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Sickle cell’s new therapy

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There is excitement in the medical community in the United States over a new treatment for sickle cell. When it becomes available to all who want it, Nigeria’s chances of benefiting are clogged by challenges, writes CHINAKA OKORO

Until the harmattan eased some days back, several medical doctors had one major worry: the effect of the severe weather on sickle cell patients. A Senior Registrar, Pulmonology Unit, Jos University Teaching Hospital (JUTH), Dr Chung-Gyang Shedrack, advised sickle cell patients to keep themselves warm during the harmattan cold.

Dr Shedrack said patients, who suffered from sickle cell and were anaemic, were likely to experience worsening of their condition during the unfriendly weather.

He said: “Sickle cell patients are prone to a crisis in this period of cold; they will have to stay very warm, take a lot of fluid, go for medical checks with their doctor and take medications adequately so that they do not have to worsen of the crisis.”

Dr. Shedrack’s advice should become useless if only Nigeria can benefit from a gene therapy that scientists hope will cure the disease.

A New York Times’ report at the weekend shows that the therapy has successfully worked on Helen Obando, a 16-year-old American girl. It is believed that soon the therapy will be available to more Americans.

For Nigerians, the wait will be longer. The best the country has now is stem cell transplant, whose first recipient in Nigeria is Matthew Ndik. He benefitted from the treatment in 2011 when he was seven years old. The Obando family also considered the bone marrow transplant before they heard about a “complex procedure to flip a genetic switch so the body produces healthy blood”. Helen’s elder sister, Haylee, got a bone marrow transplant. The donor was a sibling who luckily was not anaemic.

A 2011 report by Ms Joke Kujenya, then of The Nation, indicated that the cost of bone marrow transplant is killing. Ndik’s parents could only afford it because they did not have to pay for it. The transplant was done at the University of Benin Teaching Hospital (UBTH), Benin, Edo State.

Haematologist Dr. Nosakhare Bazuaye, who operated on Ndik, said the journey to achieving the feat began in 2009.

“Now, let me go to how the story started in 2009,” Dr. Bazuaye said. “We went for the training at the Basel University in Switzerland about two years ago. On returning to Nigeria, we met the Chief Medical Director (CMD), Professor Michael Ibadin, and then, the process of setting up, a temporary Stem Cell Transplant (SCT) unit, a four-bedded isolation room, began in earnest. The essence is to prove to Nigerians that we can do Stem Cell Transplant in our country.

“Let me quickly tell us the parameters with which young Matthew Ndik, 7, became the first SCT recipient. He was a seven years old child who has a previous history of Cerebrovascular Accident (CVA) that is, stroke, of which he already had three severe episodes. The first episode left him with a one-sided paralysis. And the second episode took his speech. And he was able to survive because if he had had the third stroke, he would have died. By then, his Stable Porcine Circovirus (PCV), at which he operates, was 20per cent. And we had to knock off his bone marrow. But we don’t have to crush his bone like some medical colleagues have asked me.

“But if there had been no donor for Matthew, forget it, none of us would be gathered here today. So, please permit me to ask us all to recognise someone very unique to the success of this experiment. That is the donor, the elder brother Matthew, Emmanuel Ndik. I don’t think that without his cooperation and willingness to donate his bone marrow, we can be here celebrating the 100days success at all.

“I do this because the donor for a transplant recipient is very important in this whole process. I must tell you that the day we were to take him to the theatre, I asked him how scared he was for the operation. He said that this was something he had to do as a last resort to save his brother’s life. And in about five minutes we were done. And so, for those who are scared, by the time were done Emmanuel, he went back to school the third day. His own is to show us that donors are safe and the complications are less. So they have nothing to worry about.”

Significantly, not many Nigerian patients have taken advantage of this treatment, whose cost is unfriendly.

The gene therapy

With Nigerians still unable to fully take advantage of the stem cell transplant, a better treatment is taking shape in the United States.

Dr. Francis S. Collins, the director of the National Institutes of Health described the breakthrough as “an exhilarating success story for those of us who have waited and hoped for this day”.

The New York Times’ report said: “For more than a half-century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into a rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the disease’s horrifying hallmark: episodes of agony that begin in babyhood.

“Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts say the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African-American, often poor or of modest means.

“The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.”

Behind the new therapy is a scientist at Boston Children’s Hospital, Dr. Stuart Orkin, 73, who has laboured against the disease since he was a medical resident in the 1970s. A federal grant has made the trial possible.

If the therapy shuts down the disease in her and another teenager in the same clinical trial at Boston Children’s, The New York Times said, scientists will soon begin testing it in even younger children. Two other gene therapy trials for the sickle cell are also underway in the United States. One aims to flip the genetic switch; the other is to add a new gene. Such cures are expected to cost $1 million or more, raising questions about affordability.

Verdict

As the world awaits the final verdict from the Food and Drug Administration, there is excitement in the medical community but as usual, it will take ages for it to make sense to Nigerian patients, who are first disadvantaged by the huge cost.

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