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Philanthropic support is critical to driving forward our health research. Philanthropy enhances and often leverages other traditional funding streams, enabling us to accelerate the pace at which lifesaving innovations, invaluable insights, and new treatments for patients are discovered, developed, and deployed at UCL.
Professor Mark Emberton OBE, Dean of UCL Faculty of Medical Sciences, says: “Philanthropy provides the space for the creation and refinement of new ideas, the time to test theories effectively and allows researchers to explore the limits of what we know to be possible.”
This support often has a catalytic effect, allowing investigators to achieve proof of concept and the knowledge base needed to secure traditional research funding. In the fast-moving field of health research, our teams need to be able to capitalise on new opportunities as they emerge: it is philanthropic support that powers this.
T-ALL immunotherapies
Before his death, Alviar had crowdfunded to pay for treatment which was only available in Singapore and a portion of the funds raised remained unspent. In 2022, Alviar’s family decided to donate the remaining funds to UCL to support the T-ALL research programme led by Professor Marc Mansour in the UCL Cancer Institute.
Professor Mansour’s team is trying to understand the ‘nuts and bolts’ of T-ALL biology – a subtype of ALL which has proved more challenging to treat than other types of the disease for which there now exist NHS-licenced immunotherapies. The group’s priorities include furthering our understanding of the genetic causes of T-ALL; uncovering the structure of the gene responsible for 80% of T-ALL cases; understanding how T-ALL cells become resistant to chemotherapy; developing immunotherapies for T-ALL; and identifying genetic targets that could lead to new treatments.
Thanks to the unrestricted gift from the Cohen family, Professor Mansour is able to empower his team to respond to unexpected opportunities as and when they arise.
- UCL News: New immunotherapy target offers hope for treatment of T-cell leukaemia (August 2022)
Establishing a Charcot Marie Tooth disease patient registry
The gift was used to pilot the first national patient registry for Charcot Marie Tooth disease. The initial funding and pilot have enabled Professor Reilly to leverage further funding to develop the registry.
Charcot Marie Tooth disease (CMT) is part of a heterogeneous group of hereditary neuropathies which collectively are the most common inherited neuromuscular conditions. There are currently no therapies available to treat CMT. However, there have been major advances in our understanding of the genes which cause CMT leading to a range of potential therapies.
A comprehensive registry will enable Professor Reilly and her team to identify patients who are suitable for clinical trials. This involves knowing who and where the patients are with each genetic subtype and having a registry with enough detail to be able to gauge how many suitable patients there are available for each clinical trial. It also provides a portal for patients to enter their own data as well as clinicians entering data.
Using patient registries to define the natural history of other progressive diseases such as Spinal Muscle Atrophy and Duchenne Muscular Dystrophy has been crucial in the introduction of gene therapies and the assessment of efficacy, safety and toxicity of therapies; and the new Charcot Marie Tooth registry has the potential to prove equally impactful for this patient group.
Images:
- T-ALL immunotherapies: NCI visuals Killer T cells surround cancer cell
Social prescribing research project to address children and young people’s mental health
Social prescribing focuses on connecting people to non-medical forms of support within the community including skills development and training programmes; peer support and befriending schemes; and social, cultural and community activities to empower individuals and address social determinants of ill-health.
The INSPYRE project, led by Dr Daisy Fancourt, will trial offering immediate social prescribing treatments to children and young people referred to services for moderate mental health conditions, such as anxiety, depression or PTSD. Currently, many children and young people face long waits for mental health services, during which time their mental health often deteriorates. The INSPYRE social prescribing pathway will be offered as children and young people wait for their treatments. The project will run at a number of sites in England and Wales.