Two Americans who suffered from sickle cell anemia told the story of their struggle and relief from many years of pain, after receiving innovative gene therapies that were recently licensed by health authorities in the United States.
The two former patients, Tisha Samuels and Jamie Olager, hope to raise awareness of these gene therapies, so that others can use them, especially since their cost may reach $3.1 million per person.
Patients with sickle cell disease have a mutation that affects hemoglobin, an oxygen-carrying protein in red blood cells, which then takes on a sickle shape, limiting blood flow and oxygen delivery.
Sickle cell anemia is a genetic disease that affects about 100,000 people in the United States and 20 million people worldwide. It mainly affects people with dark skin. Scientists attribute one of the reasons for this to the fact that the sickle cell trait protects against malaria.
Treatment “Livgenia”
Tisha Samuels was born in 1982, just before newborn screenings for sickle cell anemia began, but she was diagnosed at the age of two. At the age of seven, she suffered a serious bout of anemia, and at 13, a stroke forced her to undergo monthly blood transfusions.
As Samuels became a young adult, she lost a friend who battled sickle cell disease, and who often ended up in the same hospital where she was being treated.
The young woman continued her studies at the prestigious Howard University, hoping to become a doctor, but she was forced to drop out due to her illness, saying that the illness “led her to give up her dreams.”
After getting married at a young age, Tisha needed 8 hours of injections every night, but in 2018, her life took a new turn, as she became one of the first to receive an experimental treatment at the time.
This treatment, currently marketed as Lyfgenia, 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.
Tisha lost her hair and was hospitalized with a nosebleed that lasted 16 hours, then regained her energy, saying it was “like coming back to life.”
It takes months for platelets to return to normal, and each patient participating in the clinical trial undergoes 15 years of follow-up.
CRISPR scissors
The young man, Jimmy Olagir (38 years old), has a similar background, as his first memories of his illness go back to when he was eight years old, when he was playing football in Nigeria, his native country, but he was forced to stop every 5 minutes to rest and drink.
After a while, his parents sent him to live with his aunt in New Jersey in the United States, where health care was better, but he lived a difficult childhood, as he failed to complete his studies, had to have his gallbladder removed, and suffered a heart attack.
At his worst, he spent 80% of his time in bed, but moving to Atlanta, where it is warmer, helped him, as is the case for many sickle cell patients.
In 2019, when he heard about a clinical trial using CRISPR molecular scissors, he registered himself and received a “magical” message informing him of his acceptance of this trial.
Thanks to the modified stem cells he receives with this treatment called Casgevy, Jamie is now “living the dream.” He became a father of three children, thanks to in vitro fertilization, and runs several small companies, as he seeks to popularize this treatment, especially in Africa, where access to it is still a “distant dream.”
John Tisdale of the US Institutes of Health (NIH), who led the trial in which Tisha Samuels participated, said that the next step is to make the treatment less burdensome and expensive for patients, noting that there is uncertainty about the coverage of these gene therapies by private health insurance, but the program ” Medicaid has announced that it will cover these costs starting next year.
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