Update – Jack Fowler

photo-3Hello Everyone,

As many of you have noted, we’ve been quiet of late on news of Jack Fowler.  While we haven’t stopped working on behalf of Jack – work that has been relentless, stressful, and heartbreaking, we went quiet publicly while we worked some back-channel routes to see if Shire would and could be forthcoming and interested in saving this little boys life.

Unfortunately, Shire has continued to rebuff our efforts and continues to put Jack’s life in jeopardy.  Jack faces a steep decline, and his parents are beside themselves with heartache and heartbreak.  It seems unbelievable to me that a drug exists that has the potential to save him, yet it’s being withheld due to reasons that the pharmaceutical company cannot really stand behind.

We will be redoubling our efforts of the coming days and weeks.  Please stay tuned on how you can help #SaveJack.

In the meantime, drop over and read this article.  It’s something that Shire should read as well.  It’s all well and good for the FDA to have a plan.  But if big-pharma doesn’t want anything to do with it, our kids will continue to die needlessly.

I’ll be in touch with you all soon.

With Love,


Family fights to get drug for dying 6-year-old son

BY MONIFA THOMAS Staff Reporter February 15, 2014 1:26AM


dt.common.streams.StreamServer.clsShould a dying child receive an unproven experimental drug even if the patient doesn’t fit within a carefully designed clinical trial?

More than 53,000 people who have signed an online petition on behalf of a Mundelein boy say yes, absolutely. But Shire, the Ireland-based pharmaceutical company that owns the drug, says it’s a complicated matter and has refused to make the drug available to the child.

At the heart of this moral and ethical dilemma is 6-year-old Jack Fowler. Jack has a rare disorder called Hunter syndrome, or MPS II, that is expected to kill him because he lacks the enzyme needed to break down cellular waste in his body. Ten to 20 years is the usual life expectancy of someone with the disease, but Jack’s more severe type means he may have less time.

An estimated 2,000 patients — nearly all boys — are affected by the disease worldwide.

The hope is that the drug in question, SHP-609, can for the first time, slow or halt the progression of the disease in the brain. It is going through clinical trials, which means it hasn’t been approved by the Food and Drug Administration yet. The Phase II/III stages are just starting, and FDA approval happens after Phase III.

So Jack’s parents, Jason and Jamie Fowler, have been trying to get a “compassionate use” approval for Jack, which the FDA allows on a case-by-case basis. Clearance for such use means Jack’s case would not influence outcomes in the clinical trial, so an adverse reaction by Jack, for example, would not count against the drug.

For two years, the Fowlers have pleaded with Shire to let their son be part of the clinical trial or get the drug through compassionate use, including during a brief meeting with the company’s CEO, Dr. Flemming Ornskov, in a Chicago airport hotel in January. An advocate for the Fowlers, Andrew McFadyen of The Isaac Foundation, a nonprofit group focused on treatments for rare diseases, started the meeting by stating, “An immoral decision remains immoral if delivered in ones face,” and said that if Shire was still choosing death over saving a life then the meeting was over, according to Jamie Fowler.

Shire “delivered the same grim news, so we upped and walked out,” Jamie Fowler said in an email.

Dr. Barbara K. Burton, Jack’s geneticist at Lurie Children’s Hospital of Chicago and one of the investigators for Shire’s clinical trial, said there’s no evidence one way or another that the drug will save Jack’s life. “It’s conceivable that it would hasten his death,” Burton said, but added, “What I do know is that without [any treatment beyond what he’s getting now], he is definitely going to die.”

For that reason, she supports the Fowlers’ position.

Shire said it has compassion for the Fowler family. Yet “we believe that expanding access to SHP-609 beyond the clinical trial can put the overall development at risk and delay or eliminate the opportunity to make a safe, approved treatment more widely available to the global Hunter syndrome community,” Ornskov said.

An FDA spokeswoman said that of the 940 submitted requests for expanded access for such investigational drugs between October 2011 and September 2012, all but four cases were allowed to proceed.

Unknown adverse events usually do not prevent a compassionate use request from being granted, but that is determined on each case, the FDA added. The agency could not comment specifically on Jack’s case.

Dr. Michael Caplan, pediatrics department chairman at NorthShore University HealthSystem, who is knowledgeable about clinical trials, also did not speak about Jack’s situation, but he said experimental drugs usually aren’t given to patients outside clinical trials until Phase III is complete. Safety is the issue, Caplan said.

Shire didn’t say for sure whether that might be a possibility for Jack after the Phase III is done, but a spokeswoman noted that Shire has done that in the past.

Lewis Smith, an associate vice president of research and a medicine professor at Northwestern University Feinberg School of Medicine, said, “these are moral, ethical dilemmas.”

Companies typically are very concerned about allowing people who are not in the clinical trial access to the drug, because “they’re very concerned about whether that would muddy their results, because this is a rare disease,” Smith said.

Yet, it’s an emotional issue, especially when a child is involved, Smith said.

That’s clear when Jamie Fowler talks about how Jack’s cognitive deterioration, which came with the disease, is slowly taking away his personality. Gone is his ability to say “sis” for his sister, Juliet. Fowler dreads the same fate for “mom.” “It’s heartbreaking,” Fowler said, crying.

They’ve turned to social media, such as Facebook, to try to get Shire to reconsider. An online petition hopes to hit 75,000 signatures by the end of the month; there are more than 50,000 names so far. The family also started doing media interviews to get the word out about their case.

Nothing has worked so far, but the Fowlers said they aren’t stopping until Jack gets the drug to see if it can save his life.

“We won’t sit by and watch our son die,” Jamie said.

Contributing: Chris Fusco

Email: mjthomas@suntimes.com

Twitter: @MonifaThomas1

Jack Fowler – Update

image-27Hi Everyone,

I thought I would take a moment to update you on our latest attempt, our latest plea, to Shire Pharmaceuticals to open communication and connect with us about Jack Fowler, Expanded Access, and doing what is right for the Hunter Community as a whole.

Yesterday evening, I sent Shire CEO Flemming Ornskov and Head of R and D Phil Vickers an invitation to participate in a teleconference with Jack’s physician (and Internationally renowned MPS expert) Dr. Barbara Burton, and with Dr. Emil Kakkis, an expert in rare diseases who has a wealth of experience in the pharmaceutical world and with expanded access requests to save children’s lives.  The purpose of the call was to ensure that all parties are on the same page moving forward, with the hope that a collaborative discussion can take place to best meet the needs of everyone involved.

I have not had a response from Shire, though I remain hopeful that they will agree to participate.  At some point, someone at that company needs to look at this situation with a different lens.  At some point, someone at Shire will have to look back at their company credo and work to “Be As Brave As The People” they treat.  I’m hoping that some point is soon.

I’ll keep you updated as things progress.  In the meantime, I’ve been busy speaking with numerous media outlets and scheduling in radio appearances.  I can confirm that Jamie and Jason Fowler, Jack’s parents, and The Isaac Foundation will appear on a Boston Call-In Show in late February, from 9 – 10 pm.  This show is carried over 38 states, so chances are we’ll be on the radio somewhere close to you.

We know that our case to #SaveJack is solid.  We have everything in place to save this little boy’s life.  We only need Shire to sponsor our effort, something we all know they should do.

With Thanks,


Boston Globe – A Few Notes

photo-3Hi Everyone,
Jack Fowler’s case was presented in the Boston Globe today. And while it’s nice to get some exposure, I truly feel the editor at the Globe should be held accountable for the biased and single-sided view the article portrayed.

Nowhere in the article was the FDA’s position put forward – a position we have been open and transparent about presenting since day 1. That position is simple – access to investigational medicines outside of the clinical trial setting have NO BEARING on clinical trial results. In fact, there are cases where expanded use have helped to speed up the process. Indeed, the FDA guidelines say the following:

“There are examples in which FDA has made use of adverse events information from expanded access use in the safety assessment of a drug. There are a small number of cases in which an important adverse event was first identified during expanded access use and those adverse events were in clouded in product labelling. This is not a negative from a public health perspective – the sooner important adverse events are identified the better. Even from the sponsor’s viewpoint, early discovery of a rare adverse event is, on the whole, a benefit. Although adverse events first identified during expanded use of a certain drugs have been included in the drugs’ approval product labelling, we are unaware of any cases in which adverse event information obtained from expanded access use has resulted in denial of approval for a product.”

As I have previously noted, the FDA has been in contact with The Isaac Foundation regarding expanded access, and I have assurances that any adverse events that happen outside of the clinical trial setting will not hinder the clinical trial for any reason.  Why the Boston Globe editor did not see fit to ensure that information was presented is beyond me, is unfortunate, and leaves an incomplete picture of the story as a whole.

In addition, no quotes were sought from families who have children currently participating in the trial.  The trial is currently in Phase II/III and has lasted over 4 years.  Families are reporting incredible results in their children – their lives have been saved.  The treatment works, and the reversal of devastating symptoms these children have been experience is proof positive. Moreover, the treatment is safe!  Over 4 years of clinical trial has seen NO ADVERSE EVENTS REPORTED.  None.  Zero.  A few words from families who have experience dealing with this treatment would have shed light on the trial, the effectiveness of the treatment, and the safety of administering that treatment to our kids.

Finally, with all due respect to Dr. Muenzer, I have to  object to his comment that “compassionate use will destroy a trial.”  That statement flies in the face of all evidence the FDA has provided us, and flies in the face of the entire purpose of compassionate use for our kids.  Expanded use guidelines were clarified by the FDA a few short years ago, and they were clarified so that physicians and pharmaceutical companies alike could fully understand that compassionate use does not affect trials.  Their guidelines ensure that expanded access is provided as a treatment option for patients, not a data collection option for principal investigators or pharmaceutical companies.  In short – any expanded use will not impact the trial.  Not by any stretch.  And to further this point, I emailed Janet Woodcock at the FDA for a direct statement on the matter.  She promptly replied and said “…in our collective knowledge here at CDER, adverse events occurring during the development program have not delayed the programs.  In one case, we know the drug development was actually accelerated.”  One would have expected this information would be available to anyone who asked, including Shire Pharmaceuticals.

It is beyond me why the Boston Globe would neglect to present these viewpoints in their story.  Journalism is supposed to present an objective viewpoint on a trending topic.  It’s purview is to educate the public and allow for open discussion.  For that discussion to take place – for any fair and open discussion to take place – all the viewpoints need to be laid out.  Sadly, important information was precluded from the story, and I hope this entry helps clarify the matter once again.

Finally – Phil Vickers – never in this world would it be “ethically wrong” to try to save a child’s life.  EVER.

On another note, I emailed Shire CEO Flemming Ornskov yesterday to ask, once again, for a collaborative solution to this matter.  I am hopeful that Flemming and his team will agree to sit down directly with the FDA, Dr. Muenzer, and Dr. Burton to work through this matter, set a proper policy and guidelines for access to this investigational drug, and work to do what is best for our children.  ALL of our children.

With thanks for your continued support,

The Isaac Foundation

A Response to Shire

flemming2Hi Everyone,

Last night on NBC news, Shire Pharmaceuticals released a statement for the media (no personal interviews, sadly).  Our response to their statement is included below.

Once again, I’m calling on Shire to publicly address our case, point by point, with their rationale for not supporting expanded access use for Jack Fowler.  I’m happy to present the document publicly, and I call on Flemming Ornskov to be open, transparent, and honest with the Fowler family and the public, and provide the reasons that Shire cannot live up to their public motto by “Being Brave” to #SaveJack.


Shire’s statement reads:

We remain deeply sympathetic to Jack Fowler and his family. Patients have been, and will always be, at the heart of what we do, and have driven our desire to research potential new therapies for more than 20 years. Guidance from regulatory agencies is that expanded access to investigational medicines and devices outside of a clinical study setting should be based on the existence of compelling evidence of efficacy, or the drug’s benefit, and safety. Efficacy is typically studied in later stage trials; these data are not yet available for our investigational treatment as we have only just initiated our Phase II/III study. Shire does support, and has provided, expanded access at the appropriate stage in the development process-when clinically validated efficacy and safety information is available. We remain focused on completing our Phase II/III trial with the hope of making our investigational treatment available to as many Hunter syndrome patients as possible, as quickly as possible.

Source: http://www.nbcchicago.com/news/local/Parents-Fight-To-Get-Drug-For-Sick-Son-242704721.html#ixzz2rsxvW0L8

A few notes (in order of rebuttal):

1.  “Patients have been, and will always be, at the heart of what we do…”

  • Flemming Ornskov specifically said to the Fowler family, and I was present to witness this statement: “We don’t deal with patients.  We don’t deal with families.  We are in the business of developing products.”

2.  ” Guidance from regulatory agencies is that expanded access to investigational medicines and devices outside of a clinical study setting should be based on the existence of compelling evidence of efficacy…”

Richard Klein, a member of the Office of Special Health Issues at the FDA (the regulatory body authority that guides expanded access protocol) has stated a few things regarding efficacy (source information at bottom):

  • “Confidence of safety is more important than efficacy.”  
  • With regards to safety confidence, “for a patient with an immediate life-threatening condition, evidentiary burden is low.”  
  • Efficacy (and safety) of early phase investigational drugs not proven – and sometimes not known; however, might be given in hope of direct benefit to patient.”

3. “Shire does support, and has provided, expanded access at the appropriate stage in the development process-when clinically validated efficacy and safety information is available.”

  • Shire did not have a compassionate (or expanded use) policy in place when we approached them about Jack Fowler.  I have the transcript of our teleconference where we questioned them on this fact, and they agreed that there should be something in place but that there was not.  While they may have allowed this type of request in the past, they have had not set criteria to allow expanded access for patients until January of 2014, long after we approached them to #SaveJack.  Incidentally, those criteria are riddled with errors, and I will post our response to that policy soon.

4.  “We remain focused on completing our Phase II/III trial…”

Expanded Access to an investigation drug outside the clinical trial setting can HELP with clinical trials and, in some cases, speed up approval of a drug.  As well, any adverse affects that transpire while a patient is receiving Expanded Access to a medication DOES NOT IMPACT THE CLINICAL TRIAL in the slightest.  See below, direct from the FDA:

  • In an email to The Isaac Foundation, Janet Woodcock from the FDA stated, “…in our collective knowledge here at CDER, adverse events occurring during the development program have not delayed the programs.  In one case, we know the drug development was actually accelerated.”
  • The FDA has made a very clear statement on this matter, and included this information in their report on Expanded Access.   This report states: “Although adverse events first identified during expanded access use of certain drugs have been included in the drugs’ approved product labeling, we are unaware of any cases in which adverse event information obtained from expanded access use has resulted in denial of approval for a product.”
  • Former FDA Director Dr. Lester Crawford stated “The FDA, categorically, does not attach special significance to adverse events reported from such expanded access program as (one critic) has tried to join.  We recognize that these programs involve less-controlled use of new drugs, and we assess the reported data accordingly.  The development of a new medication is not slowed by side effects occurring outside clinical trials.”

Source from Richard Klein: http://www.fda.gov/downloads/ForIndustry/DevelopingProductsforRareDiseasesConditions/OOPDNewsArchive/UCM294794.pdf


Shire has released there guidelines for compassionate use (or expanded use) of their products. They didn’t have any criteria before we approached them, which continues to astound me. Regardless, their criteria is riddled with issues and inaccuracies, and I’ll post our response to it tomorrow.

In the meantime, the info graphic included below is a quote from Shire’s CEO Flemming Ornskov. Jamie Fowler, Jack’s mom, posted it on her page today, and it’s quite telling.

Till tomorrow,


An Open Letter To Flemming Ornskov – A Passionate and Poignant Message

photo-7The letter below is posted on Deb Purcell’s blog site over at treypurcell.com.  Deb is the director of our MPS II Fund, is a passionate advocate for children suffering from MPS, has been responsible for bringing so much awareness to the disease throughout Canada, and is one of the kindest and most caring people I have ever met.

Deb’s son Trey has MPS II and is currently enrolled in Shire’s clinical trial.  Her letter below was hand-delivered to Flemming Ornskov by myself and the Fowler Family during our now infamous 30 second meeting with the CEO.

Sadly, I have a hard time believing that he read it.  And I have a hard time believing he took the huge binders with over 32,000 signatures and comments from people around the globe supporting Jack.  Or the other letters from parents who have children currently enrolled in the trial.  And most sad of all, I expect he didn’t look or read the note that Jack’s mom left him.

Regardless, the message is poinient and is beautifully written.  And because our site has been visited an unbelievable amount of times throughout this campaign, I’m including it below for you to read, comment, and share.

Oh – and Shire has been spending the bulk of their day visiting our site and reading your comments and posts.  Perhaps Flemming will take a moment to read the thoughts below while he’s here.

Thanks, as always, for your love, care, and support.

The Isaac Foundation.

Originally Published On TreyPurell.com

Posted on  by 

Before I get on with the letter that Jamie and Jason Fowler hand delivered to Flemming Ornskov at their brief meeting in Chicago last Saturday, January 18 (more can be read at Jack’s new website: http://www.savejack.ca/), I am bursting with hopeful news. Due to new and close connections our MPS II community has developed with the FDA, this past week, our #SaveJack team connected directly with the FDA on one very important point. If an adverse event happened to Jack, would or could the FDA shut down the trial? This is a quote directly from the FDA this week:

‘As far as I know, and in our collective knowledge here at CDER, adverse events occurring during the development program have not delayed the programs.  In one case, we know the drug development was actually accelerated.’

Former FDA Commissioner Dr. Lester Crawford stated:

“The FDA, categorically, does not attach special significance to adverse events reported from such expanded access program as (one critic) has tried to join.  We recognize that these programs involve less-controlled use of new drugs, and we assess the reported data accordingly.  The development of a new medication is not slowed by side effects occurring outside clinical trials.”

In my eyes, this has been the only question holding us back. I can see from Shire’s perspective why they would not approve a drug for a child if it could threaten their clinical trial and shut down treatment for many other children (not including the financial burden they must look at). However, with the above answer straight from the horses mouth, there is no good reason not to submit an application to the FDA for Jack Fowler to receive Intrathecal Elaprase.

It will not harm their trial. A doctor and institution are ready. The finances are in place if there are any issues with funding. We just need Shire to say yes. With this new news, I am so so so hopeful that there is no longer anything holding Shire back from saving Jack’s life.

Please please please Flemming Ornskov and Phil Vickers, the decision has been made so easy for you!! Just say yes! And finally, here’s the letter:

Dear. Dr. Ornskov,

My name is Deb Purcell and I am the mother of a boy, Trey, who lives with MPS II. The long version of our journey is on our website at www.treypurcell.com. The short(er) version, I will share with you here.

Trey was diagnosed unexpectedly at 23 months of age on February 14, 2006. That diagnosis uprooted our lives. It changed everything about what it means to be a parent, what it means to be alive. It took my husband and I at least a year to adjust to what it meant to be parents of a child with MPS II. Most of our days were filled with tears and the only reasons we could find to live were our two children at the time, Trey and his 3-month old brother Avery.

One year after diagnosis, after a huge battle with our government to access the drug, Trey began IV Elaprase. With Elaprase giving us hope, life improved. However, as you know, 2/3 of kids with Hunter’s are cognitively affected. Beginning when Trey was two, every six months we traveled to North Carolina to have Trey’s IQ tested. With each test, we were told that the chances of Trey’s brain being affected were reduced. When Trey first tested for the Intrathecal trial at the age of five in 2009, Dr. Muenzer told us Trey’s brain would be okay. His IQ was stable and Trey was beyond the age at which his IQ would decline. When Trey was six, I asked Dr. Muenzer to test Trey one last time, to be sure. It was then we were told Trey’s brain was affected and Trey would be screened for the trial.

I cannot explain to you what it is like to live for four and a half years, wondering if my son would live or die. Because that is the difference between the severe and attenuated form of Hunter Syndrome to a parent. Attenuated means college, jobs, traveling, LIFE. Possibility. Severe means g-tubes, seizures, wheelchairs, safe rooms, vegetative state, and DEATH.

For four and a half years, every moment of my life was taken up by fear. If Trey had a toilet accident, if he hit his brother, if he forgot a word, if he put his shirt or pants or shoes on backwards, if he had a tantrum, if he didn’t finish a puzzle or scribbled on paper instead of drawing, if he choked on food, if he woke in the night, I was seized by terror and the possibility that Trey’s brain was affected. Even when Trey did great, when he learned a new word, when he shared, when he learned to cut with a knife or ride a bike or put his hearing aids in, I wondered if that meant his brain was okay. For four and a half years, I lived with indescribable fear.

When we learned Trey’s brain was affected, I had the second biggest collapse of my life, the first being upon diagnosis. When I got back up after that, I had something to fight for. The Intrathecal trial.

I planned how often to call and email Dr. Muenzer so that he would know we want Trey in the trial and are dedicated and not giving up, but not so much that I would put him off. We stopped turning on any movies or TV and hired daily tutors to keep Trey’s cognition high enough long enough so he would not drop below inclusion criteria. I interviewed doctors about symptoms of hydrocephalus so I could have some indication of whether or not Trey’s intracranial pressure (ICP) would exclude him. We did anything and everything we could- I could tell you more in person- to ensure Trey would qualify for the trial.

That qualifying trip to UNC was the hardest time of my life. There was a drug that would save my son’s life, and an arbitrary number would tell us whether or not he would get it. Waiting four hours while Trey was under general anesthetic for the qualifying tests is indescribable. I have never been so close to a panic attack. For the first two hours I distracted myself with reading and practicing yoga. As we got closer to the time when Dr. Muenzer would walk in that door and give us the news, I could only sit and stare in front of me. I could not talk to my mom or husband. Every time the waiting room door opened, my stomach jumped and my heart dropped. I began to feel nauseous and sweaty and shaky and after an hour and a half of this, I thought I was going to throw up, pass out or explode (I’m not exaggerating). When Dr. Muenzer walked in and told us Trey qualified, I collapsed with relief. Trey got to live. Trey would live. I couldn’t believe it. It took me months to understand what that really meant. Trey gets to live.

Life since Trey qualified for the IT trial has changed completely. Instead of a life of fear and loss and sadness, our family is LIVING. Really living. I don’t have to wonder what skill or word Trey will lose today or push away the thought of when he will die and what song I will play at his funeral.

When Trey first started receiving IT drug, I recorded every new thing he did. This was easy to notice because Trey had stopped making big gains. He lost 25 IQ points between August 2010 when his IQ qualified him for the trial and 14 months later when he got his first dose of IT drug in October of 2011. At that point Trey was down to one-syllabyl words and 3-4 word sentences. His proprioceptive and vestibular needs led him to hit and bang anything he could get his hands on and spank any bum within his reach. His younger siblings were not safe in a room alone with him.

Now? You really need to speak with my family and those who worked with him before and after. They will cry. EVERY LAST ONE OF THEM. There have been so many changes in Trey as a result of this IT drug, I could wrote a thesis on it, but here are a few:

I can’t even count how many words are in Trey’s sentences. His language has grown so incredibly that he uses complete sentences (he used to skip small words like ‘it’ ‘as’ ‘the’), joins sentences with the word because (never did he do that before) and uses descriptive language. Trey’s speech therapist noted that before IT drug, when she tried to play a simple game with him, he would grab pieces because he couldn’t wait and throw the game when he was done because he was frustrated and didn’t know how to say he was done. About six months ago Trey threw the game and when she asked why, he said ‘Because I am done with the game and I wanted to throw the game so the pieces would go over your head.’ More recently, Trey told her he didn’t want to play so they stopped, and about two months ago, she pushed Trey even farther, telling him she wasn’t done playing so he had to wait, so he sat and waited. A month ago he kept playing until the game was over, even though he was done.

It’s small details like these that are noticeable and some I don’t even notice right away. It was random one day that I suddenly realized Trey no longer has a need to drum and hit, that he no longer smacks people’s bums.

He began to hug us. Before the trial, I could tell Trey wanted to hug, but he would lean in and back right out again. He couldn’t hug. Now we hug for hours, which as a mom told her child was going to die, is by far the best perk.

Trey is learning to swim. He is learning his letter sounds and learning to read. I never thought he would. Trey now understands one-to-one correlation with numbers and is learning to add and subtract. No matter how many times I tried, he never made this connection with numbers before.

Our family is learning ASL and for the first time Trey has the attention to sit for an hour lesson and actually attempts to form signs with his hands.

The biggest change is quality of life. Trey can be alone with his siblings, he understands not to touch sharp knives or touch hot stoves. I can tell him to go to bed and he will. For the first time, Trey is beginning to understand and make connections. When I was trying to figure out if Trey’s brain was affected or not, Melissa Hogan described ‘passing conversations’ to me and it became clear as day. When you talk to a child who is cognitively affected, it’s like you’re two cars on a highway passing each other. The connection is missed. Trey was just like that. Every day now I see new connections being made in Trey’s brain. I feel like I can see the IT Idursulfase clearing new pathways in his brain. It is so so very awesome. Trey has a progressive disease that is no longer progressive and is actually reversing itself. I keep wondering how I got so lucky and if this really is for real.

Jamie could have been me and I could have been her. Jack could have qualified for the IT trial and Trey could have been excluded. It was the flip of a coin. Chance. What would I want her to do for me if I were in her shoes? How hard would I need her to fight for my son’s life? These are questions I ask myself.

I will fight for Jack and I will fight for Trey because they are both my boys. This means fighting until Jack gets access to drug because as a parent, it is incomprehensible to me that anyone thinks another parent could give up on their child’s life. It also means fighting to get this drug approved if anyone gets in the way. If there is one thing MPS parents learn how to do better than anything else, it is to fight. We have been fighting systems, institutions, governments, schools, hospitals, insurance and pharmaceutical companies, and injustice since the day our children were diagnosed.

What would I do to get this drug approved? ANYTHING. What would you do to keep your child alive? ANYTHING. This drug has saved Trey’s life. I would do anything to get this drug approved because it is the only thing keeping Trey alive. This is not even a mild exaggeration. That some ponder its efficacy is shocking. It makes me realize that those people have no connection to families with Hunter Syndrome. This drug is a miracle.


Deb Purcell


Request for Collaborative Approach – An Open Letter To Flemming Ornskov

vickers-quote---fowlerDear Mr. Ornskov and Mr. Vickers,

I am writing to request an opportunity for you to review information we have gathered to support an Individual, emergency use, expanded access application for Jack Fowler.  I am very confident that the document we have prepared will present a very compelling case to allow access to your investigational drug SHP-609 outside of the clinical trial setting.  After consultations with the FDA and some of the most experienced and powerful people throughout the pharmaceutical world, I truly believe that allowing this application to move forward to the FDA can and will work to meet the the best interests of the patient, the Hunter community as a whole, and Shire plc.  We are firm in our belief that the information we have gathered to support this application enhances the clinical trial process, which in turn is a direct benefit to Shire.


If you are interested in working collaboratively to meet the needs of the entire patient community that you hope to represent, please indicate in writing if you will accept our document that lays out our position.  I would very much like to receive your feedback and counter arguments against supporting this application, with hopes that we can work together to come to a resolution.  Perhaps an open and honest discussion could follow as we continue to find the best way to meet the needs of this patient while supporting and enhancing the clinical trial that is currently underway.


I look forward to hearing from you with a response within the next 24 hours.


Very Sincerely,


Andrew McFadyen

The Isaac Foundation
“Love, Laughter, and Hope”


Meeting With Shire – Update

flemmingHi Everyone,

It’s with a heavy heart, and with anger and frustration, that I update you on the meeting that took place this morning between the Fowler family, The Isaac Foundation, and Shire Pharmaceuticals.  The meeting was organized after Shire’s decision to deny Jack Fowler the life saving treatment he immediately requires.

This meeting was supposed to take place at the Fowler home, 45 minutes outside of the city of Chicago.  It was set to take place at 1:30 p.m.  However, a few days before the meeting, Shire’s CEO Flemming Ornskov abruptly changed the location of the meeting to the Chicago airport, and changed the time to an unseemly 7:30 am.  The Fowler family struggled to put child care in place.  And when you are dealing with a special-needs child, that is no easy task.

We made it to the meeting on time, and brought Jack in with us to meet with the Shire team.  Present members for Shire were CEO Flemming Ornskov and Head of Research and Development, Phil Vickers.

The Isaac Foundation began the meeting by thanking Shire for taking the time to meet with the Fowler family.  We expressed that the purpose of the meeting from our point of view was to discover how we could work collaboratively with Shire Pharmaceuticals so that we can find the best treatment options for Jack Fowler and in a timely fashion.  We expressed that if Shire was present to simply reiterate their position from December and deny Jack the treatment he needs, then the meeting would need to come to an abrupt end.  I made very clear to Mr. Ornskov one very simple fact – an immoral decision is still immoral, even if it’s delivered while looking us in the eye and said to our face.

Flemming looked at us and said “We are not changing our decision.  I guess this meeting is over.”  With that, the hopes of the Fowler family were dashed, and our hope to work together with Shire to save this little boy was ended.   We left a large print out of the 32,000 signatures that were signed in the online petition, as well as letters of support from a high percentage of the families currently participating in the clinical trial of the drug that Jack desperately needs.

As we were leaving, Jack walked around the table and gave Flemming a hug.  Close to tears, Jack’s mom said “If he could talk, he would be asking you to Be Brave, like your motto says, and save him.”  It was one of the most heartbreaking moments of my life – watching a sweet little boy who doesn’t have any idea what is going on give a hug to the man who just gave him a certain death sentence.  I told Flemming that I had a hard time understanding how he can go home and look his children in the face after that moment.  He just looked at us and said “It was nice to meet the family.”

The facts of this case remain the same, and are very clear.  Jack Fowler needs access to a drug that will save his life.  He easily qualifies for individual use access through the FDA’s Expanded Use guidelines.  It is those guidelines that decide whether any investigational drug is safe for use outside the bounds of a clinical trial.  The question of whether enough safety data exists to proceed or not doesn’t rest with Shire Pharmaceuticals, nor does it rest with any pharmaceutical undergoing the same process.  It rests with the FDA first and foremost, and it rests with the physician in charge of treating the patient.  All Shire has to do is begin the application process on Jack’s behalf and leave the decision to the FDA.  When I stated this very clearly to Mr. Ornskov and asked if he would submit the application, he flatly refused.

There are things in this world that many people would be better off not knowing.  What lurks in the minds of pharmaceutical decision makers should be at the top of everyone’s list.  To have the ability to provide help and support, to be able to save the life of a precious little boy, and then choose not to, shows a callous disregard toward life.  That callous disregard is a painful reminder of what Big Pharma is after – money, product, fortune, and fame.  Don’t ever be fooled that the patient comes first.  As Flemming so plainly stated before I gave my introduction – “We don’t work with patients.  We don’t work with families.  We are in the business of developing product.”

With that, there’s nothing left to be said.

Our press release goes out early this week.  Stay tuned on how you can help us #SaveJack.  We will never quit when the life of a child hangs in the balance.

Thank you for your ongoing and tremendous support.

With Love,

The Isaac Foundation