[On-screen text: Roberta C. G. Azbell, MD
Hematology
OSUCCC – James]

Roberta C. G. Azbell, MD: Sickle cell disease is an inherited blood disorder, so people with sickle cell disease are born with it. It mainly causes pretty significant anemia and can cause severe pain throughout a person's life. Due to the expertise that our team has here managing people with sickle cell disease, we were able to treat our first person with sickle cell disease with a new gene therapy product.

[On-screen text: Khaled Alsheebani
Patient]

Khaled Alsheebani, speaking in Arabic, translated by Rawya Abuhajleh, who is offscreen: I was diagnosed when I was a kid. My family was told that I have the sickle cell anemia. My life was very restricted with my disease. It was very hard for me to be like my friends and my siblings practice any kind of sports or to go to the gym or to do any hobbies.

[On-screen text: Milly Valverde
Director of Destination Medicine
Ohio State Wexner Medical Center]

Milly Valverde: Destination Medicine is our international and out of state patient program for the Ohio State Wexner Medical Center. So we care coordinate for patients that travel to our medical center. In the 12 years, we now count over 120 different countries that we have welcomed patients. Khaled came to us from UAE, that's his home country. Their citizens are provided the option of receiving care out of their country when it's appropriate, and they provide full support for this. That conversation led to a referral of Khaled to our center, a formal referral, and that's when services started a few months later. 

[On-screen text: Marcos de Lima, MD
Cell Therapy Program
OSUCCC – James]

Marcos de Lima, MD: So this was our first gene therapy for sickle cell. It is a fairly busy and long schedule. This particular patient, basically almost a year with us. What this does to the cells is essentially recap how we make blood before we're born. And this fetal hemoglobin, it's not necessarily we're not correcting the actual sickle cell defect. We're making these cells produce more fetal hemoglobin, which in turn holds more efficiently to oxygen. So this change is enough to make those cells less likely to burst as they do normally. So the expectation with that is that he will not show the usual manifestations of sickle cell, the crisis, the chest pains. And they are the first one to tell you when these things start calming down. This doesn't come from us, right? This is the message that comes from the patient, not from the doctor or the nurses.

Roberta C. G. Azbell, MD: When we ask ourselves what it means to cure a patient with sickle cell disease, would it mean looking at labs and seeing no sickle hemoglobin? Or would it be, I was able to stay out of the hospital for years and I was able to go back to school or go back to work or start a family? And I know Khaled talked about all of those things, being excited to go back home and start a family and do some of the things that we, I think, call normal for a 20 something year old. Those were the signs to me that it was working and that we might be able to say, "Wow, I think we've cured him."

[On-screen text: 
The James
The Ohio State University Comprehensive Cancer Center
For more information, visit: cancer.osu.edu]