My Name’5 Doddie Foundation Business Club

28 Feb 2024

My Name’5 Doddie Foundation was established with one clear goal: to
expedite the development of new treatments for motor neuron disease (MND) and
ultimately, a cure. 2023 was an epic year of fundraising for the Foundation and
saw the launch of our new research strategy “Catalysing a Cure”.
Thanks to the extraordinary efforts of our supporters, today we are able to
announce a 6 million pound investment into research in 2024, beginning with the
launch of the Discovery Network and the reopening of our flagship Advancing
Treatments Award. Expression of interest applications are now open and will
close at midday on the 17th May. £4 million investment to establish the
Discovery Network The Discovery Network will provide the substantial funding and
innovative environment needed to encourage breakthroughs in our understanding of
MND that could lead to fundamentally new approaches to treating the disease.
Multidisciplinary teams will take on bold ambitious projects to better
understand MND biology and identify new targets for treatments. Underpinned by a
model born out of the Milken Institute and applied in Parkinson’s and
bipolar disorder, teams will collaborate in an open transparent network to
stimulate innovation, spark discoveries and speed up progress. In our research
strategy, we said that we’d drive the adoption of new approaches and new
ways of thinking to make faster progress together - this network does just
that. £1.75 million investment in our Advancing Treatments Awards The
Advancing Treatments Award is one of our annual competitive awards that supports
the next stage of research, translating breakthroughs into potential treatments.
It will support the validation of new targets and the pre-clinical development
of promising treatment approaches. Last year, we invested almost £1.3
million in three exciting projects through this scheme, which will be announced
soon. This year we are increasing the amount of funding available to £1.75
million. To ensure we’re supporting only the very best science, all
projects must be underpinned by the Guiding Principles for Drug Discovery and
Development in ALS we published last year with the MND Association and Medicines
Discovery Catapult. £250,000 investment in our Catalyst Awards Later this
year, we’ll also be re-opening applications to our Catalyst Award, our
smaller awards which enable researchers to bring early-stage promising ideas to
life. In our last Catalyst Award round, we awarded up to £225,000 across
three innovative projects which are currently being contracted and will be
announced in the coming months. This year, we will be investing up to
£250,000 into the Catalyst Awards which will open in Autumn. Jessica Lee,
Director of Research at My Name’5 Doddie Foundation said “When
Doddie established the Foundation in 2017, he wanted to make a step change and
fund the smartest, most efficient research to speed up the development of new
treatments. Research takes a long time, but by investing in innovative projects
and new funding models, we are striving to make faster progress and bring
effective treatment options to people living with MND as quickly as possible.
This is only made possible by our committed supporters, for whom we are so
thankful.” For more information about the funding available, please visit
our funding opportunities page.



My Name’5 Doddie Foundation Business Club

23 Feb 2024

Tofersen receives positive opinion from European Medicines Agency Committee We
welcome the positive opinion on tofersen (Qalsody) by the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)
and what this means for people with the SOD1 type of motor neuron disease (MND)
in Europe. The Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) has announced a positive opinion for the
approval of tofersen for the treatment of the SOD1 type of MND. The CHMP panel
reviews the scientific evidence and forms an opinion on whether new drugs should
be approved in Europe. The committee based their review on data from the phase 3
VALOR trial and its open label extension. The EMA has recommended to the
European Commission (EC) that tofersen is given marketing authorisation for
treating people with the SOD1 type of MND in the European Union. The
CHMP’s recommendation will be reviewed by the EC with a final decision
expected in the second quarter of 2024. A positive EC decision would mean that
tofersen is approved as the first treatment for the SOD1 type of MND across all
27 European Union member states, as well as Iceland, Liechtenstein, Norway, and
Northern Ireland. The three MND charities in the UK – My Name’5
Doddie Foundation, MND Association and MND Scotland - will continue to work
together to understand this outcome and what it means for people with the SOD1
type of MND in the UK, and our next steps. More about tofersen and what this
means for the UK Tofersen has been developed by Biogen for use in people with
the SOD1 type of MND.  SOD1-MND is a rare genetic subtype of MND,
accounting for 2% of total MND cases. Everyone has the SOD1 gene and it contains
the instructions for making an enzyme important for keeping the cells of our
bodies healthy.  In people with SOD1-MND, the DNA code for SOD1 contains a
mistake, meaning that their cells can’t make this enzyme properly and
instead makes a mutant version that is damaging. Tofersen stops the faulty SOD1
code from being used to make SOD1 protein, reducing the amount of toxic material
being made. In April 2023, tofersen was approved in the USA for treating people
with the SOD1 type of MND.  This news from the EMA means it may be made
available to people in Europe (depending on final authorisation by the EC). At
the moment, tofersen is not approved for use in the UK. Biogen, the company that
has created it, has provided the following update: Following Brexit, marketing
authorisations for medicines in Great Britain (England, Scotland and Wales) are
determined by the Medicines and Healthcare products Regulatory Agency (MHRA). We
are in dialogue with the MHRA, National Institute for Health and Care Excellence
(NICE) and the Scottish Medicines Consortium (SMC) on the most appropriate
access route for tofersen in the UK.



My Name’5 Doddie Foundation Business Club

09 Jan 2024

For the first time there is a realistic possibility of new treatments for motor
neuron disease (MND) emerging over the coming years. As the three main UK MND
charities – My Name’5 Doddie Foundation, The MND Association, and
MND Scotland - naturally we are all focused on getting proven treatments to
people with MND as soon as practically possible. The best and most efficient way
to do that is to work together, combining knowledge, expertise and our
voices. As we enter the new year, our priority activities are: Build a
comprehensive database of new medicines and repurposed drugs currently being
researched in clinical trials Share our database with and provide regular
updates to regulators, healthcare providers, other Government agencies and
relevant charity coalitions Continue to work with clinicians and healthcare
providers to best prepare for the expedient uptake of new and/or repurposed
medicines (eg tofersen, interleukin-2) once there is sufficient evidence of
efficacy and safety Ensure people with MND are at the heart of regulatory and
approval processes (for example health technology appraisals) when decisions are
made about the value and affordability of new medicines by building
collaborative relationships with stakeholders Collaborate with people with MND,
neurologists, Health Technology Appraisal experts and others to ensure our work
is informed and credible A successful outcome of this work will be timely and
equitable access to proven and effective treatments for people living with
MND. We are driven by this joint ambition, and by sharing knowledge, expertise
and resources we hope to accelerate our progress. We will jointly share our
activities with the MND community.



My Name’5 Doddie Foundation Business Club