Patients suffering from a rare bleeding disorder that prevent proper clotting of the blood during injury may very well be treated with a combination of stem cells with gene therapy in the near future. Experiments on sheep show that administering stem cells that posses a copy of a gene coding for a missing protein in haemophilia, ameliorate the disease. The injected stem cells produce a clotting factor, and are able to migrate to a site of inflammation, which is a sign for injury, and consequently bleeding. Although the therapy has not yet been tested on humans, it is a fine example of how controversial therapies such as stem cells and gene therapy can help us.
In haemophilia A, the clotting factor VIII is missing, which results in prolonged bleeding after injury. There are many factors involved in the process of blood clotting, and a missing factor VIII is the most common cause of haemophilia. Because the blood does not clot, patients can easily bleed out, highlighting the need for a proper therapy for this currently untreatable disease. While the scientists could not cure haemophilia in sheep, their novel therapy was able to decrease the severity of the symptoms, which is possibly enough to save a patient's life in the case of bleeding.
For their experiments, scientists genetically modified mesenchymal stem cells. These are adult stem cells, committed to a cellular lineage that is supposed to form connective tissue, to stitch cells together. However, researchers have found that these stem cells can relatively easy be persuaded to develop into other cells, which may come in handy for regenerative medicine. In this case, the mesenchymal population was likely chosen for their ability to migrate: because injury can happen everywhere in the body, the repair cells need to be mobilized and ready to travel when the body is in need of clotting factors.
The main problem of injecting sheep with modified stem cells is that the immune system develops a response against clotting factor VIII, because it is recognized as a foreign molecule. For the therapy to remain effective, scientists need to find a way around the immune system. Else, the therapy will likely not be working anymore after the first treatment.
One of the most famous cases of haemophilia is the one of Alexei Nikolaevich, son of the Russian tsar. Born in 1904, he was diagnosed with haemophilia B, which differs slightly from haemophilia A in terms of missing clotting factors. According to historic tales, he was once saved from haemophiliac complications by the famous Rasputin, who served as an advisor of the tsar. In the end, it did not help him, as the whole family was murdered in 1918, in the bolshevik revolution that started the Soviet Union.
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For their experiments, scientists genetically modified mesenchymal stem cells. These are adult stem cells, committed to a cellular lineage that is supposed to form connective tissue, to stitch cells together. However, researchers have found that these stem cells can relatively easy be persuaded to develop into other cells, which may come in handy for regenerative medicine. In this case, the mesenchymal population was likely chosen for their ability to migrate: because injury can happen everywhere in the body, the repair cells need to be mobilized and ready to travel when the body is in need of clotting factors.
The main problem of injecting sheep with modified stem cells is that the immune system develops a response against clotting factor VIII, because it is recognized as a foreign molecule. For the therapy to remain effective, scientists need to find a way around the immune system. Else, the therapy will likely not be working anymore after the first treatment.
Having haemophilia A this means that his body, his immune system, fights off the medicine – the infused factor VIII. The alternative clotting factors that we used to control bleeding were much less effective. His medical condition was heart broken. Despite our visit to several doctors his health wasn't getting better. He was subjected to different medications, by many doctors for treatment without the assurance of having a permanent cure. Having a son with hemophilia and then, the added challenge of an inhibitor, is not always easy. Suspicion that something wasn’t right began when he was 6 months old due to the heavy amount of bruising which started showing up all over his legs. As a parent, I would love to fix things and make everything better. Fortunately, there have been other positive alternative (Herbal Medicine) though few people doesn't know the challenges of living with hemophilia. If your child is having same problem, do not expose him/her to more danger, use a herbal remedy that is safe and effective. Contact him directly for more info with his email address: sharmash116@gmail.com
ReplyDeleteThanks to Dr Williams I am so happy today, my wife have been suffering from hemophilia for the past 8 years now, and i have spent a lot on western drugs which has all proved abortive, i have tried all means in life to get my wife out of this , but there was no answer until i decided to try herbal solution and i found Dr Williams online and i contacted him and after she took his medication as instructed, my wife is now completely cure from hemophilia within those week of usage, i am so much happy, thanks to Dr Williams for helping me get my wife life back again without any form of crisis, i promise to tell your name and good deeds to the whole world,if you have someone with hemophilia you can email him on drwilliams098675@gmail.com for more information .
ReplyDeleteALL THANKS TO DR WILLIAMS WITH HIS HERB MY WIFE WAS COMPLETELY CURED FROM HEMOPHILIA .
ReplyDeleteIs my pleasure to comment on this site and i thank the admin of this site for his/her great work so far.one of the happiest moment in my life was when the doctor confirm that my wife is been free from this illness. this awesome moments makes me the happiest man on earth, I really don't know how to thank DR WILLIAMS for helping my wife get cured for over how many year of suffering from hemophilia. i came across DR Williams contact through a headline news on internet about how DR Williams help a woman to get cured of her hemophilia and so many other with similar body problem ,i contacted him and he told me how to get his herb,few day later he sent me the herbal portion which my wife take every morning for 28 days, and his medicine was able to cure her completely from hemophilia. and now she very mush okay without any side effects whatsoever, If you have hemophilia, you can contact him on his email address drwilliams098675@gmail.com for advice and for his product.
I am Sophie from Canada, I once suffered from a terrible and chronic hemophilia ,since 8 years now , the doctor told me there was no permanent cure i was given medications to slow down its progress, i constantly felt my health was deteriorating as i constantly go out of breath,and this illness was really terrible especially when am going out with my friends, i have this constant disorder for about 30 years, this was really a terrible illness ,on thin one day that i was going through the internet,and i came across a post of Mrs kate on how his husband was been cured from hemophilia through the help of Dr Williams herbal product, I contacted this herbal doctor via his email and explain everything to him and make purchase of his product,few days later he sent me the herbal medicine through courier service, when i received the herbal medicine, i used it for 1 months as prescribed by Dr Williams and i was totally cured within those week of usage,on thin now i have not experience any sign or characteristics again . for more information you can email him on drwilliams098675@gmail.com for help
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