Biotech company CRISPR Therapeutics is set to treat Europe's patients with the CRISPR gene-editing tool this year following regulatory approval for trials.
Researchers will apply the much-hyped tool to patients with beta thalassaemia, an inherited blood disorder that affects the body's production of hemoglobin—a protein red blood cells use to carry oxygen around the body—U.K.'s Telegraph newspaper reported.
The condition is treatable, but can cause bone deformities, fatigue and severe anaemia. Patients with severe anemia from thalassaemia may undergo blood transfusions every two weeks, the CDC reports. Beta thalassemia is relatively common worldwide, according to the U.S. National Library of Medicine, with thousands of children born with the condition each year.
CRISPR (clustered regularly interspaced short palindromic repeats) is a tool which researchers can use to modify DNA sequences and alter the function of genes. Also known as CRISPR-Cas9, it was inspired by genetic defense mechanisms found in bacteria.
Bacteria carry a kind of memory bank of deadly viruses in strands of genetic code. If they spy one of these deadly viruses nearby, they send out an enzyme to cut away the dangerous bit of code. Scientists Jennifer Doudna and Emmanuelle Charpentier recognised the potential for this mechanism to edit human genes—targeting and slicing rogue DNA. An RNA molecule leads an enzyme called Cas9 to the DNA strands in need of cutting.
Read more: Gene editing could stop cancer, diabetes—and bioterrorism
In the new beta thalassaemia research, scientists will harvest certain stem cells from patients and engineer them in the lab. The team aim to increase levels of fetal hemoglobin—a form of the oxygen-transport protein found in babies but stifled in adults. After the lab work is complete, the stem cells will be returned to the patient's body via transfusion—a process known as a stem cell transplant. It is hoped the cells will then begin producing a healthy supply of hemoglobin.
With this research, beta thalassaemia will become the first human disease treated with CRISPR gene-editing technology in Europe. A specific location for the first trial has not yet been announced.
China already has a number of CRISPR trials ongoing to treat various conditions. "As of the end of February 2018 there were nine registered clinical studies testing CRISPR-edited cells to treat various cancers and HIV infection in China," Goldman Sachs analyst Salveen Richter told Endpoints News. In the U.S., she said, there was only one.
A lack of regulation, Endpoints News reported, was driving China's use of the technology.
The University of Pennsylvania "has opened a clinical trial for the treatment of cancer in patients with melanoma, sarcoma, and multiple myeloma," a spokesperson told the Telegraph. "Findings from the trial will be shared at an appropriate time via medical meeting presentation or peer-reviewed publication."
Read more: CRISPR Gene-editing—scientists edit human embryos to safely remove disease
Last year, Brian Madeux of California became the first person in the world to undergo gene editing in the body. Scientists used a tool called zinc finger nucleases on Madeaux, who has Hunter syndrome.
Helen O'Neill, a geneticist from University College London (UCL), told the Telegraph: "Certainly, 2018 promises to be the big year for clinical trials using CRISPR based genome editing. Results presented by [CRISPR Therapeutics] at a haematology meeting showed that the method dramatically increased fetal hemoglobin in beta thalassemia patients' cells. The therapy successfully edited over 90 per cent of blood stem cells removed from patients, which were retransfused."
Robin Lovell-Badge, group leader at London's Francis Crick Institute, added: "Assuming they do get going, we will look back and think that this is the real beginning of gene therapy. It will obviously be critical to check that there are few and hopefully no problems with the new methods, but as far as I know, the preclinical trials look very promising."
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