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Stem Cells are the Only Hope For a 17 Month Old Toddler Fighting a Rare Disease Named After Her!

Many of you might have heard about rare diseases whose rarity is strengthened by the low number of people affected by them. One such incident took place recently in the USA, where a disease was named “Winslet’s disease” after the girl who was born with this rare condition. In fact, Winslet Seoighe is the only human being in the world with this disease.

Winslet was born with an enlarged heart, an enlarged spleen, a liver that was three times its normal size and several organs that were gradually dying. Apart from all these abnormalities, she had only 33% blood in her body. Very soon her doctors found out that Winslet’s body was not producing blood at all.

Although her bone marrow generated red blood cells, these were not getting transported into the blood. Dr. Stan Goldman, Winslet’s doctor, thinks that a mutation in red blood cell membrane proteins might be the cause of this condition.

She was given blood transfusions once in a month.

As an alternative to blood transfusions, which if performed continuously can lead to organ failure from metal toxicity due to the accumulation of iron, doctors have suggested stem cell transplantation in Winslet’s case.

It was a trial and error scenario as the doctors could not predict the outcomes of this transplant.

Winslet’s bone marrow was destroyed using chemotherapy and replaced with a donor’s bone marrow which was obtained within two weeks after the search began for a perfect match. The donor stem cells were put in a bag and transferred to her body through veins using intravenous therapy. Since the cells had markers, they migrated to her bone marrow for engraftment (incorporation of grafted tissue into recipient’s body).

Her hemoglobin levels reached normal levels after this bone marrow transplant, for the first time after her birth indicating increasing levels of blood.

The promising results seen so far have proved the potential of stem cells to some extent in treating diseases.

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