People living with Sickle Cell Disease (SCD) in Nigeria have lamented that the high cost of medication and poor access to comprehensive care are weighing heavily on them and their families.
They also decried the lack of access to new and advanced medica therapies that have been proven to cure the disease in other countries such as gene therapy, and bone marrow transplant, reports Daily Trust.
The patients also said till date, they face neglect, and stigma in employment among other challenges.
Ahead of this year’s World Sickle Cell Day, people suffering from the disease are calling on government to improve access to healthcare, subsidize essential medications, and strengthen public education on genotype compatibility.
The World Sickle Cell Day is commemorated on June 19 every year.
They called for the establishment of a national agency for the control of sickle cell disease, and a center that exclusively caters for patients.
Medical experts have also said there is need for increased investments and adoption of new therapies for sickle cell disease such as gene therapy in high burden countries such as Nigeria. They said it will go a long way to provide succour for people suffering from sickle cell disease.
Prof. Obiageli Nnodu, a haematologist, said gene therapy is a type of treatment in which an abnormal disease causing gene is taken out of a cell and replaced by a healthy gene to correct the deficiency.
She said, “It Involves altering the genetic material within cells to correct underlying genetic defects. Sometimes, a functional copy of a faulty gene is added such as Beta Globin A in sickle cell disease to make non-sickle red cells or by disabling a disease-causing gene.”
Sickle cell disease is a genetic blood disorder that affects a person’s red blood cells and passed on from parents.
The disorder causes normal round and flexible blood cells to become stiff and sickle shaped, which in turn stops the blood cells and the oxygen they carry from moving freely around the body.
According to the Coordinating Minister of Health and Social Welfare, Prof. Muhammad Ali Pate, Nigeria stands out as the most sickle cell disease endemic country in Africa and globally, ahead of India and the Democratic Republic of Congo, with an annual infant death of 100,000 representing 8% of infant mortality.
The minister said in many countries of the African region including Nigeria, 10%–40% of the population carries the sickle-cell gene resulting in estimated prevalence of at least 2%.
He said in Nigeria, sickle cell disease contributes significantly to both child and adult morbidity and mortality.
“The 2018 NDHS report put the prevalence of the disease in the country at 1% while 25% of Nigerians are healthy carriers of the disease,” he said.
Prof. Nnodu, who is also the director of the Centre of Excellence For Sickle Cell Disease Research & Training, (CESRTA) said sickle cell disease exerts a huge economic burden for individuals and their families because of the catastrophic health expenditures that it poses due to frequent crisis and complications involving many organ systems over the life course.
“Some complications such as stroke and chronic kidney disease, chronic leg ulcers, priapism also prevent an individual with sickle cell disease from making meaningful contributions to their families in various ways,” she said.
‘Our experiences living with sickle cell disease’
Nazifi Dawud, a journalist in his mid-40s, said sickle cell disease’s lifelong management and the associated cost pose serious financial challenges to patients like him and families with children or adults suffering from it.
He said routine drugs such as hydroxyurea, folic acid and hydrochloride tablets, known to improve the quality of life of sickle cell disease patients, are expensive.
He urged the government to subside or make the drugs and treatment of the disease free. He also recommended the establishment of a national agency for the control of sickle cell disease and a center that would exclusively cater for patients.
Dawud believes that developing new therapies and improving quality of life for the patients remain critical to correcting the generic defect that leads to sickle cell disease.
“The high cost of treating sickle cell disease in Nigeria is evident in the need for frequent blood transfusions, regular hospital visits, and the use of various drugs and supportive therapies.
“It is not uncommon for a single blood transfusion session to cost between N20,000 and N50,000.The cost of medical care including the cost of drugs and medical consultations, further adds to the financial burdens for families,” he said.
He said patients are often stigmatised and many face discrimination in employment, education and access to health services leading to unintended consequences.
Another patient, Abdullahi Sani Bashir, who is on admission at the Aminu Kano Teaching Hospital said a single dose of injection cost him N35,000 in addition to other drugs and hospital bills.
Bashir, who is in his 20s, said his fragile condition has made it difficult for him to pursue education beyond secondary school, so he embraced mobile phone sales business to eke out a living.
Bashir has not been attending his mobile phone business since his latest hospitalisation, thereby taking a toll on his income and life savings. This, he said, happens oftentimes when his business needs his attention.
In Edo State, sickle cell sufferers said they are facing challenges in accessing drugs and quality healthcare in the state.
A sickle cell disease sufferer, and president of Edo State Sickle Cell Club, Charles Edigin, 56, said they were neglected and that the immediate past administration increased money for accessing treatment and drugs.
He said the club and other critical stakeholders are discussing with the new administration to address the neglect of sufferers in the area of drug subsidies and access to quality healthcare.
“For now, the government has reduced the cost of drugs and test a little and also restored the 24-hour service that was scraped by the immediate past government.
Charles, who is also the founder, Edigi-Charles Foundation for Sickle Cell, noted that the amount of money a sufferer spends in a month depends on the number of sick cell crisis experienced.
“Sufferers’ crises are different from one another. For me, I have not fallen into crisis in the last two to three months, but some can have a crisis every two to three days; others may be two or three weeks in a month.
“When you are admitted at the hospital, it will cost about N50,000, N100,000, N150,000 and above, depending on the severity of the crisis, coupled with the high cost of drugs.”
Tomi, a 26-year-old female living with sickle cell disease in Lagos, shared that she did not understand her condition while growing up, and often engaged in strenuous activities without complications. However, as she matured, she became more aware of the limitations imposed by her genotype.
Tomi described frequent pain crises, difficulties accessing proper care, and emotional strain. “There are general hospitals near me, but I have to go all the way to Idi-Araba to get proper sickle cell facilities,” she said. She also highlighted challenges in sourcing essential medications such as hydroxyurea.
She called on government authorities to subsidise sickle cell medications, equip general hospitals with specialised facilities, and intensify public sensitization on genotype compatibility to reduce the incidence of sickle cell births.
Another respondent, Barnabas Obande, a data engineer in Lagos, described living with sickle cell has “a normal routine on most days,” but said crises often lead to prolonged hospital admissions and severe pain.
“During a crisis, the pain is excruciating, especially in the joints and bones,” he said. “I once spent over a month in the hospital and was unconscious for seven days.”
Obande raised concerns about healthcare delivery, stating that some public hospitals lack adequate personnel trained in managing sickle cell complications.
He urged policymakers to prioritise awareness campaigns and expand support for foundations working with sickle cell patients.
Dr Aminu Sudawa, a medical doctor at the Kano State Centre for Disease Control, advised patients to avoid common infections like malaria which may worsen their conditions.
Sudawa also advised patients to form a community of sickle cell disease patients to share information among themselves and mobilise blood donors for patients.
Dr Sudawa said the mandatory premarital test introduced for intending couples in Kano is a significant move to reduce the burden of the disease.
How investments in gene therapy can provide succour for sickle cell disease sufferers in Nigeria
Jimi Olaghere received gene therapy treatment for sickle cell disease four years ago in the United States. Since then, he has been cured of the disease.
Narrating his experience to our correspondent, he said he lived with sickle cell disease for 35 years before undergoing gene therapy, adding that it has completely changed his life.
The Atlanta-based technology entrepreneur said he lived with pain crises and frequent emergency room visits but that today, he is “virtually pain-free.”
He said, “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 trial that has completely changed my life. I have travelled around the world telling my story, but this is the most important destination, Nigeria, where I feel like my story needs to be heard, because this is where I am from.
“So I am just really excited to come home and really show the community the impact of these gene therapies and other curative therapies. From what my life was like to what my life is right now, I’m excited to share this story.”
He said the treatment is, however, not a ‘walk in a park’, adding that it took him nearly a year to recover.
“But if you have lived with sickle cell disease, you have already survived worse,” he added.
Olaghere added that he was able to climb Mount Kilimanjaro after treatment as a result of gene therapy.
Prof. Jennifer Adair, co-founder of the Global Gene Therapy Initiative (GGTI) and a faculty member at the University of Massachusetts Chan Medical School, said there was urgent need to introduce curative treatments such as gene therapy and bone marrow transplants in countries with the highest burden of sickle cell disease, such as Nigeria.
She said, “Jimi has had a tremendous response following gene therapy treatment, including living mostly pain-free since that treatment happened. Since then, he has been thriving physically and trying to manage the emotional changes that come with such a large transition.”
Prof. Adair highlighted that it is important that those affected see that curative options are not science fiction, but real and increasingly within reach.
She said, “It is so important for us to let people of Nigeria, especially those warriors who live here in-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 was speaking during the recent 5th Global Sickle Cell Congress in Abuja.
On the benefits of gene therapy for sickle cell disease patients, Prof. Nnodu said gene therapy is transformational, reduces the frequency of crisis, and restoring health and wellbeing.
“Gene therapy corrects the abnormal gene, reduces its production in the body and restores the individual to health and normal living,” she stated.
Also, Prof. Adair, who is also the vice chair, Department of Genetic and Cellular Medicine, UMass Chan Medical School, said as a medical professional, she has witnessed the transformation in the lives of children and adults offered the opportunity to undergo gene therapy and other curative treatments.
She said, “Many of us have taken care of sickle cell patients for most of our careers, and we were certainly happy to see some of the incremental steps that have occurred in terms of improving outcomes for sickle cell disease. But the last five to 10 years, seeing this opportunity to apply science in a way that would allow the patient to be their own donor and conceivably have a long-term effect with controlling their sickle cell disease, at some point would have been almost unimaginable.”
She added that till date, no gene therapy clinical trial for sickle cell disease has taken place in Africa, where the majority of patients are.
Dr Alexis Thompson, a physician-scientist at the Children’s Hospital of Philadelphia and a lead investigator in US gene therapy trials, said current gene therapy clinical trials have focused on individuals between the ages of 12 and 35, adding that patients outside the age range could still benefit from the therapies.
She said, “But with earlier screening and better interventions, more patients could become eligible over time.”
Alexis, who is also a former president of the American Association for Haematology, said fertility preservation is recommended before the procedure.
She also applauded gene therapy treatment 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.”
Dr Thompson said, like all other treatments, there were some risks such as infertility, infection and rare cases of cancer, but that, “This isn’t for everyone, but for many, the promise of a life without crisis, pain is worth the trade-offs.”
She added that most patients who participated in their trials travelled far away from home, even within the United States, to undergo gene therapy, adding, “In the future, that should not be the case. They should be able to get these therapies in a way that actually does not require that they disrupt their lives as much as they have had so far.”
Affordability of gene therapy
Despite its curative effects, there are concerns about equitable access to gene therapy treatments in Nigeria and other countries in sub-Saharan Africa.
Before recent advancements in gene therapy, bone marrow transplant was the only cure for sickle cell disease.
Nigeria has a few centres for stem cell therapy for sickle cell disease, but most patients have not been able to access the treatment due to the high costs.
A mother of two children suffering from sickle cell disease said meeting the cost of hospital bills during crises has not been easy, not to mention expensive treatments like bone marrow transplant and gene therapy.
Another patient, Ibrahim, said he first heard of the treatment in the news in 2023 when the FDA approved two gene therapy treatments.
While saying it is a silver lining for reducing the burden of the disease in the country, he noted that most sufferers and their families in Nigeria cannot afford the cost of treatment.
On the affordability of the procedure, Prof. Adair said government’s involvement through local production and training will help reduce the cost of gene therapy.
“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,” she said.
She acknowledged that the current cost of the gene therapy treatment Olaghere received ranged from $2 million to $3 million in the US.
However, she said the US cost is not reflective of what the cost will be in every country.
The medical expert cited the example of Uganda, where the same therapy could be cost-effective at approximately $40,000, saying it represents a starting point for investment.
“Now, $40,000 is still a lot of money. We don’t think that will be the ultimate price, but it is a starting point that could be leveraged to give access to the patients who could most benefit from gene therapy now to help implement the clinical research and the access for patients moving forward.”
She also said Nigeria is part of the GGTI, a coalition of nations committed to building local capacity for research, regulation, and access to gene-based treatments.
She said the GGTI also supports knowledge transfer, including training programmes between Nigeria, India and the US, and promotes access to technical information in local languages.
Nigeria has the foundation for investments in gene therapy, such as clinicians, researchers and patient advocates, among others, she said, adding, “We are working to support clinicians who want to learn how to manufacture and administer these therapies locally.”
How FG is responding to sickle cell disease
The Coordinating Minister of Health, Prof. Muhammad Ali Pate, said the government recognises the huge burden of socio-economic and psychological effects of sickle cell disease, and has instituted policies and several strategic interventions to address the challenges.
Speaking during a World Sickle-Cell Day event, he said, the government has put in place policies and interventions to improve care for sufferers in the country.
He said they include the development of the first national guidelines for the prevention and control of the disease and the Universal Newborn Screening policy adopted to ensure that all children born in Nigeria are screened for sickle cell disease, among others.”
