BIRMINGHAM — Danielle Jamison, of Islandton, S.C., suffers from sickle cell anemia, a condition that causes pain so severe it used to send her to the hospital two or three times a year.
"If you've ever stubbed your toe, imagine that ten times worse in your back or chest," Jamison said.
The episodes happened when misshaped blood cells clumped together inside vessels, blocking blood flow to organs and triggering crippling pain. In addition to the misery, those events cause permanent damage that often shortens the lives of the estimated 100,000 people afflicted with the disease, most of them African-Americans.
But two years ago, she began taking an experimental medication in a study led in part by a doctor and researcher at UAB. It's one of two new drugs for sickle cell approved in November by the U.S. Food and Drug Administration – the first new treatments to hit the market in three decades.
Dr. Julie Kanter, a hematologist at UAB, has treated sickle cell patients for more than 10 years. She became involved in the study because she believed the treatment, Adakveo, might effectively target one of the factors that causes 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."
The drug has worked wonders for Jamison. Since she started taking it two years ago, she hasn't had a single pain crisis that required hospitalization – her longest stretch as an adult with sickle cell.
"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% decrease in painful episodes, and significantly longer stretches between hospitalizations.
Kanter said sickle cell patients can range across a wide spectrum, from those who have professional careers to others so disabled they are unable to work. During her training in hematology, she noticed a shortage of treatment options for sickle cell patients. Only one drug had been approved to reduce the disease's hallmark pain crises.
These bouts of intense pain typically require hospitalization and IV opioids, Kanter said. That can be expensive for both patients and medical systems, prompting the push for more effective treatments.
Adakveo reduced the number of pain episodes during the trial. The treatment is now available for patients age 16 and older as a monthly infusion administered at a doctor's office.
Each vial has a wholesale cost of $2,357, and individual patients will typically require three to four vials every month. That adds up to about $100,000 a year.
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."
Much of Kanter's research focuses on helping sickle cell patients access care. Having a new treatment could help extend lives and keep people out of the hospital, as well as bring them to the doctor for regular checkups.
"I've been saying for a while that I really consider it a gamechanger," Kanter said. "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."