Shared Leadership Delivers Impact
For my last Message from the President, I had the opportunity to connect with the Co-Chairs of the ISSCR’s Best Practices for the Development of Pluripotent Stem Cell-Derived Therapies Jacqueline Barry and Kapil Bharti and the Co-Chairs of the Embryo Models Working Group Amander Clark and Janet Rossant to discuss these strategic initiatives. The Society often assembles members from across the world with specific expertise for short-term projects. These two groups are among them and have produced exceptional work on behalf of the field.
Q: Describe the work of the Best Practices Task Force.
KB: We set out to develop a resource that reinforces how to conduct rigorous research and how to take research and translate into clinical practice and safe products. We got together to figure out how we could put together a best practice document to go through this pipeline, mainly focusing on pluripotent stem cells. We wanted it to be jurisdictionally neutral so that it is applicable across the globe.
Q: ISSCR started with basic research standards and now developed this best practices roadmap. Can you comment on the thoughts and energy on how this change over time to produce such a comprehensive resource?
KB: When we met as a steering committee two years ago, I didn’t think we would get to this level of detail in the instruction. When we went through what we needed using a whiteboard, we knew it would nearly require becoming world global expert in each region. Where we ended up is such an amazing document that takes you from a higher-level view to click to get deeper to learn more and more. You won’t find such a comprehensive document anywhere else. There may be guidelines in various sectors, but until now the information has remained fragmented.
JB: I remember we figured out we need a roadmap – a navigation tool. We know that regulatory review during the process of therapy development can be daunting and a massive undertaking. So, the interactive concept we came up with offers a wealth of information from people with different expertise and allows users to be able to access exactly what they need during their development journey.
Q: Can you speak about the composition of the members of the task force?
JB: It was very important to incorporate as many jurisdictions as we could in this document. We needed the task force members to have deep knowledge in their specialty areas such as clinical trials, and a willingness to share that knowledge. I think we found amazing people within our membership who are committed to working with one another. People who didn’t know each other at the start of this project formed a closeness, and we are all now bound together by this common goal. This bond led to more than the output of this document – we created a group of people who will be able to continue this international network beyond this specific project and who are committed to this mission.
KB: What we saw was colleagues coming from 14 different countries who believed in shared leadership and who put it to work for impactful outcomes.
Q: What is your hope for the resource now that the work is nearly completed and soon to be launched?
JB: The ultimate outcome is to see an acceleration of stem cell-derived trials and ultimately licensure. How do we get there? I can see industry scientists using it as part of a pitch for investments, then I can see scientists in the lab who look to go to industry in order to build their knowledge, and I can envision people who are working on clinical trials using it internally. So really three groups – nascent lab scientists entering industry, reinforcing the internal knowledge in a biotech company, and a resource needed for investors.
KP: When I got funding approved for my work, I went to the Food and Drug Administration (FDA) website to download documents as guidance. After a few pages, I couldn’t understand anything, and I would feel frustrated for days. Colleagues recommended I to talk to various consultants. Through this project and the resource we developed, I hope to keep this from happening to anyone else so that access to critical information is much more streamlined. What we did reduces mistakes, reduces cost, and increases the pipeline of more therapies going to patients.
Q: What drives you to invest in/give so much of your time and talent to the ISSCR and the work of the committee? Describe the value you see in this volunteer work?
KB: I’ll start sharing my story. I had an IND approved for a trial that was shut down when everything was shut down during COVID and many people started reaching out to me by email and phone calls asking questions and I realized that many of the conversations were redundant, and we could learn from each other. I ended up starting a consortium where people could connect and discuss problems and solutions every two months sharing openly in a precompetitive space. We realized the cell therapy field was growing so quickly, and I felt that ISSCR, in the way that it engages with the community both on the research side and the patient side, had the right platform to disseminate this knowledge and information to the larger audience. It just gets me excited as we hope that by sharing our experience, we can stop reinventing the wheel and make the whole roadmap available to enable more cell therapies and help more patients.
JB: My first thought about this question is, well why wouldn’t you?! As Kapil says, we’ve got this knowledge, and we want to move these products into patients. If you don’t share the knowledge, it’s going to take so much longer for us to get to where we all want to get to. I think with collaboration and working together, we achieve so much more than just working on a project. You really feel you are bringing together the knowledge based that will help the community. And I think ISSCR is such a wonderful organization and being part of that feels quite special. We’ve got this knowledge, why wouldn’t you share it?
I also had the opportunity to learn about the work of the ISSCR Embryo Models Working Group led by Amander Clark and Janet Rossant. The working group was tasked with developing a white paper that summarizes the state of the science related to rapid advances in stem cell-based embryo models (SCBEM), categorizes different types of SCBEMs and proposes updates to the 2021 ISSCR Guidelines for Stem Cell Research and Clinical Translation. This important work is led by a group of leaders with specific expertise from across the world.
Q: Describe the focus of the Embryo Models Working Group?
AC: Our working group is developing updated recommendations for researchers working with stem cell-based embryo models. The goal of these updates is to bring more clarity to the field, help guide the deliberations of researchers, oversight committees and other relevant stakeholders, and ensure continued public confidence.
Q: Can you share more about the talent and background of members of the working group and how the composition of members contributes to the work?
AC: Our working group is brimming with talent and diversity. This includes geographic diversity and a diversity of expertise. Members of our working group live and work in Australia, Canada, China, Europe, UK, and the USA. Our expertise includes science, medicine, law, bioethicists, and social sciences.
Q: Can you describe how you hope the community will use or respond to the working group’s recommendations?
AC: This working group met regularly for more than a year to craft updated consensus recommendations for research with stem cell based-embryo models. This has been a deliberative process including engagement from other ISSCR committees such as the ISSCR Public Policy and Ethics Committees as well as expert peer-review from members and non-members. This process involved active listening and exploration of diverse viewpoints by not just the working group but also the different people and committees who the working group engaged with. This work was only possible through the willingness of committee members to challenge each other while at the same time being respectful of alternate points of view. Given the careful approach taken by the group of experts in the group, we hope that the new recommendations will be well received by the community of researcher’s and stakeholders. It was an honor to co-chair the committee with Janet Rossant who is an expert consensus builder and valued developmental biologist.
Q: What drives you to invest in/give so much of your time and talent to the ISSCR and the work of the committee? Describe the value you see in this volunteer work?
AC: In value laden areas of science such as stem cell-based embryo models, it is important that regulations are crafted so that scientists are supported by a framework that facilitates impactful research. It is equally important that the public is also supported from the perspective that these unique areas of science have rationalized and thoughtful limits.
It has been my privilege throughout this last year to learn more about the work of our members and volunteer leaders who selflessly give their time and talent to the ISSCR as we address the largest opportunities and challenges in our field. I am full of gratitude for the opportunity highlight t e accomplishments of our leaders and share them with our community.
It has been my privilege throughout this last year to learn more about the work of our members and volunteer leaders who selflessly give their time and talent to the ISSCR as we address the largest opportunities and challenges in our field. I am full of gratitude for the opportunity highlight t e accomplishments of our leaders and share them with our community.
Best Practices Steering Committee: Kapil Bharti, Co-chair, Jacqueline Barry, Co-chair, Ricardo Baptista, Melissa Carpenter, Derek Hei, Ana Hidalgo-Simon, Deborah Hursh, Jung-Hyun Kim, Tenneille Ludwig, Hideyuki Okano, John Rasko, Yoji Sato, Glyn Stacey, Clive Svendsen. Starting Materials Working Group: Co-chair, Kapil Bharti, Co-chair, Tenneille Ludwig, Jacqueline Barry, Catharina Brandsten, Elizabeth Csaszar, Indumathi Mariappan, Mark Tomishima, Alex Zhang. Banking Working Group: Co-chair, Jung-Hyun Kim, Co-chair, Glyn Stacey, Elsa Abranches, Jie Hao, Hyunyoung Kim, Joanne Mountford. Ancillary/Raw Materials Working Group: Co-chair, Ricardo Baptista, Co-chair. Uma Lakshmipathy, Elsa Abranches, Kevin Bruce, Tamar Harel-Adar, Annelie Persson, Anat Shnaiderman, Shuyan Wang, Claudia Zylberberg. Regulatory Working Group: Co-chair, Jacqueline Barry, Co-Chair, Deborah Hursh, Michela Gabaldo, Kilian Kelly, John Rasko, Ana Hidalgo-Simon, Yoji Sato, Sowmya Viswanathan. Preclinical Working Group: Co-chair, Jeanne Loring, Co-chair, Yoji Sato, Joy A. Cavagnaro, Lila Collins, Shawna Jackman, Cristelle Monville, Andras Nagy, Michaela Sharpe, Clive Svendsen. Clinical Working Group: Co-chair, Claire Henchcliffe, Co-chair, John Rasko, Roger Barker, Stanley Lazic, Graziella Pellegrini, Alan Trounson, Sowmya Viswanathan, Kirsty Wydenbach. Drug Substance/Drug Product Working Group: Co-chair, Derek Hei, Co-chair, Jennifer Hollands, Ricardo Baptista, Melissa Carpenter, Jennifer Dashnau, Kate Fynes, Mashiro Kino-oka, Dhruv Sareen. Consultants: Roger Barker and Michaela Sharpe. ISSCR Staff Liaisons: Jack Mosher, Chris Barry, Denise DeVilla, Tyler Lamb, Kendra Prutton.
Embryo Models Working Group: Amander Clark, Co-chair, Janet Rossant, Co-chair, Peter Rugg-Gunn, Heidi Cook Andersen, Jan Zylicz, Sarah Franklin, Deb Mathews, Hongmei Wang, Xiaomei Zhai, Rosario Isasi, Vincent Pasque, Patrick Tam. ISSCR Staff liaison: Tyler Lamb.
Acknowledgements: I am grateful to Kym Kilbourne for her continuous contributions and partnership in co-creating these monthly messages.