Psilocybin for ALS

Overseen By Betsy Mosmiller

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Johns Hopkins University

Must not be taking: Atypical antidepressants, Tricyclics, MAOIs, others

Disqualifiers: Severe speech impairments, Cardiovascular conditions, Epilepsy, Renal disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 2

Duration: 8 weeks

24 Participants Needed

Trial Summary

What is the purpose of this trial?

This study aims to study the feasibility of psilocybin therapy for patients with Amyotropic Lateral Sclerosis (ALS) with depressed mood. The secondary objective is to assess its impact on depression, quality of life, hopelessness, and functional status in this patient population.

Will I have to stop taking my current medications?

If you are currently taking certain antidepressants, MAOIs, or other specified medications, you will need to stop them to participate in the trial. The protocol lists specific medications that cannot be taken regularly with psilocybin.

What data supports the effectiveness of the treatment Psilocybin for ALS?

Research suggests that psychedelic-assisted psychotherapy, which includes psilocybin, may help reduce psychiatric and existential distress in ALS patients, similar to its effects in other conditions. Additionally, psychedelics have shown potential neuroprotective properties that could be beneficial for ALS, warranting further investigation. ^{1 2 3 4 5}

Is psilocybin safe for use in humans?

There is no specific safety data on psilocybin for ALS, but psilocybin has been studied for other conditions and is generally considered safe in controlled settings. Common side effects can include temporary changes in mood, perception, and thought, but serious adverse effects are rare when used responsibly under medical supervision. ^{2 6 7 8 9}

How does the drug Psilocybin differ from other treatments for ALS?

Psilocybin is unique because it is a psychedelic drug that may help alleviate psychological distress in ALS patients, unlike traditional treatments that focus on physical symptoms. It has potential neuroprotective properties and could address both psychiatric and neuropathological aspects of ALS, which are not typically targeted by existing therapies. ^{1 2 10 11 12}

Research Team

Ambereen K Mehta, MD, MPH, FAAHPM

Principal Investigator

Johns Hopkins University

Eligibility Criteria

This trial is for adults over 18 with ALS who meet specific diagnostic criteria, have a lung function measure (FVC) above 50%, can swallow tablets, and show significant depressive symptoms. It's not suitable for those who cannot take psilocybin orally or have conditions that exclude them from the study.

Inclusion Criteria

My condition meets the criteria for a diagnosis of ALS.

My lung function is more than half of what is considered normal.

I can swallow tablets by mouth, even if I have a feeding tube.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive psilocybin therapy over an 8-week course, including two psilocybin sessions at weeks 4 and 6

8 weeks

2 psilocybin sessions

Follow-up

Participants are monitored for safety and effectiveness after treatment with assessments at 1 week, 1 month, 3 months, and 6 months post-treatment

6 months

Treatment Details

Interventions

• Psilocybin

Trial Overview The trial is testing the safety and effects of psilocybin therapy on mood in ALS patients. It aims to see if this treatment can improve depression, quality of life, hopelessness, and functional status over six months.

Participant Groups

Group I: Psilocybin Experimental Treatment 1 Intervention

All subjects will be in the same, open label arm

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials

2,366

Recruited

15,160,000+

United States Department of Defense

Collaborator

Trials

940

Recruited

339,000+

Findings from Research

A new self-reported ALS disability scale called the Rasch-Built Overall ALS Disability Scale (ROADS) was developed, consisting of 28 questions that showed improved item targeting and high test-retest reliability (0.97) compared to the existing ALS Functional Rating Scale (ALSFRS-R).

The ROADS scale demonstrated better psychometric properties and responsiveness, making it a potentially valuable tool for assessing disability in ALS patients during clinical trials and in clinical settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development and Validation of the Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS). Fournier, CN., Bedlack, R., Quinn, C., et al.[2021]

In a study of 153 ALS patients, riluzole was found to have an acceptable safety profile, with 50.3% experiencing adverse effects, primarily gastrointestinal issues, hepatotoxicity, and fatigue.

Riluzole's mechanism of action suggests it may also be beneficial for other neurodegenerative diseases related to glutamate excitotoxicity, warranting further investigation and monitoring for its use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Adverse efects of riluzole (Rilutek) in the treatment of amyotrophic lateral sclerosis]. Roch-Torreilles, I., Camu, W., Hillaire-Buys, D.[2013]

A signal was detected indicating a potential link between statin use and amyotrophic lateral sclerosis (ALS) based on reports in the FDA's adverse event reporting system, with 91 cases identified.

However, a comprehensive analysis of 41 long-term clinical trials involving around 200,000 patient-years of exposure showed no increased incidence of ALS in patients treated with statins compared to those on placebo, suggesting that the initial signal may not indicate a true causal relationship.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evaluation of a data mining signal for amyotrophic lateral sclerosis and statins detected in FDA's spontaneous adverse event reporting system. Colman, E., Szarfman, A., Wyeth, J., et al.[2019]

In a one-year double-blind trial involving 22 patients with amyotrophic lateral sclerosis (ALS), those treated with branched-chain amino acids (12 g L-leucine, 8 g L-isoleucine, and 6.4 g L-valine) maintained muscle strength and walking ability, unlike the placebo group which experienced a decline.

The results suggest that branched-chain amino acids may provide a therapeutic benefit in preserving muscle function in ALS patients, highlighting their potential role in managing this progressive disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pilot trial of branched-chain aminoacids in amyotrophic lateral sclerosis. Plaitakis, A., Smith, J., Mandeli, J., et al.[2019]

References

1. United States pubmed.ncbi.nlm.nih.gov

Exploring the Potential Utility of Psychedelic Therapy for Patients With Amyotrophic Lateral Sclerosis. [2023]

2. France pubmed.ncbi.nlm.nih.gov

Cannabis for the treatment of amyotrophic lateral sclerosis: What is the patients' view? [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Survey of cannabis use in patients with amyotrophic lateral sclerosis. [2017]

4. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Single-dose pharmacokinetics and tolerability of oral delta-9- tetrahydrocannabinol in patients with amyotrophic lateral sclerosis. [2015]

5. United States pubmed.ncbi.nlm.nih.gov

Development and Validation of the Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS). [2021]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Herbal medicine for amyotrophic lateral sclerosis: A systematic review and meta-analysis. [2022]

7. France pubmed.ncbi.nlm.nih.gov

[Adverse efects of riluzole (Rilutek) in the treatment of amyotrophic lateral sclerosis]. [2013]

8. England pubmed.ncbi.nlm.nih.gov

A phase II trial of talampanel in subjects with amyotrophic lateral sclerosis. [2021]

9. England pubmed.ncbi.nlm.nih.gov

An evaluation of a data mining signal for amyotrophic lateral sclerosis and statins detected in FDA's spontaneous adverse event reporting system. [2019]

10. England pubmed.ncbi.nlm.nih.gov

Pilot trial of branched-chain aminoacids in amyotrophic lateral sclerosis. [2019]

11. United States pubmed.ncbi.nlm.nih.gov

Fungal Neurotoxins and Sporadic Amyotrophic Lateral Sclerosis. [2020]

12. England pubmed.ncbi.nlm.nih.gov

Reversible sub-acute motor neuron syndrome after mushroom intoxication masquerading as amyotrophic lateral sclerosis. [2022]