Augusto and Michaela Odone founded The Myelin Project in 1989. Their son, Lorenzo of the movie “Lorenzo’s Oil,” was diagnosed at the age of six with adrenoleukodystrophy, or ALD. This rare disease, which is caused by a genetic mutation that results in a breakdown of the neurological system, leads to an accumulation of fatty acids that damages the myelin sheath, which insulates every nerve of the brain and spinal cord. Like the plastic sheath around an electrical wire, myelin assures the proper conduction of nerve impulses from one part of the body to another. When this sheath is damaged, the results can be devastating.
The Myelin Project and Oliver’s Army are now co-funding the Augusto Odone New Investigator Award. Young scientists embarking on their research careers are invited to submit their plans for a project aimed at finding new treatments for adrenomyeloneuropathy (AMN). AMN and the more well known X-linked adrenoleukodystrophy (or X-ALD) are debilitating disorders caused by a mutation in the same gene (known as the ALD gene). While X-ALD has been the focus of much research, very little is known about AMN.
“We want to get the word out to scientists everywhere that this major research grant is available from our organization,” said Candace Root, executive director, The Myelin Project. “If a researcher has submitted for federal stimulus money and did not receive a grant we want them to know that other grant opportunites are available. We plan to fund this grant in the fall and our call for proposals is open now.”
About Adrenoleukodystrophy or X-ALD: This genetic disease affects about one-third of boys who carry the X-ALD gene, causing loss of most normal neurologic functions within one or two years. Fewer than 20 years ago, X-ALD was invariably fatal. Thanks, in part, to The Myelin Project, substantial progress has been made in treating X-ALD. Treating pre-symptomatic boys with Lorenzo’s Oil significantly reduces the chances that X-ALD will develop. For those boys who develop symptoms despite Lorenzo’s Oil therapy, there remains the option of a bone marrow or cord blood transplant. While still risky, the success rates of bone marrow transplantation have improved significantly in recent years.
About Adrenomyeloneuropathy or AMN: This disorder affects the other two-thirds of the males and about one-half of the females who carry the X-ALD gene. AMN is part of the complex of diseases caused by mutations in the ALD gene. In an affected family in which two boys have the same mutation, one may develop ALD and die early, while the other may remain asymptomatic until his 20’s when he develops AMN. X-ALD and AMN have quite different symptoms. AMN is characterized by loss of spinal cord and peripheral nerve function resulting in stiffness, ataxia, weakness to paralysis and sometimes cognitive defects showing that in some men with AMN, the brain is also involved. In men, symptoms of AMN most often develop between 20 and 30 years of age, when they are just beginning to build a career and start a family. Women become symptomatic later in life, in their 40’s or 50’s, and while the symptoms are usually less severe than men with AMN, they can be serious and debilitating.
For more information contact: Candace Root, Executive Director, The Myelin Project, (800) 869-3546, https://www.myelin.org