Last night, a talking point to discredit scientific spending in the United States was misused creating additional confusion surrounding research, and specifically research using mice to advance important therapeutics and understanding of diseases and conditions.
HIE is no stranger to mouse models to advance research. The Vannucci model, funded by the NIH, sas been the “gold standard” for decades. That, combined with other preclinical animal models for HIE like the rat, zebrafish, fruitfly, sheep, pig, rabbit and primate models, all contribute in different ways to understanding the best ways to decrease the incidence and impact of neonatal and pediatric-acquired hypoxic ischemic encephalopathy.
To set the record straight, a misspeak by the United States president referencing transgenic mouse research and misstating it to be transgender research, is the crux of the issue. Transgenic mouse models are similar to the Vannucci model, but are able to study genetic factors for the host of genetic disorders that are also in great need of research advancement.
So, what is this great history of using the Vannucci mouse model for HIE, and what’s the importance?
Two words: safety and efficacy.
All of these clinical trials go through regulatory agencies around the world. In the US, it’s the FDA. In Europe, it’s the EMA. In Canada, it’s Health Canada. In the UK, it’s the Medicines and Healthcare products Regulatory Agency.
Regulation is paramount to ensure safety is at the forefront. Rigorous standards and comprehensive data are required and reviewed by each regulatory agency to determine how safe and effective a potential trial is. They also monitor for adverse events.
Before you get to human trials, there is a lot of basic science through lab research that is done to generate safety and efficacy data — learning what works and doesn’t, and what has the most promise to move to human trials.
The preclinical lab, meaning before something is tested in human beings, exists to ensure we understand the most we can about a potential therapy or medicine. Over the years, many things have and have not translated from animal model research to humans. One of the successes for HIE has been therapeutic hypothermia, or cooling, either via head cooling cap or whole-body cooling methods.
But, cooling has limitations.
Over the last 10 years, cooling has be fit tested — longer, colder, shorter, different gestational ages, and other methods. Recently, a large NIH-funded multi-center study evaluating if it was safe to cool premature babies with HIE between 33-35 weeks showed no improvement in neurodevelopmental outcomes, and an 87% increase in mortality compared to the group that was not cooled. So, in this case, cooling actually was harmful.
Some other medicines have moved through to human clinical trials, and most have not translated to humans to be effective, so regulatory agencies are now stricter on the process and data, most requiring both small animal model and large animal model data. This requires additional time and funding, but is critically important to know what is best to move to human trials.
Currently, there are several studies underway, and more planned. Hope for HIE is collaborating on several studies to ensure the patient-family perspective is included early and often through the whole development process. Our award-winning Halo of Support model also ensures families are connected to support right from enrollment through the end of the study.
Government-funded research in preclinical labs is critically important for HIE to continue to advance. Misinformation helps no one, and the potential harm on our community is significant. We have a lot to advocate for to protect if we want research to move forward.
Research is a symbiotic relationship between researchers, clinicians, regulatory, biosciences, patients and families: no one can do this research and advance HIE without the entities listed.
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