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Resources and Reading
The Right To Try Law
The Right to Try Act (RTT) is a way for people who have been diagnosed with life-threatening conditions, who have exhausted the normal treatments.
Right to Try Law
https://en.wikipedia.org/wiki/Right-to-try_law
FDA on Right to Try
https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try
Q & A on Federat Right to Try
https://righttotry.org/rtt-faq/
However, the controversy over the ethics of giving terminally ill the "Right to Try" continues, and some agencies are still discussing whether "Right to Try" is "safe" enough for the terminally ill to attempt. I will leave it up to the reader on whether that logic makes any sense. https://ashpublications.org/ashclinicalnews/news/4120/The-Realities-of-Right-to-Try
We see this as an improvement over the previous
FDA rules for "expanded-access", or "compassionate-use," which required that a physician believe that a patient will benefit from an experimental treatment, and then make a request from the FDA to apply for a new drug (IND) application for the therapy. Then the drug manufacturer would decide if they are "able and willing" to provide the product for expanded-access use.
Medical Tourism
Medical tourism is a term coined to describe traveling to other places to get treatment that you can't afford in your location, or perhaps it is not allowed. For example, many people get cosmetic surgery outside of the US, due to the cost of doing it in the US.
We are interested in this, because it gives the seriously ill another way to get treatment. If something like a biologic or exosome treatment for your disease is not yet approved in the US, you may consider traveling elsewhere to get this. Millions of US residents travel to another country for medical care every year.
If you are considering traveling to get care, we highly recommend that you do some reading ahead of time to reduce your risks.
Even the FDA understands the issue, and has guidance on their web site if you are considering a medical tourism option. https://www.magazine.medicaltourism.com/article/right-to-try-fda-reaffirms-what-medical-tourism-patients-know
The CDC also has guidance on medical tourism and advice on what risks may be encountered, and how to minimize your risks. https://wwwnc.cdc.gov/travel/page/medical-tourism
The AMA has opinions on the subject, and point out that many patients "travel on their own initiative, with or without consulting their physician." This is highly discouraged, as the primary physician is put into a difficult position of doing after care, without any input beforehand. https://code-medical-ethics.ama-assn.org/ethics-opinions/medical-tourism
Orthobiologics
Regenerative medicine is the idea of using living or functional tissue to heal, replace, or restore function to a target tissue. It could be platelet-rich plasma, growth factors, cytokines, blood products, adipose extracts, and other natural biological factors.
It can be from the patient themselves (autologous) or from a donor (allogenic).
There are many types of treatments, and hundreds of clinical trials testing the safety and effectiveness of these factors.
Here is what the Mayo clinic web site says about this area.
Ethically Sourced
We only research ethically sourced biologics. There is some history in this field, where this was not always true. However, we only focus on treatments made from the patients own body tissues, or donor material from ethical sources. Here is a recent 2022 review with a little history of one such treatment, and what diseases they have been tested against.
https://www.nature.com/articles/s41392-022-01134-4
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MASH NASH Liver Disease
The liver is a vital organ located in the abdomen. It's like a busy factory, working hard to keep our bodies healthy. One of its main jobs is to clean our blood by filtering out harmful substances. It also helps us digest food by producing bile, a fluid that breaks down fats.
Additionally, the liver stores important nutrients like vitamins and sugars for later use.
Everything you eat, drink, or breathe could potentially have toxins in it, and your liver protects you from these.
So when your liver has problems, your overall health can be heavily impacted. One emerging liver disease is Metabolic dysfunction-associated steatotic liver disease (MASLD), also known as nonalcoholic steatohepatitis (NASH) or nonalcoholic fatty liver disease (NAFLD).
There is at least one novel biologic therapy as a Potential Treatment of Non-Alcoholic Steatohepatitis-Related End-Stage Liver Disease: A Narrative Review
https://link.springer.com/article/10.1007/s40778-024-00241-y
Liver Transplants
The standard of care for some diseases is an organ transplant. [4] There are a few things wrong with this.
1.There are not enough organs.
2.Surgery like that is dangerous.
3.Surgery like that is very expensive
And because of those issues, people die every year. People who have a liver transplant have an 89% percent chance of living after one year. The five-year survival rate is 75 percentTrusted Source. [1][2]
About 8,000 liver transplant surgeries are performed in the United States every year per the American Liver Foundation, which translates to about 880 deaths after a year, even if they receive the liver transplant. In 2015, an estimated 14000 people were waiting. In 2018, 8,200 liver transplants were performed in the U.S. and about 12,800 people were waiting.[3] Many people are on the waiting list, but will not receive one. Even with the new idea of using partial livers transplanted from living donors, there is still not enough livers to go around.
References:
[1] https://www.healthline.com/health/liver-transplant-survival
[2] five-year liver surgery survival rate is 75 percentTrusted Source.
[3] https://www.mayoclinic.org/tests-procedures/liver-transplant/about/pac-20384842
[4] https://standardofcare.com/liver-transplant/
Skeptical? You Should Be
Biologic Therapies are new and exciting. Some of the results sound impossible. If you are a good consumer of information, you are right to be skeptical. Our media today is full of doubtful promises and snake oil.
So if you are skeptical, that is a positive sign. You should be. You should demand evidence, not emotion.
How can you tell an unsubstantiated emotional promise from scientific truth?
I believe in science. If you show me a peer reviewed research article saying the xyz helps mice, I will say, "Okay. That is promising. But does it work on humans?"
If it doesn't at least work on mice, I get really skeptical.
If you show me some good solid research that xyz works on humans, I say, "Great! Is there a clinical trial? Does it usually work, or 50% or what?"
Learn to look up research on scholar.google.com. Learn to search for clinical trials on clinicaltrials.gov. It's an option for you - it can be daunting at first, but with a little practice, you can get good at it. And yes, research articles can have a lot of technical jargon, but you can look up the words, one at a time, and you can get there.
Now, you should also care about your risk to benefit calculation. If I am perfectly healthy, I will demand a lot of proof that a new drug is both safe and effective. However, as I have gotten older, this has changed. If you are closer to dying, like me, you still care about effectiveness, but a little less about safety. And if you are diagnosed as terminal, the risk to benefit ratio is very different.
And if you think about it, this is the basis for the Right to Try law. People who are dying are willing to accept more risk, for a possible cure, and should be allowed to make that choice. In the United States you have this right.
Hepatic Encephalopathy
If you have had liver disease for a long time, you may be told at some point that you have HE, Hepatic Encephalopathy. Or you may have noticed problems with your memory, concentration and thinking abilities, and you may need to ask about it.
There are many causes of high ammonia levels associated with HE. These include high protein intake, infections with certain bacteria that create urease, malignancies, renal failure, gastrointestinal bleeding, gastric bypass, and others.
However, HE is a treatable condition, and you can find out more from your MD or by reading online. Here is one article of interest by the Liver Foundation.
We have some anecdotal information that stem cell therapy can help with this, and there is research that concluded this as well. [1]
References
[1] Patel M, Patel A, Kshatriya P. Mesenchymal Stem Cell Therapy for Hepatic Encephalopathy Due to Advance Liver Cirrhosis: Case Study. Ann Clin Gastroenterol Hepatol. 2024; 8(1): 017-020. Available from: https://dx.doi.org/10.29328/journal.acgh.1001046.
