Specific Treatments

The following is more info about specific potential treatments. Last updated 13 February 2021.

Drugs - Approved by FDA

• Riluzole

  • Basic info at WebMD: https://www.webmd.com/drugs/2/drug-12146/rilutek-oral/details

  • BHV-0223: Dissolving tablet containing riluzole. BioHaven Pharmaceuticals plan to file for FDA approval in 2018: http://biohavenpharma.com/bhv-0223-for-amyotrophic-lateral-sclerosis-als/

• Edaravone / Radicava

  • Vendor web site for patients: https://www.radicava.com/patient/

  • Customer support: https://www.radicava.com/patient/support/searchlight-support/

  • Article: https://alsnewstoday.com/news-posts/2020/11/17/radicava-suggests-more-real-world-data-needed-to-determine-efficacy-of-edaravone-in-als/

  • Article about follow-up study: https://alsnewstoday.com/2020/03/30/radicava-effective-slowing-als-progression-across-1-year-use-patients-trial-data/

  • Alternative (not-so-positive) view: https://www.healthnewsreview.org/2017/09/facts-about-the-new-als-drug-radicava-that-youre-unlikely-to-see-in-news-stories/

  • ALSA webinars - http://www.alsa.org/research/radicava/

    • The webinar of 7/25/17 is most useful: https://cc.callinfo.com/cc/playback/Playback.do?id=6z4isz

  • Oral version from Treeway - hoping to start a phase 3 trial soon:

    • News item: https://alsnewstoday.com/2018/05/31/als-therapy-candidate-oral-edavarone-shows-promise-clinical-trial/

    • Vendor press release: http://treeway.nl/press-release-treeway-announces-promising-data-from-phase-i-clinical-trial-of-lead-program-tw001-for-als/

    • http://treeway.nl/tw001-oral-edaravone-adore/

  • I took Radicava from November 2017 through November 2018. I had a port "installed" - that was an out-patient procedure. We had a nurse do port access (2x per 10-day course), but we did daily infusions and port de-access ourselves. No side effects. Didn't notice any positive effects, so stopped after one year. I participate in the Precision Medicine Program at the ALS Therapy Development Institute: https://www.als.net/precision-medicine/ and have since November 2016. I have data about my condition and progression monthly since then up until now. I don’t see any difference between my progression for the year before I was on Radicava, the year I was on Radicava, or the more than a year since I stopped Radicava.

Drugs - In Clinical Trials (see also "Stem Cells" below)

• Reldesemtiv (CK-107) (phase 2 trial complete)

  • Vendor web site: https://cytokinetics.com/reldesemtiv/

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03160898

  • Trial web site: https://www.fortitude-als.com/

  • Article reporting phase 2 results: https://alsnewstoday.com/2019/05/09/reldesemtiv-reduces-lung-function-decline-als-progression-cytokinetics/

  • Article re: orphan drug status: https://alsnewstoday.com/2019/12/19/reldesemtiv-earns-fdas-orphan-drug-designation-for-als-treatment/

  • NEALS webinar: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/fortitude-als-a-phase-2-study-of-ck107-to-treat-respiratory-and-motor-funct

  • Podcast at ALS-TDI: https://www.als.net/endpoints/ (the one entitled "Cytokinetics and Fast Skeletal Muscle Activators")

  • I participated in this trial. The trial site was in Syracuse (SUNY Upstate Medical). Required seven visits (including screening) over 16 weeks. Was easy to participate. I had no side effects. During the trial, I didn't think it was helping; however, after finishing the trial, I seemed to have increased weakness in my arms and legs, so maybe it was helping! I found out after the trial, that I was on the middle dose, 300 mg twice a day.

• Ibudilast (phase 2 trial completed; had positive results; phase 3 trial soon):

  • Phase 2 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT02238626

  • Article about Phase 2 results: https://alsnewstoday.com/2019/12/13/ibudilast-most-effective-in-people-with-short-history-of-als-analysis-suggests/

  • News story: https://alsnewstoday.com/2018/07/11/phase-2-als-trial-shows-ibudilast-rilutek-improves-function/

  • Phase 3 trial news story: https://alsnewstoday.com/2018/09/27/fda-approves-phase-3-plan-ibudilast-potential-als-treatment/

  • Phase 3 trial to begin soon: https://alsnewstoday.com/2019/04/17/fda-approves-pivotal-phase-2b-3-trial-ibudilast-als-patients/

  • Update (November 2019): https://alsnewstoday.com/2019/11/05/patent-application-for-ibudilast-as-treatment-for-als-approved-in-canada/

• Levosimendan (ODM-109) (phase 2 trial missed endpoints; phase 3 trial at higher dose fully enrolled):

  • Phase 3 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/study/NCT03505021

  • News article: https://alsnewstoday.com/2019/12/12/orion-presents-data-oral-levosimendan-mechanisms-als-disease-biomarkers/

  • NEALS webinar: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/the-refals-trial-a-phase-3-trial-of-oral-levosimendan-on-respiratory-functi

  • News story: https://www.als.net/news/orion-pharma-aims-to-enroll-450-people-with-als-in-phase-3-clinical-trial/

• TUDCA (phase 3 trial recruiting)

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03800524

  • Article: https://alsnewstoday.com/tudca-tauroursodeoxycholic-acid/

  • (see also AMX-0035)

• AMX-0035 (phase 2 trial recruitment complete; has an open-label extension)

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03127514

  • Open label extension: https://clinicaltrials.gov/ct2/show/NCT03488524

  • News about petition for early approval: https://alsnewstoday.com/news-posts/2020/11/23/petition-calling-for-swift-approval-of-amx0035-delivered-to-the-fda-amylyx/

  • News item about phase 2 results: https://www.businesswire.com/news/home/20191217005248/en/Amylyx-Pharmaceuticals-Announces-AMX0035-Demonstrated-Statistically-Significant

  • Article about phase 2 trial: https://alsnewstoday.com/2019/10/24/severe-als-patients-included-in-centaur-trial-to-maximize-results-webinar/

  • NEALS webinar: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/the-centaur-trial-a-phase-2-trial-of-amx0035-for-the-treatment-of-als-2

• Arimoclomol (phase 3 trial enrollment complete)

  • News article: https://alsnewstoday.com/arimoclomol-fda-fast-track-als-treatment+

  • News article: https://alsnewstoday.com/2019/07/22/enrollment-complete-orarials-01-trial-arimoclomol-als/

  • ALSA blog post (has links to additional info): http://web.alsa.org/site/PageNavigator/blog_081318.html

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03491462

• BIIB067 (phase 3 trial recruiting)

  • Phase 3 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT02623699

• Masitinib (phase 3 trial in US not yet started)

  • Vendor web site: http://www.ab-science.com/en/human-medicine/masitinib-in-neurodegenerative-disease

  • Phase 2 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT02588677

  • Phase 3 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03127267

  • Article about phase 3 trial: https://alsnewstoday.com/2020/04/03/fda-clears-ab-science-to-open-phase-3-trial-of-masitinib-add-on-als-treatment/

  • European agency denies application (23 April 2018): https://alsnewstoday.com/2018/04/23/ema-issues-negative-opinion-masitinib-conditional-marketing-authorization/

  • Pre-clinical study info: (2 May 2019) https://alsnewstoday.com/2019/05/02/masitinib-can-prevent-several-cellular-mechanism-involved-in-als/

  • Update 6/13/19: https://alsnewstoday.com/2019/06/11/ab-science-masitinib-study-test-oral-treatment-mildly-severe-als-ems-validated/

  • Update 7/11/19: https://alsnewstoday.com/2019/07/10/add-on-masitinib-slows-progression-of-als-final-phase-2-3-trial-results-show/

  • Other info: https://alsnewstoday.com/masitinib/

• Cu-ATSM (phase 2/3 trial recruiting)

  • Article: https://www.als.net/news/drugs-als-tdi-has-validated-part-1-copper-atsm/

  • Podcast: https://www.als.net/endpoints/64/

  • Informative blog post: http://web.alsa.org/site/PageNavigator/blog_061219.html

  • Article about phase 2/3 trial: https://alsnewstoday.com/2019/10/16/first-patient-enrolled-in-phase-2-3-trial-of-cuatsm/

  • Phase 2/3 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/study/NCT04082832 and

  • Phase 1 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT02870634

  • Vendor web page: https://colmeddev.com/procypra/

  • Report from ALS Untangled (Nov. 30, 2017): https://www.tandfonline.com/doi/pdf/10.1080/21678421.2017.1406733?needAccess=true&

  • Article about Phase 1 results: https://alsnewstoday.com/2019/01/10/cuatsm-may-slow-als-progression-improve-cognition-respiration-phase-1-trial/

• Ultomiris (Phase 3 trial not yet recruiting)

  • News article: https://alsnewstoday.com/2020/01/15/alexion-to-initiate-phase-3-trial-of-ultomiris-for-familiar-sporadic-als-patients/

  • Press release: https://news.alexion.com/press-release/product-news/alexion-announces-planned-initiation-pivotal-phase-3-study-ultomiris-ravu

• Methylcobalamin (vitamin B-12) (Phase 2/3 trial did not meet endpoints)

  • News article: https://alsnewstoday.com/2019/01/17/high-dose-vitamin-b12-helpful-early-diagnosis/

• Fasudil (Phase 2 trial recruiting)

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03792490

  • News article: https://alsnewstoday.com/2020/04/09/fasudil-shows-promising-results-in-3-als-patients-treated-under-compassionate-use/

• ALS platform trial (not yet recruiting; hope to start March 2020)

  • Status Update, including Impact of COVID-19 (April 2019): https://www.massgeneral.org/neurology/als/news/2020-03-25-platform-webinar

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT04297683

  • Press Release: https://www.massgeneral.org/about/pressrelease.aspx?id=2454

  • Webinar: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/accelerating-therapy-development-and-expanding-access

  • List of sites participating: https://www.massgeneral.org/neurology/als/research/platform-trial-sites

• Info About Active and Upcoming Trials

  • Impact of COVID-19 on ALS trials: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/impact-of-covid-19-on-als-clinical-research

  • ALS Research Update from ALS-TDI (January 2020): https://www.als.net/als-webinars/archive/01232020/

  • ALS Clinical Trial Pipeline Series from NEALS (December 2019) : https://www.neals.org/for-people-with-als-caregivers/educational-webinars/als-clinical-trial-pipeline-series-winter-2019

  • ALS Research Update from ALS-TDI (September 2018): https://www.als.net/als-webinars/archive/09252018/

  • ALS Research Update from ALS-TDI (June 2018): https://www.als.net/als-webinars/archive/06132018/

  • Top 10 Things to Watch in 2018 in ALS Research (January 2018): https://www.als.net/als-webinars/archive/01242018/

Stem Cells

• Article summarizing a review of stem cell therapies for ALS: https://alsnewstoday.com/2018/10/31/stem-cell-therapy-safe-als-patients-evaluating-effectiveness-next/

• Article warning about un-proven stem cell therapies: https://jamanetwork.com/journals/jama/article-abstract/2703448

• Webinar on stem cell therapies for ALS: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/update-on-stem-cell-trials-in-als-nurown-and-other-studies

• NurOwn (phase 3 trial in progress; patient dosing complete)

  • Vendor web site: https://brainstorm-cell.com/pipeline/#als

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03280056

  • Article about Manufacturing of NurOwn: https://alsnewstoday.com/news-posts/2021/02/12/brainstorm-fda-discuss-manufacturing-plans-nurown-potential-als-cell-therapy/

  • Phase 3 results: https://alsnewstoday.com/news-posts/2020/11/18/nurown-fails-to-slow-disease-progression-in-als-patients-top-line-data-show/

  • Expanded Access: https://alsnewstoday.com/news-posts/2020/12/16/nurown-may-be-given-to-early-als-patients-in-us-who-finished-phase-3-trial/

  • Phase 3 dosing complete: https://alsnewstoday.com/2020/07/06/200-als-patients-finish-dosing-phase-3-trial-nurown-cell-therapy/

  • Press release about FDA meeting: http://ir.brainstorm-cell.com/news-releases/news-release-details/brainstorm-cell-therapeutics-and-fda-agree-potential-nurownr

  • Press release about phase 3 trial: http://ir.brainstorm-cell.com/news-releases/news-release-details/nurownr-data-safety-monitoring-board-recommends-als-phase-3

  • Press release about publication of phase 2 results: http://ir.brainstorm-cell.com/news-releases/news-release-details/brainstorm-cell-therapeutics-announces-publication-nurownr-als

  • Other info: https://alsadotorg.wordpress.com/2017/07/20/update-from-brainstorm-cell-therapeutics-on-upcoming-phase-iii-nurown-clinical-trial/

  • News story: https://alsnewstoday.com/2017/10/17/als-phase-3-clinical-trial-nurown-enrolls-first-patients-brainstorm-cell-therapeutics-announces/

  • NurOwn and right-to-try:

    • https://alsnewstoday.com/2018/06/29/als-therapy-nurown-not-available-under-new-policy-brainstorm-decides/

    • https://brainstorm-cell.com/patients-caregivers/#policies

    • https://goldwaterinstitute.org/article/former-navy-pilot-with-als-receives-treatment-under-federal-right-to-try-law/

• Neuralstem NSI-566 (phase 2 trial complete; phase 3 trial coming)

  • Phase 2 trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT01730716

  • News story about phase 3 trial: https://alsnewstoday.com/2020/04/13/potential-stem-cell-therapy-nsi-566-moving-into-phase-3-trial-seneca-says/

• Tregs (phase 2a trial recruiting)

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT04055623

  • News story about phase 2 trial: https://strongly.mda.org/the-als-association-als-finding-a-cure-and-mda-co-fund-clinical-trial-grant-to-study-restoring-immune-system-function-to-treat-als/

  • News story about phase 1 results: https://alsnewstoday.com/2018/06/07/study-reveals-potential-treg-immune-cells-slow-als-progression/

• AstroRx (phase 1/2 in progress)

  • Trial info on clinicaltrials.gov: https://www.clinicaltrials.gov/ct2/show/NCT03482050

  • Early results:

    • https://alsnewstoday.com/news-posts/2020/12/17/astrorx-stem-cell-therapy-at-higher-dose-still-showing-transient-reductions-als-progression-trial/

    • https://alsnewstoday.com/2020/01/13/astrorx-daily-functions-als-patients-trial-results/

    • https://alsnewstoday.com/2020/08/05/astrorx-slows-als-progression-at-all-doses-tested-trial-shows/

  • News story: https://alsnewstoday.com/2020/01/10/kadimastem-finishes-treating-second-group-als-patients-astrorx-phase-1-2a-trial/

  • News story: https://alsnewstoday.com/2019/11/20/kadimastem-seeks-to-amend-protocol-of-its-trial-testing-als-stem-cell-therapy-astrorx/

Exercise

• NEALS Webinar: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/exercise-and-als-a-discussion-of-research-and-practical-recommendations

Nutritional Supplements

• List at ALS tuttu (Finnish association for supporting ALS research): https://alstuttu.org/wiki/index.php?title=Information_on_nutritional_supplements_people_with_ALS_have_been_taking

• ALS Untangled (NOTE: only a few of the treatments reviewed are nutritional supplements): http://www.alsuntangled.com/completed.html

• Lunasin

  • Trial info on clinicaltrials.gov: https://clinicaltrials.gov/show/NCT02709330

  • Poster with study results: https://f1000research.com/posters/6-2118

  • NEALS webinar about results: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/lazarus-by-lunasin-untangling-an-als-x-file

  • ALS tuttu: https://alstuttu.org/wiki/index.php?title=Special:MyLanguage/Lunasin

• Curcumin

  • Review done by ALS Untangled: https://www.tandfonline.com/doi/pdf/10.1080/21678421.2018.1440738

  • Thread at ALS-TDI (has lots of references to related journal articles): https://www.als.net/forum/?g=posts&t=44482

  • More recent article: https://www.aimsci.com/ros/html/?page_id=764

  • ALS tuttu: https://alstuttu.org/wiki/index.php?title=Special:MyLanguage/Curcumin

  • Info at PatientsLikeMe: https://www.patientslikeme.com/treatments/show/532

  • I tried Theracurmin, 300 mg, 2 tablets per day (180 mg curcumin) for about 45 days, with no noticeable good or bad effects.

Other

• Various trials run by Dr. Richard Bedlack at Duke University:

  • News Story: https://www.msn.com/en-us/health/wellness/duke-university-hospital-slated-to-start-als-clinical-trials-before-the-end-of-the-year/vi-BBWF6G1

  • Webinar: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/postcards-from-the-edge-updates-on-six-unusual-als-projects

• Things to be wary of in alternative or off-label products: https://www.tandfonline.com/doi/pdf/10.1080/21678421.2020.1765518

• Info about "Unproven Treatments" at MNDA: https://www.mndassociation.org/research/mnd-research-and-you/research-into-treatments/unproven-treatments/

• Cannabis

  • NEALS webinar: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/cannabinoid-use-in-als-only-symptom-management-or-more

  • Paper surveying the research: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5270417/?report=classic

  • Presentation at recent conference: https://www.youtube.com/watch?v=ypkG2IRaXSU&feature=youtu.be

  • Forum post: https://www.patientslikeme.com/forum/plm/topics/152527 - In addition to the initial post (which has some info), I thought this post (by user "bicyclist", posted on Jan 10, 2018 08:55PM) summarized things quite well: https://www.patientslikeme.com/forum/plm/topics/152527?post_id=2430471#post-2430471

  • ALS Untangled review (BUT note that it's from 2012): http://www.tandfonline.com/doi/full/10.3109/17482968.2012.687264

  • Info about NYS Medical Marijuana program: https://www.health.ny.gov/regulations/medical_marijuana/

  • Info at PatientsLikeMe: https://www.patientslikeme.com/treatments/show/4924-cannabis-side-effects-and-efficacy

• RCH4 - this seems to be very controversial. Lots of noise, not much objective info. As of October 17, 2019, it is the top vote-getter for the next review on ALS Untangled. (It looks like they are no longer accepting new patients; looks like website has not been updated for a couple of years.)

  • "Charity's" web site: http://www.als-new-drug.com/

  • https://rch4als.com

  • https://www.alsforums.com/community/threads/public-data-on-rch4-ndx-peptides.42704/

  • Info at PatientsLikeMe: https://www.patientslikeme.com/treatments/show/28469

  • https://www.als.net/forum/?g=posts&m=421270

  • http://forum.mndassociation.org/showthread.php?8046-Rch4

  • https://www.patientslikeme.com/treatments/show/28469-rch4-side-effects-and-efficacy

  • https://www.patientslikeme.com/forum/als/topics/145055

  • http://www.alsforums.com/forum/als-research-news/36113-rch4-drug-stopped-dr-bedlack.html