50 years ago, enzyme injections showed promise for treating a rare disease
Excerpt from the April 28, 1973 difficulty of Science Information
Extra Tales from Science Information on Well being & Medication
First profitable enzyme remedy in people — Science 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.
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).
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