Public health experts have emphasised the urgent need for Nigeria to adopt life-changing gene therapy for the treatment of sickle cell disease.
Official data shows that approximately 4 million Nigerians, about 2% of the population are living with the condition.
Prof. Jennifer Adair, co-founder of the Global Gene Therapy Initiative (GGTI) and faculty member at the University of Massachusetts Chan Medical School, stressed the urgent need to introduce curative treatments such as gene therapy and bone marrow transplants in countries bearing the highest burden of sickle cell disease.
She made this call in Abuja during a press briefing at the 5th Global Sickle Cell Congress, alongside Dr. Alexis Thompson, a physician-scientist at the Children’s Hospital of Philadelphia and a lead investigator in U.S. gene therapy trials, and Jimmy Olaghere, a Nigerian-born recipient of the groundbreaking gene therapy.
Emphasizing the need for Nigeria to formally adopt gene therapy with appropriate administrative structures and legislative support, she noted that embracing the therapy is essential not only for its many benefits but also for advancing scientific progress in the country.
Gene therapy is a medical approach that treats or prevents disease by modifying a person’s genes. It typically involves replacing, repairing, or regulating defective genes—often by introducing a healthy copy to replace a faulty or missing one—thereby addressing the root cause of the condition.
“Nigeria bears the highest number of sickle cell patients globally. It is vital that those affected, ‘warriors,’ as they are often called, to see that curative options are not science fiction, but real and increasingly within reach,” she said.
She noted that gene therapy is not entirely new to Nigeria, as the country is already part of the GGTI, a coalition of nations committed to building local capacity for research, regulation, and access to gene-based treatments.
According to her, among the first six countries in the network, Uganda has already passed legislation to support bone marrow transplants and gene editing.
Adair noted that Nigeria has the necessary foundation, scientists, clinicians, regulators, and patient advocates but must align these elements into a cohesive national strategy.
She said Nigeria would not be alone in the process as GGTI also supports knowledge transfer, including training programs between Nigeria, India, and the U.S., and promotes access to technical information in local languages.
“We are working to support clinicians who want to learn how to manufacture and administer these therapies locally,” she said.
Addressing concerns about affordability in countries with limited healthcare funding, Adair acknowledged the high cost of gene therapy in the U.S., currently ranging between $2 million and $3 million.
However, she noted that the figures are based on value-based pricing models rather than actual manufacturing costs.
She cited an example from Uganda, where the same therapy could be cost-effective at approximately $40,000, adding, “While $40,000 is still a steep cost, it represents a starting point for investment.
“With local manufacturing, the cost can fall significantly”.
Adair referenced the Christian Medical College in Vellore, India, where a similar therapy for cancer is produced for about $35,000, despite importing many materials.
“With local production and training, prices will drop and capacity will grow,” she added.
On her part, Dr. Thompson described the last decade in hematology as revolutionary, saying, “What was once unthinkable, using a patient’s own genetically modified cells to control or even cure sickle cell disease is now a reality”.
While acknowledging the risks, including infertility, infection, and rare cases of cancer, she emphasized that many patients have experienced life-changing results.
“This isn’t for everyone, but for many, the promise of a life without crisis pain is worth the trade-offs,” she explained.
She added that not all sickle cell patients will qualify for gene therapy, noting that eligibility depends on factors such as age, severity of disease, organ health, and willingness to undergo intensive conditioning.
“But with earlier screening and better interventions, more patients could become eligible over time,” she said.
American-born Jimi Olaghere, who underwent a successful Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-based gene therapy in the United States four years ago, was diagnosed with sickle cell disease before birth.
The Atlanta-based technology entrepreneur shared his personal testimony on the effectiveness of the therapy, saying, “I lived 35 years with pain crises and frequent emergency room visits. Today, I’m virtually pain-free.
“I’ve told my story around the world, but this is the most meaningful stop for me because this is home.
“The therapy changed my life. From constant hospital visits to climbing Mount Kilimanjaro, the difference is unimaginable.”
Still, he cautioned that the process is not easy, saying, “There’s chemotherapy, hair loss, isolation, it took me nearly a year to recover. But if you’ve lived with sickle cell, you’ve already survived worse”.
Highlighting the scale of Nigeria’s burden, Prof. Obiageli Nnodu, Special Adviser on Sickle Cell Disease to the Coordinating Minister of Health and Chair of the Congress’ Local Organising Committee, noted that data from the 2018 National Demographic and Health Survey showed that around 2% of Nigerians live with the disease, an estimated 4 million people.
She said the staggering figure highlights the need to integrate advanced therapies like gene therapy into Nigeria’s healthcare system.
“Nigeria continues to bear one of the highest rates of sickle cell disease globally. We cannot ignore curative innovations that have the potential to transform lives,” she said.
