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Uganda to start trials on gene therapy for sickle cell treatment

Sickle cell patient under treatment. Often they require bone marrow transplants. File Photo

Kampala, Uganda | THE INDEPENDENT | A team of scientists is to embark on a research trial about the new sickle cell treatment and some other complicated diseases.

Dr. Martin Ongol, the Acting Executive Secretary Uganda National Council for Science and Technology-UNCST, says that the gene therapy treatment technology was discovered several years back in some other countries like the USA.

According to Ongol, Uganda will be taking on the research trials at the Joint Clinical Research Centre-JCRC to help patients who besides living with the pain forever, also have to meet a lot of costs for treatment.

Dr. Ongol explains that with the new gene therapy treatment technology, a small piece of the bone marrow from a normal person is inserted into the bone marrow of a sickle cell patient.

Dr. Ongol says that currently, the council is looking into the regulatory environment, ethics, and practice in Uganda to ensure that the research does not cause harm to the participants in the study. He says that the process will last six months to come up with the guidelines.

He also says that facilities will be set up for training, bringing expertise from abroad, infrastructure, and systems.

Currently, the only option for long-term survival for sickle cell patients is the replacement of unrelated Hematopoietic Stem Cells-HSCs, the bone-marrow-based progenitors of all blood cell types from a healthy and immunologically compatible donor.

However, patients can be given pain killers to ease pain, antibiotics for infections, and anti-malarial drugs for malaria.

Ruth Nankanja Mukiibi, the Executive Director of Uganda Sickle Cell Association says they are eagerly waiting to embrace the new treatment technology.

According to Ministry of Health statistics, of the 1,600,000 births in Uganda per year, 33,000 babies are suspected to be born with sickle cell disease. Of these, 80 percent die before the age of five. Those who survive beyond five, hardly make it to their 18th birthday. Worldwide, 400,000 babies are estimated to be born with the disease. Of these, half are from Sub-Saharan Africa.

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