BE brave….What does that mean to you?
I have been sick to my stomach terrified for the last 7
weeks. I’ll get to that later. Don’t worry Jonah is fine.
I went to Kirsten Gillabrand’s kick off presidential rally
today, in an attempt to distract myself from my nightmares. Kirsten held her
rally underneath the Trump Tower at 60th st and Central Park West.
Just for that I had to go, but seriously I did want to hear what she had to
say. What better way than to hear it in person.
I arrived
early assuming the place would be mobbed. I was surprised that I was able to
walk right up to the front of the stage. Kirsten’s most prevalent running
topics are: Gun control, Dreamers, LGBTQ, Sexual assault and Climate. To me
these are no-brainers. I wanted to
hear her underlying positions.
A journalist from the Washington something or
rather, it wasn’t the Post came up to me for a few questions. I had noticed the
journalist making his way in my direction, he stopped and asked a female
teenager some questions, she shied away. I looked at her mom, I swear it was
Elizabeth Perkins and yes, I had to google her to find out what her name
was. The only movie that my brain could
place her face in was the Flinstones Movie.
The journalist asked me if I was going to vote for
Gillabrand? I responded: “probably”.
Press: What does that mean probably?
Me: Well I don’t know yet, we only just got started.
Press: Do you like what Gillabrand stands for?: Gun control,
Climate/Environment, Dreamers.
Me: Well of course I do, but who wouldn’t? What I want to
know is where she stands on the needs of children with special needs.(I realized later that a lot of people wouldn't agree)
My response was something to that affect. I go into detail
after I caught his interest.
You can’t start with: “Hi I’m Jill, my son is dying of an
ultra-rare genetic disease, called Sanfilippo Syndrome specifically Sanfilippo
Syndrome type C, which is under the umbrella of Mucopolysaccharidosis
syndromes, MPS for short. My son, Jonah has MPS IIIC, you see there are 6
different forms of MPS and they all have subtypes, I think there are 20 forms
of MPS in total. Jonah has MPSIII a.k.a. Sanfilippo Syndrome type C. MPSIII or
more commonly known as Sanfilippo Syndrome which has 4 subtypes, maybe 5:
A,B,C,D and sometimes E. I want Gillabrand to support federal funding to the
NIH and FDA and support not bash pharmaceutical companies that aspire to create
treatments for ultra-rare diseases.”
I’ll lead with: “Hi I’m Jill, my son is mentally impaired and
I can’t find a public school that fits our needs.”
The opening speakers for Gillabrand were awesome. I was most
impressed by the “dreamers”, both speeches were delivered by young women. They
belted their stories of perseverance against all odds over the crowd of people
all the way back to the Trump supporters heckling them. I wanted to be like
these young women, their strength and drive to win resonated within me. I fed
off their words. Gillabrand’s campaign theme is bravery. Be Brave. Brave enough
to stand up for what you believe in to stand up for yourself. Or to stand up
for those whose voices aren’t being heard.
I fear the worst I fear that Phoenix Nest will lose our NIH
grant. This past fall Phoenix Nest won an SBIR (Small Business Innovation Research ) grant from the
NIH (National Institute of Health). Among funding research, I was able to rent an office and hire a new
employee. For the first time in 10 years I took a salary. Jeremy’s tv series he had been working on
ended. He has been home taking care of Jonah. It has been awesome having
him home helping me. With Jeremy home I don’t have to wrap everything up at
2:30 to pick Jonah up from school. I don’t have to work the entire weekend to
make-up for the work week. Jeremy absolutely loves being home with Jonah. For a
few months our lives completely changed for the better. Jonah thrives on having
us both home before bedtime.
I regret not putting more effort into finding a financial
partner. I have been weak, it’s heartbreaking to be told time and time again: “Sanfilippo
syndrome, especially type C is just too rare to fit into our pipeline.”
I took the easy way out and relied on the grants to float me
along. Early February my co-founder, CEO and grant writer gave his resignation.
It was hard to see my friend leave. I pushed him to look at an alternative to surviving off of
grants. For those of you that have or had to live pay check to pay check you
know how hard it is not knowing if you are going to be able to pay the rent at
the end of the month. This is how I feel, with the exception that if I don’t
pay the rent children will die, one of them being mine.
My CEO was also a primary investigator (PI) on our
5.6million dollar grant. The NIH is not pleased that he resigned and asked to
be replaced on the grant. The NIH froze the grant until I find a suitable
replacement. The NIH left PN with a small amount of funds, which has gotten us
through February and most of March. Jeremy and I have had to make ends meet at
the company with our savings.
Our Investigational New Drug (IND) meeting is planned for
the 23rd of April. Our research and clinical team will be going to
the FDA (Food and Drug Administration) to find out what they think of our plans
for clinical trial. With the grant funding, PN hired Srikanth
Singamsetty as our Associate Director of Translational Science. Sri’s first
priority was to write the Pre-IND for our MPS IIID ERT program, in the Pre-IND
we lay out what we want to do in our clinical trial and we ask the FDA direct
questions. After we receive the FDA’s feed-back we will address all their comments
and then submit our IND package. Upon approval we will be allowed to have the
clinical trial. Sri is now writing our IIIC PreIND letter. I have also scheduled a
meeting in Manchester, UK with our gene therapy scientists and clinical team at
Manchester’s, Children’s Hospital. We will start the planning of our IIIC
trial. Unfortunately, I had to cancel my trip, Jonah finally received his MRI
date, which is scheduled for the day after I would have arrived back from the
UK. I’ll call in by video. Sri is still waiting on his Visa so that he can go
in person. Sri isn’t a US citizen, he’s here on a work visa. In the US
non-citizens have to apply for a travel Visa to leave the country. This process
only takes about a month. Sri’s just trying to help save American lives. No
biggie we can wait a month for approval to fly to the UK and back. That’s
sarcasm.
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| This is what BRAVE looks like. Parents of terminally Ill children, fighting for their child's life |
