WHY CHARGE FOR EXPERIMENTAL TREATMENT?
Why is this hospital charging for an experimental treatment, its criminal. If this proved to be a treatment for NP-C and other neurological disorders the hospital would stand to make billions of dollars.I cant understand why, when they are using our kids as "guinea pigs", that we have to pay these enormous sums for a trial.If it was 100% guareenteed to be successful then I could agree but not while its a trial. The very fact that somebody is making money out of Kids who have no other option is in itself criminal. These people should take a long hard look at themselves in the mirror and ask if this is right and moral.
I have huge admiration for Aaditya and his family who are going through this at this moment, but basically the hospital is making money out him. If its successful are they going to return the families money as a thank you for making them billions?........Now please dont get me wrong I hope with all my heart that it is successful and it does prove to be a kind of treatment but, come on, charging thousands for an experiment and using the life of a helpless child, that's not right.
Duriya, please let us know how exactly Aaditya is doing and coping with the cyclodextrin infusions. How often does this happen?
Carl.
I have huge admiration for Aaditya and his family who are going through this at this moment, but basically the hospital is making money out him. If its successful are they going to return the families money as a thank you for making them billions?........Now please dont get me wrong I hope with all my heart that it is successful and it does prove to be a kind of treatment but, come on, charging thousands for an experiment and using the life of a helpless child, that's not right.
Duriya, please let us know how exactly Aaditya is doing and coping with the cyclodextrin infusions. How often does this happen?
Carl.
Comment
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Comment by Ravi Dasgupta on February 23, 2009 at 7:27pm -
Dear Carl,
This is Aaditya's father Ravi. I am responding to your post based on my 15 years of experience in the Pharma / Biotech industry .
Clinical trials as you rightly say are free for those who take the unproven medicine. The traials are backed by a sponsor ( a Phama / Biotech company that has developed the experimental drug and is paying for its clinical developments so that it can take the drug to the market ).
In the case of Aaditya's stem cell transplant there is no sponsor company claiming that it has a possible cure for NPC and enroling patients for its clinical trial.
Duriya in her reading had found to the possibility of using mesenchymal stem cells and thereafter we had found Stempeutics who could provide the stem cells for us and BGS Global who were ready to conduct the procedure for us.
I do agree that there is a possibility of more business for both these entities especially as stem cell therapy is not allowed in the US and many other places ; but this does not mean they should do this for free.
In the end nothing in this world is "free". Even the free clinical trial is paid for by someone. Since this is not a clinical trial & there is no company backing it its natural that someone ( we ) needed to pay.
I am overwhelmed by the response Duriya and Shama had got for her fund raisers for Aaditya. As his father I had expected to bear the brunt of the stem cell therapy cost but instead it was entirely taken care of by hope for Aaditya funds + some help from my sister.
A similar thing happened in the last 2 days when Duriya & I had appealed to some of our friends to contribute towards Sawson's surgery. We have so far collected $ 4000/- and I expect we will get some more before the day is through.
I dont think we need to worry about money for our kids : God will make a way.
regards,
RCD
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Comment by duriya on February 22, 2009 at 3:43pm -
Carl,
I understand your frustration. But you have to understand that the hospital did not come to us. We went to the hospital. If this was done in the US billions would have been spent, and a lot of kids lives lost before we would even be at this point. Being Indian, Aaditya and his family had no relief in getting Miglustat for him. $10,000 per month for Miglustat!!! Who was making billions on that? We do what we have to do and leave the rest to God. The doctors have been very kind and extremely helpful. And I have a deep respect for them. The system is the same where ever you go. We will get through it. Aaditya is doing well but we are not seeing any changes from the cyclodextrin as yet. He has had 3 infusions so far.
Hang in there, it will be okay.
Duriya
About
Niemann-Pick Disease
Niemann-Pick disease is an inherited condition involving lipid metabolism (the breakdown and use of fats and cholesterol in the body) in which harmful amounts of lipids accumulate in the spleen, liver, lungs, bone marrow, and brain.
There are three variants of Niemann-Pick disease based on the genetic cause and the symptoms exhibited by the patient. All variants are inherited in an autosomal recessive pattern.
Mutations in the NPC1, NPC2, and SMPD1 genes cause Niemann-Pick disease.
This condition is inherited in an autosomal recessive pattern, which means two copies of the gene must be altered for a person to be affected by the disorder. Most often, the parents of a child with an autosomal recessive disorder are not affected but are carriers of one copy of the altered gene. If both parents are carriers, there is a one in four, or 25%, chance with each pregnancy for an affected child. Genetic counseling and genetic testing is recommended for families who may be carriers of Niemann-Pick.
Type C is characterized by onset in childhood, although infant and adult onsets are possible. Other signs include severe liver disease, breathing difficulties, developmental delay, seizures, increased muscle tone (dystonia), lack of coordination, problems with feeding, and an inability to move the eyes vertically. People with this disorder can survive into adulthood. The incidence of Niemann-Pick disease, type C is estimated to be 1 in 150,000 people. The disease occurs more frequently in people of French-Acadian descent in Nova Scotia.
Mutations in either the NPC1 or NPC2 gene cause Niemann-Pick disease, type C. The NPC1 gene produces a protein that is located in membranes inside the cell and is involved in the movement of cholesterol and lipids within cells. A deficiency of this protein leads to the abnormal build up of lipids and cholesterol within cell membranes.
The molecular basis for this disease is extremely complex due to the role that endosome formation has on affected patients. Recently, three theories have attempted to explain the buildup of cholesterol in the lysosomes of affected patients of Niemann-Pick Disease Type C due to the malfunction of the protein NPC-1.
* The contention by Neufel et al is that the buildup of mannose 6-phosphate receptors (MPRs) in the late endosome suggests that the retrograde breakdown of cholesterol via the Trans Golgi Network cannot occur.[1]
* Another theory suggests that the blockage of retrograde cholesterol breakdown in the late endosome is due to decreased membrane elasticity and thus the return vesicles of cholesterol to the Trans Golgi Network cannot bud and form.
The support of these theories has considerable evidence using mutant proteins in vitro to determine the buildup of cholesterol in the lysosomes. Researchers have also discovered that the NPC-1 protein may function as a pump of cholesterol.[2]
The overall effect of a malfunction in NPC-1 is that low levels or an absence of the protein lead to the abnormal accumulation of lipids and cholesterol in the cells of people with this condition.
There are three variants of Niemann-Pick disease based on the genetic cause and the symptoms exhibited by the patient. All variants are inherited in an autosomal recessive pattern.
Mutations in the NPC1, NPC2, and SMPD1 genes cause Niemann-Pick disease.
This condition is inherited in an autosomal recessive pattern, which means two copies of the gene must be altered for a person to be affected by the disorder. Most often, the parents of a child with an autosomal recessive disorder are not affected but are carriers of one copy of the altered gene. If both parents are carriers, there is a one in four, or 25%, chance with each pregnancy for an affected child. Genetic counseling and genetic testing is recommended for families who may be carriers of Niemann-Pick.
Type C is characterized by onset in childhood, although infant and adult onsets are possible. Other signs include severe liver disease, breathing difficulties, developmental delay, seizures, increased muscle tone (dystonia), lack of coordination, problems with feeding, and an inability to move the eyes vertically. People with this disorder can survive into adulthood. The incidence of Niemann-Pick disease, type C is estimated to be 1 in 150,000 people. The disease occurs more frequently in people of French-Acadian descent in Nova Scotia.
Mutations in either the NPC1 or NPC2 gene cause Niemann-Pick disease, type C. The NPC1 gene produces a protein that is located in membranes inside the cell and is involved in the movement of cholesterol and lipids within cells. A deficiency of this protein leads to the abnormal build up of lipids and cholesterol within cell membranes.
The molecular basis for this disease is extremely complex due to the role that endosome formation has on affected patients. Recently, three theories have attempted to explain the buildup of cholesterol in the lysosomes of affected patients of Niemann-Pick Disease Type C due to the malfunction of the protein NPC-1.
* The contention by Neufel et al is that the buildup of mannose 6-phosphate receptors (MPRs) in the late endosome suggests that the retrograde breakdown of cholesterol via the Trans Golgi Network cannot occur.[1]
* Another theory suggests that the blockage of retrograde cholesterol breakdown in the late endosome is due to decreased membrane elasticity and thus the return vesicles of cholesterol to the Trans Golgi Network cannot bud and form.
The support of these theories has considerable evidence using mutant proteins in vitro to determine the buildup of cholesterol in the lysosomes. Researchers have also discovered that the NPC-1 protein may function as a pump of cholesterol.[2]
The overall effect of a malfunction in NPC-1 is that low levels or an absence of the protein lead to the abnormal accumulation of lipids and cholesterol in the cells of people with this condition.
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