50 years ago, enzyme injections showed promise for treating a rare disease


Excerpt from the April 28, 1973 difficulty of Science Information

An illustration of two doctors carrying a giant syringe on their shoulders.

At the moment, a number of uncommon illnesses are handled through enzyme substitute remedy, and scientists have even discovered a solution to deal with one illness earlier than an individual is even born.

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First profitable enzyme remedy in peopleScience Information, April 28, 1973

Fabry’s illness … [is caused by] a defective fat-metabolizing enzyme known as alpha-galactosidase. As a result of the irregular enzyme doesn’t break down lipids within the blood because it ought to, they accumulate within the physique. [Researchers] tried to purify the enzyme in its regular type from human placental tissue. 4 months in the past they acquired sufficient of the enzyme to inject into two sufferers…. The outcomes look promising.

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Although the trial confirmed that enzyme substitute remedy may work, it took a long time earlier than such remedies have been cleared to be used by the U.S. Meals and Drug Administration. In 1991, an injection to deal with a distinct uncommon inherited dysfunction known as Gaucher illness turned the primary to achieve approval (SN: 11/23/74; SN: 5/25/91). Approval for an enzyme substitute remedy for Fabry’s illness adopted in 2003. Scientists are nonetheless growing enzyme therapies. Final yr, researchers reported treating a growing fetus for Pompe illness, which causes extreme coronary heart and muscle defects (SN: 11/9/22).

Maria Temming

Beforehand the workers author for bodily sciences at Science Information, Maria Temming is the assistant editor at Science Information Explores. She has bachelor’s levels in physics and English, and a grasp’s in science writing.

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