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a-cure-for-beta thalassemia-from-our-own-bodies

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love and prayers:
Dr. Robert H. Broyles, a medical school professor and researcher in Oklahoma City, has discovered a protein that is grown in our own bodies that will silence the sickle cell gene and activate a normal gene in its place. This cure is very specific for the cause, easy to deliver, and beneficial to the whole body.  Our vision is a global one: our goal is to get this cure to each of the millions of people world-wide who have SCD and/or Beta-Thalassemia.
"The approaches of gene therapy and/or a stem cell transplant have the potential to cure sickle cell permanently. But, neither of these procedures has been perfected. Both of these treatments can go wrong and kill the patient. If either or both of these treatments were completely ready for clinical application today, there would still be a major obstacle to their wide-spread use. That obstacle is that gene therapy and stem cell therapy are very, very expensive; and both can be performed only in large, major medical centers. Because of the great expense associated with each of these therapies, 99.99% of the people who need them are unlikely to receive them for many years to come. And banking cord blood is also very expensive, so that this is done only when there is a closely matched person who needs those stem cells.
Our therapy, called gene regulation therapy (GRT), is different. No genes are altered, as they are in gene therapy. No stem cells are required. GRT is very inexpensive and easy to deliver. With our plan, more than 80% of the people in the world with sickle cell or beta-thalassemia can be treated for the rest of their lives – and lead normal lives – at a cost all can afford. The treatment shuts off the sickle cell gene and activates the fetal hemoglobin gene in its place, which nature has shown us is a “cure.”
We would very much like to have your help in raising the awareness, support, and money we need to push this cure through clinical trials. Please email me at robert-broyles@ouhsc.edu, and I will send you files from which you can print brochures and other materials you will need to do an effective fundraising campaign.
Thank you very, very much!
Sincerely,
Robert H. Broyles, Ph.D.
President, SCCF"

http://sicklecellcurefoundation.org/2009/08/press-release/

We are a research foundation; and this is the first year in our 4 1/2-year existance that our research has received significant funding, thanks to the Bill & Melinda Gates Foundation. So, I have been spending most of my time in the lab. Our animal trials in mice got underway November 1, 2010. We hope to start clinical trials in humans with sickle cell within this year (2011).

Sharmin:
This sounds very interesting and promising.  I would love to learn more.  How is this gene regulation treatment delivered to the patient?  Is it a drug?  Does it require multiple treatments? 

Thanks for sharing,

Sharmin

love and prayers:
the treatment should be good for bet-thalassemia, as well.
the treatment should partially silence the mutated beta globin gene; AND it should also stimulate fetal hemoglobin production (that is, the gamma globin  gene will be activated), which should help greatly to relieve the globinchain imbalance that results in premature red cell destruction.

Some people have asked, “Your ‘cure’ works in theory, but does it work in practice?” The answer is, “Yes!!!” The key point is that fetal hemoglobin (HbF) has been shown to be preventive for sickle cell disease in nature, in many labs around the world, in many, many repeated experiments and patient trials over the last thirty years. The problem is that no one has figured out how to stimulate this hemoglobin switch in humans without gene therapy, a bone marrow/stem cell transplant, or use of toxic drugs – all of which are dangerous, expensive, and not available to 99.99% of the world’s sufferers. This is what we have now done: found a safe way to induce the HbF switch. We have found a natural, small, stable human protein that will produce this Hb switch (a) in a test tube, (b) in living primate and human cells, (c) in transgenic mice carrying and expressing the human H-ferritin gene, and (d) in forming red bllod cells obtained from pediatric sickle cell patients. All in all, we have performed almost 200 experiments of 50 different types/designs, each experiment replicated 2-to-10 times by 2, 3, or 5 different investigators – all consistently pointing to this same conclusion and its safety. And we have devised 4 ways to deliver this cure! Now, we need the funds to prove its safety in animals, so that we can then start human trials. We can provide the hope, the science, and the safety; you can help us get the necessary funds.

Our cure has been peer-reviewed via Publications & Patents, Grants Awarded, and invitations for International Presentations.

Publications & Patents: PDF files of a List of 25 Publications; a key paper that established the principle of the cure; and our Patent issued in the U.S., the European Union(10 countries), and Australia, are attached.

Publications List
PNAS 2001
US Patent #7,517,669 (FtHgenetransduction)
Grants Awarded: A PDF file of an NIH-form 4-page bio-sketch is attached, with the last two pages listing NIH and other nationally-competitive grants. Dr. Broyles’ curriculum vitae is also attached.

Dr. Robert Broyles CV
Grants (pp3&4) & NIH biosketch
International presentations: PDF files of abstracts of nine invited, international presentations (2003 through 2009) are attached.

2003 IBIS Bethesda.pdf
2004 ISSCR Boston.pdf
2005 IBIS Prague.pdf
2006 ASH Orlando.pdf
2006 NSCD Memphis.pdf
2007 IBIS Kyoto.pdf
2007 SFRBM Washington, DC.pdf
2008 SCDAA NewOrleans.pdf
2009 IBIS Porto.pdf

http://sicklecellcurefoundation.org/2009/11/peer-review-validations-of-sccfs-discovery-our-credentials/

love and prayers:
i hope it works too!!

love and prayers:
"...this treatment will have to be given several times a year; and it will have to be taken for the rest of the patient’s life. But “the rest of the patient’s life” will then become a normal life span; and the pain should become a thing of the past. The treatments will be very inexpensive, about 10% of what it now costs for inferior, partially effective treatment. And there should be no side effects."
Robert H. Broyles, Ph.D.
President, SCCF


The treatment should be easy to deliver, as a protein directly into the blood or marrow, where red cell precursors have receptors on their surface that recognize this protein and internalize it. We have also discovered a plant compound that will activate expression of this protein in human cells, and the plant compound will be tested for both safety and effectiveness in humans with sickle cell or beta-thalassemia.

http://sicklecellcurefoundation.org/2009/07/our-research/

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