There are several examples of dramatic genomic changes that appear when cells are reprogrammed. Studies of some trinucleotide repeat diseases have reported changes in the repeat length following reprogramming. Specifically, in Friedreich ataxia, the GAA/TTC triplet repeat length in the FXN (frataxin) gene appeared to change following reprogramming of patient fibroblasts
Although the probability of an FDA-approved hPSC-derived cell therapy causing harm to a patient appears to be low, the consequences of adverse events are enormous. There is an important lesson from the failures in early gene therapy trials. If even one patient is harmed in an FDA-approved trial using hPSC derivatives, all further trials will be in serious jeopardy, and the promise of stem cell therapy will be put on indefinite hold.
Abstract
Human pluripotent stem cells (hPSCs) are known to acquire genomic changes as they proliferate and differentiate. Despite concerns that these changes will compromise the safety of hPSC-derived cell therapy, there is currently scant evidence linking the known hPSC genomic abnormalities with malignancy. For the successful use of hPSCs for clinical applications, we will need to learn to distinguish between innocuous genomic aberrations and those that may cause tumors. To minimize any effects of acquired mutations on cell therapy, we strongly recommend that cells destined for transplant be monitored throughout their preparation using a high-resolution method such as SNP genotyping.
Introduction
In the 15 years since the derivation of human pluripotent stem cells (hPSCs)2 was first reported, the research community has been moving steadily toward the goal of using these remarkable cells as replacements for tissues that are lost as a result of heart disease, diabetes, neurodegenerative disease, and skeletal and muscular disorders and injuries. The progress toward therapies has required incremental improvements in culture and differentiation methods, GMP and industrial-scale cell culture, legal permissions, and considerable investment from both public and private entities. At the same time that new hPSC therapies are being launched, concerns regarding their safety remain a major hurdle. In the worst case scenario, the transplanted cells may harm the patient by becoming cancerous or inducing cancers. For cell types derived from hPSCs, there are two major concerns about tumorigenicity. The first is intrinsic to pluripotency; undifferentiated hPSCs generate teratomas, germ cell tumors that are usually benign, when they are transplanted to immunodeficient mice. Although all of the clinical applications require the cells to be differentiated, there is concern about residual undifferentiated cells in transplanted populations. Second, mutations in transplanted cells are a major concern because genomic mutations are associated with tumorigenicity. In this minireview, we summarize the available information about instability in the genomes of hPSCs and consider the potential impact of genomic instability on the safety of current and future hPSC-derived transplantation therapies.
I had my adipose fat cells removed and injected into my nasal cavity. Since it is my own stem cell do you think it is safe? The theory is the stem cells will travel up smell nerves into the brain I have OPCA.
Any Stem Cell therapy can invoke some contraversial views.
There is a RISK with putting anything into your body. (but at least if anything is resourced from your own body, at least you know its history and its makeup !)
I think that there is alot more research to be done on this issue.
The reason I went with injecting my own adult stem cells is for my body rejecting it. I read if you use someone else's stem cell you might get tumors. Do you agree?
I had my adipose fat cells removed and injected into my nasal cavity. Since it is my own stem cell do you think it is safe? The theory is the stem cells will travel up smell nerves into the brain I have OPCA.
Although, now that I take a closer look at the website, since the therapies offered are the stem cell center are not yet FDA-approved, there is a certain buyer-beware aspect to the situation. I guess the best way to think of it is experimental treatment. Have you seen improvement?
I have some improvements my sister-in-law who is a teacher of deaf and hard of hearing brother-in-law and mother-in-law noticed my speech is better I donot slur
as much, I went to my PT Feb 7th she put me through a battery of test and finally ended up on a test booth hooked to a computer.
I stand on a foot plates that move it checks your reflexes and balance she said I have not digressed in 2 months since the last time she tested me. I got the stem cell treatment Oct. 10th the treating doctor said it will take 6 months so I just over
4 months. I feel a little steadier on my feet but I still have gait disorder. When I was searching for a stem cell center the man at the phone was very knowledgeable and my consultation with the treating doctor went well.
The only regret I have is I should have ask for nerve growth factor. dancermom said:
Although, now that I take a closer look at the website, since the therapies offered are the stem cell center are not yet FDA-approved, there is a certain buyer-beware aspect to the situation. I guess the best way to think of it is experimental treatment. Have you seen improvement?
I would like to describe the procedure for the stem cell treatment. First they numbed the back side both sides near the hips. Waited an hour for the local anesthetic to work. Then used a long syringe and sucked out my fat cells along my waist in the back.
They told me to come back in 4 hours so they could treat the stem cells with vitamins. When I came back they injected a enzyme to loosen my pores in the nasal cavity. Then they injected the stem cells in the back of my neck for neck pain from a accident I was in and injected the rest into my nasal cavity and I sniffed at the same time.
The theory is the stem cells will travel up the smell nerve pores into the brain and repair my damaged cerebellum.
The reason I went for this experimental treatment is I am 72 and donot have time to wait. Since it looked safe and not invasive I went for it. My wife went with me and met the doctor and staff and felt they were nice and legit. They have
one price $7600 and any follow up treatment $7100. So the price is reasonable.
dancermom said:
Wow, that is fascinating, Steve. I look forward to hearing more about these treatments and their effects. Thanks for the information.