CURRENT STATUS
Neurodegeneration has crossed from symptomatic-only management into genuine disease modification. Two anti-amyloid antibodies (lecanemab, donanemab) are FDA-approved and measurably slow Alzheimer's progression; tofersen is approved for SOD1-ALS; blood-based p-tau217 has transformed diagnosis. Tau-targeted antisense oligonucleotides, stem-cell replacement for Parkinson's, LRRK2 inhibitors and BTK inhibitors for progressive MS are in late-phase trials. The field's defining shift is from treating symptoms to targeting the underlying proteinopathies and genetics.
KEY BREAKTHROUGHS
Lecanemab (Leqembi) β first anti-amyloid antibody to win full FDA approval, slows Alzheimer's decline ~27%
Donanemab (Kisunla) β FDA-approved 2024, slows Alzheimer's progression up to 35% in early-stage patients
Tofersen (Qalsody) β first ASO approved for SOD1-ALS, lowers neurofilament and slows decline
Blood tests (p-tau217) now detect Alzheimer's pathology years before symptoms β diagnosis transformed
AI-COMPRESSED PIPELINE
AI TOOLS ACCELERATING CURES
KEY ORGANIZATIONS
KEY CLINICAL TRIALS
TRAILBLAZER-ALZ 3 β Donanemab for Preclinical Alzheimer's Prevention (Phase III)
ViewEli Lilly
Testing whether donanemab can delay or prevent the onset of symptomatic Alzheimer's in cognitively normal people who already show amyloid pathology on blood tests and PET. A landmark prevention trial β intervening before irreversible neuronal loss occurs.
BlueSky β Tau-Targeting ASO (BIIB080) in Early Alzheimer's (Phase II)
ViewBiogen / Ionis Pharmaceuticals
An antisense oligonucleotide that lowers production of tau protein β the second major driver of Alzheimer's after amyloid. Delivered intrathecally, BIIB080 aims to reduce tau tangles directly, a mechanism distinct from antibody clearance.
Bemdaneprocel β Stem Cell Therapy for Parkinson's (Phase I/II)
ViewBlueRock Therapeutics (Bayer)
Transplanting dopamine-producing neurons derived from human pluripotent stem cells directly into the brain to replace cells lost in Parkinson's. Aims to restore the dopamine system rather than just managing symptoms with levodopa.
TIMELINE ESTIMATE
Alzheimer's disease-modification: available now, improving. Parkinson's cell therapy: 4β7 years. ALS gene therapy: expanding now. MS remyelination: 5β8 years.