“Gene therapy” for “sickle cell anemia” is a journey of struggle

Two Americans who suffered from sickle cell anemia narrated to Agence France-Presse their journey of struggle against this disease, after benefiting from innovative treatments, albeit very difficult and expensive, that ended long years of pain.

The two former patients hope to raise awareness of these gene therapies, which have recently been licensed by US health authorities, so that others can use them.

But its high cost — up to $3.1 million per patient — can limit accessibility.

Tisha Samuels was born in 1982, just before newborn screenings began for sickle cell anemia, a genetic disease that affects about 100,000 people in the United States and 20 million people worldwide.

It mainly affects black people. Scientists say one reason for this is that the sickle cell trait protects against malaria.

Patients with sickle cell disease have a mutation that affects hemoglobin — an oxygen-carrying protein in red blood cells. The red blood cells then take on a sickle shape, restricting blood flow and oxygen delivery.

The potential consequences are tragic, and include anemia, pain attacks, organ damage, and even premature death.

Tisha Samuels was diagnosed at the age of two. At seven years old, she suffered a serious bout of anemia, and at thirteen, a stroke forced her to undergo monthly blood transfusions.

Tisha continued her studies at the prestigious Howard University, hoping to become a doctor, but she was forced to drop out due to her illness.

She says that the illness forced her to “give up on dreams.”

In 2018, her life took a new turn: she became one of the first to receive an experimental treatment at the time.

This treatment, currently marketed as “Levgenia,” uses a harmless virus to carry a healthy copy of the gene that produces hemoglobin into cells.

Doctors first collect the patient’s stem cells. Then comes the difficult part, which is removing the stem cells from the patient’s spinal cord through chemotherapy, making room for the modified cells.

Jimmy Olaguer, 38, has a similar story. His first memories of his illness date back to when he was eight years old.

At the time, he was playing football in Nigeria, his native country, but he had to stop every five minutes to rest and drink.

“I asked my mother, ‘Why am I different?’” he recalls.

Thanks to the modified stem cells he receives with this treatment, called “CasGevi,” Jimmy Olguerre is now “living a dream.”

He became a father of three children, thanks to in vitro fertilization, and runs several small businesses.

Olaguerre also seeks to popularize this treatment, especially in Africa, where access to it is still a distant dream.

For John Tisdale of the US Institutes of Health (NIH), who led the trial involving Tisha Samuels, the next step is to make treatment less burdensome for patients – and less expensive.

There is still uncertainty about whether these gene therapies will be covered by private health insurance.

But the public Medicaid program announced that it will cover these costs starting next year.

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