Yael, 18, suffers from cystic fibrosis (CF) with pancreatic insufficiency and CF-related diabetes. At 14, she developed a serious lung disease and required intensive daily treatment. When the news of a new miracle drug called Trikafta came to light, it was disappointing that the drug wasn’t approved for Yael’s particular mutation under the Israeli health basket. The drug costs $24,000 a month.
Undeterred, Yael started a national campaign to try the pills, asking for samples from patients whose mutations were approved for treatment. At the Cystic Fibrosis Center at Hadassah Hospital Mount Scopus, the doctors agreed to create a clinical trial for her. Within a few days, her coughing stopped, her weight improved, and she was no longer insulin dependent. When news of the trial went out from Israel, a doctor in France and another in the US started treating their patients who shared Yael’s mutation. Eventually, the US Food and Drug Administration approved Trikafta for a further 94 mutations.
Rather than testing drugs on a patient — in this case, on the organs of a CF patient — Hadassah researchers can conduct the tests in a lab using organoids, miniaturized and simplified versions of cells or tissue produced in vitro in three dimensions that mimic the key functional, structural and biological complexity of those cells or tissue.
As doctors at the Cystic Fibrosis Center continue delineating additional mutations for which the drug would be effective, trials can be carried out on these organoids. If their lab proves that Trikafta works for an individual patient, health funds will cover the cost of the drug, thereby changing their life forever.
Says Dr. Liron Birimberg-Schwartz, clinical director of the Organoid Center at Hadassah Hospital Ein Kerem, “This is the ultimate personalized medicine.”
