FDA clears troculeucel for expanded access program
NK cell treatment shows benefit for Alzheimer's
NKGen Biotech said it’s been cleared to launch an expanded access program (EAP) that will allow patients in the U.S. with neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS), to access its experimental therapy troculeucel outside clinical trials.
EAPs, or compassionate use programs, allow people with serious or life-threatening conditions to receive experimental therapies when no comparable or effective treatment options are available.
“We will begin enrolling patients as soon as possible, but the speed of enrollment will depend on funding,” Paul Y. Song, MD, NKGen’s chairman and CEO, said in a company press release.
NKGen is testing the treatment in clinical trials enrolling people with moderate-stage Alzheimer’s disease, another neurodegenerative condition. The EAP will allow the company to provide it to 20 patients with less common neurodegenerative diseases, many of which lack effective treatment.
The EAP will be open to people with the related disorder frontotemporal dementia, early-stage Alzheimer’s, Parkinson’s disease, multiple system atrophy, progressive supranuclear palsy, corticobasal degeneration, multiple sclerosis, and Lewy body dementia. It will run across multiple centers in the U.S., and all participants will be given the experimental therapy.
Expanded access program follows promising trial results
Brain inflammation and the buildup of protein clumps are key mechanisms underlying a range of neurodegenerative diseases, including ALS. Troculeucel, also known as SNK01, is a cell-based therapy containing natural killer (NK) cells, which are believed to help with these two mechanisms.
NK cells are immune cells that are often dysfunctional in people with neurodegenerative conditions. However, these cells have been found to identify and eliminate self-reactive T-cells that drive inflammation and to reduce protein accumulation.
NK cells also can help eliminate damaged nerve cells after injury, which may help clear cellular debris and prevent further inflammation.
The experimental therapy uses NK cells collected from a patient’s own blood, which are grown and activated in the lab to increase their therapeutic potential. These cells can be frozen and stored before being infused back into the patient by intravenous (into-the-vein) injection. According to the company, a single collection procedure may yield enough doses to cover 4-6 months of weekly treatments.
In a small Phase 1 clinical trial, troculeucel was safe and well tolerated in people with Alzheimer’s when given every three weeks at doses ranging from 1 billion to 4 billion cells.
It also showed signs of crossing the blood-brain barrier, a selective barrier that protects the brain from potentially harmful substances in the bloodstream.
Once in the brain, the therapy reduced markers of neuroinflammation and lowered the amount of abnormal protein in the cerebrospinal fluid, the liquid surrounding the brain and spinal cord.
“There is a common element of autoimmune neuroinflammation in all of these neurodegenerative diseases due to autoreactive T cells,” Song said. “We believe there is ample scientific and clinical rationale to offer this to patients with other neurodegenerative diseases for which there is no effective therapy and little hope.”
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