A recent article in the journal Nature elaborated on the work of Dr. Ted Love who left early retirement in 2013 to devote his talents and experience to the development of a new drug to defeat sickle cell disease (SCD).
His efforts at Global Blood Therapeutics as CEO were rewarded last year when the FDA approved the newly developed drug voxelotor (Oxbryta) for SCD.
About SCD
SCD is a genetic condition. Red blood cells change into a sickle shape and block blood flow and oxygen. Occasionally these modified cells burst, increasing the risk of infections, stroke, organ damage, and causing severe pain.
The life expectancy of SCD patients is reduced by about thirty years. SCD mostly affects Hispanics and African Americans.
According to the Center for Disease Control, SCD affects millions of people worldwide. SCD occurs in one in 365 African American infants and one in 16,300 Hispanic births in the U.S.
About Hydroxyurea
Until work began on Oxbryta several years ago, SCD patients were left with only one drug, Hydroxyurea, which came with multiple side effects. The only other option was a bone marrow procedure that was rarely used.
About Oxbryta (voxelotor)
The function of Oxbryta, an anti-sickling drug, is twofold. It prevents hemoglobin from forming clumps and also prevents cells from becoming sickle-shaped.
When anti-sickling drugs were slipped into cells of SCD mice, the anti-sickling hemoglobin was able to enter ninety-nine percent of red blood cells, and key symptoms were reversed.
The HOPE 2021 Study
The HOPE study of Oxbryta involved 274 SCD patients ages 12 to 65. Results of the study showed that up to 89% of those taking Oxbryta had a substantial increase in hemoglobin and fewer incidents of anemia.
Seventy-four percent of patients taking Oxbryta were found to be either moderately or considerably improved. Patients in the placebo cohort, however, were only forty-seven percent improved.
An Opportunity to Serve
Dr. Love recalls that his motivation for his work on the development of the drug Oxbryta goes back forty years. It was about that time that Dr. Love witnessed people who had sickle cell disease (SCD) being treated with skepticism and as if they were just searching for drugs.
Fast forward to 2013 when Dr. Love happened to see an article about an experimental drug being developed for SCD called Oxbryta (voxelotor). At that very moment, he decided he must join the effort to develop the drug.
A Patient’s Life With Sickle Beta-Cell Thalassemia
Michelle Parr, of Pittsburgh, PA, was twenty-five years old when she was told she had Sickle Beta-Cell Thalassemia, a subtype of SCD. By the time Michelle was forty years of age, she was spending at least one week in the hospital four times a year. Michelle required frequent blood transfusions and additional oxygen.
In 2017 Michelle heard about a clinical trial for Oxbryta and joined the study immediately. Within just a few days her condition improved. She continued to have much more energy and much less pain.
Eventually, she was well enough to become an advocate for the disease. Michelle also worked long-term for an SCD awareness organization in the Pittsburgh area. Michelle hopes others with SCD experience the same positive results.
