"We fight the bacteria, guns a blazing!" says Johannesburg-based investment banker Cheri Nel.
Nel has cystic fibrosis (CF) and refers to an ongoing stand-off between bacteria and immune cells in her lungs. She has check-ups with her specialist doctor, Dr Cathy Baird, every six weeks or at the slightest hint of a cold, as any bacteria or virus could be deadly.
"I have to be hypervigilant because otherwise the bacteria will really colonise my lungs and stay there forever. I always say bacteria is on the lookout for CF lungs like, fantastic, we'll go live there! CF lungs offer ideal conditions for bacteria to really nest in," she says.
Fortified by a small pharmacy of pills and syrups (Nel takes 25 different prescription medicines a month), daily nebulizing treatments, and occasional small insulin jabs, she has now also launched a legal stand-off against United States biotech giant Vertex Pharmaceuticals, in a bid to bring their CF "miracle drug" Trikafta to South Africa.
Over the past two years, Nel exchanged emails with Vertex executives and even had a virtual meeting with company representatives in Boston, but they wouldn't budge. Their responses, according to her, allude to bureaucratic red tape in developing countries.
The altercation came to a head on February 7 when Nel filed a 123-page founding affidavit before the Court of the Commissioner of Patents, a specialist court of the Gauteng Division of the High Court of South Africa, accusing Vertex of patent abuse and of infringing on the constitutional rights of South African CF patients to access the life-saving drug. Including four additional expert affidavits and numerous annexures, the total submitted document was 1 834 pages long. (Spotlight reported on the case in-depth here.)
In the landmark court case, Nel is fighting to have Trikafta or generic equivalents made available in South Africa, which might well set a precedent for poor countries around the world.
On March 7, Vertex filed its intention to oppose. Since then, the company asked for an extension to file their answering affidavit, with a date still to be decided. But the clock is ticking.
"I know of two patients who are literally gasping for air," says Nel. "If they don't get the drug in the next month, they're going to die."
In her affidavit, Nel says at least 30 South Africans have died of CF since Trikafta was first approved for use in the United States in 2019.
'An awful lot of money'
So far, Nel has ploughed R2 million of her own money into the court case. "Yes, this lawsuit is costing me an awful lot of money. I am busy setting up a crowdsourcing page to try and reimburse some of the legal fees. I mean, one person can't carry all of this," she says.
According to The New York Times, Vertex has reported more than $17 billion in sales for Trikafta since 2019.
While Nel is reaching deep into her own pockets, the pharmaceutical company is raking in profits. According to The New York Times, Vertex has reported more than $17 billion in sales for Trikafta since 2019.
Nel describes herself as "curious and information hungry", saying that she has researched the matter from patient, healthcare, legal, and scientific points of view. She notes that while profit is important for incentivising pharmaceutical companies to push for excellence in scientific research and development, profit margins need to be balanced against human rights imperatives.
Trikafta contains three precision medications - ivacaftor, tezacaftor, and elexcaftor, known as CFTR modulators - which treat CF symptoms while also targeting the specific genetic defect that causes the condition.
Released in the United States in 2019, and later in Canada, Europe, Australia, and New Zealand, the drug is not available in developing countries, including South Africa. Vertex registered patents for Trikafta in South Africa, but hasn't applied for registration with the South African Health Products Regulatory Authority (SAHPRA) and is not producing or distributing the drug here, effectively blocking potential generic competitors.
In response to earlier questions from Spotlight, a spokesperson for Vertex indicated that the company does not intend to register Trikafta in South Africa, but would supply it via Section 21 authorisations - a legal mechanism for importing unregistered medicines into the country.
Responding to this, Nel explains, "All medicines, including generics in South Africa, must be registered with SAHPRA. If a medicine is not registered with SAHPRA and a patient needs to use it, said patient needs to apply for a Section 21 approval."
She says that this application is cumbersome and would have to be repeated every six months at a cost of R350 each time. "It is an admin-intensive process made by a treating medical practitioner and must include a laundry list of details. The authorisation for the Section 21 medicine only lasts for six months and so CF patients and their treating practitioners will need to reapply every six months. Also, medical aids are not obliged to compensate patients for Section 21 medicines. In fact, some medical aids explicitly exclude Section 21 drugs."
CF causes severe illness. People with it suffer frequent, serious lung infections. We are hospitalised regularly, we endure the indignity of invasive treatments, exhausting daily regimens, liver and pancreatic damage, lung failure, and some of us may ultimately require a lung transplant to avoid dying. - Cheri Nel
The cost of the medicines is also a major factor. In the United States, Vertex charges around R5 million per year for Trikafta.
'People didn't really know what CF was'
Speaking to Spotlight over Zoom from her office in Sandton, Nel's vitality is striking. Relaying her CF journey, which started in Upington when she was diagnosed at the age of six, she laughs and jokes, dropping an occasional expletive. Her parents suspected something was wrong when, as a pre-schooler, Nel ate more than her father - sometimes up to three plates of food - without putting on weight.
"I was diagnosed, but people didn't really know what CF was back then, so my family never treated it as a big deal," she says. "CF wasn't a part of my identity and I did not get any special treatment growing up. The first time I got really sick was in my twenties, at university [Stellenbosch University], when I had to be hospitalised." (Spotlight recently reported on plans for a new CF screening programme in South Africa.)
Today, Nel lives in Sandton with her husband Rob and their three dogs. The couple married in 2014, with Nel being honest about her condition from the get-go. Incidentally, her husband's late father was a doctor. "After first meeting me, my late father-in-law told my husband, no way, she can't be sick?"
Nel likes to socialise over a glass of wine and is a keen runner. Photographs show her life as fast-paced and playful; punctuated with bright smiles, poolside bikinis, and road trips. One picture shows her in a unicorn jumpsuit at a pyjama party with girlfriends.
High stakes
Despite her sense of humour, the stakes here are life and death. At 38 years old, Nel is very fortunate - many CF patients do not live into their thirties.
CF is caused by defective genes resulting in excessive salt secretion in cells. This leads to thickened mucus which cannot be removed by normal body functions, which in turn clogs narrow ducts and passage ways, severely damaging the respiratory and digestive systems, leading to premature death. Symptoms include cough, lung infection, and the inability to gain weight. It can also cause secondary conditions, such as diabetes. (Spotlight previously reported on how CF is currently treated in South Africa.)
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"I have run a marathon," says Nel. "I do think that I'm a slow runner, like insanely slow. And I am very curious to see that if I get to use this Trikafta, how much better would I feel? Would I run faster? Because I've never known any different [from] how I have felt all my life. And everyone who starts taking this drug says how much better they felt in general."
In 2020, the South Africa Cystic Fibrosis Association (SACFA) reported that, on average, people in South Africa with CF live to be 27.5 years old. SACFA notes 525 registered CF patients in South Africa, estimating the real number to be higher due to misdiagnosis.
"CF causes severe illness," says Nel. "People with it suffer frequent, serious lung infections. We are hospitalised regularly, we endure the indignity of invasive treatments, exhausting daily regimens, liver and pancreatic damage, lung failure, and some of us may ultimately require a lung transplant to avoid dying. Many of us die early, often in childhood, and well before our time. Life expectancy is short."
Despite this stark medical reality, and now also her involvement in a major court case, Nel's demeanour remains positive, at times verging on jubilant. She captions a photograph in which she toasts drinks with a friend - "Mimosas forever!"
*This article is part of a Spotlight special series on CF. In previous parts, we took an in-depth look at the landmark CF court case in South Africa, unpacked plans for a new CF screening programme, and asked what CF treatment is like in the absence of new breakthrough medicines.
