By Franca Ofili

As the country has an estimated four million Nigerians living with sickle cell disease, health experts emphasise the need for Nigeria to adopt gene therapy for treatment of sickle cell disease.

Prof. Jennifer Adair, Co-founder, Global Gene Therapy Initiative, made the call during a news briefing at the 5th Global Sickle Cell Congress in Abuja.

According to her, gene therapy is a new curative treatment for sickle cell disease which uses the patient’s own bone marrow cells, negating the need for an unaffected donor.

She said that gene therapy was the use of genetic material to prevent, treat and cure disease.

“The transformative potential of gene therapy comes from its targeting of underlying causes of disease rather than treatment of symptoms.

“To date, no gene therapy clinical trial for sickle cell has taken place in Africa where the majority of patients are,” Adair said.

She said that the congress was necessary as Nigeria bore the largest proportion of patients living with sickle cell disease in the world.

“It is so important to us to let people of Nigeria, especially those warriors who live in the country, see the possibilities for curative therapies such as gene therapy and also bone marrow transplant as possibilities for a better future for themselves,” she said.

According to her, the two days congress focused on addressing the global burden of sickle cell disease, including discussions on gene therapy, bone marrow transplants, and other emerging treatments.

Also speaking, Dr Alexis Thompson, a Physician Scientist in the United States at the Children’s Hospital of Philadelphia, said that some incremental steps have occurred in terms of improving outcomes for sickle cell disease.

Thompson said that for the last five to ten years, they saw opportunity to apply science in a way that would allow the patients to be their own donor.

“They will also conceivably have a long-term effect with controlling their sickle cell disease,” Thompson said.

According to her, they have done the trials for both of the approved products at her institution as well as some of the ones that are currently in clinical trial.

“It is a field that we are far from over in terms of looking at innovation, but what is most extraordinary is the transformation in children and adults’ lives by giving the opportunity to undergo gene therapy and other curative treatments.

Again, a patient treated for sickle cell disease by gene therapy, Mr Jimi Olaghere said that he had a comprehensive screening and care for sickle cell disease.

Olaghere said that as a result to find cure to the disease, he enrolled in a gene therapy trial.

“Before I was born, my mom did not have access to prenatal screening and fortunately, she was able to fly to the U.S. and get that prenatal screening done.

“That prenatal screening confirmed that I would have sickle cell disease, so my mother decided to give birth to me there.

“And fast forward to 35 years after being born, I got fortunate to participate in a clustered regularly interspaced short palindromic repeats (CRISPR-based) gene therapy that has completely changed my life.

“CRISPR-based gene therapy utilises CRISPR technology to precisely edit genes and correct genetic defects that cause diseases.

“So, I am excited to come home and show the community the impact of these gene therapies and just curative therapies across board,” he said. (NAN)(www.nannews.ng)

Edited by Vivian Ihechu