A mother of four, she is the first American patient. Having your genes edited with controversial CRISPR technology to treat your sickle cell disease says the technology is working.
Victoria Gray, 34, of Forest, Mississippi, has suffered from the debilitating blood disorder throughout her life.
She was in so much pain at one point that she couldn’t even lift a spoon, let alone care for her children.
Sickle cell disease affects up to 100,000 Americans, a disproportionate number of whom are black, such as Gray, and while treatments can alleviate symptoms, they do nothing to address the underlying disease.
But the genetically modified cells that doctors have inserted into his body appear to be reversing and alleviating the symptoms of the debilitating disease, NPR reported.
Tests show no signs that Gray’s DNA has changed, and she says she no longer experiences severe episodes of pain that would leave her hospitalized several times a year.
34-year-old Victoria Gray (left and right) of Forest, Mississippi, became the first American patient to use CRISPR to treat her sickle cell disease. The gene editing tool created a new fetal hemoglobin, the oxygen transporter protein, to cancel out the faulty hemoglobin that caused its red blood cells to bind in blood vessels.
Gray says that she no longer suffers from severe episodes of pain that would leave her hospitalized and that she has not needed blood transfusions or narcotics. Photo Shows: Gray with three of her children.
‘It is wonderful. It’s the change I’ve been waiting for my whole life, ” Gray told NPR.
“It is difficult to express in words the joy I feel: to be grateful for such a great change.” It has been amazing ‘
Sickle cell disease is an inherited disorder that affects the way in which red blood cells circulate oxygen throughout the body.
The disorder causes the red blood cells to have a crescent or sickle shape, rather than the normal round shape.
Sickle cells die prematurely, causing a shortage of red blood cells. When they pass through the blood vessels, they join and block the vessels, preventing blood from flowing properly.
The accumulation of sickle cells that occurs periodically in Gray’s blood vessels often leaves her in immense pain.
HOW DOES THE CRISPR DNA EDITION WORK?
The CRISPR gene editing technique is increasingly being used in health research because it can change the building blocks of the body.
At a basic level, CRISPR works as a DNA cutting and pasting operation.
Technically called CRISPR-Cas9, the process involves sending new DNA strands and enzymes to organisms to edit their genes.
In humans, genes act as blueprints for many processes and characteristics in the body: they dictate everything from eye and hair color to whether or not you have cancer.
The components of CRISPR-Cas9, the DNA sequence, and the enzymes necessary to implant it, are often sent to the body on the back of a harmless virus so that scientists can control where they go.
Cas9 enzymes can cut DNA strands, effectively shut down a gene, or remove sections of DNA to replace them with CRISPRs, which are new sections sent to change the gene and have an effect that they have been preprogrammed to produce.
But the process is controversial because it could be used to change babies in the womb, initially to treat disease, but it could lead to an increase in ‘designer babies’ as doctors offer ways to change the DNA of embryos.
Source: Broad institute
According to the Centers for Disease Control and Prevention, the disease is much more common among African-Americans, and an estimated one in 13 is born with the trait.
About 365 African-American and black babies are born with the disease itself, and many will die at age 40.
Treatment is primarily focused on relieving symptoms, such as pain and infections, through blood transfusions and pain relievers.
But this new experimental treatment, which Gray received on July 2, 2019, is different.
The scientists removed cells from his bone marrow and edited a gene to make new fetal hemoglobin, the oxygen-carrying protein.
This is usually only produced by fetuses in the womb to obtain oxygen from their mothers’ blood.
If it works, the fetal protein could be a lifelong solution for Gray and hundreds of thousands more sickle cell patients worldwide by compensating for the faulty hemoglobin his body produces.
Blood tests seen by NPR showed that about 46 percent of the hemoglobin in Gray’s system is fetal hemoglobin, and 99.7 percent of his red blood cells contain some of it.
The team says they would have considered the treatment successful if only 20 percent of the hemoglobin in their system was fetal hemoglobin.
For the past two years, Gray has told NPR that she would be hospitalized about seven times a year because of her pain, and that she would need regular blood transfusions or prescription narcotics.
Now, she no longer has severe episodes of pain, has not had to be hospitalized, and has not required any blood transfusion.
“High school graduations, college graduations, weddings, grandchildren, I thought I wouldn’t see any of that,” Gray told the radio station.
“Now I will be there to help my daughters choose their wedding dresses and we will be able to take family vacations and they will have their mother every step of the way.”
Gray’s condition means that many of his red blood cells are sticky sickles (center), rather than smooth, round, oxygen-rich discs.
In the fall of 2019, Gray’s husband, who is in the National Guard, was sent to Washington.
She says she doesn’t know how she would have been able to care for her three children alone had it not been for treatment.
“Since my treatment, I have been able to do everything for myself, everything for my children,” Gray told NPR.
“And it has been a joy not only for me, but also for the people around me in my life.”
Although animal and petri dish tests have been successful enough to get the go-ahead from the Food and Drug Administration for live human trials, it has never been done before.
It’s possible that CRISPR may make unwanted changes to DNA, for the worse, so scientists say they will need to follow Gray for years to file a lawsuit.