The use of stem cells to develop treatments is a promising area, but unfortunately, it typically uses animals to test the safety and efficacy of the treatment before applying it to human patients. In a recent study of Hirschsprung disease – a devastating intestinal birth defect – scientists bypassed animal experiments to improve both the speed and quality of the outcomes.

Rather than implant stem cells into animals, they used human transplanted tissue from Hirschsprung patients to confirm the treatment’s safety and effectiveness. With the knowledge acquired, they will now proceed to clinical trials.

In their publication, they describe how standard animal experiments in this area have “slowed efforts to date” and how using a human transplant approach is better because “observations achieved through this approach are directly translatable.”

Human cells and tissues provide answers that animal experiments cannot. In a field heavily dominated by animal experiments, it’s exciting to see this and other studies affirming that human tissue leads the way for deriving important and life-saving information.

Important step forward in stem cell therapy for rare bowel disease

Hirschsprung disease is a rare bowel disease affecting infants and children, characterized by the absence of intestinal nerve cells, resulting in poor motility, severe constipation and high risk of infection. Current treatment requires multiple surgeries and lifelong symptoms often persist despite the surgeries. 

Researchers at the University College London are using stem cell therapy in innovative and human relevant research. While typical stem cell research uses animals to test the therapy, the current study bypassed using animals by transplanting nerve precursors derived from stem cells into sample tissues donated from patients with Hirschsprung disease. 

Results showed that the transplanted gut samples exhibited an increased ability to contract, indicating the potential for this stem cell treatment to safely, less invasively and more effectively treat Hirschsprung patients. The research team will now pursue clinical trials to further develop the treatment.

Preserving breast tissue outside of body will aid cancer research

Researchers from the University of Manchester in the UK have developed a way to preserve breast tissue for up to a week, allowing them to test potential cancer treatments on individual breast tissue that accurately models human biology.

They were able to preserve breast tissue outside the body after testing different hydrogel formulas. The preserved tissue modeled normal structure and cell type and most importantly responded to drugs in the same way as normal breast tissue.

Researcher Hannah Harrison from U Manchester said, “This breakthrough means that researchers will be able to test new drugs in the lab with far greater accuracy, which should mean fewer drugs failing at clinical trials and ultimately better results for women affected by this terrible disease. It’s a hugely exciting development in animal-free research which puts us in a really strong place to find new drugs to prevent breast cancer. This is a real game-changer for breast cancer research in many ways.”

Crohn's discovery could lead to better treatments for devastating condition

Crohn’s disease is a debilitating inflammation of the digestive tract that can affect children as well as adults. In children particularly, relapses are common, despite anti-inflammatory treatments that reduce some symptoms.

To learn more about why children with Crohn’s relapse, scientists at the University of Virginia Health System conducted an entirely human-based study rather than conduct outdated and inapplicable animal experiments.

They compared intestinal biopsy samples from children with Crohn’s who had gone into remission with those of a healthy control group. Their analysis of these two intestinal sample groups revealed significant changes in immune cells linked to inflammation, as well as an altered bacterial composition (microbiome) in the Crohn’s group. 

Based on these findings, they theorize that therapies to normalize the gut microbiome in children with Crohn’s may help prevent recurrences and perhaps even cure the disease. Future research will explore this possibility.

Study compares RNA editing between postmortem and living human brain

Studying RNA editing in human cells and tissues provides vital information on normal function and disease because RNA carries the instructions for DNA’s genetic blueprint. Modern scientists are studying neurological diseases by examining RNA editing in post-mortem human brain tissue, but this method has limitations because a lack of oxygen impairs the post-mortem tissue’s cellular and genetic activity. 

To circumvent this problem, researchers at the Icahn School of Medicine at Mount Sinai Hospital obtained  brain tissue samples donated from dorsolateral prefrontal cortex of patients undergoing deep brain stimulation for neurological illness. 

RNA editing in these living brain tissues was examined and compared with postmortem samples, and matched for demographic and clinical variables, allowing scientists to identify significant differences in RNA editing activity. 

Studying both post-mortem and living human brain tissue will allow scientists to improve knowledge of intricate brain functions at the cellular level. This information will advance progress in studying neurological disorders like Parkinsons and other brain conditions by conferring knowledge unobtainable from cruel brain experiments on animals.