By Sandra L. Burr, LL.M. Candidate
A local television station recently aired the movie Lorenzo's Oil. You may recall this movie, starring Nick Nolte and Susan Sarandon, was based on the true story of Lorenzo Odone's battle with akrenoleukodystrophy (ALD) and the efforts of his parents, Michaela and Augusto, to discover the cause of and a cure for the disease. Following the movie credits is a reference to THE MYELIN PROJECT. Seeing the movie again left me wondering about Lorenzo's progress, the outcome of his parents efforts, and what, if anything, The Myelin Project actually accomplished.
Lorenzo was diagnoised with ALD at age five. ALD is an inheritable metabolic disease in which a gene mutation causes the production of a malfunctioning protein in peroxisomes. Peroxisomes are used by cells to break down very long chain fatty acids (VLCSFAs) and when malfunctioning, result in a build up of VLCSFAs. This build up causes demyelination of cells within the nervous system resulting in the slowing and ultimate stopage of nerve impulses. The disease progresses from hyperactivity through aophasia, blindness, hearing loss, paralysis, loss of speech, dementia to death. ALD affects only males and is inherited from the mother. Anna Arnaudo, The Human and Scientific Story of Adrenoleukodystrophy, available at http://serendip.brynmawr.edu/bb/neuro/neur00/web/Arnaudo.html
As chronicled in the movie, Michaela
and Augusto Odone, although laypersons, were unwilling to accept the medical
community's lack of answers or treatment options for their son. Through
intensive research the Odones determined that a mixture of oleic and euric
acids would inhibit ALD develpment. This mixture is known as Lorenzo's
Oil. However, the oil does not repair or remyelinate damaged nerve axioms.
Bradley J. Stith, Ph.D., The Use of the Movie "Lorenzo's Oil" as a Teaching
Tool, available at
While searching for answers to their son's illness, the Odones were frustrated by the complexities, political infighting, and slow pace of medical research related to ALD and remyelination. In hopes of moving research forward, the Odones created The Myelin Project. The mission of the project is to accelerate research on myelin repair by creating a framework to encourge researchers to cooperate effectively, provide prompt financing, and allow families dealing with a demyelinating disease to interact with the scientific community. Funds raised by the project are used to finance clinically oriented experiments in remyelination research. Basic research and studies are excluded. Funding requests are processed in a matter of weeks using a fast track "no red tape" approach. As of December 2001, funds totaling $4,000,000 have financed 28 experiments, including the historically significant Yale University Schwann Cell Transplant Trial. The Myelin Project, Introduction, available at http://www.myelin.org/introduction.html.
The purpose of the Yale trial is to assess the viability and safety of transplanting Schwann cells into the brains of MS patients. Schwann cells produce myelin in the peripheral nervious system. The Yale trial uses autologous transplantation in which cells are harvested from the patient's own body and implanted into the brain.This is the first such transplantation in medical history. The first patient, a 53-year-old woman, received one million cells in July 2001. A follow-up biopsy, performed in January 2002, confirmed no abnormalites and reseachers are awaiting test results to determine whether the transplanted cells have survived and whether they are producing myelin. A second trial patient was transplanted on March 7, 2002, and the remaining three scheduled transplant procedures will be performed over the next six months. Each patient will receive progressively more cells with the final two patients receiving 10 million cells each. See Update: Schwann Cell Transplantation Trial in MS available at http://www.myelin.org/schwann_cell_update.html.
Should the Yale trial prove successful, it is anticipated similiar procedures will be used in other demyelinating diseases. Additional research, funded by The Myelin Project, is currently being conducted to determine if olfactory ensheathing cells and oligodendrocyte precursors derived from stem cells are viable for transplant. See Update: Schwann Cell Transplantation Trial in MS available at http://www.myelin.org/schwann_cell_update.html.
As to Lorenzo, he will celebrate his 24th birthday on May 29, 2002 and continues to communicate through the use of eye and finger movement. Although Michaela Odone died from cancer in June 2000, Augusto continues to lead The Myelin Project. Materials from The Myelin Project reference the aphorism "fortune favors the brave." See http://www.myelin.org. Lets hope it continues to do so.