MDS 2021 | Achieving success in Parkinson’s trials: defining subgroups and improving drug delivery
Etienne Hirsch, PhD, Institut du Cerveau-ICM, Inserm, Sorbonne Université, Paris, France, discusses the critical next steps to improve success in clinical trials of neuroprotective agents for Parkinson’s disease. Prof. Hirsch talks on the need to select defined patient subgroups with underlying biology that would make them suitable for the intervention being assessed. Parkinson’s disease encompasses many subtypes, each with different underlying biology and thus, applying the same approach to treat all patients cannot be successful. To achieve this, better biomarkers are needed to characterize these subgroups. Additionally, Prof. Hirsch highlights the importance of improving the brain biodistribution of trialed drugs. Delivering agents to the specific regions in which degeneration occurs may improve their efficacy and limit the peripheral side effects that hamper their use. This interview took place during the 2021 International Congress of Parkinson’s Disease and Movement Disorders.
Transcript (edited for clarity)
I would say two next steps in parallel. The first one is clearly to better select the patients. I think we are doing—sorry for all the community—but we are doing a stupid job. Imagine that we are treating a patient with diabetes. Of course, you would try to understand whether the patient is responsive to insulin or not. If you give insulin to all the diabetic patients, it’s just stupid and we are doing exactly the same in Parkinson’s disease...
I would say two next steps in parallel. The first one is clearly to better select the patients. I think we are doing—sorry for all the community—but we are doing a stupid job. Imagine that we are treating a patient with diabetes. Of course, you would try to understand whether the patient is responsive to insulin or not. If you give insulin to all the diabetic patients, it’s just stupid and we are doing exactly the same in Parkinson’s disease. So, one way to really improve the clinical trial is to work with selected population of patients. And for that, we need better biomarkers to characterize those patients. So, this is one way to improve the clinical trial.
The other way is that most of the drugs we are using are not going to the right place in the brain. And the pharma industry needs to have better ways to bring the molecule first into the brain. If we are taking as an example, the anti-inflammatory drugs, PD patients are very often elderly people and anti-inflammatory drug is one of the best drug to kill the elderly people due to medication. If you have stomach bleeding, it’s just a catastrophe. And the reason for the failure is that we have the side effects, which very often are peripheral effects, and the drug might have a fantastic effect in the brain if you get rid of the side effects which are very often peripheral effects. So, it’s a challenge for the drug industry and the biotechs.
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