Experts in the diagnostic technique of Corneal Confocal Microscopy or CCM gathered at Weill Cornell Medicine-Qatar (WCM-Q) to discuss its use in clinical trials. The pioneering technique can detect major neurodegenerative disease by examining the small nerves of the eye. 

The two-day conference, supported by Qatar National Research Fund (QNRF) drew leading authorities in CCM from universities, research institutes and hospitals in Qatar, the UK, Italy, Germany, Australia, Singapore, China, Canada, the US, and elsewhere to explore the applications of this rapid, ophthalmic test in the diagnosis and assessment of treatment effect in various neurodegenerative diseases.

CCM can accurately quantify nerve damage and repair in many central neurodegenerative diseases like Parkinson’s disease, dementia, multiple sclerosis, autism and schizophrenia, as well as peripheral neuropathies in patients with diabetes, HIV and chemotherapy for cancer. CCM can also used to investigate nerve damage in amyloid neuropathy (nerve damage caused by build-up of a rogue protein), Friedrich’s ataxia (an inherited degenerative condition), and idiopathic small fibre neuropathy (a genetic condition characterised by severe pain).

Advantages and disadvantages

The expert speakers discussed a wide variety of topics, beginning with a history of the development of CCM, and ranging through the use of CCM in clinical trials, the potential for using CCM in conjunction with AI, and the advantages and challenges of using CCM.

A major issue identified by most speakers was the lack of effective US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved therapies for neurodegenerative diseases, identifying inadequate end points as a major reason for multiple failed clinical trials. The main theme of the conference was whether CCM could be used as a ‘surrogate endpoint’ in clinical trials of new treatments for neurodegenerative diseases, enabling FDA approval.

WCM-Q speakers

  • Dr Rayaz Malik (Professor of Medicine / Assistant Dean for Clinical Investigations and a pioneer in the development of CCM)
  • Dr Ioannis Petropoulos (Assistant Professor of Research in Medicine)
  • Dr Georgios Ponirakis (Clinical Researcher and Lab Supervisor)

Other speakers

  • Professor Andrew Boulton (President, International Diabetes Federation / Professor of Medicine, University of Manchester, UK)
  • Professor Nathan Efron (Queensland University of Technology, Brisbane, Australia)
  • Professor Ashfaq Shuaib (University of Alberta, Canada)
  • Professor Giuseppe Lauria (Professor of Neurology, Milan)
  • Professor Roy Freeman (Director, Center for Autonomic and Peripheral Nerve Disorders, Harvard Medical School, Boston)
Dr Rayaz Malik, one of the pioneers of CCM
Dr Rayaz Malik, one of the pioneers of CCM technique

According to Dr Malik, the key advantages of CCM is that it is fast, non-invasive and reliably detects neurodegeneration.

We need to make it more widely available and incorporate AI-based technology to diagnose different neurodegenerative diseases and to use it in multicentre trials of new therapies.

The conference titled, Corneal Confocal Microscopy: A Surrogate Endpoint for Neurodegeneration in Clinical Trials, concluded with agreement on the issuance of a Doha CCM consensus statement – a document identifying the key challenges, opportunities and actions for the wider adoption of CCM for the assessment of peripheral and central neurodegenerative disease, which is to be submitted for publication to the Journal of the Peripheral Nervous System.

The participants also reached an agreement to establish a multi-centre consortium to work collaboratively on studies utilising CCM to investigate peripheral and central neurodegenerative diseases.

The event was also supported by Proctor and Gamble and Heidelberg Engineering.


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