Ask the Pediatrician: The big stories

Q: As the year draws to an end, it’s a common journalistic tradition to ask what the biggest story was in a particular area of the news. Do you have a single issue that stands out?

A: I do, but as it happens there are two of them and I really can’t choose between them. So, I will try to be brief. The first was a report in October about the first malaria vaccine, which has proved to be fairly effective. It was tested in seven African countries in children 5-17 months old, in places where current preventive techniques such as mosquito nets and oral medication are in common use. The results showed that children who received the vaccine had only half the total cases and only half the life-threatening cases as children who did not get the vaccine. In 2009, about 700,000 children in Africa died from malaria. Further improvements in the vaccine are likely to follow.

Q: Wonderful! So, tell us about the second story.

A: The second story is about gene therapy for a condition called Hemophilia B, an inheritable disease that causes those who have it to bleed easily causing arthritis and other difficulties and sometimes death. Until earlier this month, the only preventive treatment was to harvest the protein called Factor 9 which these patients could not make themselves from donated blood, at a cost of $300,000 per year per patient. A new technique has shown effectiveness by injecting a live virus carrying a working gene into a vein which carries the virus into liver cells which when infected use that new gene to make enough Factor 9 for up to 21 months, so far. The trick is to get just enough virus into the body without detection by the immune system which would kill the virus. This is the first time this technique has succeeded after many, many failures. This treatment is estimated to cost roughly $30,000.

Q: So, these two new breakthroughs are related.

A: Exactly! The first studied vaccine was given against small pox in 1796, using a milder virus than the smallpox virus found in cows. Smallpox itself had been used by Dr. Zabdiel Boylston and Rev. Cotton Mather as a preventive in Boston in 1721 which was quite effective but 2 of every 100 patients died. Now we are using viruses to carry genes which cure entirely unrelated illnesses. The old and the new, always a good tale.

Helpful Links:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1200696/
article on malaria vaccine

http://www.nytimes.com/2011/12/11/health/research/hemophilia-b-gene-therapy-breakthrough.html?_r=1
article on hemophilia B genetic treatment