Overview of Stem Cell Therapy
For 25 years, researchers have made ambitious claims about the transformative power of stem cells in medicine. These extraordinary cells, first extracted from human embryos in the late 1990s, have a remarkable capability: they could theoretically turn into any type of tissue in the human body. The concept was enticing—envision having spare parts for any bodily failure. Nevertheless, the transition from laboratory breakthroughs to real-world applications has proven to be significantly more difficult than expected.
The domain faced years of exaggeration, letdowns, and even exploitation by unethical clinics. However, after decades of meticulous research, stem cell therapy is finally starting to fulfill its groundbreaking promise. Two innovative treatments for epilepsy and type 1 diabetes are yielding extraordinary results that are positively impacting patients’ lives.
Mechanism of Stem Cell Therapy
Stem cell therapy leverages the innate potential of these adaptable cells to evolve into specialized tissues. Researchers utilize either embryonic stem cells or iPSCs (adult cells reprogrammed to act like embryonic cells). In meticulously controlled lab environments, they direct these stem cells to transform into the specific cell types required for treatment.
In the case of epilepsy therapy, stem cells develop into specialized neurons that generate GABA, a neurotransmitter that aids in calming excessive brain activity. For diabetes therapy, stem cells are transformed into beta islet cells—the insulin-producing cells typically found in the pancreas but that are destroyed in type 1 diabetes patients.
Once generated, these laboratory-grown cells are implanted into patients. The transplanted cells then merge with existing tissues and commence their designated functions. The aim is to achieve a “functional cure” where the patient’s body can self-regulate independently of continuous medical intervention.
Breakthrough Epilepsy Treatment: The Journey of Justin Graves
Justin Graves was running a scuba diving shop in Louisville, Kentucky, when epilepsy abruptly turned his life upside down. At 22, he received a diagnosis of temporal lobe epilepsy. The condition stripped him of everything—federal regulations prevent anyone with a seizure history from diving, ending his career. Unable to drive, he had to move and accept whatever jobs were available along bus routes.
By 2023, at 39 years old and two-and-a-half years sober, Graves was suffering from one to two seizures each day. When his physicians at UC San Diego proposed an experimental stem cell therapy, he readily consented. The treatment, known as NRTX-1001 and developed by Neurona Therapeutics, involved injecting thousands of lab-created neurons directly into his brain.
In July 2023, neurosurgeon Dr. Sharona Ben-Haim navigated a ceramic needle into his hippocampus and delivered thousands of inhibitory interneurons cultivated from stem cells. These specialized cells release GABA, which might help reduce the electrical storms that cause epileptic seizures.
The outcomes surpassed expectations. Within a few weeks, Graves observed a significant decline in his seizures. By early 2025, he was experiencing seizures only about once a week, in contrast to the daily occurrences before the treatment. Other patients noted similar success—one woman in Oregon went eight straight months without a seizure. Among the first five volunteers treated, four reported an eighty percent or higher reduction in seizure frequency. Patients also demonstrated cognitive enhancements, especially in memory.
For Dr. Ben-Haim, this signifies a potential paradigm shift. Conventional surgical methods often entail removing or damaging problematic brain tissue, which carries substantial risks like memory loss and vision impairment. Providing a definitive treatment without harming brain tissue could transform epilepsy management.
Revolutionary Diabetes Therapy: Achieving a Functional Cure
Type 1 diabetes is an autoimmune disorder where the body attacks and eliminates insulin-producing beta cells in the pancreas. In the absence of these cells, patients struggle to regulate blood glucose levels and must depend on constant monitoring and multiple daily insulin injections to survive.
In June 2024, Vertex Pharmaceuticals revealed astonishing results. Twelve individuals with type 1 diabetes who received an experimental stem cell treatment known as VX-880 (later renamed zimislecel) were generating sufficient amounts of their own insulin to maintain healthy blood glucose levels ninety days post-treatment. Remarkably, three patients monitored for a full year no longer required insulin injections whatsoever.
The treatment includes differentiating stem cells into functional beta islet cells, subsequently administering them through a single infusion. All twelve patients who received the complete dose met the recommended blood sugar targets and maintained healthy glucose levels. Their bodies could now self-regulate in a manner they hadn’t been able to since their diagnosis.
This signifies a “functional cure.” Patients must