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?

World Hunger: Crisis and a plan of action

According to UN food and agriculture agencies, the number of hungry people in the world has fallen over the past decade. However, the number still stands at 805 million, a figure representing one ninth of the global population.

Some countries have been able to improve their domestic figures, but the number of undernourished people is a still a problem that needs urgent international attention.

Crisis

The fight against world hunger in 2014 has had numerous setbacks. For instance, the Ebola virus has taken its toll on food supplies reaching the affected countries.

Elsewhere, conflicts in Syria, South Sudan and the Central African Republic have increased hunger in those countries; with the need for aid clear to see.

Plan of action

Solving the issue of world hunger is not just in the interests of those affected, but is also good for the rest of the world. When a country is suffering from hunger throughout its people, this can cause a basic lack of productivity.

Economically, this hinders trading opportunities, costs millions of dollars and is eventually leads to even worse poverty. The desperation caused by such situations can lead to conflict, an issue, which can cost intervening parties millions of their own.

Looking after our own

However, it is not merely out of self-interest that it is important to try and fight world hunger. Humankind must learn to protect each other from all harm. Is world hunger an issue you want on your conscience? Or do you want to beat it?

We can help

There is no straightforward solution to combating this issue, every suffering country has troubles to contend with, and privileged nations must be willing to help.

Countries affected by disease must receive more medical attention. Only by getting the healthy back on their feet can the fight against hunger begin.

Development

In underdeveloped countries, where there is not enough food to go around, aid packages are a necessity; it is also important to promote sustainability.

We must make sure that people are taught how to gather their own food and treat their sick, so they can survive once the aid packages cease to arrive.

Finally, governments must learn to stand up to those who oppress their people. The issue of countries with economic wealth, yet a huge divide between rich and poor, need to be addressed. There is no reason for millions of people to be starving every day.

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.

Ebola death toll: are we doing enough?

Since March this year, over 3500 people in western Africa have been affected by the Ebola virus, with over 1,800 people suffering fatalities. The virus, which is contagious, has affected the countries of Guinea, Liberia, Nigeria, Senegal and Sierra Leone in this most recent outbreak.

Discovered in 1976, the causes for Ebola are not clearly defined, but are said to involve contact with an infected animal’s bodily fluids. Amongst humans, the disease is spread by bodily fluids and secretions – and because there is no vaccine, measures must be taken to slow contagion.

What more can we do?

According to the World Health Organisation (WHO), £360m is the very least needed to combat the current outbreak. They have warned that up to 20,000 people could be infected before the virus is brought under control.

Medecin Sans Frontiers (MSF), the international medical aid group, has stated that the global response to the crisis has been inadequate and that military intervention is necessary in the fight against Ebola. MSF believe that the only way to bring the outbreak under control is for military and civilian teams capable of dealing with a biological disaster to be immediately deployed.

These calls have been heeded by the French, who have sent 20 experts to the infected area – the UK must follow suit.  Although the foreign office has already donated well over £3m to the relief effort, more expertise and personnel is needed in the region.

Will there ever be a cure?

Ebola outbreaks have been sporadically appearing across Africa for over 35 years, yet the search for a vaccine is still ongoing. New medical research has uncovered possible ideas for trials, but there is no answer yet.

How can we stop the death toll?

If Western powers join together to provided financial aid and personnel then the death toll will slowly decrease. As the disease is not spread by regular everyday contact (such as shaking hands), in theory it is relatively simple to slow the transmission of the disease. This has not proved to be the case, primarily because the people in the region are afraid and do not know the correct preventative measures. These residents should be educated in how to stop the spread of the disease.

World superpowers should be investing their resources in Western Africa, because there is no telling how far the virus could spread. The region needs to be able to both treat and educate its people, and they cannot do it on their own.

Ashya King: lessons to be learned

The parents of 5 year old Ashya King were released from a Spanish prison after a European arrest warrant against them was cancelled; this arose following their actions to smuggle him out of Southampton General Hospital and travel to Spain, believing he was not getting the best care and that his condition would deteriorate. They wanted Ashya, who has a brain tumour, to receive treatment at the Proton Therapy Centre in Prague.

However, the full force of the Law immediately swung into action, involving the Crown Prosecution Service (CPS) and the Hampshire Constabulary. The couple were arrested, handcuffed, separated from their son and thrown into prison, simply for loving their son and wanting the best possible treatment for him.

Justice for the Kings
Only after a sustained cry of public protest ensued, involving the Prime Minister, were the parents re-united with their desperately ill son. By now the child was confused, depressed and lying alone in a foreign hospital, adding to the family’s anguish.

Mr King said: “We want to help our son get through this bad time because he hasn’t got too many months to live and we’re locked away in a cell…”

Mrs King, said: “I just want to wet his mouth because he can’t drink through his mouth, I want to brush his teeth, I want to turn him side to side every 15 minutes because he can’t move.”

Yet, he could still see and feel emotions. Would deprivation of a mother and father’s comfort help Ashya? How would the little boy’s confidence and trust be impacted long term? The story has touched the hearts of people worldwide.

We have since learnt that the private clinic in the Czech Republic can treat Ashya.

Duty of care v parents love
The CPS said the risk to Ashya’s life “was not as great or immediate as had been originally thought.” The parents had ordered specialist foods to care for Ashya, and had managed to charge the food pump.

Doctors have a duty of care to do the best for their patients. Do loving, devoted parents also have a right to disagree with their decisions? Isn’t there a case for decent common sense to prevail?

On the other hand, what sort of outcry would have happened if the doctors had let him go without a word and the boy had died? What would this say about his medical care?

The parents have won their brave fight against the institutions yet only after the healthcare system, the police and prosecutors were ruthless in their pursuit of them.

Was their only crime to opt out of receiving NHS care? Having allowed their son to be treated by the NHS they couldn’t escape its clutches and were punished if they dared to disobey the rules? Was this the fault of the doctors or a heavy handed NHS?

Should we question a healthcare system that, instead of apologising to the parents when they thought the service was not good enough, reacted with an unpleasant uproar?

Clearly, there are some lessons to be learned.

HIFA2015: to achieve goals – more support is required

“Our shared vision is a world where people are no longer dying for lack of healthcare knowledge.” Statement on HIFA2015 website.

HIFA2015 (Healthcare Information for All by 2015) is a global network of more than 10,000 members which aims to improve the availability of lifesaving information in developing nations. Based in the United Kingdom, HIFA2015 is now supported across 167 countries by at least 2,000 separate organisations. These include the British Medical Association, the Royal College of Midwives and Publication Integrity & Ethics (PIE).

The aim is to stop thousands of people dying each day for want of simple, low-cost interventions. HIFA2015 not only recognises the need to provide professionals with improved information – it also promotes the value of educating anyone who provides basic care. Campaigners behind the organisation believe that access to relevant, reliable and user-friendly health information is vital in meeting both the World Health Organisation’s goal of ‘Health For All’ and the United Nation’s ‘Millennium Development Goals’.

Are these goals achievable? Will every mother, care worker and doctor of the worlds’ poorest countries really have access to sufficient information?

According to several key figures in the medical world, the answer is yes. These people include BMJ editor Fiona Goodlee who wrote an essay calling for universal healthcare information in 2004 and partly inspired the foundation of the group. This is only possible, however, if more support is gained and adequate funding is secured.

The good work that HIFA2015 promotes can already be seen taking immediate effect with the start-up of several other organisations. Just one example of this is Doctoori. This UK-based organisation focuses on the development of the healthcare sector in the Arab nations with online content provided by the NHS. “HIFA has been a massive inspiration in founding www.doctoori.net. It has reinforced the need to bring high quality, reliable and accessible health information into the Arabic language,” said Dr Zain Sikafi, CEO and Founder of Doctoori.

As a proud supporter of HIFA2015, the Publication Integrity & Ethics believes that the organisation needs a much stronger backing if it is to achieve its lifesaving ambitions. The HIFA strategy can only succeed in improving global healthcare knowledge in 2015 and beyond if the message is spread on a mass scale.

Just one of the statements on the HIFA2015 website states that 7 out of 10 African children with malaria receive mismanaged treatment. That is 2,000 young lives lost each day in Africa alone. HIFA2015’s mission is not only important, it is urgent.