Yesterday, we talked about the promise (lost) of stem cells.  Which, if we were allowed to employ, would offer hope for those folks who need organ replacement- or for sufferers of Parkinson’s disease.

But, there still may be hope for Parkinson’s patients- even without stem cell therapy.  From a study that is ongoing in the United Kingdom.  Interestingly, the study is funded by Parkinson’s UK and the Cure Parkinson’s Trust.   Parkinson’s UK, founded in 1969, was originally known as the Parkinson’s Disease Society. Cure Parkinson’s Trust, a much younger charity (started in 2005), is devoted to finding a cure for the dreaded disease.  The government is also involved (North Bristol NHS Trust) in this study.

The hope is that GDNF (glial cell line derived neurotrophic factor) will regenerate the dying brain cells that abound in patients with Parkison’s, and in so doing, will improve their condition. There are several issues- not only if the GDNF works- but how to get the factor to the brain.  After all, our brains have significant controls to keep out foreign materials (such as the blood-brain barrier).

This study involves 41 patients (ranging in age from 35 to 75 y) who had four tubes inserted into their brain (via robotic surgery).  The delivery system allows GDNF to be infused to the particular areas of the brain that have the diseased cells.  This is actually the second phase of the trials- initially only 6 subjects were involved to discern that the treatment would be safe; then, we sought to prove it’s effective over a 9 month double-blind study.  (You do recall that in double-blind, we don’t know who got a placebo and who didn’t.  Now, after the initial phase (phase 3), everyone is receiving the drug.)

All subjects were provided PET (positive emission tomography) brain scans at the end of the first phase.  Not surprisingly, those who received the placebo manifest no change, while those who received the GDNF provided a 100% improvement in the affected brain regions. That should mean the treatment is certainly “awakening” the damaged cells- perhaps even restoring them to full function.  Symptomology has ranged from marked improvement to only moderate after 18 months, though.

In the study, over the course of 80 weeks (after some 3 years of patient screening), patients had the GDNF infused every 4 weeks (for a total of 20 injections).  However, the failure to improve symptomology is thought to be related that symptoms lag the brain changes; as such with continued therapy, it is hoped that the shaking and other symptoms will evaporate from the patients’ behaviors with time.  Right now, the key assessments of motor response, daily living activities, among others demonstrate the difference between the placebo and therapy cohorts.

The study is headed by Dr. Allan Whone, along with Dr. Steven Gill (who developed the infusion device, the port [called Convection Enhanced Delivery, as manufactured by Renishaw] which allows the drugs to be infused to the brain, with an opening behind the ear). The GDNF is produced by MedGenesis Therapeutix.   The results (crediting thirty authors) were reported both in the Journal of Parkinson’s Disease   and in Brain.

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