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

CHICAGO SUN-TIMES

Should 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

Hi 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

jamie fowler

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

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

Meeting With Shire – Update