The lab
What really goes on at the science end of treatment discovery? WORDS BY
John Pinching
H IT S E LEC TI O N
H IT TO LE AD
LE AD O P TI M I SATI O N
To develop treatments that can tackle the underlying causes of Parkinson’s we need to change the way brain cells work. The key is finding small molecules that target the process that has gone wrong in the cell. Up to half a million drug-like molecules are screened, quickly identifying any that may have potential.
To discover more about the chosen molecules, they are tested to identify those with the most promise. Any that fail, or cause problems in other organs, or are known to produce serious side effects, are discarded. Each molecule is tested for strength and it must be soluble in water. Biotech company Oncodesign is currently developing a class of compounds in the Hit to Lead phase that prevent the enzyme LRRK2 from activating. LRRK2 is often found in genes that are associated with Parkinson’s.
Now the group has been whittled down to molecules with genuine promise, it’s time to start developing them into drugs – this step needs skilled scientists who are specialists in drug design. They painstakingly tweak the structure of the remaining molecules to achieve the best possible effects, while minimising any negative reactions. Throughout this process, molecules are refined and then tested repeatedly – this can go on for years.
C LI N I C A L TR I A L S
C LI N I C A L TR I A L S
The new drug is ready for clinical trials on a small number of people, testing for safety, side effects and best dose. Studies involve a small group who may be healthy volunteers or people with the condition. Two different Parkinson’s vaccines are currently being tested in early stage trials – one developed by US firm Prothena, and the other by Austrian company Affiris.
These studies include a comparison group who receive a placebo, allowing researchers to see whether people who receive the treatment do better than those that ‘imagine’ they have taken it. Exenatide, a drug that is currently used to treat type 2 diabetes, has shown potential for slowing the course of Parkinson’s. A phase 2 trial of the drug is now complete and results are expected in 2017.
PHASE 1
1 4 | PH A R M A FI EL D.CO.U K
PHASE 2