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Clinical trial shows possible breakthrough for sickle cell disease

PEMBROKE PARK, Fla. – An ongoing clinical trial for a new gene therapy for sickle cell disease continues to show promising results.

Sickle cell disease is a painful and debilitating genetic blood disorder that has limited treatment options.

The trial involves an experimental gene editing cell therapy that modifies a person’s own stem cells to correct the mutation responsible for the disease.

“We are seeing really very encouraging results where this patient that is almost a year now form the gene therapy has had no pain crisis that occurred after receiving the modified cells and also in the other patient and also hear from other colleagues who are participating in this clinic trial who are showing similar results for their patients,” said Dr. Rabi Hanna, Principal Investigator at Cleveland Clinic Children’s Hospital, one of the sites for the clinical trial.

Along with pain relief from the disease, initial results showed new white blood cells in all patients at about four weeks with no severe side effects.

Similar benefits have recently been seen in patients enrolled in other clinical trial sites.

The nationwide study is slated to continue through 2024.

STUDY QUESTIONS OPIOID BENEFIT FOR BACK PAIN

And an Australian study of 310 people suffering from neck or lower back pain found opioids weren’t any more helpful than the placebo.

Researchers also found opioids appear to cause harm even after short-term, sensible use.

The opioid group had worse mental health scores and more reports of nausea, dizziness and constipation compared to the placebo group.

Opioid medications are one of the most typically prescribed treatments worldwide for treating lower back pain.


About the Authors
Kristi Krueger headshot

Kristi Krueger has built a solid reputation as an award-winning medical reporter and effervescent anchor. She joined Local 10 in August 1993. After many years co-anchoring the 6 p.m. and 11 p.m., Kristi now co-anchors the noon newscasts, giving her more time in the evening with her family.

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