Sunday, May 17, 2009

Getting the Rx Cure: It's a Numbers Game

Maybe it's a sign of how far science and medicine have come. Today, there are a lot of treatments for medical conditions. So when someone we know or love is diagnosed with a condition that has no cure or treatment or baffles doctors we wonder, "Well, why don't they know how to treat that?"

It's a legitimate question, one I've even asked myself. The problem is the assumptions behind the question:
  1. that medicine knows everything there is to know about the human body and how it works
  2. that medicine knows everything there is to know about all disease causing entities---bacterial, viral, fungal, prion, genetic, etc.---and how they work
I know that, despite how doctors may talk and act, they don't know everything.

For some really complex systems in the body, like the brain and nervous system, what is known is dwarfed by what is not known. For some diseases, like HIV/AIDS and Alzheimer's, even if there was a cure tomorrow, there is no technology to repair the damage to the human body that these conditions inflict as part of the disease process; there is no way to repair a damaged immune system lacking T-cells or a brain full of amyloid plaques and neurofibrillary tangles.

Then there is the process of how medical discoveries are made. Among the many steps, somewhere near the beginning of the process a medical researcher must step forward and identify a problem or issue to explore, usually something that holds their interest, sustains their curiosity, drives them to find answers and fuels a willingness to "jump through the hoops" of the research process. Next are the dual issues of money and need. The medical researcher needs money to conduct their research and, at a very basic human needs level, they need to bring home a paycheck. Part of the process to acquire money is to prove need: how many people are affected by the condition that is being researched and how dire is their situation. Somewhere someone is thinking about the "big picture" and the "payoff" too, i.e. what is the probably outcome of the research---a medication, a surgery, a procedure---and how much money will it generate.

It's a lot of hurdles to jump through just to get the research started.

Once the problem is identified, need established and funding secured the science can begin. Scientific discovery is a lot like life: the more risks you take the more you get in return. In the long run, it has lead to all the treatments we rely on today. In the short run, it means a lot of trial and error and a lot of money, time, energy and resources wasted.

I know a little bit about all of this first hand as I have been following the progress of potential antiviral treatments for Hepatitis C (HCV). I began in earnest following the HCV drugs in the research pipeline in 2005, when it was suggested to me that my fibromyalgia symptoms might be related to my chronic HCV infection and I needed to consider treating the HCV. I have been waiting for the antiviral drugs in the pipeline to make it out of clinical trials and into the pharmacy.

Periodically I log onto the HCV Advocate website and the webpage Hepatitis C Treatments in Current Clinical Development. I have cut and pasted from this webpage because I think the graphical view of this table alone paints a picture of the road to medical discovery. Take a look at the columns below. Phase I is right out of the test tube and Phase IV is in the pharmacy, ready to prescribe:

Phase I Phase II Phase III Phase IV
HCV/MF59 Oral Interferon alpha Viramidine Infergen/
Bavituximab (Tarvacin) Civacir Albuferon Nexavar
IL-29 (Type III Interferon) Omega Interferon ZADAXIN® (thymalfasin or thymosin alpha 1)

NOV-205 PF-03491390 (formerly IDN-6556) VX 950 (telaprevir)

ITMN-191 IC41 SCH 503034 (boceprevir)

Belerofon (oral) MX-3253 (Celgosivir) Eltrombopag

R7128 DEBIO-025

A-831 GV1001


TG4040 BLX-883 (Locteron)

ChronVac-R MitoQ

IMO-2125 SOV-07

LGD-4665 Alinia (nitazoxanide)

CF102 VCH-759

VCH-916 Oglufanide disodium

VX-500 TMC435350

PF-00868554 GGI-5005 (Tarmogen)

SPC3649 (LNA-antimiRTM-122) CTS-1027

ABT-333 SCV-07

ANA598 SCH900518 (518)

IDX184 BMS-790052

CYT107 ITX5061

PHX1766 A-832

VX-813 GS 9190

ANA773 Oral Interferon

BI 201335 CF102

SCY-635 GS9450



So as of May 7, 2009:
  • 29 drugs in Phase 1
  • 27 drugs in Phase 2
  • 6 drugs in Phase 3
  • 2 drugs in Phase 4 (in addition to the "standard" treatment of Pegylated interferon and Ribavirin)
  • 41 additional drugs made it to human clinical trials but those studies have been canceled (not shown in table above).
As you can see, medical research starts with lots of promising ideas, but the closer you get to the finish line (Phase IV), the list gets smaller and smaller.

It seems to me that it's all a numbers game:
  • how many researchers are interested in finding out more about a certain medical condition and possible treatments
  • how many people have this condition
  • how much money will it take to develop a treatment
  • how many treatments will be tried before they find the one(s) that work
  • how much money can be made from a successful medication once it reaches the pharmacy
I know that the way this process works leaves some medical conditions with less attention, less funding and less hope for patients. I have a very goood friend who has been diagnosed with LAM, or lymphangioleiomyomatosis (lim-FAN-je-o-LI-o-MI-o-ma-TO-sis) which is a rare and fatal lung disease affecting women in their 30s and 40s. LAM affects about 10,000 women in the United States and very little money goes to research for treatments that can help prolong the current prognosis of 10 to 20 years from diagnosis.

My discussion of this topic is not meant as an endorsement of the way medical research decisions are made. Rather it is food for thought, because if there is going to be health care reform it will affect medical research funding. If you only have so much money to pay for health care, then how do you decide what to pay for? How much goes to medical research? How do you decided which conditions to focus on? How do you play the Rx cure numbers game?

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