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Some thoughts on the FDA Workshop on M.E and CFS
As often happens when I attend a government meeting on our disease, I am left with more questions than answers.
The purpose was to facilitate drug development for our disease, and I don't think we made very much progress there. There's always someone to say "no," to put down an idea. It gets frustrating.
So here are some random impressions that could be productive:
1. We were informed there is money at NIH to develop and validate tests, questionnaires, and endpoints for drug testing. If you could get something verified, the drug company can just go ahead and use it. Otherwise, they don't have much to go on. Perhaps patient groups should think about tests that should be verified and getting this accomplished.
2. In the absence of validated tests, particularly biomarkers, we're left with questionnaires. Questionnaires pose a LOT of problems, but more so with our disease because cognitive dysfunction is a primary symptom. I think FDA was beginning to "get" this. We should use that to emphasize the need for objective testing.
3. Sometimes we can be our own worst enemies. We need whatever biomarkers we can get. There were patients complaining that VO2 MAX testing is unethical. Well, just how are you going to show the significance of our disability without walking on the edge? Off Ampligen, my VO2 MAX score is 14-15 - which is in the seriously disabled, dangerous range. (More on that later.). But I'd do it tomorrow if it meant someone would take our symptoms seriously, or I could get a drug passed.
4. I don't know how to get around this one, but those of us who have been really sick for a very long time may require a long period of treatment before we show much improvement. That's expensive. But it needs to be taken into account nevertheless.
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Now, those were thoughts on testing for drug approval. Here are a few things that occurred to me just listening to what was said.
1. IT'S ALL ABOUT THE DEFINITION. I think that's self-evident to anyone reading this, but it about drove me crazy on the second day. I do not want to be in a "cooperative" data sharing effort with someone (e.g., the CDC) who uses the Reeves or "empiric Fukuda (2003)" definition. I want to be in studies that use the M.E. (2011) international research definition or the M.E./CFS (2003) Canadian consensus definition. For those of you in the UK, that means beware of a research program being set up that cooperates with those who use the Oxford definition. It will really skew the results because the ill-defined Oxford cohort will outnumber and swallow up any group defined by a more biomedical definition. When it comes to drug approval, we need to convince drug companies to go for a definition that gives you the most homogeneity.
2. What is the REAL meaning of those VO2 MAX (CPET) scores? Everyone talks about using the VO2 MAX stress test to prove disability, or using the 2-day version to demonstrate the biological nature of post-exertional collapse. But I want to take this a step further. I knew I was really sick, but I was honestly shocked at how low I scored on that test. WHAT DOES IT MEAN TO SCORE 15 OR BELOW ON A VO2 MAX STRESS TEST? It means THERE IS SOMETHING SERIOUSLY WRONG WITH YOUR CARDIOPULMONARY FUNCTION.
To push this further, when I scored so badly I had a mess of active viruses in my blood serum (EBV, HHV-6A, CMV, HHV-7, and Coxsackie B). I had active CMV and HHV-6A in my spinal fluid and classic symptoms of encephalitis, so I think it's a good bet that I had viral encephalitis.
But did I also have viral myocarditis? Lenny Jason has suggested we are more likely to die of a heart attack prematurely than die of suicide. IS THERE MORE CARDIAC DAMAGE IN THS DISEASE THAN RESEARCHERS HAVE REALIZED? How do we get someone to pay attention to this information?
[For the record, most of the encephalitic symptoms clear up for me within six months on Ampligen. But my CPET scores don't budge for at least a year. Then they do start getting better, and after 4 years on Ampligen my CPET score was actually normal. Perhaps that helps explain why it seems to take much longer to get my stamina back.]
3. Where is the rest of the iceberg? How many years have we been told that only 15-20% of patients with this disorder have a diagnosis? Where is the 80-85%?
Someone asked a panel about the distribution of this disease by ethnicity. Beth Unger of CDC responded (and I agree with her response) that evidence shows this disease is at the equal opportunity - and that African-Americans and Latin Americans may actually have a slightly higher prevalence rate.
THERE WERE NO PEOPLE OF COLOR AT THIS CONFERENCE (AFAIK) except for one person who was not a patient. People of color are grossly under-represented in the diagnosed population; I don't want to think how badly they are represented in the practices of the handful of specialists who actually know what they are doing.
Could we find out? Could we get an accounting from the clinician experts as to what percent of their patient population with this disease are white? How about income? I think we already know the answer to this.
WHO WOULD BE INTERESTED IN THIS PROBLEM? Are there political groups who might be interested in the neglect of patients of color?
And WHERE ARE THE 80-85%? I was an invalid. Someone would have had to take care of me, and someone did. What happens to a single mother of two when she gets a serous case of this disease and remains untreated?
What has happened to patients I knew well online in the 1990s who have just disappeared?
The larger question, of course - the elephant in the room - has yet to be addressed by anyone in the government:
WHERE IS THE SENSE OF URGENCY? What is it going to take to hear that word spoken by someone in a position to actually change things?
