SCD affects approximately 100,000 people in the United States and 20 million people worldwide.
Washington:
Their stories are divided into before and after.
First, those long years were filled with pain every moment—school, relationships, work.
Then, after painful treatments, it felt like the miracle of life after sickle cell disease (SCD).
Two Americans whose lives have been improved by a newly approved treatment told AFP they hope others will benefit too.
But the eye-watering cost — up to $3.1 million per course of treatment — may limit access to other patients.
-“It’s like coming alive”-
Tesha Samuels was born in 1982, just before prenatal screening for SCD, an inherited red blood cell disorder, was invented.
SCD affects approximately 100,000 people in the United States and 20 million people worldwide.
Most people with the disease are black. Scientists say this is because sickle cell trait evolved to protect people exposed to malaria and therefore have a higher risk of developing SCD.
People with this disease have abnormalities in their hemoglobin (the molecule that carries oxygen), making their red blood cells stiff and C-shaped, like a sickle.
Complications include anemia, severe pain, organ damage, and premature death.
Tessa, who was diagnosed with the disease when she was two years old, recalls her childhood experiences in and out of the hospital.
She developed life-threatening anemia at age 7 and suffered a stroke at 13, resulting in monthly blood transfusions.
Tessa said it was “a shame for black kids to go to the hospital and say they were in pain,” which led her to wait until things became unbearable.
As a young adult, Tessa watched the disease claim the life of a close friend named Muhammad, a “sickle cell warrior” who was often admitted to the same hospital as her.
She began studying at the prestigious Howard University in hopes of becoming a doctor, but her health forced her to drop out. She then tried community college, but SCD again meant she couldn’t complete her degree.
“You scale down your dreams based on your sickle cell abilities,” Tessa said.
As a newlywed in her twenties, she was frustrated by the eight hours of intravenous drips she needed every night to control her condition.
But in 2018, her life took a turn when she became one of the first people to receive an experimental gene therapy.
The program, now marketed as Lyfgenia, uses a modified virus to deliver a functional version of the hemoglobin-producing gene. .
First, doctors extract stem cells from bone marrow and then modify them in the laboratory. Next comes the hardest part – chemotherapy, clearing the way for the treated cells to return.
As well as losing all her hair, the chemotherapy treatment caused Tesha to have a nosebleed for 16 hours, causing her to need intensive care.
Her recovery was further complicated because it took several months for her platelets (vital for blood clotting) to recover.
But when they did, her energy levels soared.
“It’s almost like being alive,” Tessa said. “A new life is before you. What do I want to do with it?”
Tessa went back to school to finish her degree.
She also started her own advocacy group, Journeys with ExSCellence, to spread the word about the treatment in the black community.
“This looks like a cure, but we like to call it ‘transformative,'” said John Tisdale, a physician-scientist at the National Institutes of Health who led Tessa’s involvement test.
Tisdale emphasized that each patient will need to be monitored for 15 years to complete the study.
– Childhood Struggle –
Jimi Olaghere’s first memory of sickle cell disease dates back to when he was eight years old, playing soccer with other kids in his native Nigeria and needing to stop every five minutes to rest and drink water.
“I asked my mother, why am I different?” he remembers.
His parents sent him to live with his aunt in New Jersey, where there was better health care, but his childhood was still difficult.
Unable to finish college, Jimmy, 38, found his illness too burdensome for most romantic partners until he found a wife willing to take on the challenge.
The disease also takes a terrible toll.
His gallbladder was removed, he suffered a heart attack and developed blood clots in his lungs. At his worst, he recalled, he spent 80 percent of his time in bed.
Moving to the warmer climate of Atlanta brought some relief, as it does for many people with SCD.
Then, in 2019, he heard about clinical trials of CRISPR gene therapy. He applied to take the qualifying test and received a “magical” voicemail telling him he had taken it.
Thanks to the CRISPR-modified stem cell therapy he received (now marketed as Casgevy), Jimi is “basically living the dream now.”
Through IVF, he had three children and ran several small businesses.
Like Tessa, Jimmy speaks out in advocating for others, especially in Africa, where access to such treatment seems like a distant dream.
The next step is to make treatment less physically taxing and less expensive, said NIH’s Tisdale.
It’s unclear how much private insurance companies will pay to offset the huge cost of the surgery.
But the U.S. government-backed Medicaid program says it will start paying for treatment next year.
(This story has not been edited by NDTV staff and is auto-generated from a syndicated feed.)
