CF10 Prodrug Update – April 2020

Professor Herbie Newell CBE
Professor Herbie Newell CBE. Image source: https://www.sunderland.ac.uk/more/news/story/fight-continues-against-rare-condition-930

CF10 (Cystinosis Foundation 10) is the code name for the prodrug developed by the late Professor Roz Anderson at Sunderland University with over £1m of funding from Cystinosis Foundation UK. Roz worked closely with cystinosis patients and before her untimely death in 2018 was able to secure a £1.6m MRC grant to take the project through the pre-clinical development ready for clinical trials – one of the highest graded grants that have been awarded through the MRC in the last two years.

Roz’s work is being continued by colleague and friend, world leading cancer researcher, Professor Herbie Newell, who has a background in bringing drugs from discovery to market. Professor Newell attended Cystinosis Foundation UK’s recent trustee meeting to provide an update. He is determined to bring to fruition Roz Anderson’s dream of a more effective treatment without the unpleasant side effects of cysteamine.

The team aim to complete their pre-clinical trials in safety and toxology by the end of 2020, having scaled up the production of CF10, and will finish the paperwork for clinical trial authorisation. The next year will be spent formulating the drug into a doseable form and applying for funding for clinical trials from funding bodies. The team hope to be able to start clinical trials in the first patients in 2022/2023.

In advance of the clinical trials the team are planning a pharmacological and logistics study (PALS) as a ‘dry run’ to iron out any problems before the main trials start. This study, involving approximately 6 patients, will take place at Birmingham Queen Elizabeth Hospital under the direction of Dr Graham Lipkin.

The impact of CF10 could be considerable on patients who are currently taking cysteamine in its present form.

  • Less dosage required because the prodrug directly targets the cells where it is needed
  • Reduction from four times to once or twice a day
  • Reduction/elimination of halitosis and body odour
  • Reduction/elimination of nausea, vomiting and gastrointestinal tract issues

Cystinosis Foundation UK has funded a special machine designed to measure hydrogen sulphide, methanethiol and dimethyl sulphide in the breath. We are also preparing to fund a ground-breaking handheld confocal microscope to give more accurate measure of cystine levels in body tissue. Both these machines will help deliver quick results of the effectiveness of CF10 in clinical trials hopefully providing the information needed to bring the drug to market.

We are very proud of the work we have funded over the last 10 years and very grateful for all those who raise money for the charity. We are delighted in the way the project is moving forward and confident in the way that Herbie is continuing to engage with the cystinosis community, he recently gave a talk at the Virtual International Cystinosis Conference, which took place on 25th April 2020.

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