Researchers are making meaningful progress toward understanding ALS and developing effective treatments, but there are only a few FDA-approved drugs that just have modest effects. As a result, some families look for experimental therapies with the hope that they might slow, stop, or reverse ALS.
There are three ways people can access experimental therapies:
Because most people living with ALS do not qualify for or have access to clinical trials or EAPs, some families decide to search online for alternative options. While this desire is understandable, it is important to understand the potential risks and learn how to make informed decisions.
- Clinical trials test new drugs that have shown promise in the lab but have not yet been proven to be effective. These trials need participants who have been diagnosed with ALS and meet certain inclusion criteria. Though there is no guarantee that these drugs will be safe or effective, mainstream clinical trials have strict safety measures and independent oversight rules in an effort to protect participants. Participants should not have to pay to participate.
- Expanded Access Programs (EAPs) allow people who don’t qualify for a clinical trial to still receive the drug being offered in the trial. However, EAP opportunities for ALS are fewer in number and geographically more restricted than trials.
- Alternative and off-label treatments (AOTs) can come in the form of prescription drugs approved for other purposes, stem cell therapy, special diets, dietary supplements like vitamins, minerals, and herbs, healing energy, and more. Some of these products and treatments are sold or offered in “trials” and have a large upfront cost. Unlike prescription drugs, alternative treatments and natural supplements are not regulated, so there are few requirements in place to ensure their quality or safety. And because there is no centralized database for recording the outcomes of people self-experimenting with AOTs, the world does not learn from this and the field does not move forward.
Because most people living with ALS do not qualify for or have access to clinical trials or EAPs, some families decide to search online for alternative options. While this desire is understandable, it is important to understand the potential risks and learn how to make informed decisions.
Understanding Potential Risks
It may be possible to find a supplement that shows promise or an alternative practitioner who has a treatment that might have some beneficial effects, but you must exercise great caution because these options often lack safety monitoring and independent oversight.
Unfortunately, the claims of those selling AOTs (“completely safe”, “guaranteed”, “no risk”) are often not supported by credible evidence. There are plenty of examples of people who have experienced physical harm such as blood clots, infections, tumors, and even death while self-experimenting with AOTs. There are also examples of people spending thousands, hundreds of thousands, and even a million dollars on ineffective AOTs when that money could have been used for home care, medical equipment, and other things to improve quality of life.
Some of the people who advertise AOTs run for-profit clinics and don’t have the proper scientific background or training.
Unfortunately, the claims of those selling AOTs (“completely safe”, “guaranteed”, “no risk”) are often not supported by credible evidence. There are plenty of examples of people who have experienced physical harm such as blood clots, infections, tumors, and even death while self-experimenting with AOTs. There are also examples of people spending thousands, hundreds of thousands, and even a million dollars on ineffective AOTs when that money could have been used for home care, medical equipment, and other things to improve quality of life.
Some of the people who advertise AOTs run for-profit clinics and don’t have the proper scientific background or training.
Making Informed Decisions
If you are interested in AOTs, the best place to start is usually with your neurologist—ideally one who specializes in ALS and works at an ALS clinic. You can ask for recommendations or ask about an option that you are considering. Keep in mind that not all doctors or neurologists will be familiar with AOTs and some may not feel comfortable discussing them.
Standard treatment options for ALS include taking FDA-approved drugs and using proven non-drug interventions like noninvasive ventilation, which can help you live longer than any approved drug.
Standard treatment options for ALS include taking FDA-approved drugs and using proven non-drug interventions like noninvasive ventilation, which can help you live longer than any approved drug.
ALSUntangled
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There is a trusted website called ALSUntangled that can help you make informed decisions about AOTs for ALS. Duke ALS Clinic neurologist Dr. Richard Bedlack and over 100 clinicians and scientists from around the world objectively review and assign letter grades to specific AOTs of interest to the ALS community. You can continue to check the website to find out which AOTs currently show the most promise.
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Another helpful resource is ALSUntangled’s ten red flags, which is a list of things to be wary of when you come across an alternative or off-label product. The more red flags a product has, the more skeptical you should be. Here are a few of the things to look out for:
You can learn more about all ten red flags in the published paper ALSUntangled 56: “ten red flags” - things to be wary of in alternative or off-label products.
Ultimately, you will need to weigh the potential benefits and risks of trying any alternative or off-label treatment. Keep in mind that if something sounds too good to be true, it probably is.
- A large out-of-pocket cost
- One treatment that is advertised for multiple incurable conditions
- Lack of safety and scientific oversight
- An absent or limited informed consent process
- Lack of evidence for why this treatment might be effective
- The only evidence of benefits is anecdotes
- A proponent or practitioner who doesn’t have relevant training or publications
You can learn more about all ten red flags in the published paper ALSUntangled 56: “ten red flags” - things to be wary of in alternative or off-label products.
Ultimately, you will need to weigh the potential benefits and risks of trying any alternative or off-label treatment. Keep in mind that if something sounds too good to be true, it probably is.
Resources
Your ALS Guide is an educational website. We do not offer medical advice and are not promoting the use of alternative or off-label treatments or products for ALS. For medical advice and guidance, always speak with your licensed healthcare providers.
This page was reviewed by Duke ALS Clinic neurologist Dr. Richard Bedlack.
This page was reviewed by Duke ALS Clinic neurologist Dr. Richard Bedlack.
