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 (Illinois) 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 one’s 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.

Written by Monifa Thomas for the Chicago Sun-Times, February 16, 2014. Additional contributions by Chris Fusco.

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