Former President Donald Trump signed legislation into law to make it easier for people with life-threatening illnesses to try drugs and treatments that aren’t yet fully approved by the Food and Drug Administration. This policy is called “Right to Try”.
A good step in the right direction, but what does it matter if drug companies won’t sell you the experimental drug or if hospitals refuse to administer it?
Luckily for a little man with a rare disease, Denver’s Children’s Hospital and CU Anschutz Medical Campus have the courage to put patients first when others don’t.
Imagine your child has a disease so rare it has no name. Heck, ALS, also known as Lou Gehrig’s disease, gets two.
Six-year-old Maxwell Freed has a neurological condition so unusual it’s characterized only by its genetic location, SLC6A1. It has resulted in severe physical and developmental delays and about 40 seizures a day.
Max’s mother, Amber, began investigating the condition only to find nothing was found. She found out the name of a doctor working on gene therapy for rare diseases, but he could not be reached.
She went after him like a bounty hunter. When she learned he was attending a medical conference in Washington DC, she hopped on a last-minute flight and sandbagged him.
dr Steven Gray of the University of Texas Southwestern Medical Center gave her hope for a gene therapy treatment for Maxwell to potentially repair his DNA.
There were just a few minor hurdles: the FDA didn’t approve it, drug companies might not sell the treatment, and there might not be a hospital that would approve the treatment.
And one more little thing: It could cost up to $7 million, which Amber and her husband shamefully didn’t have lying around.
Amber decided to “fight like a mother” for her son by starting a GoFundMe page and running a non-stop PR campaign. She raised the million dollars needed to get the process started.
Bigger headaches came from the pharmaceutical complex. Right to Try does not mean pharmaceutical companies have to participate. They are very reluctant to experiment with drugs they may already have on the shelf.
Congress says you have a right to try, but a much more powerful branch of government, the regulatory state, is holding you back.
Drug companies don’t want to risk angering the FDA for fear they’ll tie up potentially profitable drugs to the death of bureaucracy. Why launch a drug that could only help a handful of people when it threatens the pharmaceutical company’s viability?
So Amber went to war with the doctors and the drug companies. To control Max’s seizures, she found a drug that she knew would work, but they wouldn’t give it to her. She asked other patients for additional doses. During video conferences with doctors and pharmaceutical companies, the bottle of the desired drug would always be there in front of the camera.
When asked how she got it. She said, “Oh, they fell off a truck. I guess we’ll have to use them if we can’t get your meds officially.” Amazingly, after sharing this story with pharmaceutical journals and TV stations, they broke down and gave her the meds to successfully control Max’s seizures.
After successful clinical trials, gene therapy is about to be applied to her son.
Amber’s family lives in Dallas, which has some of the best medical care networks in the country. But Max may not be able to get the treatment there.
For the same reasons that the pharmaceutical industry doesn’t want to upset the FDA just to save a handful of lives, hospitals don’t want to jeopardize their status with procedures that haven’t been fully vetted.
And that’s where Children’s Hospital Colorado and CU Anschutz Medical come in. You are ready to put Max first.
This fall, Team Maxwell will fly to Denver for treatment. If successful, he could live a long and normal life.
There are many heroes in this story namely Amber Freed who will overcome any obstacle to save her child.
dr Gray cared more about this tiny patient than about his practice.
And finally, the brave medical system on the Anschutz campus is ready to put children before bureaucracy.
There is only one villain in this story – the unaccountable, bloated regulatory state that puts the paperwork before the 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 in Colorado Politics on Sundays.