Cancer immunotherapy is so hot right now. Signals bloggers have covered CAR T cells (here and here), but haven’t given some other exciting immunotherapies their time to shine. I aim to correct that. A normal part of the immune system, checkpoints are a mechanism to prevent the immune response from running rampant and destroying host cells. Some cancers have developed ways to evade the immune system by promoting checkpoint signalling. Hence, the development of checkpoint…

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CRISPR/Cas9 from the NIH image library, found on Flickr Over the past four weeks, I’ve discussed numerous aspects of gene therapy in an attempt to convey my excitement for this field moving forward. The convergence of numerous advances in basic biology (as described here and here) and clinical trials (as explained here and here) has placed gene therapy back in the driver’s seat as one of the most promising technologies in the cellular therapy and…

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source: https://www.mdpi.com/1422-0067/17/1/75/htm The second post in this series on gene therapy and blood stem cells might give readers a small sense of the absolutely enormous research investment (time, money, people, etc.) required to find a way to expand blood stem cells for therapeutic purposes. Ever since the first bone marrow transplantations in the late 1950s, where the regenerative potency of blood stem cells was powerfully realized, researchers have been keen to develop methods to create…

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Image courtesy of Wikimedia As mentioned in the introductory post to this series on blood stem cell gene therapy, several significant issues existed with the first set of clinical trials in the gene therapy world. It became abundantly clear that simply delivering the gene target on its own would not be sufficient for curing disease. Rather, these early trials inspired a great need to investigate the human body’s response to the gene delivery by viruses,…

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An illustration of red blood cells from Wikimedia Blood stem cells are the fundamental component of regenerative medicine applications involving the blood and immune system. Despite significant efforts and investment, the research community has largely failed to maintain fully functional blood stem cells for substantial periods of time outside the body. As a result, the limiting factor for cell and gene therapies is often obtaining a sufficient number of blood stem cells to seed the…

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The novel DNA-binding molecule combines multiple tools into a single synthetic platform, like a chemical Swiss knife. Illustration by Mindy Takamiya (CC BY 4.0) Over the coming weeks, I will be writing a series of blog entries on gene therapy using blood stem cells as a base cellular product. In the last few years we have witnessed an incredible return to the headlines for gene therapy now that safer, more accurate tools have been developed and…

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Starting work on a new research project can be overwhelming. This is especially true in the stem cell field, with its complexities and rapid evolution. It is quite easy to get lost while conducting literature searches or looking for specific protocols, making it feel difficult to get started. This Alice in Wonderland quote comes to mind: “It takes all the running you can do, to keep in the same place. If you want to get…

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Síofradh McMahon is the Senior Manager, Clinical Translation and Regulatory Affairs at CCRM. Siofradh works with academic and industry partners to advance promising products and technologies to commercialization. Having worked worldwide, Síofradh brings more than a decade of experience in pharmaceutical and biotech regulatory affairs and policy. She is an active member of the Alliance for Regenerative Medicine’s Regulatory and Value & Access working groups, and lends her expertise to groups in Canada too. Photo credit Alexey Hulsov, Pixaby…

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On most days, scientific discoveries are not cover stories or even top headlines. That hasn’t been the case since COVID-19 hit our collective consciousness. We are being inundated with science news, which would be great under differ circumstances. But communicating new science during a crisis appears to have its own challenges. The scientific community has banded together incredibly well to discover everything it can about COVID-19. Researchers are working tirelessly to get information out and…

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MERS-Coronavirus particle, National Institute of Allergy and Infectious Diseases Earlier this month, Chinese media reported that doctors in Yunnan cured a patient from coronavirus with a stem cell transplant. But did they really and do stem cells actually hold promise against the new virus wreaking havoc across the world? The global pandemic has sent scientists racing to find a cure. There are more than one hundred clinical trials testing potential treatments and vaccines, most of…

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