Sickle Cell Treatment Has Breakthrough

Feb 6, 2017

Individuals struggling with Sickle Cell Disease may be on the cusp of a breakthrough after an international, year-long drug trial ended last year.

One of the most active test areas was in eastern North Carolina and one of its participants was Majara Mooring of Greenville.

She, like millions around the globe, live with a disease that causes hidden, though intense pain.

“Because it can be to the point where I can’t walk and one time I just told myself ‘look, just cut my limb off. I will feel better if you just cut it off, the pain will go away.”

Mooring was diagnosed with Sickle Cell Disease as an infant. It causes blood cells, usually a disc shape, to harden and take on the form of its namesake. Blood clots are common and it can shorten the average lifespan by decades.

Mooring said hospital visits have been common throughout her life. Very common.

"Sickle Cell" by Hertz Nazaire - an artist who also Sickle Cell Anemia
Credit Hertz Nazaire / Kreyolicious

“Starting out with it, I was hospitalized, I could say, every other month. I was in there on my birthdays, every holiday. It was to the point where – I have a twin brother – that doesn’t have the sickle cell disease but he has a trait. But it was to the point where they would also have a bed for him in my hospital room.”

Last year, she and 197 other Sickle Cell Patients from three countries participated in a trial for a new, intravenous treatment called “crizanlizumab” [pronounced: Cris-AN-liz-a-mab]. The trial was sponsored by Selexys Pharmaceuticals.

Dr. Darla Liles at the Brody School of Medicine helped facilitate the Greenville leg of the crizanlizumab  trial, which included 11 local patients – Mooring among them. Liles also oversees a Greenville Sickle Cell day clinic that treats 750 patients from across the region

“In the 1990s there was a large study performed with hydroxyurea and hydroxyurea is the only medicine the FDA has approved that really alters the crisis the number of crisis people have and so we try to put all our patients on hydroxyurea. The problem is that it’s not always an easy medicine to take.”

Hydroxyurea comes in pill form and Liles says patients have to take it multiple times a day over a long period of time for it to work.

“It’s not something that automatically makes a difference that you can’t see the difference in a week or a month. It takes six months or a year for it to alter the types of red cells that are being made by your body. And so patients are sitting there, taking it every day and really seeing no benefit.”

Research on the disease has been slow, in part, because it impacts relatively few people – about 100,000 within the United States. It’s inherited and found primarily in individuals with sub-Saharan African, Caribbean, South and Central American ancestry.

According to the CDC, 1 in 365 black children are diagnosed with sickle cell disease. 1 and 13 carry the trait.

“If you look at the map for where malaria is and the map of where sickle cell trait, they really overlap. And so that protective effect is what really puts it there and in the Mediterranean and other places in that region.”  

The new treatment, Crizanlizumab, reduces the ability of red blood cells to stick to blood vessel walls which can cause fatal blood clots. The 52-week-long trial provided patients with either a high dose of the trial drug, a low dose of the trial drug, or a placebo.

The results exceeded expectations.

“Almost 50 percent of people who are on active drug in the highest dose did not have a crisis compared…to the placebo group. And there were actually 24 people in the highest dose of the drug that had no admissions, no crisis during that 1 year period.”

Liles said this study signals a new era for Sickle Cell research and greater hope for those living with the disease.

“If you take someone that has two or three crisis…per year and you can completely prevent it you’ve saved yourselves several weeks of hospitalization out of that year...you know, sickle cell crisis has a heavy disease burden so…financially, if the insurance company doesn’t have to pay for an admission where you’re spending $500-a-day on the hospital bed and all the medicines and IVs and everything else it’s thousands of dollars to society and to the patient.”

Because the pain felt by sickle cell patients can be hard to predict, Liles said the results of the study could be recorded in retrospect. Patients like Majara Mooring realized they hadn’t been to the hospital as often as they used to.

“After the trial was over, my doctor – which is Dr. Liles – we sat down and talked. She was like ‘well, how (have) you been feeling? And I said ‘I’m fine’ and she said ‘you haven’t been in the hospital’ and I said ‘you know, sitting here, thinking about that, you’re right.’”

The pain didn’t go away completely, Mooring said but through previously prescribed medication and coping techniques developed over a lifetime, she was able to take care of herself and avoid visits to the ER.

“So, I do hope this is a cure, I hope this the beginning of helping not only me but other adults and kids that are out there suffering with sickle cell disease.”

Liles said those hospital visits can be long and excruciating.

“I think that one of the problems that the patients perceive is when they have one of these crisis, they come in the emergency room and people say ‘well your arm’s not broken, you’re not bleeding anywhere. I can’t see your pain.”

In light of the study’s success, Liles and Mooring hope this new treatment brings increased awareness to Sickle Cell Disease – which impacts communities that continue to fight for recognition – though the ailments aren’t always visible.