Gene therapy: ‘Tame HIV’ used to cure disease

A pioneering treatment to correct errors in DNA, has transformed the lives of six boys with a deadly genetic disease, doctors declare. For people suffering Wiskott-Aldrich syndrome (WAS), a defective immune system leaves people susceptible to infections and bleeding.

A British and French study, published in JAMA, has used tamed HIV to correct the irregularities. A child wheelchair-user now has free mobility and there are improvements in other cases. The syndrome affects up to 10 children in every 1 million, almost exclusively boys.

Patients suffering the condition find that tiny bumps and scrapes can lead to wounds that are slow to close. Eczema is often present and they face repeat infections including pneumonia as well as some cancers and autoimmune diseases. It arises from an error in the genetic code that contains the building instructions for a key element in the immune system, a protein called WAS. The main therapy is bone marrow transplant; however that can only be considered when the donor is a close tissue match, such as from a sibling.

Great Ormond Street Hospital, in London, and Necker Children’s Hospital, in France have conducted trials to remove a part of the children’s bone marrow and purify it to find the cells that regenerate the immune system. Afterwards, the corrected bone marrow cells were then inserted back in the children. The outcome was so positive that in six out of seven boys, the therapy was a success. It reversed symptoms and drastically cut the number of nights spent in hospital.

Daniel Wheeler, who is now 15 and from Bristol, was the first British patient. His older brother died from the same condition when he was two-and-a-half. Their mum Sarah told the BBC News website: “Daniel was in and out of hospital, he had frequent infections of ear, chest, flare-ups and bruised joints, and lots of operations.”

Prof Adrian Thrasher, from Great Ormond Street Hospital, told the BBC News website: “I think it is very significant, it is another clear and powerful demonstration that a gene therapy approach is an effective one.” Prof Ian Alexander from the Gene Therapy Research Unit at Sydney’s Children’s Medical Research Institute in Australia said although the work was promising, it was “still early days”.

When all medicine, even Paracetamol, has risks, couldn’t interfering with genetic code bring huge dangers? Several trials have been abandoned as patients developed leukaemia when the modification turned healthy cells cancerous. But those trials did show one thing – the underlying principle worked. Could gene therapy be about to come in from the cold?

Stem cell breakthrough for Parkinson’s disease

Swedish scientists from Lund University have found that stem cell treatment can be used to heal the damage in the brain caused by Parkinson’s disease. The disease, which affects body control and movement, is caused by loss of nerve cells which control the chemical dopamine – essential for these cognitive functions. Parkinson’s UK have come out to say that the research is at a very early stage, but the news is a welcome breakthrough for advocates of stem cell research.

Ethical dilemma
One of the reasons that stem cell research is only now becoming a viable research technique is the ethical issues which have surrounded it for so many years. Up until 2007, stem cell research used tissue from aborted embryos to obtain material to study. This obviously posed an ethical dilemma as it involved obtaining aborted foetuses for scientific use, a process which caused significant outrage. Thankfully, in the last 7 years stem cell researchers have started using a new technique called Induced Pluripotent Stem Cells (iPS). This method allows scientists to obtain the cells they need, without the need for an aborted embryo, placating many critics.

Playing God
The other issues that surround stem cell research surround all types of health research: should we be playing God? What will the information be used for? Divisive as it is, research into terminal diseases has helped save thousands of people and we probably can’t afford to ignore the opportunities that stem cells offer.

Clones
The second concern is that in the future scientists will use the aforementioned iPS technique to create human clones, and at the rate research is progressing this concern could become reality. Can we trust scientists to act morally? Would cloning necessarily be a bad thing?

Present versus future
These questions are difficult to answer, since they concern something that is not yet possible. However, it must be said that because stem cell research has the potential to save thousands of lives scientists have a duty to continue. This new Parkinson’s breakthrough is only the beginning and it is hard to argue against a form of research which can be so medically productive. When it does happen, we will have to ask ourselves if we can ethically justify the scientific creation of human life. The issue of clones is something that will need to be confronted when the time comes, but for the moment it is not the primary function of stem cell research.