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🧠NEUROLOGY

Neurodegenerative Disease

Alzheimer's, Parkinson's, ALS, MS — from "untreatable decline" to the first disease-modifying therapies

~55 million people with dementia globally; ~10 million with Parkinson's; ~2.8 million with MS; ~140,000 living with ALS

CURRENT STATUS

The first disease-modifying drugs have arrived. Lecanemab (Leqembi) and donanemab (Kisunla) are FDA-approved and measurably slow Alzheimer's. Tofersen (Qalsody) treats SOD1-ALS. BTK inhibitors and CAR-T are reshaping MS. Gene and cell therapies for Parkinson's are in trials. AI is decoding protein misfolding and finding targets that eluded researchers for decades.

KEY BREAKTHROUGHS

Lecanemab (Leqembi) — first anti-amyloid antibody to win full FDA approval, slows Alzheimer's decline ~27%

Donanemab (Kisunla) — FDA-approved 2024, slows Alzheimer's progression up to 35% in early-stage patients

Tofersen (Qalsody) — first ASO approved for SOD1-ALS, lowers neurofilament and slows decline

Blood tests (p-tau217) now detect Alzheimer's pathology years before symptoms — diagnosis transformed

AI-COMPRESSED PIPELINE

AI TOOLS ACCELERATING CURES

Protein Misfolding SimulationNeuroimaging Biomarker AIDigital Cognitive PhenotypingAntisense Oligonucleotide Design

KEY ORGANIZATIONS

Eisai / BiogenEli LillyIonis PharmaceuticalsRoche / GenentechDenali Therapeutics

ACTIVELY RECRUITING TRIALS

TRAILBLAZER-ALZ 3 — Donanemab for Preclinical Alzheimer's Prevention (Phase III)

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Eli 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 — intervening before irreversible neuronal loss occurs.

👥 ~3,300 cognitively unimpaired participants at risk📍 US and global — multicenter

BlueSky — Tau-Targeting ASO (BIIB080) in Early Alzheimer's (Phase II)

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Biogen / Ionis Pharmaceuticals

An antisense oligonucleotide that lowers production of tau protein — the second major driver of Alzheimer's after amyloid. Delivered intrathecally, BIIB080 aims to reduce tau tangles directly, a mechanism distinct from antibody clearance.

👥 ~735 participants with early Alzheimer's📍 US, EU, Asia — multicenter

Bemdaneprocel — Stem Cell Therapy for Parkinson's (Phase I/II)

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BlueRock 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.

👥 ~24 participants with advanced Parkinson's📍 US and Canada

TIMELINE ESTIMATE

Alzheimer's disease-modifying therapy: Available now (Leqembi, Kisunla). ALS genetic-subtype therapies: 2–4 years. Parkinson's cell/gene therapy: 4–7 years. Broad neuroprotection & prevention: 6–12 years.

EXPLORE FULL CURE PROTOCOL