In any treatment, in any therapy, there are risks and benefits.
Its up to every individual, and in this case, parent, to determine if the benefits outweigh the risks.
After speaking to Dr. McGuire and Emile, we have a lot of information.
To start off, there are risks.
The treatment involves an injection, done under sedation, directly into the retina. As with any surgical procedure, there are inherent risks.
The major risks involve the anesthesia. Patients can react badly to anesthesia. The injection itself can cause a tear in the retina, or the injection can be placed in the wrong site.
There is a small chance the virus could spread to other tissues in the body, it could damage the DNA, or it could interact with other viruses. None of this has been observed in the patients that were treated.
There is also a risk of cataract development.
In animals, in dosages 10 times greater than the human dose, inflammation of the eyes was seen, and the cells eventually died.
Lastly, there is the risk of the agent causing damage to the eyes in which it was injected.
The good news is that the first patients who were treated, in this or other studies, showed none of these side effects. A few older patients, over the age of 30, did develop cataracts, but this was surgically corrected. A tear in the retina can also be treated, should it occur, right after the virus is administered.
In terms of benefit, the results can vary from patient to patient.
One of the main determining factors is the amount of scar tissue in the retina. As the cells cease to function, over time, the body may start to scar over the cells, because the body feels they are no longer needed.
The amount of scar tissue in the retina at the time of administration can affect the results. With younger patients, you assume less scar tissue. Some of the tests Nomi went through were to determine the amount of scar tissue he has, if any.
Another key factor is the visual cortex of the brain. The visual cortex of the brain "learns" based on what is seen. Typically, this learning period is strongest at birth and rises rapidly, and starts to peak in adolescence. By age 8, the visual cortex can learn no more.
Nomaan will be 6 in October. So his young age benefits him. His scar tissue should be, hopefully, at a minimum. His visual cortex still has the ability to learn.
The injection itself can only help cells that it directly comes into contact with. The surgeon can't inject the virus into the entire retina. He/She injects the virus into the central 1/5 of the retina.
Following the injection, the patient begins to see better after about 7 days and continue to improve over the next 30 days. ... After 30 days, there is usually no additional improvement.
The biggest improvement is in the response to light. It was described to us as if you were sitting in a dark room, and someone slowly increased the light using a dimmer switch.
Naturally this helps somewhat with visual acuity and color recognition as well. Peripheral vision does not increase as much.
This all isn't to say that every patient reacts the same way. Again, it varies from patient to patient. Especially taking into account how much vision the patient already has... and there is no way to know if one patient will benefit more or less than the next.
Lastly, the first group of patients were treated over 5 years ago. Since that time, it has been observed that visual function has not decreased... that's to say the gains made did not diminish. The Retina itself continued to thin however. LCA causes thinning of the retina. So despite the improvement in vision, the retina continues to thin... form generally follows function, so it is postulated that the vision may eventually begin to deteriorate again.
There are other studies taking place world wide, and the data is still coming in... So things are changing constantly.
There is even a oral drug trial underway, in its preliminary stages. The QLT1 trial involves oral administration of a retinoid compound, meant to supplement the form of vitamin A needed by the retina. The jury is still out, because large amounts of retinoid compounds are toxic. But its another step in the right direction towards a cure.
So as parents, how do we take all of this information in, and make a decision that we feel is best for our son.
This is a degenerative disease. As of two years ago, Nomaan had already lost 90% of his retinal function. This procedure may restore some of that vision. If it doesn't last, perhaps it will buy him enough time until a more permanent solution is discovered. If it doesn't work at all, or makes the condition worse, at least we tried something.
We have to take this chance, if given the opportunity.
My wife and I, and our families, have faith in God. We pray that he does what is best for Nomi. We leave our son in his hands. If he is meant to have this procedure, he will be selected. If he is meant to benefit, he will, and it will last as long as God wishes it to. All we can do is continue to pray and support Nomi in any way we can.
If he is not selected, he does not benefit, or it doesn't last, we will still love him. We will all care for him, and make sure he grows up to be a strong, independent man, with a family of his own. His parents, his brother, his cousins, everyone, will always be there for him to lean on, should he need it.
Inshallah, he will be selected, and this will be a long lasting success. And the data collected by the researchers will be used to help countless others.
Mom and Dad love you Nomi. Its our love for you that has helped us make this decision on your behalf. One day, when you are older, and you look back at this, we hope you smile.
Its up to every individual, and in this case, parent, to determine if the benefits outweigh the risks.
After speaking to Dr. McGuire and Emile, we have a lot of information.
To start off, there are risks.
The treatment involves an injection, done under sedation, directly into the retina. As with any surgical procedure, there are inherent risks.
The major risks involve the anesthesia. Patients can react badly to anesthesia. The injection itself can cause a tear in the retina, or the injection can be placed in the wrong site.
There is a small chance the virus could spread to other tissues in the body, it could damage the DNA, or it could interact with other viruses. None of this has been observed in the patients that were treated.
There is also a risk of cataract development.
In animals, in dosages 10 times greater than the human dose, inflammation of the eyes was seen, and the cells eventually died.
Lastly, there is the risk of the agent causing damage to the eyes in which it was injected.
The good news is that the first patients who were treated, in this or other studies, showed none of these side effects. A few older patients, over the age of 30, did develop cataracts, but this was surgically corrected. A tear in the retina can also be treated, should it occur, right after the virus is administered.
In terms of benefit, the results can vary from patient to patient.
One of the main determining factors is the amount of scar tissue in the retina. As the cells cease to function, over time, the body may start to scar over the cells, because the body feels they are no longer needed.
The amount of scar tissue in the retina at the time of administration can affect the results. With younger patients, you assume less scar tissue. Some of the tests Nomi went through were to determine the amount of scar tissue he has, if any.
Another key factor is the visual cortex of the brain. The visual cortex of the brain "learns" based on what is seen. Typically, this learning period is strongest at birth and rises rapidly, and starts to peak in adolescence. By age 8, the visual cortex can learn no more.
Nomaan will be 6 in October. So his young age benefits him. His scar tissue should be, hopefully, at a minimum. His visual cortex still has the ability to learn.
The injection itself can only help cells that it directly comes into contact with. The surgeon can't inject the virus into the entire retina. He/She injects the virus into the central 1/5 of the retina.
Following the injection, the patient begins to see better after about 7 days and continue to improve over the next 30 days. ... After 30 days, there is usually no additional improvement.
The biggest improvement is in the response to light. It was described to us as if you were sitting in a dark room, and someone slowly increased the light using a dimmer switch.
Naturally this helps somewhat with visual acuity and color recognition as well. Peripheral vision does not increase as much.
This all isn't to say that every patient reacts the same way. Again, it varies from patient to patient. Especially taking into account how much vision the patient already has... and there is no way to know if one patient will benefit more or less than the next.
Lastly, the first group of patients were treated over 5 years ago. Since that time, it has been observed that visual function has not decreased... that's to say the gains made did not diminish. The Retina itself continued to thin however. LCA causes thinning of the retina. So despite the improvement in vision, the retina continues to thin... form generally follows function, so it is postulated that the vision may eventually begin to deteriorate again.
There are other studies taking place world wide, and the data is still coming in... So things are changing constantly.
There is even a oral drug trial underway, in its preliminary stages. The QLT1 trial involves oral administration of a retinoid compound, meant to supplement the form of vitamin A needed by the retina. The jury is still out, because large amounts of retinoid compounds are toxic. But its another step in the right direction towards a cure.
So as parents, how do we take all of this information in, and make a decision that we feel is best for our son.
This is a degenerative disease. As of two years ago, Nomaan had already lost 90% of his retinal function. This procedure may restore some of that vision. If it doesn't last, perhaps it will buy him enough time until a more permanent solution is discovered. If it doesn't work at all, or makes the condition worse, at least we tried something.
We have to take this chance, if given the opportunity.
My wife and I, and our families, have faith in God. We pray that he does what is best for Nomi. We leave our son in his hands. If he is meant to have this procedure, he will be selected. If he is meant to benefit, he will, and it will last as long as God wishes it to. All we can do is continue to pray and support Nomi in any way we can.
If he is not selected, he does not benefit, or it doesn't last, we will still love him. We will all care for him, and make sure he grows up to be a strong, independent man, with a family of his own. His parents, his brother, his cousins, everyone, will always be there for him to lean on, should he need it.
Inshallah, he will be selected, and this will be a long lasting success. And the data collected by the researchers will be used to help countless others.
Mom and Dad love you Nomi. Its our love for you that has helped us make this decision on your behalf. One day, when you are older, and you look back at this, we hope you smile.
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