I wanted to start this blog to share our family's experience with HNPP. There is not a lot of information available so the more we can share the more we can help each other and help in the research process. We would love to hear from you too.
- Our Family.
My sister in law told me about the movie Lorenzo's Oil.
From the website:
The film is based on the true story of Augusto and Michaela Odone and their son Lorenzo. In 1984 Lorenzo came down with adrenoleukodystrophy (ALD), a rare inherited disease. Doctors said that he would lose all his functions and die within two to three years. Refusing to accept this grim verdict, the Odones set out on a mission to find a treatment for ALD and to save their child (Lorenzo survived, and is now 25 years old). In their quest for a treatment the Odones often clashed with doctors, scientists, and support groups, who were skeptical that anything could be done about ALD, much less by laypeople. Their relentless struggle tested the strength of their marriage, the depth of their beliefs, and the boundaries of conventional medicine.
The Odones haunted medical libraries, reviewed countless animal experiments, badgered researchers, questioned top doctors all over the world, and persisted until a solution came to them in a moment of inspiration. They commissioned a special type of oil from a British firm, which normalized the accumulation of very long chain fatty acids in the brain, the hallmark of ALD.
The film ends on a positive note, showing several healthy children who, having followed a course of treatment with Lorenzo's Oil, remained symptom-free. The conclusion relates that Lorenzo can communicate again by a modified sign language, and that Augusto Odone was awarded an honorary Ph.D. for his pioneering work in researching and discovering a significant treatment for ALD.
In the second chapter of the story, the Odones founded The Myelin Project in the hope of finding a way to restore the myelin sheath, which is destroyed in ALD and a host of other myelin diseases, such as multiple sclerosis. If this vital substance can be restored, patients like Lorenzo may regain function and eventually lead a normal life.
A good friend of mine has just been diagnosed with MS after a very quick functional decline. I feel deeply for her and it has plunged me back into researching what is currently being done and available. Oh, how many of us would be blessed if myelin regeneration possibilities can be discovered. It is hopeful and I plan to look in on their progress often. I thought you might like to know about The Myelin Project and what they are doing to help or even get involved.
While The Myelin Project has taken the conventional steps to advance remyelination research, we have taken the unconventional step of encouraging direct interaction between families and scientists, we continuously remind the scientists that there are people waiting for effective treatments.
Research has been focused on efforts to understand mechanisms of myelin repair, with a view to discovering therapies that will permit restoration of damaged myelin. It is anticipated that these will permit at least partial restoration of function in patients suffering from myelin loss.