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Posted for Zoe:
Children with Batten Disease Seek Stem Cell Treatment in China
At age two, Hailey Goranflo was a normal, bright and happy child. At age five she is no longer able to walk, talk, or feed herself. She has jerking spasms and seizures throughout the day and night. Her brother, Carter, nearly three, began to have seizures a few months ago. In a recent letter about her children, Miranda Goranflo writes, " Carter has still been doing okay. Hailey, on the other hand, has not. She has lost so much more strength and is so weak. She sleeps all day and all night. She is only awake for 2-3 hours a day now."
Neil and Miranda Goranflo are living the agony of seeing their two children slip away from them with little chance either will live to adulthood. Carter and Hailey have Batten Disease, a very rare inherited disorder of the nervous system.
Batten Disease is one of a type of rare disorders named Neuronal Ceriod Lipofuscinoses (NCLs). Hailey and Carter inherited a form of the disorder called Late Infantile Batten Disease.
In Batten Disease, a genetic defect prevents the body’s cells from properly eliminating the waste material that builds up in them during the normal process of metabolism. Cellular debris accumulates, damaging and killing brain cells, eventually destroying the nervous system. Most victims die from the rapidly progressing disease by the age of 12.
The sudden development of seizures, clumsiness, speech delays, behavioral disturbances, are some of the first signs of the disease taking hold of its victims. Typically, children with Batten Disease will go blind and suffer from dementia. They lose all use of their bodies, the ability to communicate, and become completely bedridden before they die.
From the moment Hailey and Carter were diagnosed last January, Neil and Miranda knew they would not watch helplessly as the disease progresses taking the lives of their children. The specialists offered the Goranflos little help or information when the children were diagnosed. “The doctor gave us a piece of paper with all the clinical trials in the U.S.” Miranda recalls, “ Unfortunately he didn’t know anything about them. The children only qualified for two of the trials, one in Oregon for stem cell therapy and in New York for gene replacement because of their specific type of the disease.
“I made the phone calls, only to find out the trials had just ended and would not risk taking Hailey anyway. They also informed me that the chances of Carter being chosen were very slim.
“The first couple of weeks after the diagnoses were very confusing. ‘Why us,’ is what we asked a lot. We immediately began researching when we got home that day. For a couple of weeks we researched what we were dealing with, tried to figure things out.”
The Goranflos found support through the Batten Disease Research and Support Association (BDRSA) The BDRSA is an international organization for the families whose children have Batten Disease. Its mission is to provide support and facilitate networking so families, doctors, and researchers can more quickly find and share information about, and treatment for, Batten Disease. There is no BDRSA branch in their home state of Kentucky, so the Goranflos joined the Ohio Chapter where they have been finding support and information. “They are really nice,” Miranda notes, “most of them have Batten’s children so they really know what we are going through.”
According to the BDRSA, the signs and symptoms of Batten Disease are usually not recognized and are often misdiagnosed as epilepsy or mental retardation. Initially, Hailey was diagnosed with a form of childhood epilepsy, her parents assured that she would outgrow it.
China is a long way from Shepherdsville, Kentucky, where the Goranflo's live. Yet, it is in China where the children can receive experimental stem cell treatment for their disease.
Through the BDRSA, Miranda learned about several hospitals in China that are providing stem cell therapy for children with Batten Disease and other neurodegenerative disorders. She says, “I asked for some numbers of other parents who have gone that route and called them right away. After talking with them and researching China and stem cells, we made a plan. That plan has kept us from going crazy.”
The plan was to raise enough funds to take Hailey and Carter to China for stem cell therapy. Despite finding there is no treatment for their children in the United States, Miranda finds, “ We have some hope now and something to look forward to. Whereas the doctors basically told us to take them home and wait for them to die, we stay very busy and focused on fundraising. If it weren’t for that...I would be so lost and feel so helpless and guilty for not trying.”
No one hospital in China could treat both the children due to their ages and because Hailey’s condition is so advanced. “I talked to mothers who have taken their children to the hospital Hailey and I are going to, Tiantan Puhua, and it is in Beijing,” says Miranda. One of them gave me the email of a nurse there. I emailed her and she got right back to me. She asked a lot of questions about us and talked to the doctors about us. They normally do not treat children under six, but will make an exception for Hailey.
“She said they could not treat Carter because he is too young. She did, however, mention another hospital, Xiaoshan, in Hangzhou. So I called them and they agreed to take him.”
Once the Goranflo’s found the children could be treated in China, their focus turned to finding ways to get them there. Miranda does not work and Neil’s income as a self-employed landscaper will not meet their family’s medical expenses. The children are on the state-funded Medicaid program. This program will not cover the treatment in China, nor any of the travel and other expenses. The only hope for slowing or reversing the progress of the disease is the stem cell therapy treatment available in China.
With help from family and friends, and others, the Goranflos are devoting themselves to raising what amounts to a fortune to get treatment for the children in China.
“My brother in Illinois,” says Miranda, “helped me set up a website. He basically set it up and I filled it in. Now I’ve gotten to where I can work on it some myself.
“ I called about twenty lawyers and one called me back. He referred me to another attorney who set up a trust fund for us for free.”
Friends, family, their church, and volunteers in the community of Shepherdsville have helped organize fundraisers for the family. According to Miranda many people have stepped into to help, “ We have had a lot of people from the church volunteer to help us. We’ve also had complete strangers helping us, collecting stuff, having benefits, yard sales, setting up booths and lemonade stands. We have a lot of great friends that have been helping us too.
“We have raised $50,000 for Hailey’s treatment,” says Miranda, “and I will be taking her on June 24th to Beijing. We already have $10,000 for Carter and need $25,000 more. Fundraising will continue for Carter while we are there and as soon as his money is raised, he and Neil will go to China for Carter’s treatment. We’re hoping that will be in July. Hailey will go for nearly two months and Carter will go for one month.”
Miranda is also well aware that stem cell therapy is experimental and the outcome not certain, “ They will be getting them through a spinal cord injection. Hailey will receive a total of 4 injections of 10,000,000 each time. I am not sure how many injections Carter will get yet. Hailey will be getting enbryonic stem cells and Carter will be getting umbilical cord stem cells. Embryonic stem cells are actually better for this type of disease, but they come with a greater risk. Sometimes, but very rarely, the stem cells can actually form into tumors.
“Hailey,” Miranda continues, “will be the fifth patient at Tiantan Puhua treated for Batten Disease (they treat other neurodegenerative diseases as well). Three of the four children treated who have Batten Disease are doing much better. We don’t have our hopes up too high, we’ll take any improvements we can get.”
...
If you would like to learn more about the Goranflos or help them get treatment for their children, you can visit their web site:
http://www.haileyandcarter.wordpress.com
Links:
Batten Disease:
http://www.ninds.nih.gov/disorders/batten/detail_batten.htm
Batten Disease Research and Support Association:
http://www.bdsra.org/
Stem Cell Therapy:
http://www.mayoclinic.com/health/stem-cells/CA00081
Tiantan Puhua Hospital:
http://www.puhuachina.com/_daifumd/dochome/html/284/texts/text_10.htm
Xiaoshan Hospital:
http://www.beike.ch/en/Treatment
@2008 Zoe L. Langley
Hailey age 5
Carter age 2.5
Picture #1 Miranda with both children - Picture #2 Neil with Carter and Clown
Children with Batten Disease Seek Stem Cell Treatment in China
At age two, Hailey Goranflo was a normal, bright and happy child. At age five she is no longer able to walk, talk, or feed herself. She has jerking spasms and seizures throughout the day and night. Her brother, Carter, nearly three, began to have seizures a few months ago. In a recent letter about her children, Miranda Goranflo writes, " Carter has still been doing okay. Hailey, on the other hand, has not. She has lost so much more strength and is so weak. She sleeps all day and all night. She is only awake for 2-3 hours a day now."
Neil and Miranda Goranflo are living the agony of seeing their two children slip away from them with little chance either will live to adulthood. Carter and Hailey have Batten Disease, a very rare inherited disorder of the nervous system.
Batten Disease is one of a type of rare disorders named Neuronal Ceriod Lipofuscinoses (NCLs). Hailey and Carter inherited a form of the disorder called Late Infantile Batten Disease.
In Batten Disease, a genetic defect prevents the body’s cells from properly eliminating the waste material that builds up in them during the normal process of metabolism. Cellular debris accumulates, damaging and killing brain cells, eventually destroying the nervous system. Most victims die from the rapidly progressing disease by the age of 12.
The sudden development of seizures, clumsiness, speech delays, behavioral disturbances, are some of the first signs of the disease taking hold of its victims. Typically, children with Batten Disease will go blind and suffer from dementia. They lose all use of their bodies, the ability to communicate, and become completely bedridden before they die.
From the moment Hailey and Carter were diagnosed last January, Neil and Miranda knew they would not watch helplessly as the disease progresses taking the lives of their children. The specialists offered the Goranflos little help or information when the children were diagnosed. “The doctor gave us a piece of paper with all the clinical trials in the U.S.” Miranda recalls, “ Unfortunately he didn’t know anything about them. The children only qualified for two of the trials, one in Oregon for stem cell therapy and in New York for gene replacement because of their specific type of the disease.
“I made the phone calls, only to find out the trials had just ended and would not risk taking Hailey anyway. They also informed me that the chances of Carter being chosen were very slim.
“The first couple of weeks after the diagnoses were very confusing. ‘Why us,’ is what we asked a lot. We immediately began researching when we got home that day. For a couple of weeks we researched what we were dealing with, tried to figure things out.”
The Goranflos found support through the Batten Disease Research and Support Association (BDRSA) The BDRSA is an international organization for the families whose children have Batten Disease. Its mission is to provide support and facilitate networking so families, doctors, and researchers can more quickly find and share information about, and treatment for, Batten Disease. There is no BDRSA branch in their home state of Kentucky, so the Goranflos joined the Ohio Chapter where they have been finding support and information. “They are really nice,” Miranda notes, “most of them have Batten’s children so they really know what we are going through.”
According to the BDRSA, the signs and symptoms of Batten Disease are usually not recognized and are often misdiagnosed as epilepsy or mental retardation. Initially, Hailey was diagnosed with a form of childhood epilepsy, her parents assured that she would outgrow it.
China is a long way from Shepherdsville, Kentucky, where the Goranflo's live. Yet, it is in China where the children can receive experimental stem cell treatment for their disease.
Through the BDRSA, Miranda learned about several hospitals in China that are providing stem cell therapy for children with Batten Disease and other neurodegenerative disorders. She says, “I asked for some numbers of other parents who have gone that route and called them right away. After talking with them and researching China and stem cells, we made a plan. That plan has kept us from going crazy.”
The plan was to raise enough funds to take Hailey and Carter to China for stem cell therapy. Despite finding there is no treatment for their children in the United States, Miranda finds, “ We have some hope now and something to look forward to. Whereas the doctors basically told us to take them home and wait for them to die, we stay very busy and focused on fundraising. If it weren’t for that...I would be so lost and feel so helpless and guilty for not trying.”
No one hospital in China could treat both the children due to their ages and because Hailey’s condition is so advanced. “I talked to mothers who have taken their children to the hospital Hailey and I are going to, Tiantan Puhua, and it is in Beijing,” says Miranda. One of them gave me the email of a nurse there. I emailed her and she got right back to me. She asked a lot of questions about us and talked to the doctors about us. They normally do not treat children under six, but will make an exception for Hailey.
“She said they could not treat Carter because he is too young. She did, however, mention another hospital, Xiaoshan, in Hangzhou. So I called them and they agreed to take him.”
Once the Goranflo’s found the children could be treated in China, their focus turned to finding ways to get them there. Miranda does not work and Neil’s income as a self-employed landscaper will not meet their family’s medical expenses. The children are on the state-funded Medicaid program. This program will not cover the treatment in China, nor any of the travel and other expenses. The only hope for slowing or reversing the progress of the disease is the stem cell therapy treatment available in China.
With help from family and friends, and others, the Goranflos are devoting themselves to raising what amounts to a fortune to get treatment for the children in China.
“My brother in Illinois,” says Miranda, “helped me set up a website. He basically set it up and I filled it in. Now I’ve gotten to where I can work on it some myself.
“ I called about twenty lawyers and one called me back. He referred me to another attorney who set up a trust fund for us for free.”
Friends, family, their church, and volunteers in the community of Shepherdsville have helped organize fundraisers for the family. According to Miranda many people have stepped into to help, “ We have had a lot of people from the church volunteer to help us. We’ve also had complete strangers helping us, collecting stuff, having benefits, yard sales, setting up booths and lemonade stands. We have a lot of great friends that have been helping us too.
“We have raised $50,000 for Hailey’s treatment,” says Miranda, “and I will be taking her on June 24th to Beijing. We already have $10,000 for Carter and need $25,000 more. Fundraising will continue for Carter while we are there and as soon as his money is raised, he and Neil will go to China for Carter’s treatment. We’re hoping that will be in July. Hailey will go for nearly two months and Carter will go for one month.”
Miranda is also well aware that stem cell therapy is experimental and the outcome not certain, “ They will be getting them through a spinal cord injection. Hailey will receive a total of 4 injections of 10,000,000 each time. I am not sure how many injections Carter will get yet. Hailey will be getting enbryonic stem cells and Carter will be getting umbilical cord stem cells. Embryonic stem cells are actually better for this type of disease, but they come with a greater risk. Sometimes, but very rarely, the stem cells can actually form into tumors.
“Hailey,” Miranda continues, “will be the fifth patient at Tiantan Puhua treated for Batten Disease (they treat other neurodegenerative diseases as well). Three of the four children treated who have Batten Disease are doing much better. We don’t have our hopes up too high, we’ll take any improvements we can get.”
...
If you would like to learn more about the Goranflos or help them get treatment for their children, you can visit their web site:
http://www.haileyandcarter.wordpress.com
Links:
Batten Disease:
http://www.ninds.nih.gov/disorders/batten/detail_batten.htm
Batten Disease Research and Support Association:
http://www.bdsra.org/
Stem Cell Therapy:
http://www.mayoclinic.com/health/stem-cells/CA00081
Tiantan Puhua Hospital:
http://www.puhuachina.com/_daifumd/dochome/html/284/texts/text_10.htm
Xiaoshan Hospital:
http://www.beike.ch/en/Treatment
@2008 Zoe L. Langley
Hailey age 5
Carter age 2.5
Picture #1 Miranda with both children - Picture #2 Neil with Carter and Clown
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