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Phase 1 Trial Research

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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:

Know more about clinical trial phase 2 research
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We are a dedicated group towards well being of people with MND and their families. We understand the difficult problems they have to face due to the disease. We are well aware of their special needs and challenges to be met. We support them in all possible ways.

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Get in Touch

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ashaekhope@gmail.com

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Addresses

Clinic Address :

Plot No: 19, Sector 40, Near Seawoods Grand Central Railway Station, Off, Palm Beach Rd, Seawoods, Navi Mumbai, Maharashtra 400706

Registered Address :
402, Guruprabha Apartment, S. B. Road, Dadar West, Mumbai – 400028, Maharashtra, India
Facebook X-twitter Youtube

We are a dedicated group towards well being of people with MND and their families. We understand the difficult problems they have to face due to the disease. We are well aware of their special needs and challenges to be met. We support them in all possible ways.

Facebook X-twitter Instagram Youtube

Get in Touch

Donate Now

+91-9819741333

+91-8104546479

ashaekhope@gmail.com

Supported by :

Quick Links

Home

About Us

MND Clinic

Donate Now

Terms & Conditions

Privacy Policy

Facebook X-twitter Youtube

Addresses

Clinic Address :

Plot No: 19, Sector 40, Near Seawoods Grand Central Railway Station, Off, Palm Beach Rd, Seawoods, Navi Mumbai, Maharashtra 400706

Registered Address :
402, Guruprabha Apartment, S. B. Road, Dadar West, Mumbai – 400028, Maharashtra, India
Copyright ©2025 All rights reserved by Asha Ek Hope Foundation.
Designed, Maintained & SEO by