On 1 September, the ISSCR submitted comments to the Food and Drug Administration’s draft guidance for Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing. ISSCR supports FDA’s desire to share the Agency’s current thinking on the type of prior knowledge (i.e., public and platform knowledge) that may be scientifically appropriate to leverage to advance product development.

I am excited to have taken on the role of ISSCR President for this year, building on Hideyuki Okano’s leadership and vision.

Many of you may think of ISSCR mainly as the place we gather for the annual and regional meetings. But it is an organization doing far more. In addition to Keith Alm and his staff, ISSCR has over 150 volunteers who contribute to setting the ethical and scientific standards that guide our field, educating physicians and patients, advocating for our science with policymakers, and nurturing the next generation of researchers.

Among the many ongoing initiatives of ISSCR, I see three areas that are particularly urgent for this coming year.

Internationally recognized stem cell scientist and regenerative medicine pioneer to lead the Society during its 25th anniversary year

The International Society for Stem Cell Research (ISSCR) today announces that Lorenz Studer, M.D., founding director of the Center for Stem Cell Biology at Memorial Sloan Kettering Cancer Center, USA, has started his term as President of the Society.

Dr. Studer is known globally for his pioneering advances in stem cell biology, regenerative medicine, and the development of stem cell-based therapies for neurodegenerative disease. His research has established widely adopted methods for generating specialized neural cell types from human pluripotent stem cells, providing foundational tools for disease modeling, drug discovery, and regenerative medicine. Dr. Studer’s laboratory has also led efforts to translate these discoveries toward clinical applications, including stem cell-derived therapies for Parkinson's disease.

Knee osteoarthritis (KOA) affects more than 1 in 10 people over 40 years of age, impairing patients’ mobility and quality of life. With limited treatment options, stem cell injections have been promoted as a treatment option, but evidence of efficacy from clinical trials or claims from clinics selling untested options often lacking. Even where well-designed clinical trials were performed, there was no clear consensus about the benefit of stem cell injections for KOA, likely due to the many variables at play.  

 A major source of variability comes from the cells used for injections. Mesenchymal stromal cells (MSCs), which can be isolated from e.g. fat or bone marrow, are frequently used to treat KOA in clinical studies. Using a patient's own cells reduces the risk of an immune response and any inadvertent transmission of pathogens but the quality and the therapeutic potential of MSCs varies between patients, contributing to heterogeneous treatment outcomes. Predicting MSC potency could be a powerful tool to achieve more consistent therapeutic in such therapeutic settings. 

To tackle the issue of MSC treatment heterogeneity, Sowmya Viswanathan from the Schroeder Arthritis Institute, University Health Institute, Canada, along with colleagues performed a long-term follow-up to a Health Canada-authorized phase I/IIa clinical trial (NCT02351011) with KOA patients receiving MSC injections, whereby the MSCs were derived from the patients’ own bone marrow. The work was published today in Stem Cell Reports.

Cancer immunotherapy using T cells has proven to be effective against certain blood and skin cancers but is less effective in tumors like colorectal cancer, which is one of the most frequent cancers worldwide. One primary consideration with T cell immunotherapy is that it needs to be tailored to each patient individually through time- and cost-intensive, procedures. The procedures involve isolation of a patient’s own T cells, their expansion and genetic engineering to target the cells towards the patient’s cancer cells. This complex process that limits the widespread availability of such treatments. Consequently, an “off-the-shelf” T cell immunotherapy – one that can be universally used safely in any person – that is safe and effective in a wide range of cancer patients, including patients with solid cancers, is highly desirable.

First episode features Drs. Yifei Miao and Anca Pașca discussing advances in tissue engineering and brain organoid research

The International Society for Stem Cell Research (ISSCR) and The Stem Cell Podcast have launched a new Early Career Research Series designed to showcase the next generation of scientists advancing stem cell research and regenerative medicine.

Created in partnership with the ISSCR Early Career Advisory Committee, the series provides a platform for outstanding early career investigators to share their latest discoveries, discuss the questions driving their research, and highlight the innovative approaches shaping the future of the field. By amplifying emerging scientific voices, the collaboration aims to increase visibility for rising investigators while making cutting-edge stem cell research accessible to the global scientific community.