2016.11.10 The Good Viruses

I wonder how this works?

Do you ever feel that way when hearing about break-through science achievements?

Gene therapy is one such promising new area - a long time in the coming.

Scientists may know what the faulty genes are and they have some idea how to repair those genes. But to get patient and therapy together is very difficult.

Even if the treatment was something so literally superficial as slapping on a skin ointment, gene therapy means putting "something" that can change genes into the organism. Read the sentence again and imagine all the things that can go wrong if this is applied to the wrong patient - or let lose by mistake.

Because these "somethings" are typically genetically modified live viruses in doses big enough to overcome the patient's immune defense. (Yes, it is called bio-medicine for a reason.)

I am thinking of a parallel when chemo therapy was first introduced. Did you know that the first chemo therapy was injecting patients with mustard gas? Nasty stuff if spread in the wrong places. Hospitals apply strict treatment and handling protocols not to harm patients or staff.

Imagine a similar protocol for dealing with bio-medical treatments. It is not something you local family practitioner is supervising.

Even within the laboratory the viruses are not supposed to be able to survive outside of strictly controlled environments. But since doctors don't know exactly which doses are needed, being able to turn the virus activity up and down - more a thermostat than a light switch - is also a requirement.

Talking to Professor in Neuroscience at the University of Lund Deniz Kirik, I learned that both in "Medical Valley" - the greater Copenhagen area that houses several medical research companies and not the least Novo Nordisk - and in California people are considering starting a center for gene therapy. Dr. Kirik holds some of the key patents for regulating what the virus will do once it is in the organism - turning "the thermostat" up and down.

Unfortunately, the "long time coming" applies even more in Dr. Kirik's main field of interest: Parkinson's Disease. As the brain is really good at keeping viruses in the blood stream out of the brain itself, gene therapy for neurological conditions will currently require injecting the virus directly into the brain less the rest of the body will be flooded by virus. Needless to say, opening the skull is more complicated than putting on an ointment, swallowing a pill, or getting an injection in your arm.

Before the treatment will be approved for general use, it needs to go through rigorous testing - a big and very expensive hurdle as no hospital yet has a dedicated gene therapy center with proper protocols and capacity to carry out late stage clinical trials. Tests have to be done when/if the hospitals willing to participate have a free operating theater and a free neurosurgeon team. (Just in case you wondered what money donated to research might be used for...)

Previous tests in the gene therapy field have stranded with typically only 10-40 patients being able to be part of the test even if they have been running for five years. At the current speed and funding level, the researchers will have retired before the treatments will - perhaps - be approved.

Imagine if some of the key players in Silicon Valley would collaborate with those in Medical Valley, step up to the plate, and get this ball in play. It is a pity if missing logistics keep known research from becoming available to suffering patients - or even curing young people before they pass on the faulty genes to the next generation.