SIBLINGS WITH Sickle cell disease (SCD) who travelled abroad for a ground breaking transplant say they have been ‘cured’.

Nabila Nakigozi Mpanga, 22, and Fauzan Ssebaggala Mpanga 26, from Newham, East London, raised money to fund the trip to India and privately underwent a Haploidentical Transplantation, which is also known as ‘Haplo Transplant’. 

The siblings stayed in India for four months and say the treatment was intensive and had serious risks of complications such as organ failure. 

However, since returning to the United Kingdom in September 2021, they claim they are now able to do every day activities that were impossible before. 

The treatment they received is now currently being researched in the UK, subject to a randomised trial by the National Institute  for Health and Care Research (NIHR). 

Speaking to The Voice, Mr Mpanga said: “There is Sickle cell in us but it is not a number that can produce symptoms.”

Since his recovery, he said he has a “renewed energy” and no longer suffers from extreme fatigue and describes how he feels now as  “a miracle”. 

He told The Voice, he is now under the care of a caner team at a London hospital, because part of his treatment abroad involved him having chemotherapy. 

As a result, he has monthly blood tests to check for viruses and monitor the levels of Sickle cell in his system. 

He said: “The last time they checked me I was like 98% so I had 2% Sickle cell

“They are sure we are at a level where they can theoretically say we have the Sickle cell trait now.” 

The Sickle cell trait (SCT) is not the same as the disease. 

Having the trait means you are a carrier of Sickle cell and have one of the genes that causes the diseases – but you do not have the condition yourself. 

People with SCT usually do not have any of the symptoms of Sickle cell disease (SCD) and live a normal life

Ms Mpanga’s experience was more worrying than her brother’s, as she became very unwell and developed Sepsis. 

When she arrived back in the UK, she also developed a BK virus, which is bleeding within the bladder and required emergency surgery. 

Following that, she spent six months in hospital and her weight plummeted from a UK dress size 10-12 to a size 4. 

“I’ve smelt death many times with Sickle cell but I definitely smelt death with this transplant,” she said. 

Ms Mpanga has recovered and says she now has energy, has regained weight and is living a “normal life.” 

Looking back at how her life was prior to the transplant she says she “can’t imagine how she was doing it” and believes her and her brother were simply living in a “survival mode” to get through each day. 

She added: “How we were living, no one should be living like that.” 

“The future is potentially brighter for young people who have sickle cell than it was 20 years ago”

“Even though we are much better it is a process that we gradually go through,” Mr Mpanga added. 

The ‘Haplo Transplant’ is very intensive and risks can cause complications like multi-organ failure or even death. 

The transplant allows patients to receive a Stem cell transplantation (SCT) from relatives who are only a 50% match, such as a sibling who only shares one parent. 

The procedure is currently not available on the NHS 

The siblings were born in Uganda, East Africa, and Ms Mpanga was diagnosed with Sickle cell at birth. 

Her brother was diagnosed at nine months. Of their four other siblings, two have not inherited the condition, whilst the other two carry the sickle cell trait.

Initially the original plan was to get their sister and brother – who have not inherited the disease – to donate but the final donations came from their mother and older brother.

Mr Mpanga said: “Because we had a half match, we still have a certain number of Sickle cell in us, remember both my mum and brother were both carriers they had the trait of sickle cell.”

As infants they were both very poorly.

During their childhood, they missed out on key events like celebrating birthdays because they spent so much time in hospital, 

Ms Mpanga said: “It was awful, as far back as I can remember, I have always been sick, I have never not had pain.

“In Uganda, I remember having a lot of stomach issues and a lot of vomiting.”

“I would cry a lot to the point the neighbours would know ‘she’s been up the whole night’.”

By the early 2000s, Mr Mpanga says the healthcare system in his home country didn’t have the right facilities to treat them and because his family came from a very humble background, it made getting sufficient healthcare very challenging. 

Pain relief

Mr Mpanga told The Voice, when he was younger he thought his parents didn’t care about his condition, but as he grew older he realised  they had done everything they could with the limited resources they had at the time. 

He said: “My parents were very cautious about what we could do and where we could go, who we could hang around with as a way to safeguard us.” 

He said the debilitating condition robbed him of a childhood as it meant he “never played” and “never did PE at school” – until he moved to the UK. 

They moved and settled in Newham, East London, when they were five and nine years old. 

The transition from life in Kampala to Newham, for Ms Mpanga was “very difficult” but enjoyed sport which helped her. 

“In Primary school I tried to fit in and did every sport, like netball and basketball,” she said. 

However, at secondary school, she would attempt to play a game of netball but would end up in the hospital.

She said: “My mum would tell me ‘you shouldn’t be doing too much’.

“It got to a point where I could no longer partake in these sports.” 

“Everything that I enjoyed, I couldn’t do,” she said sadly.

Every exam season, it would bring on a more severe Sickle cell crisis, for Ms Mpanga. 

In 2016, she took her GCSE’s in hospital while she was attached to pain relief pump. 

She said: “Every five minutes you press it and it brings a bit of Morphine into your body and I would take have to take naps.” 

Ms Mpanga did pass her exams and got all C and B grades.

Arriving in London in December, just at the start of secondary school was “hard” for Mr Mpanga too and he admits he endured some bullying. 

“I started getting sick straight away,” he recalled. 

He was prescribed medications such as hydroxyurea and he said “it done wonders” for him and within a space of six months he didn’t have a Sickle cell crisis. 

Colder

Mr Mpanga initially wanted to study medicine at university, so he would be able to help those with the disease, but unfortunately he had a severe Sickle cell crisis which damaged his jaw – which destroyed his plans. 

During his college years and time at university, he endured severe pain, struggled with fatigue which meant he was absent from his classes. 

He told The Voice, he felt like the disease came back with “vengeance to pay me back for all the time I didn’t have it.” 

Despite his medical condition he did gain a degree in accounting and finance at university. 

As the winter months in London came and went, the family noticed during the colder months they would always become more unwell. 

In October 2020, during the height of the COVID-19 pandemic the siblings travelled back to Uganda for a much-needed break, and in the hope their symptoms would ease up.

Ms Mpanga said: “Normally when we go back to Uganda we would feel a bit better, maybe it’s because of the sun or because we are less stressed, we can’t really know.

“But we would always feel a little less strain on our bodies and we would always take a little less medication.” 

At the time of the trip to Uganda, Mr Mpanga had lost his hip to necrosis and needed to walk on crutches. 

His sister also has necrosis in the spine and was beginning to also develop it in her hips.

While in Uganda, their mother told them to find Professor Christopher Ndugwa – a renowned Sickle cell specialist – who had been the family’s childhood doctor and the only person their parents trusted to treat Sickle cell in Uganda. 

Ms Mpanga said after consulting Prof Ndugwa he explained that with Haplo transplants you only need to have a fifty per cent match – which would mean a parent automatically qualifies as a match. 

“We had no idea it existed,” Ms Mpanga said. 

She told The Voice, her Ugandan doctor gave her and her brother only two years to live if they didn’t get the Haplo transplant. 

Supportive

Mr Mpanga said they were initially “very excited” after hearing about the treatment but says the enthusiasm was “short-lived” when they asked about the transplant in the UK. 

He said: “Well the doctors didn’t give us the time of day and they didn’t respect Professor Ndugwa’s opinion, they were under the assumption that they knew better. 

“He was willing to speak to them, he wrote down his credentials, he studied in Brighton and worked in Great Ormond Street Hospital for 14 years but they did not give him the time of day.” 

They said he asked the NHS about the treatment and was told it was not an option. 

Mr Mpanga disclosed to The Voice, he believes the opinion of his former doctor was dismissed because he was black and from Uganda. 

The siblings thought long and hard about the idea and weighed up all the pros and cons and made the difficult decision to go-ahead with the transplant. 

They said they felt they “had nothing to lose” and decided to launch a GoFundMe campaign  aiming to raise £60,000 to pay for their treatment. 

Mr Mpanga said: “We spoke to our parents they were resistant given what the doctors had said but because me and Nabila were united in our decision it wasn’t easy.

“We told them this is our decision, we didn’t want anyone making the decision for us especially as no one goes through the pain that we go through every day.” 

“Our parents agreed because they trusted the doctor in Uganda.” 

Ms Mpanga led the fundraising campaign and raised around £40,000 for the cost of the trip and treatment and they also used family savings. 

In total they estimate the total cost to be over £100,000. 

However, they insist they would do it again, as they now feel well and healthy and they can’t put a price on that. 

Arriving in India with British passports, Mr Mpanga told The Voice, the medics were stunned.

“They were like, how could you be coming from the UK to here?” he said. 

In September 2021, they returned to the UK and only informed their British doctors once the medical procedure was completed. 

The siblings are now being cared for and monitored by a cancer care team in London and say “they have been very supportive.” 

Awareness

Even though they are both now feeling much better and able to do everyday activities like going out with friends and going on long walks, they can’t help but think of other patients who are still suffering like they once did.  

“The thing with Sickle cell is it can never just be just about you because as much as I’m not in pain, having friends that are still going through it, given that you know that pain, you can never settle, it is always at the back of your mind,” Mr Mpanga explained. 

Mr Mpanga had a hip replacement last year and describes where he is now “as a huge shift”. 

The siblings are the co-founders of a Community Interest Company (CIC) called ‘Weren’t Born Rich’, which was founded in 2014 and aims to raise awareness about the obstacles and challenges people with Sickle cell face. 

“With Weren’t Born Rich we just want to shine a light using our own personal experiences and the community around us to support one another and actually give a voice to those with Sickle cell in schools, university, in workplaces but mainly where the policies are made,” Ms Mpanga added. 

The siblings put on yearly awareness events where people can “learn about Sickle cell” and encourage those within the black community to get tested for the disease before they consider starting families.

They are now planning to bring their awareness events across the UK and want to collaborate with therapists to provide mental health support to sickle cell patients and their families. 

Mr Mpanga said: “The aim is to address the black community, the communities that are at risk, and say its genetic condition, it’s not your fault.”

He revealed to The Voice, that having Sickle cell is still considered taboo within many African communities in the UK, with many choosing not to inform family, friends and the wider community of their condition. 

“It’s like a taboo topic they don’t want to raise it,” he said. 

The siblings also want to take their work to the NHS and key decision-makers to advocate for having mental health support for those living with Sickle cell, as they feel it is something that should be provided but currently is not. 

Mr Mpanga claims the first time he received mental health support was when he returned from India. 

He said: “The support is non-existent, the first time we had mental health support is when we came back. 

“I was given a therapist under the cancer team. 

“The sickle cell team told me they can’t fund it.”

Mr Mpanga is now calling for more funding for mental health support for Sickle cell patents as part of their care and says “my mental health has been the hardest part to heal” on this journey. 

He said he still has friends with the disease and regularly receives phone calls in the middle of the night because they are in pain and have no one else to call. 

NHS Criticisms

The siblings say they are receiving great care under the cancer team, but say the care they received prior to going to India  under the Sickle cell team was not good enough.

They believe a lack of funding, awareness and compassion is leaving patients dissatisfied with the care. 

 “There is a lot of institutionalised racism in the NHS and this is not a topic that is widely discussed but it is there, when Covid hit the first ten doctors to die were of colour, Mr Mpanga said. 

He said: “The NHS is beneficial to people if it fulfils the role which it was created for to give free healthcare to people.”

He described parts of the NHS as “overly burdened and under resourced” and this is pushing people to look into health insurance and private health care. 

He added: “I’ve lost two hips at 25. Sickle cell is underrated.

“The fact that people look normal I feel like that is the worst downfall for people with Sickle cell. 

“You look perfectly normal, a lot of people do not look like they have any problems.

“They get stigmatised and discriminated against.”

The siblings say they have witnessed nurses say that Sickle cell patients are just “here for the drugs” or that they should “just give them the Morphine and tell them to go home.”

Ms Mpanga says she was told by a nurse at a London hospital: “You don’t even look like you are in pain.” 

She said she asked: “What do you want me to look like? I’ve had sickle cell for 21 years of my life. Do you expect me to cry and fall down to my knees every time I am in pain?

“We make coping mechanisms so that my mum is not stressed and I’m not stressed. 

“I try to calm myself and compose myself, that doesn’t take anything away from the pain I am going through.” 

“If they were to say that to someone with cancer or HIV they would be dealt with,” Mr Mpanga added. 

Both siblings claim they have lost jobs because they took too much time off work because they were sick and said this is very common with those who suffer with Sickle cell because there is simply not enough awareness about the severity and impact of the disease. 

Dr Carl Reynolds is clinical advisor to the NHS Race Health Observatory leading on Sickle cell inequality. 

Trial

He said: “There is currently a large randomised trial of Haplo treatment in the UK. I am not an expert but understand that the treatment looks like a promising means of making stem cell transplant available to more sickle cell patients. All forms of stem cell transplant carry serious risk, including the risk of death, but it is becoming safer over time and if successful can be a curative treatment. Overall, I see this, and emerging gene therapies for sickle cell as very promising developments.”

He told The Voice, he believes not enough research is being done into Sickle Cell.

He said: “There is a widespread perception internationally that Sickle Cell has been relatively underfunded in comparison to other rare inherited genetic disorders such as cystic fibrosis. The NHS Race and Health Observatory have commissioned research to investigate this in the UK with a view to generating evidence for policy recommendations to address and inequalities identified.”

“Common sickle cell treatments include medicines such as folic acid, penicillin and analgesics. In selected patients, hydroxyurea, blood transfusion, crizanlizumab, and stem cell transplant are also used. Gene therapy remains a promising and rapidly developing area of research but treatments are not yet currently available in the UK outside of a research setting.” 

Dr Reynolds said he would always recommend that patients and their families speak to their Sickle cell specialists for advice. 

Speaking to The Voice, John James OBE, Chief Executive, Sickle Cell Society, said: “In England, until our intervention the NHS were only funding children for Bone Marrow and Stem Cell Transplants because they were arguing they can’t fund adults because the evidence isn’t there. 

“What we have done is got NHS England to fund adult transplants in  England.

“So today, if the commission deem it necessary an adult whose over 18 can get a Bone Marrow or Stem Cell transplant.
“However, the issue is when they made that agreement they limited it to a sibling donor. 

“You would only get funded if the donor was a sibling.

“It is very limited in terms of sibling because there are individuals who don’t have a sibling.” 

Mr James said that is progress, but said there are lots of adults in the UK who are excluded from this current policy. 

He said: “The good news for people living with Sickle cell is there will be a clinical trial for Haploidentical Transplants in the UK.”

Mr James said the purpose of the trial is to collect evidence to see if it is safe, effective and if it would be affordable for the NHS. 

He said: “Sickle cell is the fastest growing genetic blood condition, it is also the most under invested.” 

He added: “The future is potentially brighter for young people who have Sickle cell than it was 20 years ago because of the potential for gene therapy, the potential for Stem cell transplantation for children and adults.” 

He called for better the black community to give blood and continue to speak out about living with Sickle cell to raise awareness of the disease. 

He said the Sickle Cell Society has some support groups on its website which can help patients and families. 

Sickle cell causes red blood cells to form into sickle or crescent shapes and become stuck in blood vessels, causing agonising crisis episodes, and serious or even fatal long term complications including organ damage and strokes. Many patients need regular blood transfusions to stay alive. 

Last year, Sickle cell became the fastest growing genetic condition in the UK. 

As a result, the NHS is issued an urgent call for more blood donors of black heritage to help treat the disease.

According to health officials only 150 donations a day were needed five years ago. 

Demand for blood to treat sickle cell has risen by 52% over the past five years and is projected to continue to rise.