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Jon Caldara

Former President Donald Trump signed a bill to make it easier for folks with life-threatening illnesses to try drugs and treatments that are not yet fully approved by the Food and Drug Administration. This policy is called “Right to Try.”

A fine step in the right direction and all, but what does it matter if drug companies won’t sell you the experimental drug or if hospitals refuse to administer it?

Fortunately for one little man with a rare disease, Denver’s Children’s Hospital and the CU Anschutz Medical Campus have the courage to put patients first when others won’t.

Imagine your child having a disease so rare it doesn’t have a name. Heck, ALS, also called Lou Gehrig’s disease, gets two.

Six-year-old Maxwell Freed has a neurological disease so uncommon it is labeled only by its genetic location, SLC6A1. It has resulted in severe physical and developmental delays and around 40 seizures a day.

Max’s mom, Amber, started researching the condition only to find there was nothing to be found. She did unearth the name of one doctor who was working on gene therapies for rare diseases, but he couldn’t be reached.

She went after him like a bounty hunter. Learning he’d be attending a medical conference in Washington D.C., she hopped on a last-minute flight and sandbagged him.

Dr. Steven Gray of the University of Texas Southwestern Medical Center gave her hope of a gene therapy treatment for Maxwell to potentially repair his DNA.

There were only a couple of tiny hurdles: the FDA hasn’t approved it, pharmaceutical companies might not sell the treatment and there may be no hospital that would allow the treatments to go forward.

And just another tiny thing: it could cost up to $7 million, which Amber and her husband shamefully did not have lying around.

Amber decided “to fight like a mother” for her son starting a GoFundMe page and going on a non-stop public relations campaign. She raised the million dollars it takes to get the process going.

Bigger headaches came from the pharmaceutical complex. Right to Try doesn’t mean drug companies must participate. They are very hesitant to experiment with drugs they might already have on the shelf.

Congress says you have a right to try, but a much more powerful branch of government, the regulatory state, dissuades it.

Pharmaceutical companies don’t want to risk angering the FDA for fear of them tying up potentially profitable drugs in the death of red tape. Why release a drug that might help only a handful of people if it threatens the drug company’s viability?

So, off to war with the doctors and the pharmaceutical companies Amber went. To control Max’s seizures, she found a drug she knew would work but they wouldn’t give it to her. She begged other patients for extra doses. When video conferencing with the doctors and drug companies the bottle of the desired drug would always be there on camera.

When asked how she got them. She said, “Oh, they fell off a truck. Guess we’ll have to use them if we can’t get your drugs officially.” After sharing this story with pharmaceutical journals and television outlets, amazingly, they broke down and gave her the drugs to successfully control Max’s seizures.

Now after fruitful clinical trials the gene therapy is getting close to being used on her son.

Amber’s family live in Dallas, which has some of the best medical care networks in the country. Yet Max might not be able to get the treatment there.

For the same reasons pharma doesn’t want to anger the FDA to save just a handful of lives, hospitals don’t want to endanger their status with procedures that haven’t been fully vetted.

And that’s where Children’s Hospital Colorado and CU Anschutz Medical come in. They’re willing to put Max first.

This autumn, Team Maxwell will fly to Denver to be treated. If successful, he could live a long and normal life.

There are many heroes to this story, namely Amber Freed, who will conquer any obstacle to save her child.

Dr. Gray cared more about this tiny patient than about his practice.

And finally, the courageous medical system on the Anschutz campus is willing to put children before bureaucracy.

There is only one villain in this tale — the unaccountable, bloated governmental regulatory state that puts paperwork before people.

Jon Caldara is president of the Independence Institute in Denver and hosts "The Devil's Advocate with Jon Caldara" on Colorado Public Television Channel 12. His column appears Sundays in Colorado Politics.

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