Discussion Forums > Working Towards a Cure
a-cure-for-beta thalassemia-from-our-own-bodies
Sharmin:
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
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 :(
Andy Battaglia:
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.
--- Quote ---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
--- End quote ---
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.
Sharmin:
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...
Sharmin:
Andy,
Do you know how things are going with the Hemaquest trials?
Thank you so much,
Sharmin
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