Sickle Cell Research at The Children's Hospital of Philadelphia

Sickle Cell Research at The Children's Hospital of Philadelphia

Naomi Behtel: I'm Naomi Bechtel. I've had sickle cell since I was four.

Kevin Bethel: When we realized that Naomi had sickle cell, it was tough.

Rhonda Bethel: I think I cried for days. I was devastated.

Alan Flake, MD: You hear about a lot of other disorders far more frequently and in a more pronounced way than you do sickle cell disease. It's really been sort of a hidden disease over the years and one that most people are extremely unaware of.

Kim Smith-Whitley, MD: We know that one in 375 African American newborns has the disease. The current life expectancy of someone born with sickle cell disease is 42 to 48 years of age.

Rhonda Bethel: Even though my husband and I sat down with my daughter and explained to her her condition, it's still hard for me to tell her that one day she may, you know, die from this condition.

Kim Smith-Whitley, MD: It's the unpredictable nature of the disease that makes a great impact on our families. Every day, you wake up cautiously hopeful that this will be one of those days that you don't have to go to the hospital because of fever or that you don't have to go to the hospital because of pain.

Kevin Bethel: The first time, she spent almost weeks in the hospital. It was very challenging.

Rhonda Bethel: I feel forever indebted to CHOP for making us feel at home and welcome.

Alan Flake, MD: The Children's Hospital of Philadelphia is unique in many ways. It's the oldest children's hospital in the country, and that's one of the reasons that our Sickle Cell Center is so large and covers over a thousand patients.

Kim Smith-Whitley, MD: Dr. Ohene-Frempong was one of the founders of the Sickle Cell Program here at the Children's Hospital of Philadelphia, and he took a group of basic scientists, clinical researchers, and put them to the test at really trying to find a way to improve the quality of life of those living with sickle cell disease.

Alan Flake, MD: I've been at CHOP for 16 years, and the reason I stay at CHOP is because it's a very conducive environment and supportive environment to do cutting-edge research. And that relates to CHOP's mission, to generate new cures for all types of diseases.

Kim Smith-Whitley, MD: Dr. Flake's work really gives us the possibility of having children being born healthy so that they never have to experience any of the chronic impact of having sickle cell disease.

Alan Flake, MD: The idea is that we can transplant a fetus with stem cells from another donor and have those cells engraft because the fetus has the unique ability to become tolerant to those stem cells.

Kim Smith-Whitley, MD: This would be a way for couples living with sickle cell trait to have children without sickle cell disease, despite the genetics of the disease.

Alan Flake, MD: The research results are absolutely convincing that we can achieve a cure of sickle cell disease right now, in humans, with the approach that I've described. What we need is approximately $3 to $5 million to complete all phases of this research and complete the clinical trial.

Kim Smith-Whitley, MD: Unfortunately, the economic environment has been challenging and the funding for research has been challenging over the last decade.

Alan Flake, MD: Because this type of research is out of the mainstream and it's a minority disease, there's really a lack of funding available to proceed with these studies. So that's why we really need philanthropy to complete this project.

Kim Smith-Whitley, MD: With Hope on the Horizon, we would have sustainable funding for moving Dr. Flake's research forward.

Alan Flake, MD: I think it's actually a very rare opportunity that you could potentially cure a major disease like sickle cell disease for $3 to $5 million.

Kevin Bethel: We can find a cure for sickle cell, but it will take a group effort, and it will take those fundings and those dollars to pour into that research to find that answer.

Kim Smith-Whitley, MD: These children are lights, and they are wonderful in what their potential is.

Naomi Behtel: I want to be a nurse when I grow up, and I want to help sickle cell patients get through hard times.

Rhonda Bethel: It's my hopes that she live for a long time. I think my mission now is to prepare her for life and to point her in a right direction for whatever may come her way.

Kevin Bethel: And I believe the stuff that is being done here at CHOP, I believe the stuff that they are taking the lead in can really lead to a cure.

Alan Flake, MD: There's no other area of my career that is more important to me than solving this problem.

Kevin Bethel: A cure that would allow that child to be cured while in the womb is — can't be measured.