Emerging Therapies
Stay informed on new treatment options
Review status: Waiting for team review. An AI editorial check was completed on 2026-08-14 by claude-opus-5. It checks clarity, attribution and scope against the sources, and it is not a Compass team or clinical sign-off. Waiting for clinical review. Last updated 2026-08-14.
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Planning 3
- Choose trusted sources firstDecide where you will follow developments before you start reading widely.Why it matters: Choosing sources first is what keeps this informative rather than exhausting.
- Track only what matches your goalsFocus on the options relevant to your situation and what you value.Why it matters: Following everything costs a lot of energy and rarely changes a decision.
- Separate evidence from hopeNote what is proven, what looks promising, and what is still uncertain.Why it matters: Keeping the difference visible protects both realistic hope and good decisions.
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ALS/MND research is active, and it is natural to want to keep track of new treatments and developments. To follow this well, it helps to understand the difference between three things that are easy to confuse.
- Approved treatments. Treatments that have been through clinical trials, shown to be safe and beneficial enough, and approved by regulators. These are part of established care, though what is approved varies by country (see the approved treatments card).
- Experimental treatments (in research). Treatments being tested in clinical trials but not yet proven or approved. They might help, but that is exactly what the trials are still determining. People usually access these by taking part in a trial (see the clinical trials card), or sometimes through special access routes (covered later).
- Unproven treatments. Things promoted as helping ALS/MND that have not been properly tested or shown to work. Some are harmless, some are useless, and some are harmful or exploitative. These are different from experimental treatments in proper trials, and need a careful, critical eye (covered in later questions).
The research pipeline:
- New ideas move from laboratory research, through early human trials (mainly checking safety), to larger trials (testing whether they truly work), before any could be approved.
- This process is slow and careful for good reasons. It makes sure treatments are safe and genuinely helpful. That can be frustrating when you want progress faster.
- Many promising ideas do not pan out, which is a normal and important part of science, even though it is disappointing.
Why the distinctions matter to you:
- They help you tell the difference between genuine, regulated research (worth following and possibly taking part in) and unproven offerings (which need caution).
- They set realistic expectations: emerging therapies are reasons for hope, but "emerging" means "not yet proven", not "available cure".
This card helps you follow developments through trustworthy sources, evaluate claims, stay safe from exploitation, and understand legitimate access. The next question covers staying informed reliably.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 2. Many ideas that look promising early on do not survive later testing, and that is a normal part of how treatments are developed.
Supports this. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Describes the same pattern on the route from laboratory work to medicine. Something that looked promising in the laboratory may fail in animals, something that worked in animals may fail in people, and something that helps symptoms may still carry risks that rule it out.
“During this process, scientists may discover that an approach that seemed promising in the lab, does not work in animals, or that an approach that worked in animals, does not work in humans.”
9. The process by which science becomes medicine is designed to minimize harm and maximize effectivenessLink checked August 2026
Statement 2 of 2. Unproven treatments sold to people with a serious condition are not all harmless. Complications can create new health problems or make symptoms harder to manage, and the costs can be very large.
Adds context. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Sets out what can be lost by trying an unproven treatment: complications that create new health problems or make symptoms harder to manage, becoming ineligible for future trials, and out-of-pocket costs that can be enormous. It is written about unproven stem cell treatments rather than unproven ALS/MND treatments in general, and it never mentions ALS/MND.
“Complications may create new short- and long-term health problems, and/or may make your condition or symptoms more difficult to manage.”
2. There is something to lose when you try an unproven stem cell "treatment"Link checked August 2026
Used across the whole answer
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Keeping up with ALS/MND research is reasonable and can be empowering. The key is using trustworthy sources so you get an accurate picture rather than hype or misinformation.
Reliable ways to stay informed:
- Your ALS/MND team. They follow developments and can give you balanced, expert perspective on what is genuinely promising and what is relevant to you. Ask them what is new.
- Reputable MND/ALS associations. Many publish research updates and news written for patients, and are careful and credible. They are among the best places to follow developments.
- Established research organisations and registries. Some research networks and registries share updates on trials and progress.
- Specialist treatment-review services. Services like ALSUntangled review the alternative and off-label treatments people are talking about, grading the evidence for each one, with final grades set across a large international team of clinicians and scientists. These are useful for cutting through hype (covered more in the next question).
Tips for following research well:
- Favour sources that explain the evidence, acknowledge uncertainty, and avoid sensational language, over those promising breakthroughs.
- Be cautious with media headlines. News stories can overstate early findings; a lab result or small early study is a long way from a proven treatment. Ask "what stage is this at?"
- Be careful in open social media groups. They can be supportive, but also spread unverified claims and promote products. Check anything striking with your team or a reputable source.
- Ask your team about anything you read. They can tell you whether a development is real, relevant to you, and at what stage.
Keeping perspective:
- Following research can bring hope and a sense of engagement. Both are valuable.
- It can also bring frustration or false hope if sources are unreliable. Sticking to trustworthy ones protects you from the rollercoaster of hype and disappointment.
You do not have to follow research closely if you would rather not. Some people prefer to leave it to their team, which is completely fine. For those who want to keep track, reputable associations and your team are the best anchors. The next question covers evaluating specific claims.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 2. ALSUntangled reviews alternative and off-label treatments advertised for ALS, grading each one across five evidence categories, and its final grades are crowd-sourced across an international team of more than 100 clinicians and scientists from 11 countries.
Supports this. Mission & Methods — ALS Untangled · Organisation
The service's own account of what it does and who does it. It reviews alternative and off-label treatments advertised for ALS, grades each one against five evidence categories, and settles the grades across a large international group of clinicians and scientists.
“Final grades are crowd-sourced across an international team of more than 100 clinicians and scientists from across 11 different countries.”
Mission & MethodsLink checked August 2026
Statement 2 of 2. A laboratory finding or a small early study sits near the start of a long process, and is a long way from a treatment shown to work.
Supports this. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Describes the route from science to medicine as lengthy and multi-step, with laboratory work, then animal work, then people, and says findings can fall down at any of those steps. It also says the current uses of stem cells are sometimes exaggerated by the media.
“This process by which science becomes medicine is often long, but it is designed to minimize patient harm and to maximize the likelihood of effectiveness.”
9. The process by which science becomes medicine is designed to minimize harm and maximize effectivenessLink checked August 2026
Used across the whole answer
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Treatment claims come up online, from other people, and in the news. A few questions help you judge whether one is credible. This is a skill worth having, because ALS/MND attracts both genuine research and unfounded claims.
Questions to ask about any claim:
- What's the evidence? Is it based on proper studies in people, or on lab work, animal studies, or just stories and testimonials? Testimonials are not evidence. Individual stories, however sincere, cannot show a treatment works (ALS/MND varies a lot between people).
- Who is making the claim, and why? Is it an independent researcher or association, or someone selling the treatment? A financial motive is a reason for extra caution.
- Where is it published? Reputable findings appear in peer-reviewed scientific journals and are discussed by experts. They do not appear only on a product's own website or social media.
- Does it sound too good to be true? Claims of a cure, dramatic results, or "what doctors won't tell you" are classic warning signs. Real science is cautious and acknowledges uncertainty.
- What do experts say? Has it been reviewed independently? What does your ALS/MND team think?
Use independent reviews:
- Services like ALSUntangled specifically investigate treatments that people with ALS/MND are talking about, and publish independent, science-based reviews. Checking whether something has been reviewed is a great way to get a balanced view.
Classic red flags:
- Promises of a cure or guaranteed results.
- Relies on testimonials rather than studies.
- Sold for profit, especially at high cost.
- "Secret", "breakthrough", or conspiracy framing ("they don't want you to know").
- Discourages you from involving your own doctors.
- Pressure to act fast.
Always involve your ALS/MND team. Before trying anything, ask them. That includes things that seem harmless. They can help you judge the evidence and safety, including whether it might interact with your other treatments. A good team will engage openly, not dismiss you.
Keeping hope and judgement together: being careful does not mean being closed to hope. It means directing your hope toward things with real promise, and protecting yourself from disappointment and harm. The next question looks specifically at unproven treatments and paid clinics.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 3. One person's account of getting better after a treatment cannot show that the treatment was the reason.
Supports this. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Explains why a sincere account can still be wrong. Someone may feel better straight after a treatment for reasons that have nothing to do with it, including belief that it will work, other things being done at the same time, lifestyle changes and the natural ups and downs of the condition, which is why a designed trial is needed to tell.
“These testimonials can be intentionally or unintentionally misleading.”
7. Patient testimonials and other marketing provided by clinics may be misleadingLink checked August 2026
Statement 2 of 3. How a treatment reaches you, and who stands to make money from it, is one of the clearer ways to tell careful research from a sales pitch: clinics selling unproven treatments tend to market straight to patients.
Supports this. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Says the way a treatment is marketed is one of the clearest differences between doctors running responsible trials and clinics selling something. Specialists mostly see people by referral, while clinics selling treatments advertise straight to patients and tend to overstate what they offer.
“Most specialized doctors receive patient referrals, while clinics selling stem cell treatments tend to market directly to patients, often through persuasive language on the Internet, Facebook and in newspaper advertisements.”
7. Patient testimonials and other marketing provided by clinics may be misleadingLink checked August 2026
Statement 3 of 3. ALSUntangled reviews the alternative and off-label treatments people with ALS/MND are hearing about, and publishes each review with its evidence grades.
Supports this. Mission & Methods — ALS Untangled · Organisation
Says why the service exists and what it covers: alternative and off-label treatments advertised for ALS on the internet, where the information is not always accurate, reviewed to help people decide about them.
“ALSUntangled® systematically reviews alternative and off label treatments (AOTs) to try and help patients with ALS make more informed decisions about them.”
Mission & MethodsLink checked August 2026
Supports this. ALSUntangled Completed Reviews — ALS Untangled · Research review
The published library itself. Each treatment reviewed carries its grades in each evidence category and a short summary, and reviews are updated when new information appears.
“Here you will find our published reports on AOTs, along with the grades we gave each of them in different categories, short summaries, and podcasts.”
Completed ReviewsLink checked August 2026
Used across the whole answer
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This is a hard and sensitive topic. When facing ALS/MND, it is utterly human to want to try anything that might help. There are clinics and sellers who, knowingly or not, take advantage of that hope. This question is here to protect you and your family, with honesty and without judgement.
Unproven treatments offered for sale:
- Some clinics and companies offer treatments that are not proven to work for ALS/MND and are given outside properly regulated trials. These are often expensive.
- These can carry real risks: physical harm, side effects, financial loss, and sometimes ruling you out of legitimate trials afterward. "Unproven" can mean unsafe, not just ineffective.
"Stem cell" clinics specifically:
- You may encounter clinics offering stem-cell treatments for ALS/MND, often at very high cost. Some are in other countries, which is sometimes called "stem cell tourism".
- Genuine stem-cell research for ALS/MND exists, but it is at the research/trial stage. It is not a proven, available treatment. Legitimate stem-cell research happens within regulated clinical trials, which do not charge you for the experimental treatment.
- Clinics that charge large sums for "stem cell" treatments outside such trials are widely warned against by ALS/MND experts and organisations: the treatments are unproven, can be dangerous, and the costs (and travel) are borne by desperate families for no proven benefit.
A key principle, again: legitimate trials and treatments do not ask you to pay large sums to access an experimental therapy. Being asked to pay for an unproven treatment is one of the clearest warning signs. Official access programs outside a trial can involve costs of their own, so if you are offered one, ask your team to help you tell the difference (see the next question).
If you are considering something like this:
- Talk to your ALS/MND team first. You do not need to fear judgement. They would far rather discuss it openly and help you weigh it than have you take a risk uninformed. Ask them directly what they know.
- Check independent reviews (such as ALSUntangled) to see what is known about it.
- Ask the hard questions: What is the actual evidence in people? Is it regulated? Why does it cost this much? What are the risks? Could it harm me or affect my eligibility for trials?
- Beware pressure and secrecy. Both are warning signs.
This is not about taking away hope or judging anyone for considering these options. The wish to fight this condition is completely understandable. It is about making sure hope is not exploited, and that you and your family are protected from harm and loss. Your team is your ally in this. The next question covers how experimental treatments can be accessed legitimately.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 3. Taking an unproven treatment can cause new health problems, cost a great deal, and make you ineligible for trials later.
Supports this. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Sets out the same things that can be lost: complications creating new health problems or making symptoms harder to manage, becoming ineligible for future trials, and out-of-pocket costs that can be enormous and are usually not reimbursed. It is written about stem cell clinics, though the trial-eligibility point is stated for any unproven or experimental treatment.
“Receipt of one unproven or experimental treatment may make you ineligible for future clinical trials or treatment options.”
2. There is something to lose when you try an unproven stem cell "treatment"Link checked August 2026
Statement 2 of 3. Outside a short list of long-established uses, stem cell treatments are still experimental, and in the United States no stem cell product is approved for ALS/MND.
Supports this. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Says the list of diseases stem cell treatments have been shown to help is still very short, covering blood and immune disorders treated by bone marrow transplant and some bone, skin and corneal grafts, and that everything else should be treated as highly experimental.
“All other applications of stem cells are yet to be proven in clinical trials and should be considered highly experimental.”
1. Currently, very few stem cell treatments have been proven safe and effectiveLink checked August 2026
Adds context. Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes — U.S. Food and Drug Administration · Official clinical information · July 2020
The US regulator names ALS among the neurological conditions for which no stem cell or exosome product is approved, and warns that products of this kind are sometimes marketed illegally and can put people at risk. It speaks only for the United States.
“None of these products have been approved to treat any neurological disorder, such as multiple sclerosis, amyotrophic lateral sclerosis (ALS; Lou Gehrig's disease), Alzheimer's disease, Parkinson's disease, epilepsy, or stroke.”
Consumer Alert on Regenerative Medicine Products Including Stem Cells and ExosomesLink checked August 2026
Statement 3 of 3. In a responsibly run trial the participant does not pay for the experimental treatment or the monitoring that goes with it.
Supports this. 9 Things to Know About Stem Cell Treatments — International Society for Stem Cell Research · Practical guide
Lists the features of a responsibly run trial, which include independent ethics committee oversight, registration on a recognised trial registry, comparison against a control group, and no charge to the participant for the treatment or the monitoring.
“The cost of the new treatment and monitoring is not covered by the participant.”
8. An experimental treatment offered for sale is not the same as a clinical trialLink checked August 2026
Used across the whole answer
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If you want to access a treatment that is still experimental, there are legitimate, safe routes. Knowing them helps you pursue hope without falling into the traps in the previous question.
The main route: clinical trials.
- For most people, taking part in a clinical trial is the proper way to access a promising experimental treatment. Trials are regulated, monitored, and free to you for the experimental treatment (see the clinical trials card for how to find and join them).
Special / expanded access programs.
- Sometimes a treatment still in development can be accessed outside a trial through programs that go by different names in different countries. Examples include expanded access, compassionate use, or early access schemes.
- These are generally for people who cannot join a trial and have serious conditions with limited options, where a treatment shows enough early promise.
- These schemes are arranged through your medical team and the relevant regulator, with oversight that usually includes an ethics committee. They are not arranged through pay-to-access clinics.
- Not every named route carries the same checks, and some countries have separate laws with different rules. Ask your team which route you are being offered, who reviews it, and what oversight applies to it.
- Availability, rules, and eligibility vary a great deal by country, and access is not guaranteed even where such programs exist.
How to explore legitimate access:
- Ask your ALS/MND team. They are the right people to tell you what trials and access programs you might be eligible for, and to arrange them properly. They can also tell you honestly what is realistic.
- Ask your MND/ALS association about what exists where you live.
- Understand the uncertainties. Even legitimate experimental treatments are unproven. They may not help, and may have unknown effects. Going in informed matters.
The contrast that keeps you safe: legitimate experimental access runs through your medical team, regulated trials, or official programs. It does not sell you an experimental treatment at a profit, though a company may be allowed to charge to recover what the product costs it to make and supply. If a route does not look like that, treat it with the caution from the previous question. There can still be costs you carry: the medical care around the treatment, or the ethics review itself, may not be covered by your insurance or health system. Ask what you would actually have to pay before you commit.
It is completely valid to want to pursue every legitimate avenue. It is equally valid to decide trials or experimental treatments are not for you. Your team can help you understand your realistic options. The next question gives you prompts to raise.
Evidence for this answer
The sources the Compass team used to write this answer. Highlighted phrases in the answer above correspond to the statements below.
Statement 1 of 5. Access outside a trial is generally reserved for a serious condition with no satisfactory alternative, where joining a trial is not possible and the likely benefit justifies the risk. These are the United States criteria; other countries set their own.
Supports this. Expanded Access | Information for Patients — U.S. Food and Drug Administration · Official clinical information · November 2022
The US regulator's own account of expanded access, also called compassionate use. It applies when the condition is serious or life-threatening, there is no comparable or satisfactory therapy, taking part in a trial is not possible, and the possible benefit justifies the possible risk. Other countries run their own schemes on their own rules.
“Whenever possible, an investigational medical product should be used as part of a clinical trial.”
What is expanded access?Link checked August 2026
Statement 2 of 5. A treatment obtained outside a trial is still unproven: it may turn out not to work, and it may cause side effects nobody has seen yet.
Supports this. Expanded Access | Information for Patients — U.S. Food and Drug Administration · Official clinical information · November 2022
States the same uncertainty about anything supplied under expanded access. These products have not been found safe and effective for the use in question, may or may not work, and may cause side effects that were not expected.
“Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause serious side effects that were not expected.”
What is expanded access?Link checked August 2026
Statement 3 of 5. Meeting the criteria for an access programme does not mean you will get the treatment, because several other parties each have to agree.
Supports this. Expanded Access | Information for Patients — U.S. Food and Drug Administration · Official clinical information · November 2022
Says meeting the criteria is not enough. The doctor, the ethics committee and the company each have to agree, and a company is under no obligation to supply a product outside its own trials and may not have enough of it.
“Even if you meet the criteria under the law and FDA regulations, the licensed physician, the IRB, or the company may not agree that expanded access is appropriate for you.”
If I meet the criteria, will I qualify?Link checked August 2026
Statement 4 of 5. Legitimate access is not a sale at a profit. A company may cover the cost itself, but where it charges, the charge must be justified and limited to recovering what the product costs to make, research, develop and handle. This is the United States rule; other countries set their own.
Qualifies this. Expanded Access | Information for Patients — U.S. Food and Drug Administration · Official clinical information · November 2022
Adds a limit worth knowing. A company may cover the cost itself, but it is allowed to charge, and where it does the charge must be justified and no more than what it costs to make, research, develop and handle the product.
“However, if a company decides to charge for the investigational medical product, the costs must be justified and limited to those necessary to recover costs of manufacture, research, development and handling.”
What costs may be involved?Link checked August 2026
Statement 5 of 5. On a legitimate access route, costs can still fall to you. Under the United States programme the product and the medical services around it are often not covered by insurance or Medicare, and the ethics review may be charged to the patient. What is covered varies by country.
Qualifies this. Expanded Access | Information for Patients — U.S. Food and Drug Administration · Official clinical information · November 2022
Says all the costs of the investigational product and the medical services around it have to be considered, and that these are often not met by private insurance or Medicare. It describes United States cover; other countries fund this differently.
“These are often not covered by third-party payers such as private insurance or Medicare.”
What costs may be involved?Link checked August 2026
Qualifies this. Expanded Access | Information for Patients — U.S. Food and Drug Administration · Official clinical information · November 2022
Adds that the ethics review can itself be billed to the patient, depending on which board reviews the request.
“Depending on the IRB, you may have to pay for the review.”
Treatment can begin once everyone agrees to expanded access to the medical product.Link checked August 2026
Used across the whole answer
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A few questions for your ALS/MND team help you follow developments, evaluate options, and stay safe. Bring anything you have read or been told. Your team would rather discuss it with you than have you decide alone.
Staying informed
- What new treatments or research should I know about that are relevant to me?
- Where do you suggest I follow ALS/MND research reliably?
- Is there anything genuinely promising on the horizon for my situation?
Evaluating something specific
- I read/heard about [treatment]. What do you know about it, and is the evidence credible?
- Has it been independently reviewed (for example by ALSUntangled)?
- Could it help me, could it harm me, or interact with my other treatments?
About unproven or paid options
- I have seen a clinic/treatment offered for a fee. Is it legitimate and safe?
- Would trying it affect my eligibility for trials? (Worth checking before committing money or your health.)
Accessing experimental treatments
- Are there trials I could join to access promising treatments? (See clinical trials.)
- Are there expanded-access or compassionate-use programs I might be eligible for here?
- If there are, what oversight applies, and what would I actually have to pay? (Ask what is not covered.)
- What is realistic for me to pursue?
A guiding principle to remember: legitimate research and treatment go through your medical team and regulated routes, and are not sold to you at a profit. Costs can still arise on an official route, so ask what they are. When in doubt, ask your team before acting. They are on your side. The clinical trials, supplements/experimental, and approved treatments cards cover closely related ground.
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