Thursday, June 16, 2016

Unique Opportunity To Provide Input to NIH on ME/CFS

Invisible No More!

I posted last week about the opportunity to share your experience using low-dose naltrexone (LDN)with the NIH (US National Institutes of Health). Well, it turns out I posted that a little too quickly without reading all the details. What I said was perfectly accurate, but...this Request for Information (RFI) is NOT just about LDN...they are requesting input from scientists, doctors, advocates, and yes, patients, on all aspects of ME/CFS research.

This is a unique - and very rare - opportunity for patients to provide direct input to the NIH on what kinds of research you think are needed for ME/CFS, what challenges or barriers exist, and what opportunities exist. So, let NIH know - as a patient - what do you need? What priorities do you see for ME/CFS research, what treatments have helped you and need further study to make them more widely available, what needs to happen to make better, faster, more effective ME/CFS research a reality?

Otherwise, the rest of what I said is accurate -  responses to the RFI should be e-mailed and the deadline for input is June 24, 2016 (next Friday).

Here is the content of the e-mail I am sending today - I focused on treatments that have helped my son and I and how they should be more widely available to all ME/CFS patients (and the great need for educating doctors), with some notes at the end about my desire for US research into Rituximab and the need to focus research ultimately on finding a cure. I had a lot to say, but you don't need to write this much - even just a sentence or two will help.

(NOTE: for detailed information on the treatments I discuss in this e-mail and lots of links to more information, see my earlier post on Effective Treatments for ME/CFS).

Here is my e-mail - send yours today & let our voices be heard! 

"I am a patient who has had ME/CFS for over 14 years. Two years after I got sick, on March 1, 2002, both of my young sons, then ages 6 and 10, also got ME/CFS. After 10 years, one of them (whose ME/CFS was always milder) is completely recovered, but the older son, now 22, still has ME/CFS, as do I.

There is an urgent need for treatments to be officially approved in the US for ME/CFS. Having a technical background myself (ChE), I have devoted much of the past 14 years to researching and trying treatments. Although nothing so far helps a lot, we have found quite a few treatments that each help a little, and those add up to greatly improved quality of life and ability to function. Both my son and I are far more functional than most people with ME/CFS, though we are both still clearly disabled by the illness and must live our lives as defined by restrictions & limitations.

I also manage several online and in-person groups for parents whose kids have ME/CFS and so have collected information on treatments from them, as well. Many of their children are housebound or bedridden by this debilitating disease. There is an serious lack - and an urgent need - for more research into treatments for children with ME/CFS. There has never even been an ME/CFS population study of children & teens conducted!

There are millions of Americans - including many, many children and teens - suffering from ME/CFS in the US…and not a single approved treatment.

All of the treatments that have helped my son and I are currently available in the US, and many of them are off-label uses of existing medications. Research is needed to quickly assess the effectiveness of these already existing treatments in order to approve them and make them available to more people. Currently, most of these treatments are either not known by most doctors or doctors are hesitant to prescribe the drugs off-label. We have been fortunate to be able to travel to see ME/CFS experts, but that is not possible for most patients. We are also fortunate to have a primary care physician who understands ME/CFS and was willing to listen when I brought her information, so some of these treatments were prescribed by her. Most SHOULD be readily available through primary care physicians - they are only lacking the knowledge.

ALL of these simple and inexpensive treatments should be made available to ALL patients (and doctors should be trained in their use for treating ME/CFS) - I am including links to more information:
  • Correcting the sleep dysfunction characteristic of ME/CFS using low-doses of tricyclic antidepressants at bedtime to help increase serotonin and/or dopamine, as a healthy body would naturally. This has worked remarkably well for my son and I, providing deep, normal sleep every night for over 10 years now.
  • Treating Orthostatic Intolerance with a variety of medications, including (Florinef), midodrine, and various beta blockers at low doses. Note that different people respond differently to beta blockers, so any study on using them for OI should 1) start with an extremely low dose - even 1/2 or 1/4 of the lowest dose available, and 2) allow for patients in the study to try a variety of types and doses (trial and error) to find the one that works best for them. Keeping the dose low helps to prevent additional fatigue from the medication. When given the opportunity to use trial and error to try different types and doses of beta blockers, ME/CFS patients are almost always successful in finding one that helps them.
  • Treat immune dysfunction. Immune dysfunction is at the heart of ME/CFS and is the root of many (perhaps all) of its resulting symptoms. Normalizing the immune system can therefore help to improve all symptoms. Two treatments that have worked very well for us and contributed a great deal to our improvement over the years are inosine (available as a supplement in the US) and low-dose naltrexone (LDN), which uses a tiny dose (1 mg - 4.5 mg) of an already long-approved drug, naltrexone. Both of these help to normalize the immune system in ME/CFS but - again - dosing is critical as most ME/CFS patients over-respond to medications. Though LDN is typically thought of with 3 mg or 4.5 mg as ideal doses, many people with ME/CFS do better with only 1 mg or 1.5 mg. Inosine must be used according to a complicated dosing schedule in order for it to remain effective. Dozens & dozens of studies on LDN for immune disorders (but none yet for ME/CFS): More info on LDN & how it works: Inosine - note that this article refers to the name brand Imunovir, sold in Europe & Canada, but we have found the generic inosine supplement to work exactly the same for us:
  • Treat underlying infections. Perhaps the most important step in improving or even recovering from ME/CFS but one that most doctors do not understand or know about. The immune dysfunction of ME/CFS causes reactivation of latent infections, so these should be treated aggressively with antivirals or antibiotics. Common culprits in ME/CFS include mono (EBV), HHV-6, CMV, enteroviruses, Lyme disease and other tick-borne infections. My own son has 3 different tick infections and has improved greatly with treatment for those. If someone with ME/CFS has high levels of EBV or contracts mono for the first time (as is true of many teen ME/CFS patients), most doctors only know the standard protocol - no treatment and wait for it to pass. But people with ME/CFS - especially children and teens - stand to improve significantly and perhaps even recover with aggressive antiviral treatment using Valtrex or Famvir. Dr. Martin Lerner pioneered much of the existing research into treating underlying infections in ME/CFS with great success:
  • Address methylation. For most ME/CFS patients, their methylation cycle is severely dysfunctional, causing problems with energy production, adrenal function, and detox pathways. This last one is probably at least partially responsible for the fact that so many ME/CFS patients can not tolerate even small doses of medications. Often, methylation can be addressed very simply (and cheaply) with B12 folate (specific types) supplementation and results in significant improvement. This was key in my own son's improvement. Dr. Amy Yasko in the UK has written extensively about this:  Here in the US, Rich Van Konyenburg (sadly, now deceased) also did some important work in methylation, including a simplified protocol for ME/CFS patients:
For more information on all of these treatment approaches and how they have worked effectively for my son and I, see this blog post I wrote:

All of these areas need attention from NIH - to further research the effectiveness of these treatments for ME/CFS (again, keeping in mind that doses must be VERY low for most ME/CFS patients), and even more importantly, to help educate mainstream doctors about the use of these simple treatments to help improve functionality and quality of life in their ME/CFS patients. Right now, the education and information sharing is happening almost exclusively among patients, with almost no knowledge among most doctors of how to treat ME/CFS - even the simplest of facts, like that Orthostatic Intolerance is an integral part of ME/CFS and is easy to treat.

One final note: all of the treatments I mentioned here are currently available using already-approved medications and/or supplements, but the lack is in educating doctors.

There is also a significant need for continuing new research into ways to diagnose ME/CFS, its cause and exact physiology, and more effective treatments. One particular area of current focus is the use of Rituximab for ME/CFS. To date, the only studies that have been done have been in Norway (one is planned in the UK), but the results from Norway have been very promising, with 60% of ME/CFS patients improving significantly or even recovering. The US needs to get moving and do its own patient-based studies to move this research along further and fully investigate any dangers or downsides.

ME/CFS has some significant challenges that have gotten in the way of effective research in the past and could make further research useless if ignored:
  • ME/CFS is a multi-system disease that affects almost every part of the body. The traditional approach of trying to squeeze it into one single category or medical specialty doesn't work.
  • ME/CFS patients tend to over-respond to "normal" doses of medication or supplements (see notes above re: beta blockers and LDN)). Studies that use a single standardized dose to test a treatment are bound to fail. ME/CFS patients need a period of trial and error, starting with the tiniest dose available (often needing to cut pills in halves or even quarters or use a liquid) and only increasing dose as tolerated. Studies should be set up with this understanding.
  • ME/CFS is heterogenous. Different patients have different primary symptoms and respond differently to treatments. Again, allowing for some trial and error in clinical studies would help tremendously, and standardized dosing is certain to fail.
Ultimately, the goal for ME/CFS research should be not just finding treatments that help to improve symptoms a little bit but finding the cause of ME/CFS and a cure so that patients no longer need to live with a lifelong sentence of disability and limitations."

Now, go and write your own e-mail and send it before June 24! Remember, even just a few sentences would help. Let ME/CFS patients' voice be heard!


Unknown said...

What is the address to send to?

Sue Jackson said...

The e-mail link is in this post, Betsy - 3rd paragraph. Just click the link to e-mail. It is also in the RFI itself which is also linked here.