Since it was first conceptualised in the early 1970s, gene therapy has been very much considered to be an experimental technique. But recent clinical successes are proving that this cellular level approach could revolutionise the way we treat (and indeed pre-treat) diseases and disorders caused by genetic abnormalities. Here, we take a look at some of the latest breakthroughs and how gene therapy is breaking the mould in the here and now:
Gene therapy can be broadly described as treatments which allow a patient’s cells and tissues to be engineered so they function normally again. It has the potential to be used for previously incurable diseases such as leukaemia, cystic fibrosis and even blindness.
Gene therapies can be categorised in two ways – somatic gene therapy, which alters a section of DNA but will not be passed onto the patient’s children, and germline gene therapy, where the effects of the treatment will be transferred to cells that produce eggs or sperm therefore will be inherited by future generations. The effects of gene therapies – especially germline treatments – must be closely monitored to ensure that what benefits patients now, will not cause issues further down the line.
Scientists have been researching the possibilities of gene therapy for more than 45 years, and while there have been more than 2,000 clinical trials conducted in that time, successes have been hard to come by. But new techniques and technologies have improved the hit rate for gene therapy, with exciting results.
Hope for arthritis suffers
In January this year, it was reported that the US had given the go-ahead to human trials of a gene therapy to treat osteoarthritis and rheumatoid arthritis. The procedure involves injecting genes into the joints to release a protein which improves the effectiveness of existing drug treatments formulated to ease the condition.
Muscular dystrophy trial
In Columbus, Ohio, a human trial is underway to help young people suffering with Duchenne muscular dystrophy (DMD). A gene therapy infusion is being administered to trial patients via the blood so it can reach all muscles in the body, to replace the missing dystrophin protein that causes the DMD condition. Preliminary results are expected later this year and as well as having an impact on the treatment of DMD, it will also help researchers to understand more about how gene therapy can be used systemically.
Sickle cell success
In March 2017, researchers announced that a teenager in France had been cured of sickle cell disease after undergoing an experimental gene therapy, developed by Bluebird Bio. Scientists removed stem cells from the boy’s bone marrow and modified them in the lab to include a fix for the single genetic mutation which causes the disease. When the treated cells were infused back into his body, they began to make normal blood cells.
Bluebird Bio is definitely one to watch for 2018 too, as the company is on the edge of launching a one-time treatment for beta thalassemia, an inherited blood disorder which can cause severe anaemia, as well as seeking approval for a gene-based cancer therapy.
Last year, BioMarin published early clinical trial results showing that nine patients who received its gene therapy to replace the faulty gene responsible for the most common type of haemophilia were showing substantial increases in the blood clotting proteins typically absent with the disease. Meanwhile, a handful of patients with the rarer haemophilia B are already experiencing amazing cures after one time gene therapy treatments.
The price tag for progress
In 2018, Spark Therapeutics Inc plans to launch a cure for a genetic mutation which causes blindness – the drug is administered only once by injection but comes with an eye watering $425,000 price tag.
If you are interested in taking your next step – or your first step – into the world of gene therapy, call us for the low down on the great opportunities with British, European and Global biotech companies and the exciting projects you could be part of.