A Milestone in Europe

   A type of gene therapy has finally been approved in Europe: patients who suffer from acute pancreatitis will be able to have a better life thanks to a drug called Glybera.

  The drug Glybera was designed to fight an especial type of acute pancreatitis. This type of pancreatitis comes from a genetically inherited protein deficiency and affects about 300 and 750 patients in Europe. Patients who suffer from this disease are unable to process fat particles carried in their blood, leading to the inflammation of the pancreas, and the inflammation of the pancreas can cause death.

  As we know, gene therapy is the use of DNA as a medicine to treat a disease. The surprising fact here is that the drug Glybera is the first type of gene therapy ever approved in Europe. Patients who suffer from acute pancreatitis had to rigorously restrict the amount of fat they consume as the only resort to deal with the disease; now, Glybera prevents the inflammation of the pancreas, which avoids the pain associated with it, among other benefits. Patients’ lives will have a significant impact due to this.

   No matter how good this sounds, I can’t help but wonder: Isn’t this a “baby step” for gene therapy? And if so, isn’t now more likely to happen that later in time gene therapy will be used to cope with multiple diseases and that itself may lead to other uses of gene therapy? I think that even though it helps to improve people’s life, it’s dangerous. 

If you want to read more about it, click here or here.

Aidan's Law

    In June 2011, Aidan Seeger, an American 7 year-old, was diagnosed with Adrenoleukodystrophy (ALD). ALD is a rare genetic disorder, which is characterized by the loss of the myelin sheath surrounding nerve cells in the brain and the progressive dysfunction of the adrenal gland.  In most diagnosed cases, patients die from this disease. This fatality rate is because patients are diagnosed wrongly or because patients do not get a proper treatment.

   When treatment is received before symptoms, children are more likely to not suffer from ALD, but this can only be possible with early detection. Aidan, however, was diagnosed too late. He went through a bone-marrow transplant which was no effective since it only works well in patients that are treated early in the disease. In April 2012, Aidan died.

   His parents, Elisa and Bob Seeger, are now fighting to make testing for ALD mandatory in every newborn child in New York, arguing that it is not necessary for any kid to suffer when they could have early testing to avoid even the symptoms. Also, in terms of money, the treatment is much more expensive than testing- or screen for- ALD.

   In my view, it would keep children from suffering if the so-called “Aidan’s Law”- which goal is to make testing for ALD mandatory in New York-  got passed.  Wouldn’t it be simpler then if every kid had the possibility to avoid this horrible disease? Would you support Aidan's parents' cause?

 For more information, click here.