A study of an experimental drug from the company Vertex, called VX-770, successfully reduced lung problems in CF patients, and the company hopes to try for approval of the drug later this year. If all goes well, doctors may soon have their first drug to treat the cause of this devastating disease, instead of just combatting the symptoms.
Cystic fibrosis is a genetic disease that impairs lung and digestive function. In particular, the normally thin layer of mucus in the lungs thickens up and impairs breathing; this happens because patients have a faulty version of a protein that helps clear mucus.
About 1800 different mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene have been implicated in the disease. The gene encodes a molecular channel that shuttles chloride ions across cellular membranes, and people with two mutated copies develop mucus-filled lungs susceptible to infection. Few patients live to see their 30s. In 1989, CF became the first disease pinned to a specific gene mutation, without the benefit of knowing the protein first. [Nature]
This newest test was a Phase III trail of Vertex’s drug, which was funded in part by the Cystic Fibrosis Foundation. The treatment goes after one major genetic mutations that causes the disease, called G551.
If a vaccine injures a child, should the parents be allowed to sue in state court? That’s a question lawyers, vaccine makers, parents, and Congress have wrestled over for a quarter century. This week, the United States Supreme Court brought forth a ruling that keeps the status quo: No, you can’t.
The justices, voting 6-2, said a 1986 federal law preempts claims that a drugmaker should have sold a safer formulation of a vaccine. The law, designed to encourage vaccine production by limiting patient suits, channels most complaints into a company- financed no-fault system that offers limited but guaranteed payments for injuries shown to be caused by a product. [Bloomberg]
The case in question, which has been kicking around for nearly two decades, was brought by Russell and Robalee Bruesewitz on behalf of their daughter, Hannah. In 1992 she began experiencing seizures after receiving a diphtheria, pertussis and tetanus shot made by Wyeth [part of Pfizer]. At the time, her parents tried to file a claim with that government-created system.
When a special Vaccine Court within the Court of Federal Claims ruled that her injuries couldn’t be linked with the vaccine, her parents tried to move the case to Pennsylvania state court. The Third Circuit Court of Appeals eventually ruled that the claim was pre-empted by federal law, a decision upheld by the Supreme Court. [Wall Street Journal]