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Phase 1 Trial Research

Home Phase 1 Trial Research

Phase 1 Trial Research

Trametinib (SNR 1611):

Completed Phase 1/2 trial
  • Trametinib (SNR1611) inhibits the key cell survival pathway that is dysregulated in ALS.
  • Can help in preventing TDP43 accumulation.

Trametinib (SNR 1611):

Tamoxifen:

Completed Phase 1/2 trial
  • Neuroprotective
  • Regulates autophagy
  • Eliminates toxic protein aggregates & cell components
  • Slows down disease progression
  • Early administration may have better effect

Tamoxifen:

Recombinant Human Erythropoietin (EPO):

In phase 1/2
  • Neuroprotective in animal models
  • Reduces inflammation
  • Enhances survival signals
  • Prevents neuronal cell death
  • Ongoing Safety and Efficacy trial notes no adverse effects

Recombinant Human Erythropoietin (EPO):

Perampanel (Fycompa):

In phase 1/2
  • Approved by the FDA for treatment of epileptic seizures
  • Restricts Calcium ion inflow into cells

Perampanel (Fycompa):

Sinemet (Carbidopa-levodopA):

In phase 1/2
  • It is the main treatment for Parkinson’s disease and might have an effect in ALS
  • In Parkinson’s disease patients, Carbidopa levodopa markedly improves patients’ rigidity

Sinemet (Carbidopa-levodopA):

Tenofir Alafenamide (TAF):

In phase 1/2
  • Antiretroviral therapy for Human Endogenous Retrovirus-K (HERV-K) Suppression
  • Some ALS patients have a high serum level of HERV-K; reason is unknown
  • Pilot study: drug was well tolerated

Tenofir Alafenamide (TAF):

AP-101:

Enrolling in Phase 1 trial
  • AP-101 is a human monoclonal antibody that reduces misfolded superoxide dismutase-1 (SOD1)

AP-101:

Treg cells:

In phase 1
  • A type of white blood cells that prevent unnecessary inflammation once an infection has been cleared
  • ALS patients have lower levels of Tregs
  • Rate of disease progression is faster in aggressive ALS
  • Increasing the number of Tregs in the blood slowed disease progression in ALS mice

Treg cells:

CNS10-NPC-GDNF:

In phase 1
  • CNS10-NPC-GDNF are stem cells called human neural progenitor cell (hNPC)
  • Genetically engineered to produce GDNF
  • Rat studies showed
  • The transplanted cells produced human GDNF and the rat neurons were taking up the GDNF released by the hNPCs
  • vhNPCs also differentiated into astrocytes
  • After seven and a half months, the transplanted hNPCs were still intact in the spinal cord

CNS10-NPC-GDNF:

Vm202 (gene therapy):

Completed Phase 1/2 trial
  • VM202 delivers a gene that produces the HGF protein
  • HGF triggers blood vessel formation and nerve growth
  • In ALS, HGF acts directly on motor neuron cells & regenerates muscles
  • Safe and well-tolerated

Vm202 (gene therapy):

Gene therapy for Silencing SOD1: APB-102:

In phase 1
  • APB-102 uses viral vector to silence SOD-1 gene
  • Faulty SOD-1 production is reduced
PHASE 1– Trametinib, Tamoxifen, EPO, Perampanel, Sinemet, TAF, AP-101, Tregs, CNS10-NPC-GDNF, VM202, APB-102

Gene therapy for Silencing SOD1: APB-102:

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