Do I think Embryonic stems cells will cure SMA? I never believed in it .I do believe in Gene therapy. Yes, I do. I believe it will help so many kids and many other diseases. I was a listener I have never ever said I was a believer in Embryonic stem cells as a cure for SMA.
Lots of negative things being written about gene therapy. Please continue reading . This was posted by a good friend of mine on His facebook wall. Its very informative and helps you understand what is going on. I had to share this:
Here is the article that was posted from Slate:
It comes on attack at many levels showing failure, but if anything we have learned that science takes time, patience and hard work. This article was written not by a researcher but by a journalist. The Author of this article was Emily Yoffe.
She was criticized by The Daily Howler for writing about a theory based largely on numerical evidence despite having recently written an article about herself titled "The Math Moron" in which she revealed that she tested at a first-grade level in mathematics.
In regards to SMA I think it is preferable to stick to scientific articles regarding the state of research Here is a well written scientific article posted by the Howard Hughes Medical Institue:
This article clearly shows the complexity of Embryonic Stem Cell research in the area of Motor Neurons. The body is not made up of just 1 generic Motor Neuron but hundreds of specific types of motor neurons that need HOX genes and FOXp1 to make the vast array of Motor Neurons. A science that is still in its beginning stages. Once these are figured out the next step is to figure out how to "rewire" the circuitry in the human body. Each of these Specific Motor Neurons Control different types of muscles. We have yet to prove that we are capable of innervating a motor neuron. To make a full signal between spine and muscle. We are years away from making this a viable treatment for our community. This is an important field and the research must continue and improve over the coming years.
For the Gene Therapy it is a much more immediate venture. There are questions on the "window" of treatment. These questions can only be answered via clinical trial. There is so much unknown about the motor neurons that it is impossible to answer. If the motor neurons are dormant then quite possibly they can be revived using the Gene Therapy. If there is a motor neuron loss then these will have to be replaced. A typical non-sma person will loose 30-40 percent of their motor neurons over the course of their lifetime. Yet they continue to ambulate in later years.The hope is that even if there has been a loss, the remaining motor neurons can be strengthened from the current state they are in. A realistic hope is that someone like Sophia may be able to breath on their own without mechanical assistance or possibly sit up. That is realistic. To think that she is going to be "normal" and run around like other kids is probably not. So many other things have happened in her body that other therapies and treatments would certainly need to be done. ie hip dysplasia. The Gene Therapy has shown the greatest results to date on the SMA type 1 mouse model. Far superior to any other drugs or therapies. In addition the results have been extremely well in larger animal studies which are much closer to humans. Lastly these results have been repeated by 3 different independent labs using similar approaches. These findings are nothing short of remarkable. When information is given out by researchers it is important to understand about their backgrounds. The Burgess Labs at OSU have been responsible for:
1. The Carrier Screening test for SMA
2. The Genetic Diagnostic Test for SMA
3. The original SMA mouse model (it is still the most popular mouse model used in SMA research)
These researchers have an incredible understanding of Spinal Muscular Atrophy and have already made significant contributions to the advancement of our disease
I have received 2 quotes from these researchers regarding their take on the posted article:
"We have been asked to comment on the story "The Medical Revolution: Where are the Cures". Indeed there have been promises in the field of biomedical sciences that have offered cures within 5 to 10 years. There is a growing list of failures based on those promises as this article highlights. To counter, medical research and understanding of complex diseases have expanded exponentially based on these studies. What these failed studies have shown remarkably clear is that diseases such as Parkinson's, Alzheimer's and Huntington's are quite complex and that a cure may not be as simple as taking a miracle pill. One may say that this research has been a waste, but if one looks at drugs, procedures and therapies that have been developed based on the research, one can see advances to patient health. For example, there are new procedures in Parkinson's Disease that counter the disease for many years. Additionally, more potent drugs have been developed that control the disease as well. Parkinson's has not been cured, but there are new procedures and drugs that have made significant changes to the disease. Certainly more work needs to be done as Parkinson's patient's continue to experience suffering due to their disease. An outstanding article from Time Magazine gives a clear look at the development of heart transplantation, where the field has made outstanding advancements.
We are currently moving a translational gene therapy program forward for Spinal Muscular Atrophy. The results in our pre-clinical studies have been very promising. This has led us to rapidly, yet carefully advance studies to larger species to evaluate safety. We have assembled a working group of other scientists and clinicians that are collaborating to move this experimental therapeutic forward. We are actively moving the program to approach the regulatory agencies including the Food and Drug Administration. Based on our pre-clinical studies for efficacy and safety, we are encouraged that the therapy is safe, and targets the cells (motor neurons) that we are intending. The SMA community and us realize that this is an experimental therapy and will be tested first for safety and evidence for efficacy in small groups of human studies which is the translational process that any drug, therapy or procedure moves through. Everyone shares in our hope that therapies to improve the life of SMA patients will be developed and we remain committed to working to advance our studies.-Dr. Brian Kaspar
The Medical Revolution: Where are the Cures".
The article describes set backs that have occurred in particular in the field of gene therapy and stem cells. It is not a surprise that there will be set backs in the development of any new therapies. However this should be placed in the context of more conventional therapies such as drugs also not being cures or having major impacts on the diseases mentioned. This begs the question as to what therapies should be followed surely it is the lessons we learned from the approaches and whether the problems can be resolved that is critical. In the case of gene therapy a number of issues have been resolved with the AAV vectors the true test is when it is effective in a clinical trial. What are the issues first it appears wise to focus on a treatment rather than a complete cure as this is more likely to be achievable. The author mentions a series of disease but there are a number of differences in the cases presented. In the case of Parkinson’s for instance the first question which arises is the similarity between genetic forms of the disease and sporadic cases is the defect and the components to be fixed the same in all cases? We really cannot be sure and it is most often the case that the therapy is tested in a genetic model. So in the case of spinal muscular atrophy it is clear that reduced levels of SMN cause the disorder with milder patient having more SMN. So both in the mice model of SMA and in humans reduced SMN levels cause SMA. Therefore the same thing is being tested in mice and eventually in man. However an important consideration in SMA therapeutics is when the SMN levels need to be increased to reverse SMA if the clinical trial design differs from what is tested in mice then you might get different results. What we know for the relative severe SMA mice is that early postnatal introduction of SMN has a major impact on the survival of the SMA mice. So how does SMA fit into the landscape of the article? First it is clear that the genetics SMA is an advantage in that you know the cases you will be treating are due to reduced SMN, second the remarkable development of Adeno Asscoiated vectors which can get to the required target clearly resolves the delivery issue at least in mice (The AAV vector can also target the required cells in larger animals), third the treatment shows efficacy in the SMA animal model when delivered early indeed gene therapy has been the most effective treatment in these animals to date. In SMA we have three major therapeutic avenues based around increasing SMN 1) drugs that stimulate production of SMN from SMN2 we should encourage better drugs that stimulate a 3 fold increase in SMN levels, Antisense oligonucleotides that encourage SMN2 to produce sufficient SMN but are more difficult to deliver to the required cells, and AAV gene therapy. We are actively pushing forward with the Kaspar laboratory in moving the proposing gene therapy approach to clinical trials. Until one or even better more than one of these approaches works in SMA patients we need to push forward with these potential treatments and develop them further using any failures that might occur to find the method of therapy that does work. -DR. Arthur Burghes
We believe in these researchers and the contributions they have made and continue to make for our community. We will continue to educate our community and provide you with the scientific research that is happening regardless of the politics. I remain very optimistic of the real promise that is truly right around the corner.
"We believe in miracle because we live with one!!"
www.our-sma-angels.com/elizabeth
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