GDNF and Parkinson’s Disease


We have already blogged about the difficulties in treating advanced Parkinson’s Disease, and the need for new strategies including stem cell therapy.

Another therapeutic approach is use of a trophic factor to replenish or prevent the loss of those dopamine producing cells in the first place.

Glial cell-derived neurotrophic factor (GDNF) has been considered a potential therapy for Parkinson’s Disease for many years.


GDNF has been known to protect and restore these cells in animal models of Parkinson’s Disease since the 1990s.

However, human studies have shown mixed results so far:

Early small open label studies of GDNF infusions into the putamen showed promising results:

These results were not confirmed in a larger double blind study, although this study has been criticized for early termination and other issues:

Furthermore, subsequent studies have confirmed the clinical benefits of GDNF infusions in Parkinson’s patients, and have even shown a sustained benefit after the infusions were discontinued in one patient.

There are ongoing trials of direct infusions of GDNF into the putamen in the UK using a novel infusion pump:

Another potential approach is to use gene therapydelivering a virus that carries the DNA to make GDNF directly into the putamen by a single injection. The “infected” neurons will make GDNF on their own to treat the disease.

So, at this point the jury is still out on GDNF and Parkinson’s, watch this space for more information!


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