Content for Families, Carers and Professionals

CDKL5 Forum - 10-11 October 2016

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This week myself, Carol-Anne and Adrian attended the CDKL5 Forum in London, organised by the LouLou foundation. The meeting was a bit different in format to the European CDKL5 meeting. A number of biotech companies and advisors from regulatory bodies attended, so there was more focus on long-term goals and what would happen should biotech companies become involved in the development of treatments, and how these could be taken to clinical trial. Although this might still be some time away, there was a lot of discussion about lessons learned from clinical trials for other disorders and how to plan and organise in advance to try and avoid some of the setbacks that have been seen before in other trials.

A number of research labs in Europe and the USA have recently been funded through a partnership between the LouLou Foundation and the Orphan Disease Centre at the University of Pennsylvania, and they were all represented in London. A lot of these funded projects have only just recently started, so in terms of basic science there wasn’t too much in the way of new and novel data since the summer meeting in Birmingham. However, there is clearly a lot of work underway and by the time of the meeting next year it will be interesting to see what avenues have progressed significantly. Some of the groups did share preliminary data at the meeting, which is actually not that common for science meetings, but quite encouraging for the CDKL5 research community. Groups were also quite open about the sharing of reagents and animal models, which, again, is encouraging for all the researchers working on CDKL5. It definitely has the potential to help speed up progress. 

Ashley Winslow, who is the new Director of the CDKL5 Program of Excellence for the Orphan Disease Centre and the LouLou Foundation CSO, finished the meeting with a summary of the main discussion points. In terms of science, a number of gaps in our knowledge were identified and I think many of these questions are going to be addressed by various research groups over the coming year. The issue of patient data and databases also came up, and the need for this to be better collated and made available, to families, clinicians and scientists. There’s no immediate solution to this, but definitely an acknowledgement, and commitment, that this should be improved as a matter of urgency. One thing was clear from the meeting; there is now a lot of work, and a very wide range of work going on to try and drive the development of therapies for CDKL5.


  LouLou; Research; CDKL5; clinical trials