By Alex Gariti
Clinical ethics consultation lives at the intersection of medicine and moral uncertainty. It is where abstract principles meet real families, real risks, and real consequences. Sometimes the work is quiet. Sometimes it is wrenching. And sometimes, it is astonishing.
Recently, we published a case about a 9-month-old infant with cystic fibrosis (CF) that forced us to confront a difficult question: What should a physician do when a parent pursues an unapproved treatment that is risky… but working?
The Background
Baby M was born with cystic fibrosis, a genetic condition that causes thick mucus to build up in organs like the lungs and pancreas. In her case, the mucus blocked her pancreas from releasing digestive enzymes. Without those enzymes, she couldn’t properly absorb nutrients from food. This condition (called pancreatic insufficiency) is typically considered irreversible.
The standard treatment is lifelong pancreatic enzyme replacement therapy: capsules taken before every meal, every day, for life. These help with digestion, but they don’t restore a functioning pancreas.
In recent years, however, a new class of medications known as CFTR modulators has transformed CF care. These drugs help the defective protein underlying CF function more effectively. For many patients, they have dramatically improved lung function and quality of life.
There was one problem: the most effective of these drugs was not approved for children under two years old. Baby M was only nine months.
Her mother, Mrs. M, was not an ordinary parent. She read the scientific literature. She understood the drug’s mechanism. She evaluated risks. When she could not obtain the medication through traditional medical channels, she found an alternative source and purchased it herself.
Then she made an extraordinary decision.
Because some of the medication is known to pass into breastmilk, Mrs. M began taking the drug herself and breastfeeding Baby M, hoping small amounts would reach her daughter.
The Result
It worked.
Baby M’s pancreatic enzyme levels, previously in the severely deficient range, began to rise. For the first time, her pancreas showed signs of functioning.
Her medical team monitored closely for known side effects, including liver injury and cataracts. None appeared.
Months later, when enzyme levels dipped again as Baby M transitioned to solid foods, Mrs. M took another step: she began giving the medication directly to her daughter at a conservative dose. Her physician, though unable to recommend the approach, advised minimizing risk by starting at a very low dose.
Within weeks, Baby M’s pancreas was producing sufficient enzymes. She stopped enzyme replacement therapy entirely. What had been considered irreversible had, at least for now, reversed.
No side effects emerged.
The Ethical Tension
The case came to our ethics service with a pressing question: What is a physician’s obligation when a parent is using an unapproved medication in a child?
Should he try to stop her?
Report her?
Refuse to participate?
Or help reduce the risks of something he could not endorse?
This was not a case of obvious harm. The treatment posed potential risks but no observed harm. At the same time, the benefits were concrete and measurable.
In situations where parental decisions clearly harm a child, physicians must intervene. When decisions are harmless but ineffective, they may tolerate them. But what about when risk is uncertain and benefit is real?
We recommended a calibrated response: do not prescribe or formally endorse the treatment, but continue close monitoring. If harm emerged, intervene. If benefit continued without injury, maintain oversight.
In other words, harm reduction.
This approach acknowledges a difficult truth: refusing to engage does not eliminate risk. It simply removes medical oversight. When parents are determined, walking away may make a child less safe, not more.
Courage on Both Sides
It would have been easier for the physician to draw a line, to end the relationship or decline further involvement. Instead, he chose the harder path. He remained present. He monitored. He advised caution. He absorbed the moral discomfort of practicing in uncertainty.
This case is not an argument for bypassing regulatory safeguards. Clinical trials and age approvals exist for good reason. But it is a reminder that medicine sometimes advances at the bedside before it advances in policy.
Clinical ethics is rarely about choosing between good and bad. More often, it is about navigating between competing goods and uncertain risks. In this case, a mother’s persistence, a physician’s humility, and careful ethical reflection converged in a remarkable outcome.
Baby M’s story challenges us to ask: When innovation moves faster than regulation, how should medicine respond?
Sometimes, the most ethical path is not to shut the door but to stay in the room.
Author(s): Alexander Gariti, MBE, HEC-C
Affiliation(s): Baylor College of Medicine
Conflict of Interest: None to declare
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