Condition

Motor neuron disease

Clinical trials and treatment information for Motor neuron disease

273K
People Affected
50
Active Trials
22K
New Cases/Year
39K
Deaths/Year

Treatment Rankings

Ranked by evidence volume first. Medicine has spent centuries ranking things by vibes. This is an improvement.

1
Non-Invasive Ventilation (NIV)
90% Effectiveness• 95% Confidence• 85% Safety• 39 trials• 3K participants
HIGH EvidenceExcellent ValueDose: Titrated positive airway pressure based on patient need
Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
85
DangerousModerateSafe

Time to Effect

Immediate for symptom relief, months for survival benefit

Duration

Lifetime

Response Rate

70%

Remission Rate

0%

Number Needed to Treat (NNT)

3

Number Needed to Harm (NNH)

10

Common Side Effects:

Facial skin irritation: 30%
Dry mouth: 20%
Bloating/gastric distension: 15%
Mask discomfort: 10%

Annual Cost of Care

Drug Cost

$0

Monitoring

$2,800

Side Effects

$200

Total Annual

$3,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.5

ICER

$20,000/QALY

Cost per Responder

$4,286

Treatment Outcomes
Primary Outcomes
Time to Tracheostomy or Death12 months (without NIV)
+50% (+6 months)
Average Nocturnal Oxygen Saturation (SpO2)88% saturation
+4.5% (+4% saturation)
Dyspnea Severity (mMRC Scale)mMRC score: 2/4
-25% (-0.5 points)
Morning Arterial PCO250 mmHg
-10% (-5 mmHg)
Sleep Quality (PSQI Score)PSQI Score: 12/21
-33.3% (-4 points)
Secondary Benefits
Quality of Life (ALSAQ-40 Score)ALSAQ-40 score: 50/100
-10% (-5 points)
Respiratory Hospitalizations per Year1.5 hospitalizations/year
-66.7% (-1.0 hospitalization/year)
ALSFRS-R Score Decline Rate1.0 point/month decline
-50% (-0.5 points/month)
Common Side Effects
Facial skin irritation
+30%
Dry mouth
+20%
Bloating/gastric distension
+15%

Clinical Trial Phases:

Phase 4
2
Gastrostomy (PEG Tube)
85% Effectiveness• 90% Confidence• 70% Safety• 5 trials• 2.5K participants
HIGH EvidenceExcellent ValueDose: N/A (procedural)
Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
70
DangerousModerateSafe

Time to Effect

Immediate for nutritional support, long-term for aspiration prevention and quality of life

Duration

Lifetime

Response Rate

90%

Remission Rate

0%

Number Needed to Treat (NNT)

4

Number Needed to Harm (NNH)

15

Common Side Effects:

Local infection: 7%
Leakage at insertion site: 7%
Pain: 5%
Tube displacement: 3%

Annual Cost of Care

Drug Cost

$0

Monitoring

$2,000

Side Effects

$500

Total Annual

$2,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.3

ICER

$35,000/QALY

Cost per Responder

$2,778

Treatment Outcomes
Primary Outcomes
Body Weight60 kg
+2.5% (+1.5 kg)
Daily Caloric Intake1500 kcal/day
+50% (+750 kcal/day)
Serum Albumin3.2 g/dL
+15.625% (+0.5 g/dL)
Secondary Benefits
Caregiver Burden (Zarit Burden Interview score)45 points (on a 0-88 scale)
-22.2% (-10 points)
Fatigue Severity (Fatigue Severity Scale score)6.0 points (on a 1-7 scale, higher is worse)
-25% (-1.5 points)
Quality of Life (Nutrition domain score)40 points (on a 0-100 scale, higher is better)
+25% (+10 points)
Common Side Effects
Local infection
+7%
Leakage at insertion site
+7%
Pain
+5%

Clinical Trial Phases:

Phase 4
3
Tofersen (for SOD1-ALS)
60% Effectiveness• 80% Confidence• 55% Safety• 1 trials• 200 participants
MODERATE EvidencePoor ValueDose: 100 mg intrathecal injection every 4 weeks after loading doses
Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
55
DangerousModerateSafe

Time to Effect

Weeks to months for biomarker changes, longer for clinical effect

Duration

Lifetime

Response Rate

%

Remission Rate

0%

Common Side Effects:

CSF pleocytosis: 36%
Myelitis: 2%
Radiculitis: 2%
Papilledema: 1%

Annual Cost of Care

Drug Cost

$160,000

Monitoring

$10,000

Side Effects

$2,000

Total Annual

$172,000

Cost-Effectiveness

POOR

QALYs Gained

0.08

ICER

$1,000,000/QALY

Treatment Outcomes
Primary Outcomes
ALS Functional Rating Scale-Revised (ALSFRS-R)ALSFRS-R score: 38/48 points
-15.26% (-5.8 points over 28 weeks (slowing of decline))
Plasma Neurofilament Light Chain (NfL)110 pg/mL (plasma)
-45% (-49.5 pg/mL)
Slow Vital Capacity (SVC)80% of predicted
-6% (-4.8 percentage points of predicted over 28 weeks (slowing of decline))
Secondary Benefits
CSF SOD1 Protein Concentration70 ng/mL (CSF)
-35% (-24.5 ng/mL)
Muscle Strength (HHD total score)65 points (sum of 16 muscles, HHD total score)
-3.08% (-2 points over 28 weeks (slowing of decline))
Common Side Effects
CSF pleocytosis
+36%
Myelitis
+2%
Radiculitis
+2%

Clinical Trial Phases:

Phase 3Phase 4
4
Edaravone
55% Effectiveness• 85% Confidence• 65% Safety• 11 trials• 600 participants
HIGH EvidencePoor ValueDose: 60 mg IV infusion daily for 14 days, followed by 14-day break; subsequent cycles 10 of 14 days, 14-day break (Oral suspension also available)
Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
65
DangerousModerateSafe

Time to Effect

3-6 months

Duration

Lifetime (cycles)

Response Rate

%

Remission Rate

0%

Number Needed to Treat (NNT)

7

Number Needed to Harm (NNH)

10

Common Side Effects:

Contusion: 9.6%
Gait disturbance: 8.4%
Headache: 7.2%
Infusion site reaction: 7%

Annual Cost of Care

Drug Cost

$165,000

Monitoring

$7,500

Side Effects

$500

Total Annual

$173,000

Cost-Effectiveness

POOR

QALYs Gained

0.07

ICER

$500,000/QALY

Treatment Outcomes
Primary Outcomes
ALS Functional Rating Scale-Revised (ALSFRS-R) score change38/48 points
-12.6% (-4.8 points over 24 weeks)
Rate of ALSFRS-R decline1.2 points/month
-33% (-0.4 points/month)
Forced Vital Capacity (FVC) decline85% predicted
-2% (-1.7% predicted over 24 weeks)
Secondary Benefits
Urinary 8-hydroxy-2'-deoxyguanosine (8-OHdG) levels15 ng/mg creatinine
-20% (-3 ng/mg creatinine)
Quadriceps Muscle Strength (Dynamometry)50 lbs
-4% (-2 lbs over 24 weeks)
Common Side Effects
Contusion
+9.6%
Gait disturbance
+8.4%
Headache
+7.2%

Clinical Trial Phases:

Phase 3Phase 4
5
Riluzole
45% Effectiveness• 90% Confidence• 65% Safety• 43 trials• 2K participants
HIGH EvidenceGood ValueDose: 50 mg twice daily
Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
65
DangerousModerateSafe

Time to Effect

6-12 months for survival benefit

Duration

Lifetime

Response Rate

%

Remission Rate

0%

Number Needed to Treat (NNT)

8

Number Needed to Harm (NNH)

20

Common Side Effects:

Nausea: 20%
Asthenia: 14%
Liver enzyme elevation (>3x ULN): 15%
Dizziness: 11%

Annual Cost of Care

Drug Cost

$2,000

Monitoring

$200

Side Effects

$100

Total Annual

$2,300

Cost-Effectiveness

GOOD

QALYs Gained

0.1

ICER

$80,000/QALY

Treatment Outcomes
Primary Outcomes
Median Survival Time14 months (untreated median)
+14.3% (+2 months)
Rate of ALSFRS-R Score Decline1.1 points/month (untreated)
-15% (-0.16 points/month)
Tracheostomy-free Survival20 months (untreated median)
+15% (+3 months)
Secondary Benefits
Rate of Forced Vital Capacity (FVC) Decline1.8% of predicted per month (untreated)
-8% (-0.14% of predicted per month)
Plasma Neurofilament Light Chain (NfL)50 pg/mL (elevated in ALS)
-15% (-7.5 pg/mL)
Common Side Effects
Nausea
+20%
Asthenia
+14%
Liver enzyme elevation (>3x ULN)
+15%

Clinical Trial Phases:

Phase 3Phase 4