What interested you in the Lawrence Goldstein Science Policy Fellowship Program?
Policy and regulations are known to have significant impacts on therapeutic development, particularly in the cell and gene therapy field, where technological innovation is a constant state of affairs. The fellowship program provides an unprecedented opportunity for scientists/clinicians working in this field to become engaged in policy advocacy at a higher level. Not only does it allow them to stay ahead of the curve, but it also prepares them to become significant players in policy and regulatory development and to benefit from the unrivalled experience of the ISSCR Public Policy Committee.
How to do you hope to benefit from the policy fellowship program?
To be a frontline advocate for regulatory reforms that advance stem cell research and protect vulnerable patient populations from poor-quality medical science by interacting and working closely with other scientists and policy makers involved in promoting these goals.
What is the current focus of your policy research, and what do you find most rewarding about your work?
My policy research is committed to addressing the dangerous implications caused by the global rise of clinics marketing unproven stem cell therapies; in this research I am also focusing on other emerging health technologies, such as genome editing. Moreover, I lead a regulatory science and research program that concentrates on identifying and solving problems affecting development of new cell and gene therapy products. It is very rewarding to potentially be able to protect hundreds, if not thousands of patients from the misuse of life science technologies.
Do you have research interests outside of the policy arena?
I am also interested in the science of evidence synthesis; I lead a research program that focuses on the development of robust mechanisms to generate, collect, report, and evaluate clinical evidence from cell and gene therapy trials. Several reasons, such as the lack of a control group, the small number of enrolled patients, complex manufacturing processes, and heterogeneous follow up times, among other reasons, often prevent innovative products from showing strong clinical evidence of efficacy in the studies they are employed in. These unique features often challenge regulatory agencies and reimbursement bodies to carry out comprehensive benefit-risk assessments for these products, once they are approaching the market.
What led you to become involved with science policy?
As a trained physician, I have always believed in putting patients first. One can achieve this purpose, not only in the clinic, but also through being actively involved in promoting responsible health and research policies. As such, I became engaged in various initiatives that made me part of cross-disciplinary networks of scientists, policy makers and entrepreneurs engaged in raising awareness and building capacity for science-based policy-making, such as the Global Young Academy (GYA), the Arab German Young Academy (AGYA) and recently the Eisenhower Fellowship Program. Particularly, becoming an Eisenhower Fellow fueled my interest in science policy by allowing me access to noted experts and resources over a 6 week visit to the United States. This visit enhanced my ability to further investigate the case of unproven stem cell interventions and to work toward establishing a consensus within and across trans-national stakeholders to take action to limit the spread of this potentially dangerous phenomenon.
What is the most exciting aspect of your work?
Besides my research programs, I am also heading the clinical development unit which is responsible for translating promising therapeutic approaches from bench to bedside. One exciting aspect of this work is to be able to work with interdisciplinary teams to solve issues related to product manufacturing or clinical trial design (among others) that could impede the progress of the project and prevent safe and effective therapies from reaching patients. It is very rewarding to see these therapeutic agents producing effective treatment responses in clinical trials and improving patients’ lives.
What guidance would you share in talking with trainees interested in pursuing science policy?
I would advise them to get involved in policy related activities as soon as possible, and not to shy away from reaching out to experts in the field, asking them for guidance. Particularly, joining societies such as the ISSCR is a wonderful opportunity to get exposed to highly professional and well-structured programs that promote rational therapeutic development, minimize harm, and protect health and well-being. I would also encourage them to involve their local communities in policy and regulatory reforms, which can be highly impactful, as they are influencing change and are achieving success.
Do you have any mentors or individuals who have inspired you in your policy work?
The list of change makers that are true catalysts for policy and regulatory changes in the field and have influenced not just me, but generations of scientists and clinicians, is impressively long. To name few, I find the work of Leigh Turner on unproven stem cell clinics is very important and timely. I am also very motivated and impressed by the proactive approach the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration, led by Peter Marks, is taking to advance the field of regulatory science and to bring more adequately tested life-saving therapies to patients. And last, but not least, my longtime friend and collaborator Gerhard Bauer, who has worked his entire career to provide therapeutic solutions to treat different debilitating diseases, has been and continues to be a source of inspiration.
How do you spend your free time?
It is rare nowadays to have free time, but when I do, I prefer catching up with all the interesting movies and TV series that have been released. I consider cinema as an impactful art form and a powerful vehicle for culture and education.
What is something your peers would be surprised to learn about you?
I consider myself a semi-professional cook. Food is a great way to learn about other traditions and places. I like to mix elements of different culinary schools into what I call ‘multi-disciplinary cuisine’.
What do you most value about your membership and involvement with the ISSCR?
One key value to members of ISSCR is their involvement in the strong and engaging role the society plays in shaping standards and guidance for responsible and ethical practice in stem cell research. The opportunity to connect and network with outstanding peers and subject matter experts also yields additional value and opens up possibilities of collaborations.