By FRANCA OFILI

With an estimated four million Nigerians living with sickle cell disease in the country, experts have urged the government to invest in local manufacturing of gene therapy treatment.

They made the call during a media briefing at the 5th Global Sickle Cell Congress in Abuja.

Prof. Jennifer Adair, Co-founder, Global Gene Therapy Initiative, explained gene therapy to be 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 involved the use of genetic material to prevent, treat and cure disease.

According to her, if all components are manufactured locally, the cost of the gene therapy will go down much further.

“So this is the opportunity that governments have to invest locally.

“If the government invests in local economy and local manufacturing of these approaches, it can be done much more affordably and within the scale of the economy in which it is operating.

“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 been taking 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 focuses 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 at the Children’s Hospital of Philadelphia, U.S., said that some incremental steps had occurred in terms of improving outcomes for sickle cell disease.

Thompson said that in the last five to ten years, they had discovered an 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 them 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 in a bid to find cure for the disease, he had enrolled in a gene therapy trail.

“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 curative therapies across the board,” he said. (NAN)