Dr. Leslie Gordon and Dr. Scott Berns:
Although Progeria had been identified in 1886 in England by Dr. Jonathon Hutchinson, and again in 1897 by Dr. Hastings Gilford, in the summer of 1998, there were no resources, no known treatments, support groups, or any information about the disorder. There was nothing but the agonizing wait for symptoms to continue to disrupt their son’s development and daily living. Sam’s parents, together with other family members and colleagues, founded the “Progeria Research Foundation.” The Progeria Research Foundation accomplished:
o 1999 - Launched the first website and awareness campaign
o 1999 - Began obtaining funds for Research Grants
o 2000 - Scientific papers were included in medical journals
o 2001 - Brought scientists together to conduct workshops at medical conferences
o 2002 – Genetic Consortium and Medical/ Research Database was created
o 2002 – Discovered the first Pregoria gene
o 2002 - Started the first triple drug trial and first clinical drug trial
o 2003 - Created the first diagnostic testing program
In November of 2012, the Pregoria Research Foundation launched new clinical drug trials that led to the discovery of a new, first time treatment that has shown improvement in many children and has exciting and promising results. In one decade, a set of parents were able to do more than doctors and scientists had done in 126 years. And they’re still going.
For information on the foundation: www.pregoriaresearch.org
Agosto and Michaela Odone
Agosto and Michaela were the courageous parents of a precocious little boy named Lorenzo Odone, who at six years old, was diagnosed with Adrenoleukodystrophy (ALD). ALD is a rare and cruel disease that is passed on to boys from their mother. It’s a chromosomal disease that allows long-chain fatty acids to accumulate and attack the myelin sheath that surrounds the part of the brain that controls the nervous system. As the myelin is stripped away, the child will become deaf, blind, mute, paralyzed, and end their final days relying on ventilation and feeding tubes. In most cases, death results from the child choking on their own saliva.
The Odone’s were hit with Lorenzo’s diagnosis during the 1980’s. At that time, there was no treatment or information on how to prolong or prevent Lorenzo’s then, horrible and inevitable fate: death within two years of the diagnosis. There was no internet. There were no social networking outlets that could enable their immediate connection to the rest of the world in hopes that there might be someone with answers or hope. It was desperate times for parents who were basically told to go home and watch their son die.
The Odone’s were not scientists. They didn’t work in the health and medical profession. Agosto was a World Bank Economist, and Michaela was a French translator. There was one support group that shared food recipes and discussed new trials of medicine that would take years to test. Years they didn’t have. Determined, the Odone’s altered their entire home life to save their son’s. While Michaela stayed home and cared for Lorenzo, Augusto took an early retirement to research and study the disease. He visited the library and spent countless hours learning about ALD, and consulting with scientists and other specialists. Augusto, through his research, learned of studies that were conducted on rats for the reduction of fatty chains caused by ALD .
This study was surrounding the ingestion of a ucelic and erucic acid mixture (forms of olive and rapeseed oil). Augusto’s theory was that these two oils would help the cease the creation of the fatty acid chain, and and that this would delay the fatal symptoms of ALD. Augusto found a scientist that would create the oil concoction, which he would then serve the oil to his family on a dinner salad to insure it would cause no harm to them before giving it to Lorenzo.
To the chagrin of the many doctors who rejected Mr. Adone’s theory, the results were astounding. Not only did the oil mixture stop the condition from worsening, it began to reverse some of Lorenzo’s symptoms. However, Lorenzo had suffered massive damage to his myelin sheath, he would never regain speech, or the ability to move and eat independently. Still, they were able to cease ALD’s deadly progression. The oil also served to stop the disease in its tracks for newly diagnosed boys.
Together, the Odone’s had discovered the first treatment for ADL ever recorded. These parents not only saved their son’s life, they saved the lives of hundreds of other boys diagnosed with ALD.
For more information on Lorenzo and the Odone’s, visit their website: www.lorenzoandhisparents.com .
The Brown vs. Board of Education was a landmark Supreme Court case that set the precedent for many civil rights cases on education issues. This famous case blew the “Separate but Equal” ruling out of the water. It helped launch the numerous discrimination cases that would take place in the years to follow. If schools were not to discriminate against students by denying them equal access to a free and public education because of the color of their skin, then surely these schools could not discriminate against students with disabilities. They too deserve a fair shake at obtaining a quality education alongside their peers.
Oliver Brown, with the support of the NAACP, filed a class-action lawsuit against the Board of Education because his daughter, Linda Brown, was denied access to to an education in an all-white school. This incredibly important and system changing event was all due to a parent who had the courage to go against the grain and advocate for his daughter's right to an appropriate and fair education.
As parents, it’s natural for some us to feel intimidated by suits, framed degrees hanging on the walls, or the acronyms that follow a person’s last name. Please remember that these people put their pants on one leg at a time just like we do. No one has more valuable information and input about your child than you do. A parent’s input on their child’s medical, behavior and learning needs is the most important element needed for a well written report, and implementing an effective medical, learning and behavioral plan.
More importantly, a parents right to advocate for their child's health, education, and equal access to all opportunities should be supported by all of us. Because we are all entitled to live a quality life and the pursuit of happiness.