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Clinical trials set for breakthrough sickle cell gene therapy technique

Using the patients’ own bone marrow

Lisa Olivia Fitch | 8/1/2013, midnight
A sickle cell beside normal cells

Symptoms vary from person to person, but the disease often causes pain, problems breathing and over time, strokes or progressive kidney problems that can lead to death in patients before they reach age 50.

“Our hope is that we can provide a treatment which can give patients a better and longer life,” Kohn said.

The prevalence of the disease in the U.S. is approximately one in 500 African American children, according to the National Institutes of Heath. Three quarters of sickle-cell cases occur in Africa. The disease is prevalent in malaria-stricken areas, where people’s chances of survival actually increase if they carry the sickle-cell trait.

Test tube chemistry in the laboratory so far suggests that an engineered version of beta globin can prevent the SCD patient’s red cells from sickling.

As a result, you would think SCD sufferers would be lining up to participate in upcoming clinical trials. But historically, African Americans have shied away from medical trials. There’s a history there. A mistrust. A communication issue.

Kohn hopes that the communication issue is waning. A stem cell transplant process was highlighted recently, when Good Morning America host Robin Roberts announced she was undergoing this type of procedure to treat her myelodysplastics syndromes (MDS). Her sister was her donor.

Clinical trials are medical research studies used to determine whether new drugs or treatments are safe and effective. The Food and Drug Administration considers the results from clinical trials in approving a drug or treatment. Once approved, the therapy becomes available for doctors to prescribe to patients.