The promise of gene therapy has been circulating for nearly a quarter of a century, and the best that can be said of it is that its results have been very modest, and sometimes tragic.
In 1999, American teenager Jesse Gelsinger died in a clinical trial where gene therapy was used.
One year later, a team of French researchers treated 11 children with a severe immunodeficiency, achieving partial success, but two of the patients developed leukemia because of the virus -a retrovirus- used for Introducing the correct gene into their cells. Despite the great efforts of scientists, little else has been improved; at least publicly
Despite little or no success, the United States will approve in September the first form of gene therapy for commercial use.
It will be used against leukemias that do not respond to any another treatment, and this time the technique, developed by Novartis, is backed by an apparent good result in an international clinical trial.
Now hold your breath. The vector virus, used as a carrier, is a manipulated version of HIV, the AIDS virus.
Like the viruses most commonly used so far, HIV is a retrovirus. Scientists have taken copies of its genome and integrated them into the human genome. It is a special type, scientists say. It is called a lentivirus. And, unlike traditional retroviruses, modified lentiviruses have been shown to be incapable of causing leukemia, as was the case with the children’s bubble.
Scientists claim that, unlike traditional retroviruses, modified lentiviruses have been shown to be incapable of causing leukemia, as was the case with the children’s bubble.
Now sit down for the following detail. The injection that is given to the patient costs between 250,000 and 500,000 euros.
The second key is that leukemia is a disease of the lymphocytes, or white blood cells, which are the incarnation of the immune system.
This avoids having to infect a solid organ, such as the liver or the lung, with the virus carrying the correct gene, which is extremely difficult. Instead, lymphocytes are removed from the patient and transported to Novartis laboratories, where virus infection is carried out with full assurance.
The cells are then selected and injected back into the patient. Scientists claim that two-thirds of them remain cancer-free two years after treatment. These would be a seriously relevant detail if they were true.
The main obstacle is now the economic one. The price of the injection is out of reach for most patients -250,000 to 500,000 euros- according to unofficial estimates.
Scientists say that if the patient heals, he does not need the medication again.
This is just the nightmare of any financial department at Big Pharma, and is often expressed with a cold statement: curing a disease is not profitable.
Unfortunately, it is going to be the big problem with the great advances of pharmacological research that are now waiting their turn in the industry’s production pipeline.
They are increasingly effective medicines, but also more expensive. A prologue of this type of situations has taken place with drugs against hepatitis C, which some administrations have resisted tooth and nail to fund despite its proven effectiveness against this serious disease.
This economic aspect is going to be our daily bread, and public health managers should already be preparing for what is coming. These are the lights and shadows of the future.