Well today there was a conference called held by the NIH and NNPDF to update the community on the Cyclodextrin trial. The news was not what I was hoping for. They had to put the trial on a pause status due to an infection at the ommaya reservoir site in two patients and another (who is not in this trial) suffered from a brain bleed. The ommaya reservoir is like a port for a chemo patient but it is surgical placed in the front of the brain. The infection was cause by bacteria that sits on the skin that causes acne. At this time they are not sure if NPC patients are at a higher risk since this is a lipids disease or not. But they knew they couldn't continue with the trial with so many problems and not enough answers.
They put in on hold and got in contact with the FDA who in turn put an official hold on the trial and will send the NIH a letter stating what has to be done to get the trial off the hold status. The FDA was very pleased with the quick action by the team at the NIH for putting the trial on pause right away without first contacted them. The NIH will receiver the letter sometime next week and will inform the NPC community once they have a plan of action in place.
Dr. Porter did add that they did collect some promising results from the trial thus far. The bio-markers looked good and they got goo PK data. These results where from the patients receiving a very small dose of the cyclodextrin. Dr. Porter thought they would see nothing with this low of a dose, so that in itself is very hopeful!!
They do think that the infection was not drug related but device related. So he did go on to say that they will be looking at a lumbar injection as means to administer the drug. They do have concerns with how much of the drug will actually reach the brain but with the data collected from the trial he believes that they will be able to administer enough of the drug to reach the brain without causing toxicity.
There is a lot of work ahead for the NIH team but they are committed to get this trial back up and running as soon as they can. They could not give any kind of time line or dose levels at this time. I will keep everyone posted as information comes to me. Please say an extra prayer for the NPC community especially those in the trial that suffered an infection and the child who suffered a brain bleed. Thank you and God Bless!!