First of all, the long-standing pain that hits you every moment with school, relationships, work, etc.

And after painful treatments, life after sickle cell disease (SCD) felt like a miracle.

Two Americans whose lives have been changed for the better by a newly approved treatment told AFP they hope others will benefit as well.

But the eye-watering cost of up to $3.1 million per course could limit access for other patients.

“It’s like life has been breathed into me.”

Tesha Samuels was born in 1982. Just before prenatal screening for her SCD, an inherited red blood cell disease, was invented.

SCD affects approximately 100,000 people in the United States and approximately 20 million people worldwide.

Most people with this condition are black. Scientists say this is because the sickle cell trait evolved to protect people exposed to malaria, which puts them at higher risk of SCD.

People with this disease have abnormalities in the oxygen-carrying molecule hemoglobin, which causes their red blood cells to become stiff and sickle-shaped.

Complications include anemia, bouts of extreme pain, organ damage, and early death.

Tesha was diagnosed at the age of two and recalls a childhood spent in and out of the hospital.

She developed life-threatening anemia at age 7 and suffered a stroke at age 13, requiring monthly blood transfusions.

Tesha said she had to wait until things became unbearable because of “the stigma of black kids going to the hospital because they’re in pain.”

As a young adult, Tesha witnessed the disease take the life of a dear friend named Mohamed, a fellow “Sickle Cell Warrior” who was often admitted to the same hospital.

She began studying at the prestigious Howard University with the aim of becoming a doctor, but was forced to drop out due to health reasons. She then tried community college, but she was unable to graduate, again due to her SCD.

“You downgrade your dreams based on your sickle cell abilities,” Tesha said.

As a newlywed in her 20s, she was disappointed to find that she had to spend eight hours a night on IV medication to manage her condition.

But her life took a turn in 2018 when she became one of the first people to receive experimental gene therapy.

This approach (now sold as Rifgenia) uses a modified virus to deliver a functional version of the hemoglobin-producing gene. .

First, doctors remove stem cells from the bone marrow and then modify the stem cells in a laboratory. Next comes the hardest part: chemotherapy, which clears the way for the treated cells to return.

In addition to losing all her hair, Tesha suffered a 16-hour nosebleed from chemotherapy and was admitted to intensive care.

Her recovery was further complicated by the fact that it took several months for her platelets, which are essential for blood clotting, to recover.

But when they did, her energy levels skyrocketed.

“It feels like life has come to life,” Tesha said. “I have a new life ahead of me. What do I want to do with it?”

Tesha went back to school to get her degree.

She also started her own advocacy group, Journey to ExSCellence, to spread information about this treatment to the Black community.

John Tisdale, a physician-scientist at the National Institutes of Health who conducted the trial in which Tesha participated, said: “This looks like a treatment, and we would call it ‘transformative.’ .

Professor Tisdale stressed that each patient would need to be monitored for 15 years to complete the study.

childhood conflicts

Jimi Olagia’s first memory of sickle cell goes back to when he was eight years old, playing soccer with other kids in his native Nigeria and having to stop every five minutes to rest and rehydrate. .

“I asked my mom, ‘Why am I different?'” he remembers.

His parents sent him to live with an aunt in New Jersey, where better medical care was available, but his childhood remained difficult.

Jimi, 38, was unable to graduate from college and felt his illness was too much to burden most lovers, until he met a wife who was willing to accept the challenge. Ta.

This disease also took a toll.

His gallbladder had been removed and he had suffered a heart attack and blood clots in his lungs. At his worst, he recalls spending 80% of his time in bed.

Like many people with SCD, moving to the warmer climate of Atlanta brought some relief.

Then in 2019, I heard about CRISPR gene therapy clinical trials. He applied to take the test to qualify and received a “magical” voicemail letting him know he was participating.

Thanks to the CRISPR-engineered stem cell therapy he underwent (now sold as Kasgebi), Jimi is “basically living the dream”.

He has three children thanks to IVF and runs several small businesses.

Like Tesha, Jimi is speaking out for others, especially in Africa, where access to such treatment can seem like a distant dream.

NIH’s Tisdale said the next step is to make treatments less physically demanding and less expensive.

It remains unclear how much private insurance companies will pay to offset the huge costs of the procedure.

But Medicaid, the U.S. government-backed insurance program, announced it would start paying for the treatment starting next year.

© 2024 AFP