For decades, scientists believed that the adult brain was incapable of meaningful regeneration. Unlike skin or muscle, the central nervous system (CNS) appeared to lack the ability to repair itself after injury, and lost neurons were considered irreplaceable. However, cutting-edge research now demonstrates that this is no longer the case. The brain possesses dormant repair mechanisms—pathways that were active during development but have been shut down in adulthood. By reactivating these pathways, it may be possible to induce neurogenesis (the birth of new neurons), axonal repair, and synaptogenesis (the formation of new neuronal connections) after devastating injuries like stroke, traumatic brain injury, and spinal cord damage.
A growing body of evidence from both the spinal cord and the central nervous system (the CNS) shows that regeneration can be stimulated by downregulating the repressing factors that prevent neuron growth and repair. For clarity, “downregulation” means reducing the activity or number of something. In the case of a repressing factor, downregulation means lowering its levels or making it less active. A repressor is a protein that blocks a process—often by preventing a gene from being turned on. When the repressor is downregulated, it no longer effectively blocks that process, allowing the underlying function to be released or activated.
These advances open the door to new treatments that could restore function to patients with neurological injuries, potentially reversing what were once thought to be permanent disabilities.
Neurodevelopmental genes and pathways that promote neuronal growth and plasticity are switched off after early development. However, researchers have identified key molecular regulators that act as “brakes” on brain regeneration. The “brakes” act as repressors of brain regeneration. By inhibiting these repressors, the brain’s intrinsic ability to regrow neurons and axons can be reactivated.
Recent breakthroughs include:
Together, these findings confirm that neural repair is possible when the appropriate repressive factors are removed, unlocking the brain’s natural regenerative capacity.
While these discoveries are promising, translating them into effective therapies requires precise, scalable, and high-throughput screening technologies that can screen out or discover biomolecules that address a specific biomedical target by the thousands with great rapidity. One such technology has been developed by Quiver Biosciences using an all-optical electrophysiology platform. (Note: the author of this article is a co-founder of Quiver Biosciences)
Quiver has developed a human induced pluripotent stem cell (hIPSC)-derived neuronal platform that allows researchers to measure functional activity in human neurons at an unprecedented scale. This platform is uniquely capable of:
The potential of this platform is vast. Scientists can now systematically search for drugs that mimic the effects of PTBP1 suppression, Nogo-A blockade, or mGluR5modulation—treatments that could one day be used to regrow neurons, reconnect severed axons, and restore lost synapses in patients with brain injuries.
Key advantages of Quiver’s platform include:
By integrating AI and machine learning, Quiver’s platform also enables pattern recognition of successful drug candidates, identifying compounds with optimal efficacy and minimal side effects.
Scientific breakthroughs alone are not enough to bring regenerative therapies to patients. Translational research—the process of developing basic discoveries into real-world treatments—is slow, expensive, and underfunded. This is where philanthropic support can make an immediate impact.
Many of the pioneering studies on brain regeneration were initially considered too risky or unconventional for traditional funding sources. Yet, thanks to early philanthropic investments, these ideas have now been validated and are shaping the next generation of treatments. Today, funding is urgently needed to:
For the first time, we have the tools to reactivate the brain’s own repair mechanisms, offering hope for individuals with severe neurological injuries. The path forward is clear: by investing in innovative research, scaling up discovery efforts, and supporting translational studies, we can bring brain-regenerating therapies to patients faster.
The DLF raises funds and uses them to inspire, catalyze, and accelerate work towards brain regeneration. We increase public awareness of possibilities in brain regeneration through our social media, news “blasts”, and quarterly newsletter. And, we promote neuroscientific advances through consultation, networking, conferences, and seed grants. This is especially important when federal funding is limited. By supporting this work, we can help transform groundbreaking scientific insights into real treatments that restore lost function and improve lives.
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