By Heather Main
I recently attended the UCSD Health CIRM Alpha Stem Cell Clinics Network Symposium at the Sanford Consortium, San Diego.
There was of course some great academic research presented, including conflicting views on liver stem cell compartments from Roel Nusse and David Brenner, and advances in CRISPR technologies from Matthew Porteus. However, what struck me about this meeting was how well it presented patient need and churned emotions outside the normal excitement or skepticism of an academic/basic science conference.
Shirley Johnson is a name I have never heard before and while my mother is in fact a nurse, listening to Shirley made me realise how I don’t even know what my mum does when she goes to work let alone what a nurse trained in clinical trials and/or cellular therapies would do. Shirley presented how the City of Hope hospital has incorporated its alpha clinic into its day surgery, ‘in the trenches’ with the nurses instead of ‘in the ivory tower’ with the clinical research unit. How alpha clinics are dependent on the knowledge, skills, abilities and behaviours of nurses, who drive the patient experience and form the link between the doctors/researchers and patients. I left this talk with great respect for these alpha clinic nurses and a desire to call my mum.
Another great talk was from Steve Cramer, a dynamic, witty fellow describing his everyday experiences and frustrations in providing treatments to his stroke patients. He outlined the importance of rigourous pre-clinical data (Ann Neurol. 2006 Mar;59(3):467-77.1,026 experimental treatments in acute stroke. O’Collins VE1, Macleod MR, Donnan GA, Horky LL, van der Worp BH, Howells DW.) and non-linear thinking that should allow promising treatments in the absence of a mechanism of action (Neurology. 2014 Apr 8; 82(14): 1277–1286. Meta-analysis of preclinical studies of mesenchymal stromal cells for ischemic stroke Quynh Vu, BS,* Kate Xie, BA,* Mark Eckert, PhD, Weian Zhao, PhD, and Steven C. Cramer, MD). Steve was both comical and desperately serious, describing how his patients do not live in boxes with controlled environments and diets, have diverse genetic backgrounds, have non-stroke related health issues, bills to pay, immigration issues, 1/3 suffer from depression, that we will not know how treatments fare in humans until we try it in them. People like Steve are great to remind us that humans are not lab animals, that invasive procedures incur risk, that patients die from infections due to immunosuppression, that clinical trials need to be well organised and stratified to ensure benefits are not diluted in human variance.
The final talk of the day was from Adrienne Bell-Cors, founder of Axis Advocacy and the 4th mother in her family to have a child affected with sickle cell anaemia. Adrienne’s talk drove home the importance of advocates for not only supporting patients and their families but in dispersing information about science and driving research and translation through raising funds and assisting in development and approval of clinical trials towards market delivery. Cheap science is bad science she says.
As Sickle Cell Anemia is particularly prevalent in the African American community it was disturbing to be reminded of the troubles with access to good healthcare that this community has faced in recent history, that Adrienne was the first woman in her family to undergo natural menopause rather than forced sterilisation or ‘recommended’ hysterectomy before 25 years of age. The women before her were either force sterilised or ‘recommended’ by doctors to have hysterectomies before the age of 25. The history of treatment of this community makes it difficult for the patients and their families to trust medical professionals, explaining why they are not lining up to participate in clinical trials. The role of patient advocates in being cultural advisors to researchers is something that never occurred to me with such intensity.
This meeting was a demonstration of the great job CIRM is doing to recognise the importance of the patient in research translation. The advances already in CIRM funded clinical trials through alpha clinic trials is remarkable and sheds light on the future of our field.