Why are stem cell therapy and gene therapy two different things?
Stem cell therapy and gene therapy are similar in that they both aim to treat some currently incurable diseases, disorders, injuries and other medical problems not curable by drug therapy. However, this is where the similarities end because stem cell therapy is based on changing and developing particular cells in the body in order to perform a specific purpose; whereas gene therapy focuses on the manipulation or replacement of faulty or damaged genes in order to correct that gene’s inability to function as it should.
Have clinical trials in gene and stem cell therapy ever seriously hurt or killed somebody?
Yes. Sadly it is the case that there have been several deaths directly linked to clinical trials where people have died having received these types of treatment. A well known case is that of Jesse Gelsinger who died in 1999 whilst taking part in a gene therapy trial. It is important to remember however that all forms of medicine have side effects and it is not unheard of for people to have similarly severe reactions to drug therapies – and drug therapy is a thoroughly researched, thoroughly understood treatment method.
Can a person receive these therapies at the same time as undergoing drug therapy for a medical problem?
Yes. Gene therapy or stem cell therapy and drug therapy are often used side by side to treat a problem. For instance, in the case of bone marrow transplants to treat cancer, chemotherapy and radiotherapy are used to kill off the unhealthy cells before the transplant is carried out, replacing the body with healthy versions of those cells.
Do these therapies make you feel ill or give you any nasty side effects like such things as chemotherapy does?
These sorts of therapies can have very severe side effects such as death or serious disease if they are unsuccessful; however, so can drug therapy and most of us wouldn’t worry too much about that. If your treatment is successful – and for the most part it would be – then it is likely you would suffer fewer side effects than you would with traditional drug therapies which can often give the patient a great number of mild but uncomfortable side effects.
Which has had more success so far, gene therapy or stem cell therapy?
Arguably stem cell therapy has had more real success due to the fact that stem cell therapies such as the bone marrow transplant have been in commercial practice for a great number of years already and have proved successful. However, both treatments have celebrated some major successes in the research stages so it would be unfair to label gene therapy as unsuccessful.
Why are umbilical cord blood stem cells and embryonic stem cells more useful than bone marrow stem cells and peripheral cord blood stem cells?
It is not fair to say that these types of stem cells are “more useful” exactly; however it is the case that these stem cells are younger and therefore less mature – they’re basically new and free from all the effects that age has on all areas of the human body, genes and cells included. The main advantage of these cells is that they have not yet had the chance to develop a strong immune system, just as a newborn baby has not. This means that there is less chance of rejection when they are injected into a body and therefore more chance of success with the treatment.
As yet, has gene therapy actually been used to create a “designer baby”?
Yes – but as with human cloning, probably not in the way which you imagine it to be. The selection of specific embryos has not yet been used to produce a blonde-haired blue-eyed child with a groundbreaking IQ even if it is a possibility. The process has been used, however, in cases where parents want to have another child with specific DNA that will be of use to a sick child they already have. This process has been documented in a book by American author Jodi Picoult called ‘My Sister’s Keeper’ which portrays a family that did exactly this – had a child who would be a perfect genetic match to her sister who had a rare and destructive type of Leukaemia – and Picoult examines the moral and ethical issues of the concept.
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