Terra

Yes, this treatment is expected to work in adults at any life stage, as well as children. Clinical trials should allow us to determine ranges and frequency of doses, and whether all people respond equally or whether some need a higher dose, etc. But, as with all treatments that have been proposed for SCD including stem cell transplants and gene therapy, the treatment does not reverse previous damage caused by SCD. So, if taken late in life, we hope that the treatment will still stop sickle cell at that point. But other medical measures may be necessary to try to repair previous damages, depending on how severe the damages are.

All the best,
Robert H. Broyles, Ph.D.
President
SCCF

Thanks for your excellent question. The answer is “yes, depending on the genetics.” Let me explain. Since our treatment does not alter the genes in any way, the same inheritance rules will still apply. So, if one parent has SCD (two sickle genes) and the other parent has none, all the children will have sickle trait (one sickle gene) and NOT have SCD. If the second parent has trait (one sickle gene), then each child has a 50:50 chance of having SCD and a 50:50 chance of having trait. And finally, if both parents are SCD (both genes sickle), then all children they have will have SCD. But the good news is that those children could be started on our treatment before the SCD has an opportunity to express itself, and lead a normal life. That is what we are aiming for and hope will be confirmed by our animal and clinical trials.

All the best,
Robert H. Broyles, Ph.D.
President
SCCF

Thank you very, very much for your Comment and, especially, for your support. We have been aware of your foundation’s interest and support of our endeavor for some time, as you have a link on your Cure Sickle Cell website directly to our Sickle CellCure Foundation website. There are not enough thanks in this world to express the depth of our appreciation for this link and acknowledgement. Of all the sickle cell websites out there, yours is the only one that has provided such a link. And thank you for considering financial support from your foundation for our effort to get this cure into clinical trials. That is wonderful and much needed. This year, we are trying to get our most earnest supporters to consider making a donation each month, on a regular basis, which will help us keep our effort constant and continuous. We would very much welcome your partnership in this endeavor. We look forward to hearing more from you and your fine group. Many thanks!

Sincerely,

Robert H. Broyles, Ph.D.
President
The Sickle Cell Cure Foundation

My name is Larry Nixon, MD & co-founder of Cure Sickle Cell Foundation, Jackson, MS. My wife, Dee Bookert-Nixon, RN, & I have a child with SCD who serves as the motivation to our cause. When I discovered what you guys were doing, I was really excited. After I read & did my due diligence with review of literature, I now feel spiritually committed to make sure that this project is a complete success.

We started http://www.curesicklecell.org as just a site to promote research aimed at a cure because we felt there was not enough discussion in this regard. At this time I was a resident at Emory University. We moved to Mississippi and the community immediately accepted our foundation with open arms. I primary goal is to promote efforts aimed at curing SCD, but because of a lack thereof we got involved in other community activities (health imformation, educations for care-providers etc.) Needless to say we as a family & foundation are excited about your work and will be meeting with our board to see how we can support you financially. Our niche as a foundation is community relations & we have been waiting for an opportunity to organize so that we can take a cure to the national stage for financial support. We sincerely want to get involved

Thanks,
Terra