New Therapy for Battling Sickle Cell Disease Gives Hope to Younger Patients

By Linda Goelzer

A recently approved drug is giving hope to younger patients living with sickle cell disease (SCD). Crizanlizumab is intended to reduce the frequency of pain crises and limit hospitalization in adult and pediatric patients ages 16 and older with  SCD. 

Patients of Clarissa Johnson, M.D., pediatric hematologist/oncologist and other SCD specialists at Cook Children’s have participated in the clinical trial to determine the dosage for pediatric patients for Crizanlizumab, the recently approved drug marketed under the name Adakveo.

“It’s an exciting time to be in sickle cell disease care,” Dr. Johnson said. “For the first time since 1995, we have options to offer our patients. The research pipeline for sickle cell is open and this new drug is a sign of the times.”

Pain crises are complex and associated with potentially life-threatening complications of SCD. Red blood cells of people living with the genetically inherited disease are sickle-shaped, similar to a crescent moon; and cells become stiff and sticky – adhering to blood vessel walls during circulation.

Healthy red cells are rounded and pliable, designed to flow smoothly through the body and supply oxygen along the way. When sickle cells clump together, they restrict blood flow and can ignite pain and inflammation. The pain can occur suddenly and with enough severity to require days of hospitalization and dosing of powerful pain medication to endure them.

Dr. Johnson’s patient, Kambri Gaut, has suffered pain crises with hospitalization. Her parents say there is little comfort for the pain when crises do happen.

“It gets to you. Especially as a man, you want to fix it and you can’t fix this,” said Kambri’s dad, Kevin Gaut. “Now that she’s older, she can tell you what’s wrong, and what works and doesn’t work.”

Fortunately, Kambri participated in the crizanlizumab clinical trial and experienced success.

How does the drug work?

Adakveo is a monthly IV infusion therapy. Dr. Johnson would prefer something less invasive, but it is an option for her patients.

Kambri visits the infusion clinic every 21-30 days and it can mean she misses some school. Her mother, Brandi, says Kambri does well in school and it is easier to make up missed classwork online.

“She has only been hospitalized twice in the last three years while participating in the trial,” Brandi said. “It is a commitment to meet with the research team and participate in the work they need for the clinical trial; plus, receive the infusions, but we’re used to it. I have no complaints and we plan to let her keep doing it.”

We asked Kambri if she has signals when she might experience a pain episode. “The weather – when it is raining and cold,” she said. “Mostly I live my life and I drink water a lot.”

“Some patients describe sickle cell pain as a glass-cutting feeling as the cells travel through the vessels,” Dr. Johnson said. “The new drug is a manufactured antibody that targets markers on white cells that make the red cells sticky; it disrupts the clumping of the cells together which is what contributes to the greatest pain.”

Living with sickle cell disease

The 13-year-old definitely lives life to the fullest. Kambri is a cheerleader, plays volleyball, and has danced since she was 6 years old. She ran track and played basketball.

“We let her do what her body allows,” Brandi said. “If she can, she can. If not, she won’t.”

The Gauts work diligently to ensure Kambri has a support team wherever she goes. Before each school year, Brandi shares with the school nurse, a handbook about SCD that Cook Children’s and Dr. Johnson provide to patient families. The nurse, at Kambri’s new school in Cleburne, educated the coaches about Kambri’s condition and what the young athlete needed from them. The nurse explained triggers that signal SCD complications and the coaches even developed a plan, including non-verbal cues during games, when Kambri needs rest and more water.

Dr. Johnson says she prefers treating her SCD patients with Hydroxyurea, an oral medication that treats pain crises and a broader range of complications including anemia, which can be the main issue. Therefore, Dr. Johnson prefers her patients to try Hydroxyurea first and if it fails to control their pain crises, she sees Adakveo as an adjunct therapy.

“Some patients with SCD are not that anemic. This drug could be sufficient for them,” Dr. Johnson said. “We would prefer to have a [subcutaneous] form of the drug rather than infusion. We offered that feedback during the trial. The downside is researchers will not look into something like that when they have not completed the trial for infusion therapy.”

Clinical trials underway for younger patients

Currently, doctors at Cook Children’s are only treating about 20 patients with the new drug, out of the 400 patients in the SCD program and results are varied. Qualifying age and administration by infusion are limiting factors. Dr. Johnson says teenagers can have more of a say in their medication choices and some young adults like to try Adakveo even if they later decide the inconvenience factor is less attractive to them. Dr. Johnson believes the monthly infusions could be a drawback for younger children, but the clinical trial is ongoing to understand the effectiveness down to toddler age.

The new medication has provided relief to Kambri and her family. Besides the teen feeling well enough to compete in sports, we asked Kambri what else she enjoys.

“My favorite subject [in school] is science, learning about planets, chemistry, things like that,” she said. “I watch Netflix. I like being with friends and talking with friends on Facetime. Watching sports.”

“Even though this drug is not a panacea, it is great to have more in our medicine cabinet to treat sickle cell,” Dr. Johnson said. “This is more than a Band-Aid. Pain meds are a Band-Aid. This is a game-changer for us.” 

Learn more about sickle cell disease:

U.S. Centers for Disease Control and Prevention
Sickle Cell Disease Coalition
Sickle Cell Disease Association of America, Inc.