Bringing a cell therapy for Parkinson's disease into the clinic - Stefan Irion, Memorial Sloan Kettering

‘The medical control of Parkinson’s disease is just not good enough - it helps in the beginning, but eventually people become less and less responsive to the therapy and they really suffer from it…It really helps to know that you're not doing it just for the several million patients worldwide but really for family members too.’

As Program Manager at Memorial Sloan Kettering, Stefan Irion is working under the leadership of Lorenz Studer and Viviane Tabar on bringing a new cell therapy for Parkinson's disease into the clinic.

We caught up with him at Biotech Week Boston in September 2017 - watch the interview or read everything Irion said below.

‘We hope to be in the clinic in the second quarter of 2018. We've licensed the technology to a company that will support the trial of the raw therapeutics. They will support the trial financially and we will file the IND (Investigational New Drug application) coming out of the institution. That will happen early next year (2018) and then obviously there are Institutional Review Boardsto please and so forth, so it will then be a few months before we can enrol the first patient.

Parkinson's is very complex. It's mainly characterized by most people based on the motor symptoms, but there are other effects that this therapy probably won't control. Since the motor symptomatic is really what the patients are suffering from, this should be a tremendous relief for their symptoms.

We believe that a single treatment of this cell therapy will provide a lifelong benefit to the patient because they stably engraft and they can produce dopamine in situ. This is vastly different to medication that you would have to take multiple times a day. It's also different to some extent to deep brain stimulation where you have a pacemaker in your brain that requires batteries and adjustment and so forth.’

Speed of progress

‘To be honest, I think the speed that we are developing this therapy is actually remarkable given that we were literally just publishing an academic paper in 2011 and here we are in 2018 talking about a clinical trial. When I joined this field after coming out of medical school, embryonic stem cells were just discovered and a couple of years later people started talking about the use of pluripotent stem cells. So that field in my opinion has been really, really rapid. Being immersed in it I realize that nothing happens overnight like the public may expect, but I think the field is actually advancing relatively fast.’

Patient education

‘There's huge excitement for these types of therapies. It's a cell therapy that's based on embryonic stem cells so some people have a hesitation with that, but I think there's very few in my experience. The people that are affected, they are looking for treatments and they are willing to do it. For me it is fascinating how many people, without knowing too many details of what we are planning to do, will say "sure, sign me up". It really shows how desperate they are.

The Michael J. Fox Foundation and the Parkinson's Disease Foundation have a lot of patient advocates that we also work with, who really try to educate these patients so they understand what the therapy will be like, and the general enthusiasm is really great.

Parkinson’s is not an acute disease that will kill you like a heart attack, but it's a disease that you have to live with for many years. With all the other diseases that you develop with age, more and more therapies come along for those, so people have to live with Parkinson's for many years. It affects not only them but all their caretakers, their family members, their children.

I'm really hoping our therapy will bring a relief to these patients and change the quality of life for both them and their families.’

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