With an estimated four million Nigerians affected by sickle cell disease, experts have called on the federal government to invest in the local production of gene therapy treatments.
The appeal was made during a media briefing at the 5th Global Sickle Cell Congress held in Abuja.
Prof. Jennifer Adair, Co-founder of the Global Gene Therapy Initiative, described gene therapy as a new curative treatment for sickle cell disease.
She explained that it uses a patient’s own bone marrow cells, removing the need for a donor without the condition.
According to her, gene therapy involves the use of genetic material to prevent, treat, and potentially cure disease, and holds great promise for addressing Nigeria’s high burden of sickle cell disorder.
Targeting the root cause
The transformative potential of gene therapy lies in its ability to target the underlying causes of disease rather than just treating 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 further explained that if all components are manufactured locally, the cost of gene therapy would decrease significantly.
“So, this is the opportunity that governments have to invest locally. If the government invests in the 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.”
Nigeria’s burden and the importance of the congress
She said the congress was necessary because Nigeria bears the largest proportion of patients living with sickle cell disease in the world.
According to her, the two-day congress focuses on addressing the global burden of sickle cell disease, including discussions on gene therapy, bone marrow transplants, and other emerging treatments.
“It is so important to us to let the people of Nigeria, especially those warriors who live in the country, see the possibilities for curative therapies such as gene therapy and bone marrow transplant as options for a better future for themselves,” she said.
Advances in treatment and research
Also speaking, Dr. Alexis Thompson, a Physician Scientist at the Children’s Hospital of Philadelphia, U.S., said that some incremental steps have been made in improving outcomes for sickle cell disease.
“In the last five to ten years, we have discovered an opportunity to apply science in a way that allows patients to be their own donors. They will also conceivably experience a long-term effect in controlling their sickle cell disease,” she added.
According to her, her institution has conducted trials for both approved products as well as some currently in clinical trials.
“It is a field that is far from over in terms of innovation, but what is most extraordinary is the transformation in the lives of children and adults by giving them the opportunity to undergo gene therapy and other curative treatments,” Thompson explained.
Patient Experience: A life changed by gene therapy
Additionally, a patient treated for sickle cell disease with gene therapy, Mr. Jimi Olaghere, said he had undergone comprehensive screening and care for the condition.
Olaghere said that in his bid to find a cure for sickle cell disease, he enrolled in a gene therapy trial. Before he was born, his mother did not have access to prenatal screening, but she was fortunate to travel to the U.S. to get it done. The screening confirmed that he would have sickle cell disease, leading his mother to decide to give birth to him there.
Thirty-five years later, Olaghere was fortunate to participate in a CRISPR-based gene therapy trial, which completely changed his life. This gene therapy uses precise gene-editing technology to correct the genetic defects that cause diseases.
He expressed excitement about returning home to share with his community the transformative impact of these genes and curative therapies.
