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Applying Rare Disease Discoveries to Diabetes

Patients With Rare Metabolic Conditions Yield Insights Into Common Ailments

overweight patient talking with doctor

Young doctors are taught the adage, “When you hear hoofbeats, think horses, not zebras.” Just as you would not expect to find a zebra in your barn, when making a diagnosis, you generally don’t expect to find an unusual disease. However, just as there are still zebras in the world, there are also rare diseases. On Rare Disease Day, celebrated on the last day of February each year, we call attention to the 300 million people affected by them.

Defined as conditions that affect fewer than 1 in 2,000 people, rare diseases pose both challenges and opportunities to medical research. Because they are so uncommon, diagnosis can take years, and treatments may be difficult to obtain or may not yet exist. However, for IRP Lasker Clinical Research Scholar Rebecca J. Brown, M.D., M.H.Sc., the study of several rare metabolic diseases may shed light on the causes of one of the most common diseases in the U.S.

Cellular Self-Destruct Tied to Type 2 Diabetes

Study Suggests New Treatment Strategy for Increasingly Common Disease

a cell undergoing apoptosis

In the classic sci-fi film Alien, the protagonist attempts to destroy the titular monster by triggering the self-destruct mechanism on her spaceship. Our cells also sometimes need to destroy themselves in order to circumvent threats like cancer, but uncontrolled cell death can lead to disease. New IRP research suggests that preventing certain cells in the pancreas from tripping their self-destruct switch could help relieve the symptoms of type 2 diabetes.

Chemical Tag-Team Shows Promise as Diabetes Treatment

Therapeutic Strategy Enhances Natural Blood Sugar Control

home blood sugar testing device

Just like Sonny needed Cher to achieve music super-stardom and Stephen Curry needed Kevin Durant to win back-to-back NBA championships, sometimes a cell or molecule in the human body needs a partner’s assistance to work optimally. IRP researchers recently showed that a synergy between a lab-designed drug and a molecule naturally produced in the body could make for a promising therapy for type 2 diabetes.