A promising clinical signal of a novel biologic combination immunotherapy in Amyotrophic Lateral Sclerosis (ALS) has been announced following the publication of a peer-reviewed study.
ALS, also known as motor neurone disease, is a rare, terminal neurodegenerative disorder that results in the progressive loss of both upper and lower motor neurons that normally control voluntary muscle contraction. The exact cause of the disease is still not known. A small number of cases are thought to be inherited.
Data, from Coya Therapeutics, Inc., demonstrates halting of disease progression in patients with ALS at 24 weeks (6 months who were administered a combination of CTLA4-Ig Fusion Protein (CTLA4-Ig) and Low Dose Interleukin-2 (LD IL-2).
The data further indicates a consistent reduction in inflammation, oxidative stress, and enhanced Regulatory T cell (Treg) number and suppressive function in the patient group.
For the open-label study, four participants with ALS received subcutaneous injections of commercial LD IL-2 (1×106 IU/injection/day) for 5 consecutive days every 2 weeks and one subcutaneous injection of CTLA4-Ig (125 mg/mL/injection) every 2 weeks for a total of 24 treatment cycles during the 48-week treatment period.
Study participants were followed for additional 8 weeks after treatment with LD IL-2 / CTLA4-Ig.
Prior to initiating treatment, each of the patients was declining at an average rate of -1.1 points per month on the ALSFRS-R rating scale. This is a disease-specific severity score that reflects motor impairment and functional deterioration in people with ALS.
Whereas, at the end of 6 months of treatment, the mean rate of change on the ALSFRS-R was +0.04 points per month.
According to lead researcher Dr. Stanley Appel: “The ability of the combination therapy to halt clinical progression in ALS for 24 weeks provides an important proof of concept for the protective role of Tregs while simultaneously suppressing inflammation.”
Appel adds: “What is most gratifying is the concomitant reduction of the lipid peroxide 4-HNE as well as inflammatory cytokines. This study provides the basis for a large-scale double-blind placebo-controlled trial to establish potential therapeutic efficacy for ALS patients.”
During the 48-week treatment period, the therapy was well tolerated.
The research appears in the journal Frontiers in Neurology, titled “A Phase 1 Proof-of-Concept Study Evaluating Safety, Tolerability, and Biological Marker Responses with Combination Therapy of CTLA4-Ig and Interleukin-2 in Amyotrophic Lateral Sclerosis”.
