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

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).

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

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/

i hope it works too!!

"...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/

This sounds like an amazing opportunity for thalassemia and sickle anemia - I can't imagine anything better.  I hope and pray that the treatment in humans turns out to be everything you describe and better.  Would several times a year amount to 3 to 4 treatmenst?  Perhaps IV infusions of the protein?  It would be great to have something that would eliminate the need for blood - therefore iron, iron deposits - organ damage - and the need for chelation.  Also, the risk to bones and organs because the body will actually be producing its own red blood cells.

Best of luck,

Sharmin

I wish funding were easier to secure...and these treatments were more readily available so people wouldn't have to face the difficulties that they are facing today

I contacted Dr Broyles through Facebook and asked for any comments on the current status of his research. I was happy to receive a reply.

Robert H Broyles April 9 at 3:42pm
Dear Andy ,
Thank you for your message and for the alert to the thread on the website thalpal.com, much of which is taken from articles on our website http://sicklecellcurefoundation.org/. I appreciate you attention to accuracy of quote and accuracy of intent in what I say. First the current status of our work: It is going slower than we thought, mainly due to shortage of needed funds that was only partially alleviated by the grant from the Bill & Melinda Gates Foundation. We still hope that some human clinical trials can begin in 2012, including a first trial for beta-thalessia in Thailand.

We are hopeful for enough an increase in fetal Hb in beta-thalassemics to eliminate the need for transfusions; but such trials must be done carefully.

In response to your question about the similarity between our work and that of Dr. Susan Perrine: Yes, our approaches are similar in one sense - we are both trying to stimulate fetal Hb enough to "cure" the disease. Our approach at SCCF is different in that our therapeutics are more natural and (we hope and intend) much less expensive. Our first and main therapeutic, FtH, is a small, very stable protein that comes from our own bodies and which we grow up with. Therapy is achieved by delivering it to bone marrow cells which are deficient in this protein; the FtH enters the cells that are to become red blood cells, enters the nucleus, and activated the fetal Hb gene. Our second therapeutic is a plant compound that we discovered which activates production of FtH which in turn activates feal Hb. The plant compound can be given as a pill and be taken as often as needed, very inexpensively. However, we are not as far along in our testing as Dr. Perrine, and we are not as well funded.

I hope these comments help you and your readers. And I hope that many will choose to donate to our nonprofit effort on our website http://sicklecellcurefoundation.org/. Thank you.
Robert H. Broyles, PhD, President/Lead Scientist
The Sickle Cell Cure Foundation, Inc., a 501(c)(3) nonprofit research foundation

I have some immediate comments. I am quite happy to see an attempt being made to improve treatment through a naturally occurring compound. As we all know, drugs have side effects and minimizing these is extremely important and using natural compounds can help minimize unpredicted side effects. I also want to point out that the draconian budget cuts in the US, because our "leaders" absolutely refuse to tax those who possess the wealth, will do incredible damage to government funded research. Researchers like Dr Broyles will need all the private help they can get.

Thanks Andy,

I wish all of this could happen faster....but I am glad that alternatives to bmt and gene therapy are being explored.  In the end, regardless of the progress and understanding - what hope is there without funds.  The government seems to have money for everything else.  I wonder how different things would be if one of Obama's daughters had thalassemia or sickle cell anemia...

Andy,

Do you know how things are going with the Hemaquest trials?

Thank you so much,

Sharmin

Thats great......looking forward to it...I will pray that it will come soon to help all thalessemia.

It's really a shame that the donation can only be given if you have a mailing address in US. Just how are we going to get the funds through from this part of the world?? I really wish the Thalassemia societies in all our home country will do a charity drive to collect funds for this GRT research, imagine the benefits to all the thalassemics in this part of the world if successful. It's a bit controversial I can imagine to get people to donate to something that is not 100% foolproof. But, if you don't try you, you'll never know. 

hqk-1001 trials are only being carried out on thal intermedias,i hope Robert H. Broyles trials are used for thal majors too!

i know this may sound a little shady but you know how asunra and kelfer ar avialable as cheaper affordable drugs. do you think they'll make a cheaper hqk-1001 or the plant compound that has been discovered by Robert H. Broyles?

 india is great that way!!!i'm amazed they haven't made a hqk-1001 already. the sad  part is that there are some people that earn less than a dollar a day and the need for affordable drugs is very important!!!!

There are reasons why a hemoglobin inducer would be trialed on intermedias, but this does not mean that once approved, the drug wouldn't also be used for majors. I guarantee that it will be, because doctors will prescribe it once it's available, just as some US doctors had patients using desferal and Exjade together as soon as Exjade was approved. I think it would be much easier to gauge the full effects of a hemoglobin inducing drug in intermedias who could go without transfusions for much longer periods than majors can. The next generation of hemoglobin inducing drugs will free intermedias from transfusion for the most part and offer a decrease in transfusion frequency for majors. In some countries like Iran, hydroxyurea is already used to decrease transfusion dependence in majors and a better drug will be most likely used worldwide, because face it - transfusions are something of a wild card in thals and minimizing the amount of transfusions has to be the key to treatment, so the wild cards can be reduced. Less antibody reactions and less stress on the heart from transfusion are only a couple of the benefits. So, if you can cut in half the transfusion frequency, you will be doing majors a huge favor. These drugs are destined for use in majors. I would also not be surprised if the next stage of Dr Perrine's trials do include majors.

Thanks Andy,

I'm hopeful that these changes will take place soon.  It would be a dream come true.
Fewer transfusions, less iron and less chelation would equal a more normal life and quality
Of life. 

We are set to meet Dr Vichynski in August.  Hopefully
He'll have more info too.  Lil A is doing a thalassemia
Camp in LA with all thalassemia children.  He's
Really excited.  It'll be a great opportunity for him.

Sharmin