Kidney Research UK’s support for rare renal – past and future

1. Kidney Research UK’s support for rare renal – past and future Pete Storey – programme lead for health equalities and rare diseases peterstorey@kidneyresearchuk.org

2. Charity partner support 2011 BKPA (now Kidney Care UK) and Kidney Research UK funding to establish RaDaR 2015 Kidney Research UK follow-on funding 2016 PKD Charity registry support KR awards to keep the project on the NIHR Portfolio KR new funding for the re-consenting project >£550,000

5. Current projects

6. Summary of research using RaDaR

8. Some examples of impact

9. A breakthrough in understanding aHUS Eculizumab can prevent kidney failure and allow people with the condition to have successful transplants. >£300,000

10. ADPKD –£700,000 invested in Sheffield team in recent years Early work in 1980s: Dr Stephen Reeders, Oxford - £25,000 PDE4 Cyclic AMP

11. Nephronophthisis - Dr Shalabh Srivastava £227,000 ‘Exon skipping’ CEP290

12. Nephrotic Syndrome, FSGS and TRPC6 - Paving new ways to treat the condition Sticky podocytes! Dr Louise Farmer £200,000 grant Moin with Anne-Maria – her family supported the work

13. TRANSFORMING KIDNEY HEALTH THROUGH RESEARCH AND INNOVATIONKidney Research UK Draft Strategy 2020- 2030 - extract Vision & Impact We will free lives from the restrictions, fear, anxiety and life limiting nature of kidney disease Purpose Prevent kidney disease Stop, slow, reverse and repair kidney damage Transform treatments that replace, restore or regenerate kidney function • The difference this will make to people affected by or at risk of kidney disease: • Earlier detection and earlier treatment to maintain kidney health • The ned for dialysis or transplantation will be prevented or delayed • The impact of dialysis upon quality of life will be reduced • Improvements in diagnosis and monitoring • The likelihood of a transplant failing and the need to go back onto dialysis will be reduced • Kidney care will be more equitable • During the next decade there will be: • New biomarkers and technical markers to improve patient diagnosis or care • New treatments for kidney fibrosis and inflammation • Advances that double the life of a kidney transplant • A new technological advance to dialysis • A new alternative to biopsy • A reduction in renal health inequalities • We will…. • Improve understanding of kidney function & disease • Maintain kidney health & reduce risk • Improve diagnosis & detection • Develop interventions to slow progression • Make improvements to treatments • Improve healthcare delivery Goals Hereditary (rare) kidney diseases Areas of focus • Specific causes of kidney disease • Consequences & complications of kidney disease • Acute kidney Injury • Hereditary kidney diseases • Inflammation & fibrosis • Treatments for end stage kidney disease • Pregnancy • Health inequalities

14. Our support can take several forms Funding through curiosity-led grants rounds: innovation, projects, fellowships Collaboration – working with funding partners Funding and facilitation to help research proposals develop e.g. RDG meetings Supporting patient groups to meet, share information and discuss research

15. We want to hear from you!