June 4, 2021
Give me a lightning quick summary⚡
Here’s what you absolutely need to know for Drug Repurposing for Rare Diseases 2021:
• We’ve made more room for discussion to facilitate engagement, and made ample room for networking to help foster collaboration amongst attendees. We’ve even booked more speakers to bring you unique perspectives on drug repurposing, and took our beloved conference virtual AND international!
• Attendees will enjoy not one, but two interactive workshops, which will explore how to move a treatment along the drug repurposing pathway, going end to end to produce viable rare disease treatments
• Funding and regulatory flexibility both contributed to the speed in which drugs were repurposed to combat COVID-19. If we can move the bar for rare disease repurposing in each of these areas, then we will be able to drive more concepts to the clinic and deliver more repurposed drugs to patients.
• You know the old saying, “There’s two sides to every story?” Well, at Drug Repurposing for Rare Diseases 2021, you’ll get both sides of a rare repurposing story as told by Mandy Sellars (patient) and Rob Semple (clinician)
• If there is anyone who knows both sides of the story (patient & clinician) extremely well, it’s David Fajgenbaum, Co-founder & Executive Director of Castleman Disease Collaborative Network.
• Our hope is that rare disease patient groups, in particular, learn how they can get involved in drug repurposing projects and lead the way on crucial rare disease research and treatments by attending our keynote panel discussion on the AKU story!
After surviving an unprecedented year, the only logical thing to do is host the grandest Drug Repurposing for Rare Diseases Conference to date in celebration!
Our annual drug repurposing conference is returning for its 8th year and is bigger and better than ever before. We’ve made more room for discussion to facilitate engagement, and made ample room for networking to help foster collaboration amongst attendees. We’ve even booked more speakers to bring you unique perspectives on drug repurposing, and took our beloved conference virtual AND international!
15% of advocates registered from North America
18% registered from Non-UK Europe
58% registered from the UK
3% registered from Asia
1% registered from South America
Who said bigger isn’t better?
Why does drug repurposing matter?
Have you watched the news!? Drug repurposing has captured the world’s attention due to its role in delivering the first approved treatment for COVID-19. It was the existing drug, dexamethasone, which was repurposed to treat COVID-19 on a timescale that was unprecedented in drug development. The question begs: What can rare repurposing learn from COVID-19?
If nothing else, focus, funding and regulatory flexibility all contributed to the speed in which drugs were repurposed to combat COVID-19. If we can move the bar for rare disease repurposing in each of these areas, then we will be able to drive more concepts to the clinic and deliver more repurposed drugs to patients. Our two interactive workshops from LifeArc and Healx will offer attendees the chance to explore some of these issues in much greater detail.
Drug Repurposing for Rare Diseases 2021 is the rare community’s chance to come together to highlight the best examples of repurposing in the field of rare disease in a collaborative atmosphere that puts patients at the heart of research!
Go big or go home, right! We’re elevating our conference once again by introducing NEW interactive workshops. Attendees can join one of two interactive workshops, which will explore how to move a treatment along the drug repurposing pathway, going end to end to produce viable rare disease treatments. Just take a look at what LifeArc has in store for their workshop!
Taking your repurposing candidate into the clinical space and delivering it to patients is a huge challenge. Hear the outcomes from LifeArc and Pinsent Mason‘s repurposing roundtable event, and work with other rare disease community members to figure out how to plot your way through some of the roadblocks encountered along the drug repurposing route.
The aim of the session?
• Understand the best routes to drive drugs toward the patient
• Devise strategies to overcome legal, regulatory and access barriers
See what Healx has in store for their workshop now!
Meet Mandy, Rob and David
At Findacure, we never lose sight of the most important people in the room: patients. Patients are at the heart of Drug Repurposing for Rare Diseases 2021. It’s why there’s not only exhibitions from patient groups and industry, but also a dedicated patient group poster zone!
Meet three of our 2021 speakers who know a thing or two about being a patient and advocating for change.
You know the old saying, “There’s two sides to every story?” Well, at Drug Repurposing for Rare Diseases 2021, you’ll get both sides of a rare repurposing story as told by Mandy Sellars and Rob Semple.
Mandy is the first to be discovered with a rare overgrowth condition called PIK3CA-related overgrowth spectrum (PROS). PROS is a rare disease that causes the overgrowth of certain parts of the body, which in Mandy’s case, were her legs. Both her legs and feet are unable to stop growing, which has left her wheelchair bound with 17-stone legs. In an attempt to stop one of her legs from growing, she made the difficult decision to have it amputated. Mandy will take to our stage to share her story and experience of partaking in a clinical trial.
Usher in Rob Semple. For nearly 20 years, Rob’s major focus has been on single gene disorders of insulin/PI 3-kinase signalling. His work led to the discovery of mosaic PIK3CA activating mutations in forms of overgrowth, which put him directly in Mandy’s path. Rob realised that the drug, rapamycin, could help patients living with rare conditions like PROS if repurposed.
Rob took that drug and secured funding for an open-label, academic-led, low-cost clinical trial to test his hypothesis that it could help treat overgrowth patients. Mandy was recruited for the trial alongside other patients and was prescribed rapamycin, which successfully stopped her legs from growing. This remained the case for five years before the treatment stopped working.
Despite this, Rob continues to work in the field and is involved in the repurposing of a different compound to help treat PIK3CA. Rob will take our stage to share the medical side of this captivating story.
Best of both worlds
If there is anyone who knows both sides of the story extremely well, it’s David Fajgenbaum, Co-founder & Executive Director of Castleman Disease Collaborative Network.
David was a healthy third year medical student training to become an oncologist in memory of his mother when rare disease touched (and almost took) his life. David went from being a beast-like college Quarterback to receiving his last rites while in medical school and nearly dying four more times battling Castleman disease.
Castleman disease is a rare condition that’s described as a cross between cancer and an autoimmune disorder. When David was diagnosed, there was no treatment, cure or research being done to help patients. David wasn’t going to stand for that. He spearheaded an innovative approach to research through the Castleman Disease Collaborative Network (CDCN). Through this network, David has discovered a treatment that is saving his life and others. He is a national best-selling author of Chasing My Cure: A Doctor’s Race to Turn Hope Into Action!
Meet our other incredible 2021 speakers now!
Go on a 19-year AKU journey
To make a long story short…
That’s our keynote panel’s challenge!
The AKU nitisinone repurposing story highlights the power of cross-sector, international collaborations. Together, they took nitisinone from a failed trial to an EMA-approved treatment for an ultra-rare condition.
Our panel will walk attendees through:
• How a clinical trial protocol was designed for an ultra-rare condition where blinding was impossible
• How patient groups were able to support patient recruitment and retention over a 3-year phase III study
• How a core scientific team came together to deliver the pre-clinical research and developed new endpoints for the clinical trial
• The regulatory hurdles encountered and how planning early on in the process sped approval
• Why one AKU patient agreed to be part of the non-treatment arm of the trial knowing that he wouldn’t be treated with nitisinone. He made that decision for the wider benefit of AKU patients – a truly selfless act!
• The role that collaboration and strong leadership from the clinical team played in the delivery of the treatment to patients
Plus, many more insights and ideas that’ll be brought to the table. Our hope is that rare disease patient groups, in particular, learn how they can get involved in drug repurposing projects and lead the way on crucial rare disease research and treatments!
What the Drug Repurposing for Rare Diseases Conference means to Findacure
Findacure’s Drug Repurposing for Rare Diseases Conference has come a long way over the past eight years. It’s been an incredible journey helping to put drug repurposing on the map with our annual conference. COVID-19 skyrocketed the awareness of drug repurposing’s benefits and challenges, so we’re ready to take centre stage and harness its power whilst all eyes are on this cost-effective alternative to drug development.