UAB Study Leads to New FDA Approved Drug for Sickle Cell Anemia

Written by Dakota Layton on January 6, 2020

UAB hematologist, Dr. Julie Kanter, who has treated sickle cell anemia for more than 10 years became involved in a study to test the drug, Adakveo. She believed that it might effectively target one of the factors that cause blood cells to stick together. Sickle-shaped red blood cells overproduce a sticky protein, P-selectin, which is targeted by the treatment.

“P-selectin acts like a hook on the inside of the blood cell,” Kanter said. “In people who have significant inflammation, there is too much P-selectin so other cells are being hooked together. It’s really been an exciting concept to block that adhesion.”

Adakveo has proven to be effective for patients in the study including Danielle Jamison of Islandton, SC.

“I’m now able to do most normal things I couldn’t before,” Jamison said. “I can take my daughter to school and dance competitions and volunteer on the PTO. I have energy. I have daily pain but it’s not so bad.”

Participants in the study averaged a 45 percent decrease in painful episodes and significantly longer stretches between hospitalizations. Kanter stated, “I’ve been saying for a while that I really consider it a gamechanger.”

“There are many issues with sickle cell disease, and access to care is a big one. To have a drug that can be administered as an IV lessens the responsibility of the patient. It enhances the reason for patients to come to the doctor. Sometimes we see them mostly when they come to the hospital, which is bad for patients and it’s bad for our system of care.”

Adakveo is now available for patients age 16 and up as a monthly infusion that they can receive at their physician’s office. However, just one vial of Adakveo costs $2,357 and patients typically require three vials per month.

In a statement, officials from Novartis said they are committed to making the treatment accessible to patients and will provide information about financial assistance to those who are uninsured.

“Most Medicaid patients pay low, single-digit costs, per month, for their medicines including Adakveo. Patients covered by commercial health plans will have a $0 copay when they use the co-pay card we offer. Patients covered by commercial health insurance usually have an initial deductible to pay each year so they may also have to meet their deductible in order for their insurance to pay for medical care and Adakveo.”

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