“I remember so clearly what the senior hematologist in Accra told us – she said, please don’t do what other companies do, don’t come in and start something and then leave,” says Fionnuala Doyle, who was V.P. and Head of Oncology Policy & Healthcare Systems at the time. “And we gave her our word – we promised we would not let that happen.”

The hematologist was referring to a six month project, which was started in 2014 and was run by Novartis’ Executive Female Leadership Program (EFLP*), to map out a theoretical plan to improve outcomes for people with sickle cell disease in sub-Saharan Africa. 

When the six months – and the EFLP’s sponsorship – came to an end, the team had a plan in hand. Doyle and several others couldn’t let themselves walk away from what they had started, still remembering the hematologists’ plea. They had already found their own funding to visit their partners in Africa and they were so compelled by what they hoped to accomplish that they went on to find funding to keep their project alive for several years. 

Their commitment built the trust and credibility that would later be the foundation of the much more comprehensive Novartis Africa Sickle Cell Disease Program, in which they joined forces with Jonathan Spector, who leads Global Health efforts at the Novartis Institutes for BioMedical Research (NIBR).

“During those first years, we had to push and push to get funding, but it was just impossible for us to let our partners and their patients down,” says Doyle. “Then I met Jonathan, and he and Jay Bradner had the resources to do so much more – I’m so thrilled Novartis is behind this now too.”

Straightforward opportunities for dramatic improvement

The team’s first objective was to identify where to begin in Africa. Seeking countries with significant unmet medical need as well as the best chances of making a lasting difference, the team quickly identified Ghana due to its political stability, relatively established healthcare system and the enormous unmet medical need there. 

“When we went to Ghana, we saw that sickle cell disease patients are living in horrific pain, all the time,” says Doyle. “They don’t even have very simple, straightforward strategies like knowing to drink enough water and basic pain relief, which can help dramatically.”

Ghana also had no consensus treatment guidelines, so patient care varied dramatically depending on where a person lived. For example, newborn screening existed in Kumasi – Ghana’s second largest city – so parents could get their children minimal treatment. But in Accra, Ghana’s capital, no such screening existed. 

“In Accra, parents were bringing in babies as young as one or two years old who were having preventable strokes,” says Doyle. “These babies were dying because they hadn’t been screened and their parents didn’t know to look out for the basic signs of a sickle cell crisis.”

The Nkwa ji – saving lives – project

During the team’s trip to Ghana, Roland Hammond helped them meet with local hematologists, nurses, patient groups, NGOs, blood banks and politicians. It was through these meetings that they met Dr. Kwaku Ohene-Frempong, a world expert in sickle cell disease and also the architect of the screening program in Kumasi. 

Doyle and her collaborators’ plan evolved into what was then called the Nkwa ji, or saving lives, project and they all agreed that the first goal was clear – to establish consensus treatment guidelines, with a particular focus on newborn screening. From there, their objectives included making care packs with basic pain relief available, as well as facilitating affordable access to hydroxyurea. This generic medicine has been the standard of care in the EU and the USA for decades, but hydroxyurea was – and still is – prohibitively expensive in many parts of Africa. 

“None of the doctors were using hydroxyurea or even considering it because it was simply unaffordable,” says Doyle. “But when we asked whether they would use hydroxyurea if the price wasn’t an issue, nearly everyone said, ‘Oh yes! You can’t imagine the difference it would make!’”

A blueprint for the future

Doyle met Spector in 2015 and, soon thereafter, Spector attended a sickle cell disease conference in Africa, which included the first presentation of Ghana’s new consensus treatment guidelines. Spector’s subsequent support brought more and more momentum and resources to the project, allowing it to become what it is today. 

“When I saw Jonathan’s vision and I knew that Jay Bradner was behind him, I knew that something really good was happening,” says Doyle. Although she handed over leadership of the Nkwa ji project to Spector and others, she hasn’t forgotten her promise to the hematologist in Accra. “I’ve been able to pull back a little bit, but I still sometimes see her face at night.” 

Sickle Cell Facts

Sickle cell disease is an inherited blood disorder in which red blood cells are affected. Patients can go through severe periods of pain and may suffer from other complications, including stroke. It is estimated that, worldwide, more than 4 million people are suffering from the disease. Most of them live in sub-Saharan Africa. 

* EFLP participants: Ann Parniex, Ming Xu, Marina Udier, Lisa Deschamps and Karen Netherton.