When the mapping of the human genome was declared complete nearly 20 years ago, pundits hailed it as the dawn of a brave new era of genetic medicine.
Well, it took a while, but those breathless claims seem to finally be coming to fruition.
The most well-known – and controversial – manifestation of genetic medicines are the COVID-19 vaccines currently in the process of being rolled out for a mass vaccination program. But DNA medicine is also resulting in new cures – not just treatments, but cures – for other debilitating conditions.
[Victoria] Gray is the first person in the United States to be successfully treated for a genetic disorder with the help of CRISPR, a revolutionary gene-editing technique that makes it much easier to make very precise changes in DNA.
Gray is being treated for sickle cell anemia, a blood disease afflicting people of African descent. Another ethnically linked disease, beta thalassemia is being treated as well. (Isn’t it strange how diseases haven’t got the woke-science message that “race doesn’t exist”?)
At a recent meeting of the American Society for Hematology, researchers reported the latest results from the first 10 patients treated via the technique in a research study, including Gray, two other sickle cell patients and seven patients with a related blood disorder, beta thalassemia. The patients now have been followed for between three and 18 months.
All the patients appear to have responded well. The only side effects have been from the intense chemotherapy they’ve had to undergo before getting the billions of edited cells infused into their bodies.
The New England Journal of Medicine published online this month the first peer-reviewed research paper from the study, focusing on Gray and the first beta thalassemia patient who was treated.
“I’m very excited to see these results,” says Jennifer Doudna of the University of California, Berkeley, who shared the Nobel Prize this year for her role in the development of CRISPR. “Patients appear to be cured of their disease, which is simply remarkable.”
Another nine patients have also been treated, according to CRISPR Therapeutics in Cambridge, Mass., and Vertex Pharmaceuticals in Boston, two companies sponsoring the research. Those individuals haven’t been followed long enough to report any results, officials say.
But the results from the first 10 patients “represent an important scientific and medical milestone,” says Dr. David Altshuler, Vertex’s chief scientific officer.
DNA medicine, in these cases, works by subtly editing the patient’s genome to eradicate the genetic code that causes hereditary diseases to manifest. (The COVID vaccine, it should be noted, does not alter the patient’s DNA. Instead it uses mRNA – the cells’ “messenger” – to tell the patient’s cells to start a process that generates an immune response to the SARS-CoV-2 virus.)
The treatment boosted levels of a protein in the study subjects’ blood known as fetal hemoglobin. The scientists believe that protein is compensating for defective adult hemoglobin that their bodies produce because of a genetic defect they were born with. Hemoglobin is necessary for red blood cells to carry oxygen.
Analyses of samples of bone marrow cells from Gray six months after getting the treatment, then again six months later, showed the gene-edited cells had persisted the full year — a promising indication that the approach has permanently altered her DNA and could last a lifetime.
“This gives us great confidence that this can be a one-time therapy that can be a cure for life,” says Samarth Kulkarni, the CEO of CRISPR Therapeutics[…]
Gray and the two other sickle cell patients haven’t had any complications from their disease since getting the treatment, including any pain attacks or hospitalizations. Gray has also been able to wean off the powerful pain medications she’d needed most of her life.
Prior to the treatment, Gray experienced an average of seven such episodes every year. Similarly, the beta thalassemia patients haven’t needed the regular blood transfusions that had been required to keep them alive.
NPR
The advantage of treatments such as this is that they edit the patient’s own DNA by extracting stem cells from their own bone marrow and then editing it with CRISPR techniques. The most gruelling aspect for the patient is that they must then undergo chemotherapy to destroy most of their original bone marrow, which is then replaced with the gene-edited stem cells.
Experimentally treated patients like Victoria Gray will be monitored for any long-term side-effects and to make sure that the treatment is indeed permanent. Researchers are confident on both counts.
If the treatment for these genetic blood diseases is as successful as hoped, researchers will turn their attention to diseases from cancer and inherited blindness to heart disease and AIDS.
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