Opinion: Bench to Bedside

A chance discovery made in my lab 17 years ago results in the first drug that can help patients with a rare disease.

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Daniel Drucker, Samuel Lunenfeld Research Institute at Mount Sinai HospitalIMAGE COURTESY OF ANNIE TONG, MOUNT SINAI HOSPITALIt’s hard to describe the feeling that washed over me when a discovery I’d made with colleagues in Toronto in 1995 translated earlier this year into the first real meaningful treatment for patients with short bowel syndrome in the history of this disease.

We were not the first to make the connection between glucagon and bowel growth—that happened in 1971—but we re-produced the same observation, identifying GLP-2 (a gut hormone) as the active peptide critical to the development of a new drug, called teduglutide, or Gattex, manufactured by the company NPS. Our drug discovery, which began with a moment of serendipity almost 20 years ago, finally became available to patients this year.

Short bowel syndrome occurs in patients who have had half or more of their small intestine removed. As a result, these patients cannot absorb enough water, vitamins, or other nutrients required by the body. Surgery is typically performed to remove a part of the small intestine after some sort of intestinal damage due to Crohn’s disease, ...

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