’t be able to convince a venture capitalist (VC) to invest in Phoenix Nest.  I created our virtual-biotech out of necessity, our patient population isn’t large enough for a big drug company to take interest in us. Our alternative is to do it ourselves, for that I need a financial partner.  But why would a VC want to help an ultra rare disease company?  Well because, we can charge the World’s highest prices for a Sanfilippo drug.    Phew- I can breathe a little easier now!

Then it occurred to me...  I have just become one of those evil capitalists, a company that my neighbors occupied Wall St. to shame. Ugh! But what am I supposed to do?  Sit back and watch our children die?!  Because I don’t want to over charge for a treatment that I have to have to save my son?!  A treatment that would never see the light of day if it wasn’t for the parent advocate groups driving the science....

I’m sorry I don’t have a billion dollars to do it all by myself!!!!  I have to convince someone that does into helping us. I can’t apologize for being happy that we can charge exorbanent amounts of money.  Life is a double edged sword. On a side note most of the manufactures of these expensive drugs give scholarships for those that don’t have insurance.

So here is my compromise. On the slim chance that we do get VC backing and Phoenix Nest is able to discover and create a treatment that in turn makes millions and millions of dollars.  I’m going to open up a private nonprofit school for special needs kids (Phoenix Nest Refuge.)  The school will be the best, a parents dream come true and free of charge.  We’ll shame the other greedy capitalists into helping us maintain the school!

After entering the realm of the special needs world, I have met many amazing parents with wonderful kids that need help.  Our government is turning their backs on our kids.  If there isn’t a special program in our district, then our government forces us to mainstream our kids.  This is totally unexceptable, for everyone involved. So here we put a child who may not be able to communicate, walk,  sit still for more then two min.’s and is prone to outbursts- in a classroom with normal kids.  Who’s benefiting from this?  NOBODY! Not the parent, child or his classmates not to mention the administrators and the rest of the fall out effect. What a disaster.

Many parents are forced to move to different districts in hopes of getting their children into a special needs program, only to pay thousands of dollars for tuition. It’s added insult to injury, our lives are complicated enough!  We're already bankrupt from having to pay through the nose for medical expenses.   As it stands now, our governments is saying to us: “Your child will never contribute to society, so make him a ward of the state; we’ll warehouse him for you until they die and are no longer be a burden on you and society.”     (fu)

So I fantasize about creating a beautiful school with all the amenities that our special needs kids need.  Because our children deserve nothing but the best, they deserve the same quality of life as anybody else, IF NOT MORE!  Who's making all the sacrifices here?

The odds are stacked against Jonah and this generation of rare disease children with no treatments.  There are 7,000 rare diseases and only 5% have treatments, 75% of the rare disease affect children. :( BTW the definition of  a rare disease is one that afflicts 1 in 200,000 live births.  MPSIIIC affects 1 in 1.4 million.

To make all my dreams come true I need money and lots of it.   Money will never by anyone happiness but it will buy our kids a cure...  Fingers crossed I can convince a VC to give some funds to Phoenix Nest.  In the mean time JJB needs to keep our payment plan for our gene therapy.  On September 1st JJB has to pull together $90K for our third payment for the first ever treatment for MPSIIIC  :) I'm very proud of this.

I have until June 16th to raise a 2k from the text to Rare program.  JJB's 1k will be matched by the EveryLife foundation.  On top of that my friends Dad has pledged to math another $340.00 (the remainder, needed to make the 1K)  I created a birthday wish, sent out an email blast, tweeted and blogged about this fundraiser.  I can't believe we haven't gotten a 100 supporters to text rare to the number 85944.  This just says to me either nobody reads my cries for help or people just don't get it?!  If you can afford 10$ and text please do it now. We only have until the 16th! Go here now. http://www.everylifefoundation.org/texttodonate.aspx

Why do we all make promises that we can’t keep?

Why do we all make promises that we can’t keep?  We’re all waiting for a cure for our kids, we hang on every word that our scientists and clinicians say.  Reading into their statements, hearing what we want to hear, clinging to hope.

February 2011 Jeremy and I went to the WORLD conference in Vegas.  The Sanfilippo community was anxiously waiting for Zacharon to take our first treatment to clinical trial.  Little did we know then, but Zacharon had just partnered with Pfizer to help them finance the R&D for our treatment. At the conference, if you all remember from my earliest caring-bridge blog. I told our friends and family how Brett- the lead scientist and co-founder of Zacharon, introduced his son to Jeremy and I.  Brett told his son that Jeremy and I had a little boy too and he was going to help him (Jonah.)

I so desperately wanted to believe Brett, I envisioned Jonah being the first Sanfilippo child to walk away from this insidious disease unscathed.  Zacharon’s treatment, would slow the progression of the disease, buying us some time for a more aggressive treatment to be developed.  I never truly hung my hat on Brett’s proclamation... I want to believe it, either way I’m grateful for the hope that Brett gave us.  We’re still waiting, it’s been 2 years now... Fortunately there is a lot of science now and I have turned my attention to other prospects.. Don’t put all your eggs in one basket, That’s my motto!

Then there is our ever so self professed brutally honest scientist Brian Bigger. Brian goes out of his way to not make any promises. Reminding me every time I see him, that a treatment might not come in time for Jonah.  I’d love to hate him.  He’s just scared that we will forever hold him personally responsible for not single handily saving our kids.  He’s quite egotistical, that’s why I like him and trust him to go the distance.

Then there’s me... I have promised all my families that they have my word... I promise!  I won’t quit until we have a curative treatment for our kids.  Yes... deep down we all know that our kids (this generation) might not be able to benefit from the treatment that we brought to market. BUT WE DON'T NEED TO BE REMINDED! It just goes without saying... We have to have hope!  What else is there?

Today I am reminded of those that never promised anything but have provided without complaint or need of praise. My Mom & Melissa, who are making our wine tasting event happen, it's going to be beautiful and delicious.  Sean, Mari and Jen who just do it without being asked.  My husband who has risen to the occasion; laid his sadness aside and has taken over all my house wife/mom duties.  Without complaint Jeremy: cooks, cleans and attends to Jonah’s demands.  For better or worse... they are all doing what they can, so I can keep my promise.  My sincerest thanks.

In order to hold up my end of the bargain I need for JJB to raise another 20K in the next three months.  Recently the EveryLife foundation donated me their text to donate widget.  All I needed was for 100 of our supporters to text “Rare” to the number 85944. I had a month (May 16th-June 16th) to raise 1,000 dollars to be matched by EveryLife.  I honestly thought that could be done  in one day.  We have 10 days left for another 50 people to take the opportunity to help JJB raise 2K and help me keep my promise. Over a 100 people read this blog, just saying... I know not everyone can afford an extra $10 bucks (I hear that.) If you don't text follow this link.

While you’re at it you might as well purchase your tickets for “Salut, Prost Cheers... To a Cure.”  Follow the link and check out the fantastic lineup of wineries pouring.   Music by the Jake Blair Band, buffet catered by Nikki Stokes the new owner of Uncorked.  The sponsored tables will have a beautiful platter of artisanal cheeses, spreads from the Republic of Jam, bread from Pionteks Bakery and hand crafted olive oils. Plus two bottles of wine from the Stoller Estate.  JJB wine glasses on the house. Seriously... get a table. Invite 8 of your friends to go in on it with you and have the time of your life (designate a driver!).  If you have a company, your logo will be plastered every where.

I'll be 39 on Sunday!  How I got here I don't know, I do know that this is going to be a huge year!  A week after my Birthday I'll be in Boston rubbing elbows with VC's, pitching Phoenix Nest to anyone who'll listen.  Jeremy and Jonah will meet up with me in Boston and we'll all drive out to Cape Cod.  The Burke's rented a cottage on the beach, great place to celebrate Fathers Day.  Can't wait to relax with my friends and watch Jillian, Lindsey and Kelsey dote on Jonah.  Then I'll run home repack and head to our patient population meeting in the Netherlands.   See my birthday wish.
Jill