By Isobel
My attendance at the Beacon Drug Repurposing for Rare Diseases Conference on Monday 10th October was well worth the very early start.
Thank you Beacon for all you do to educate, inform and connect people in the area of rare diseases and the potential remedies for these.
There were presentations from very many interesting organisations and individuals talking about the development, regulation of and deployment of drugs used for treating diseases for which they weren’t originally intended.
In pemphigus and pemphigoid, all our current treatments fall into the category of “re-purposed”.
In addition to the interesting talks and discussions, there was time to meet people who work in various capacities in this area.
The talks included:
· An explanation about how drug re-purposing works today by Dr Rick Thompson of Beacon
· How the potential of drugs in one area is identified and this is exploited in another.
· Examples of how these drugs might be used in completely different contexts with huge benefits
· Sheffield University who are exploring how patients in the UK can access “orphan” drugs
And many more.
I was particularly interested in the presentation by Rosie Lovett, head of the new-ish NHS Medicines Repurposing Programme.
Rosie Lovett
There were display stands around the centre highlighting other groups and organisations and showing other resources we can use.
Another resource
I hope to follow up with some of the presenters and others I met to explore the access that pemphigus and pemphigoid patients have to biologic solutions, particularly rituximab. Given the data showing how effective this can be in treating moderate to severe cases of pemphigus and, to a slightly lesser extent, MMP, it is disappointing that it is only approved as a third stage intervention and even then, seems to be used at a lower rate than the evidence would encourage it to be deployed.
It might also be helpful in the introduction of new treatments, for example, the Pro-tears eye gel that is about to be trialled at Birmingham Eye Hospital.
There are many possible benefits of PEM Friends attendance at the very educational conference.
Thank you,
Isobel
