Patient With Frontotemporal Dementia Treated With NKGen’s Autologous NK Cell Therapy Troculeucel in Compassionate Use Context
The company intends to pursue a full IND application for troculeucel in FTD.
Paul Y. Song, MD
Credit: NKGen
A patient has received NKGen Biotech's troculeucel (also known as SNK01), an investigational autologous natural killer (NK) cell therapy, for the treatment of frontotemporal dementia (FTD) under a single-patient compassionate use investigational new drug (IND) cleared by the FDA.1
Troculeucel is composed of patients’ own NK cells that have been expanded ex vivo, but not genetically engineered. NKGen is assessing the potential use of troculeucel in FTD in conjunction with Mario Mendez, MD, PhD, the director of the Behavioral Neurology Program at the David Geffen School of Medicine at UCLA and Jessica Rexach, MD, PhD, the director of the Rexach Lab at UCLA Department of Neurology, and intends to pursue a full IND application for troculeucel in this context. The patient’s troculeucel infusions are being administered at the Sarcoma Oncology Center and the patient, who NKGen notes has a C9orf72 gene mutation, is to be routinely assessed by Mendez at UCLA independently.
Notably, troculeucel was originally developed to treat solid tumors, and is also being evaluated for the treatment of Alzheimer disease (AD) and Parkinson disease (PD).2,3 A phase 1/2a trial (NCT06189963) for moderate AD is currently ongoing, and the therapy has received fast track designation from the FDA for this indication. It was previously evaluated for AD in an earlier phase 1 clinical trial (ASK-AD; NCT04678453), although this study was terminated early after the company received clearance of an IND application for a new trial using an improved manufacturing process for the therapy.
“While most of our experience has been in treating patients with AD and PD that there is a common theme of neuroinflammation and damage in numerous neurodegenerative diseases,” Paul Y. Song, MD, the chairman and chief executive officer of NKGen, said in a statement.1 “We have previously shown that troculeucel can cross the blood brain barrier to improve levels of amyloid, α-synuclein, and tau proteins in cerebrospinal fluid (CSF) in our treated AD patients as well as reduce neuroinflammation as shown by a reduction in glial fibrillary acidic protein (“GFAP”). Of note is that GFAP and tau are both consistently elevated in patients with FTD and we believe this supports the rationale of trying troculeucel in such patients. We are excited with the potential outcomes using our NK cell therapy in a difficult-to-treat patient population.”
In April of last year, NKGen received clearance of an IND application from the FDA for a phase 1/2a clinical trial in patients with PD.4 The cell therapy had previously been approved for a compassionate use administration for a patient with PD in February 2023.5
“We've now dosed almost, I would say, 100 patients between all of our cancer trials, AD trials, and our compassionate use trials, and [we’re] not seeing any side effect related to our treatment at all,” Song told CGTLive® in a 2024 interview focused on the use of troculeucel in PD. “There is no pretreatment, unlike a lot of cell therapies where you have to give some lymphodepletion or some type of low dose chemotherapy or even some cytokine support. We do not have to do any pretreatment, because it's your own natural killer cells that are not genetically modified. So, it's a very simple outpatient infusion in and out. And we find our patients, thus far, have been really, really tolerating [troculeucel] extremely well with no side effects.”
