Chickens Playing Their Part to Fight Rare Disease

The FDA approved a breakthrough therapy to fight a rare disease, and some may be surprised to learn that chickens are the main source of this life-saving medicine. Kanuma, produced by Alexion Pharmaceuticals, Inc., is a human biologic created by purified egg whites from genetically engineered (GE) chickens to treat humans with lysosomal acid lipase (LAL) deficiency.

Those individuals with LAL deficiency (sometimes known as Wolman disease) have little or no LAL enzyme activity. As a result, fats build up in the cells of various tissues, which can lead to liver and cardiovascular disease. The disease typically presents itself in infancy, and the life expectancy for individuals with the disease is typically <1 year.

In an attempt to create a treatment for the disease, researchers engineered chickens to produce a recombinant form of human lysosomal acid lipase (rhLAL) protein in their egg whites.

This rhLAL protein functions in place of the inactive or partially inactive LAL protein in humans with LAL deficiency. In a clinical trial, 6 of 9 infants with LAL deficiency that were treated with Kanuma were alive at 12 months of age. Treatment with rhLAL is given via intravenous infusion once a week in rapidly progressing patients 6 months of age and younger and once every other week in other patients.

The Center for Veterinary Medicine (CVM) assessed the safety of the rDNA construct, as well as the safety of the chickens and did not note any adverse outcomes in the chickens. In addition, the CVM determined that the GE chickens did not cause any significant impact on the environment, as the chickens are raised and kept in highly secure indoor facilities and both the chickens and the eggs are kept out of the food supply. Regular inspections are conducted to confirm this, as well.