NKGen Biotech administers first dose of troculeucel for FTD under FDA-cleared compassionate use

NKGen Biotech, Inc. (Nasdaq: NKGN), a clinical-stage biotechnology company specializing in natural killer cell therapy, has taken a significant step in the treatment of frontotemporal dementia (FTD) by administering the first dose of its investigational therapy, troculeucel, under an FDA-cleared compassionate use program. This development marks a potential turning point for patients suffering from neurodegenerative diseases, particularly those with no viable treatment options.

FTD affects approximately 60,000 people in the United States and is known for its progressive degeneration of the frontal and temporal lobes of the brain. Up to 40% of cases have a genetic component, with mutations such as C9orf72 playing a crucial role. The patient receiving troculeucel carries this specific gene mutation, making them a key subject in evaluating the potential of natural killer cell therapy as a frontotemporal dementia treatment. Currently, there are no disease-modifying therapies for FTD, and available treatments only manage symptoms rather than halt or reverse disease progression.

What Is Troculeucel and How Does It Work?

Troculeucel is an expanded autologous natural killer cell therapy designed to enhance the body’s immune response against neuroinflammation and protein aggregation—two key factors in the progression of neurodegenerative diseases. NKGen Biotech has previously demonstrated troculeucel’s ability to cross the blood-brain barrier in Alzheimer’s disease patients, where it was shown to reduce amyloid, α-synuclein, and tau protein levels. Given that tau protein and glial fibrillary acidic protein (GFAP) are consistently elevated in FTD, the company believes this provides a strong rationale for evaluating troculeucel in patients diagnosed with the disease.

The compassionate use of troculeucel for FTD represents an initial step toward broader clinical applications. This effort is being carried out in collaboration with leading neurology experts, including Mario Mendez, M.D., Ph.D., Director of the Behavioral Neurology Program at the David Geffen School of Medicine at UCLA, and Jessica Rexach, M.D., Ph.D., Director of the Rexach Lab at UCLA’s Department of Neurology. Their work will be crucial in assessing the therapy’s impact on FTD biomarkers and cognitive function.

How Does Natural Killer Cell Therapy Address Neurodegenerative Diseases?

Natural killer cell therapy is emerging as a promising avenue in neurodegenerative disease therapy, particularly for conditions with a strong neuroinflammatory component. The immune system’s role in brain diseases has gained increasing attention, and modulating the peripheral immune response could provide a new way to tackle conditions like FTD and Alzheimer’s disease.

Dr. Rexach highlighted that frontotemporal dementia treatment remains a major challenge due to the lack of effective disease-modifying therapies. She emphasized that a better understanding of immune modulation could lead to targeted approaches for combating neurodegenerative diseases. She further stated that by incorporating key biomarkers of both neuroinflammation and neurodegeneration, NKGen’s research could help establish new strategies for treating FTD.

Dr. Mendez echoed similar sentiments, explaining that clinicians often struggle to offer patients any meaningful treatment options. He noted that preliminary data from troculeucel’s use in Alzheimer’s patients suggest its potential to reduce tau protein levels and inflammation in cerebrospinal fluid (CSF), which could be beneficial for FTD patients as well.

What Do Clinical Studies Reveal About Troculeucel’s Potential?

This latest development follows the publication of NKGen Biotech’s Phase 1 clinical trial results in Alzheimer’s Research & Therapy, an international peer-reviewed journal. The study, which focused on troculeucel’s application in Alzheimer’s disease, demonstrated that 90% of evaluable subjects experienced either stable or improved cognitive function.

The trial further indicated that natural killer cell therapy was well-tolerated, with no drug-related adverse events reported. Additionally, researchers observed reductions in phosphorylated tau (pTau181) and GFAP levels in CSF, suggesting that the therapy had a direct impact on neuroinflammatory and neurodegenerative processes. Given the similarities between Alzheimer’s disease and FTD, these findings strengthen the rationale for expanding troculeucel’s application to a broader range of neurodegenerative diseases.

To build on these findings, NKGen has initiated a larger study with an extended dosing regimen in the United States. This expansion aims to further investigate the efficacy and safety of troculeucel in Alzheimer’s patients and could serve as a foundation for future trials targeting FTD and other conditions characterized by chronic neuroinflammation.

What’s Next for NKGen Biotech and Frontotemporal Dementia Research?

The FTD patient receiving troculeucel under compassionate use will be closely monitored by Dr. Mendez at UCLA, while infusions will be administered at the Sarcoma Oncology Center under the guidance of Dr. Sant Chawla and Dr. Erlinda Gordon. This ongoing assessment will help determine whether natural killer cell therapy can alter the disease course in FTD, providing valuable insights for potential future clinical trials.

NKGen Biotech remains committed to advancing research in frontotemporal dementia treatment, emphasizing that natural killer cell therapy could play a critical role in reshaping neurodegenerative disease management. By targeting both immune modulation and protein aggregation, the company believes troculeucel could provide a multi-faceted approach to treating conditions that have long eluded effective medical intervention.

With neurodegenerative diseases presenting significant challenges for both patients and healthcare providers, NKGen Biotech’s research offers a potential breakthrough. If successful, the findings could open new doors for treating a wide range of neurodegenerative disorders, bringing much-needed hope to those affected by conditions like FTD and Alzheimer’s disease.