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New hope for sickle cell disease patients

Treatment options for people with this disease include blood or marrow transplants.

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The treatment of sickle cell disease is currently witnessing remarkable developments, especially with the progress made in research and the development of new treatments that will give millions of patients around the world real hope of recovery, according to the specialist in pediatric hematology and oncology at Cleveland Clinic Children's Hospital, Dr. Rabea Hanna.

Hanna added that "sickle-cell disease can severely affect patients' quality of life, limiting their capabilities due to severe episodes of pain, peripheral organ damage and reduced life expectancy," noting that medications can reduce the severity of the disease and treat the symptoms it causes.

The genetic change in the DNA of people with this disease causes a chemical change in hemoglobin - a red protein responsible for transporting oxygen in the blood - which causes the cells to turn into a sickle-shaped instead of their normal round shape, which hinders their easy passage through Blood vessels, and can also block or disintegrate vessels, which leads to a reduced lifespan of red blood cells and an increase in the rate of iron storage in the liver and heart.

This can lead to serious complications for the patient, such as cirrhosis, liver failure, stroke, cardiomyopathy and heart failure, in addition to severe pain.

Treatment options for people with this disease include blood or marrow transplants, but in reality it is difficult to find a matching donor and there are significant risks associated with this type of treatment.

Dr. Hanna explained these risks: “The risk of the patient's body not accepting the donor cells ranges between 5 and 10%, and there is a risk of graft-versus-host disease, which occurs when it attacks the bone marrow of the donor or the recipient stem cells.

To avoid such a situation, the patient is given strong immunosuppressive drugs, but there is still a high risk of developing graft-versus-host disease.”

He continued, "On the other hand, gene therapy depends on the patient's own stem cells, and thus, the risks of the patient's inability to accept the cells or the possibility of developing graft-versus-host disease are eliminated."

Another benefit of gene therapy is that patients will not need immunosuppressive drugs;

So their immune system will function normally, which is especially important during a pandemic.

In addition, while patients need chemotherapy to enhance the body's readiness, whether with conventional transplants or gene therapy procedures, chemotherapy used in gene therapy is less powerful.

Dr. Hanna continued, “Gene therapy is a pioneering technology that works by replacing or disrupting disease-causing genes.

In our current clinical trial, we introduce healthy genes into the body in order to correct genetic abnormalities of red blood cells.

By enabling cells to produce more fetal hemoglobin, this treatment has the potential to treat sickle cell disease in a precise way.”

According to the data of the World Health Organization, about 5% of the world's population carries the genes of the traits that cause hemoglobin disorders, especially sickle cell disease and thalassemia. However, it should be noted that this does not mean that they will develop the disease.

It is estimated that 300,000 children are born annually with severe hemoglobin disorders.

India and some countries in the Middle East and Africa are among the countries where sickle cell disease is common.

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Among the drugs approved by the US Food and Drug Administration (FDA) during 2019 and 2021, crizanlizumab, which works to reduce severe pain and complications, and voxelotor, which helps restore the normal functions of red blood cells and deliver oxygen to tissues, thus helping to Relieve pain.

Rabea Hanna:

"Gene therapy represents a pioneering technology that works by replacing or inactivating disease-causing genes."

5%

Of the world's population carry genes for traits that cause hemoglobin disorders, especially sickle cell disease and thalassemia, and this does not mean that they will develop the disease.

300

A thousand children are born annually with hemoglobin disorders.

India and some countries in the Middle East are among the countries where the disease is spreading.

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