Dr Fiona Stewart, MPS Society trustee and chair of CSAC (Clinical Scientific Advisory Committee), looks back at a busy second half of 2024 and recalls how CSAC was able to support important future research and make meaningful connections at industry events, all for the benefit our community.
Grant funding
Well, it's been another interesting and busy six months for CSAC. We had some really interesting grant applications of which we were able to fully fund two. The first one focuses on MPS III Sanfilippo and the other revolves around the blood brain barrier. In addition, we offered partial funding in form of a 'challenge grant' for a project looking at lysosomal damage repair. In a challenge grant, the applicant is offered partial funding, provided they can find the remainder of the funding from other sources.
We felt that the quality of all three applications was excellent.
CSAC on tour
It was also great to see several CSAC members presenting at the annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Porto last September. Together with Bob, we met colleagues from other patient organisations and also had meetings with pharma companies.
It was great to see how highly the MPS Society and RDRP are regarded and how much our advice was appreciated.
Despite a very hectic schedule, we had the opportunity to meet clinical and scientific colleagues whilst trying to move forward with engaging regulators about the use of biomarkers as end points for clinical trials. As a result of this, the MPS Society is organising an international meeting for professionals in Amsterdam in March. This will provide an opportunity for clinicians, scientists, pharma colleagues and patient organisations to engage with regulators, including the EMA (European Medicines Agency) and MHRA (Medicines and Healthcare products Regulatory Agency). Sophie is doing an amazing job organising it all.
In October, Bob and I attended an event hosted by Aspire Therapeutics. They invited representatives from patient organisations and the industry to try and address areas of unmet needs. There were short presentations in the morning and then a ‘speed dating’ session in the afternoon, where patient organisations and members of the industry could engage.
We have received some great feedback from that which is very exciting.
CSAC open day
At SSIEM I handed out lots of postcards promoting our virtual CSAC open day at the start of October. We had some really informative presentations showcasing the work we have funded and also educated participants about biomarkers. Big thanks to all the speakers and also the communications team who made it happen.
Coming up in 2025
Finally, at our most recent CSAC meeting, we discussed the poster presentations at MPS Matters 2025 for which we are going to award prizes too. At the community weekend, there will also be a Dragons Den session, similar to the one held at Fabry Matters.
The main prize for this will be an amazing £5,000. Please encourage any clinicians and scientists you know to apply.
We will shortly be updating our website and sending out information with all the details. So, keep your eyes peeled and get in touch with csac@rd-rp.com if you are interested.
Firstly though, we are getting ready to attend WORLD in San Diego this February.
As you can see, it's been a busy six months for us and I'd like to say a special thank you to Harriet who makes everything work.
