Is it fair to tell a patient to be patient? Can we develop cures at warp speed?

When I talk to people about stem cell science and the timeline for turning data into treatments and cures, their reactions completely depend on whom they are.

Scientists are patient, perhaps too patient….perhaps too understanding of the many years that we are told that science takes to get something to the clinic.

Patients and patient advocates are understandably less patient.

I think on both sides, patients and scientists, we have things to teach each other about the importance of time.

As both a scientist and a cancer patient/survivor, I kind of have one foot in each boat as the old expression goes. I can see both sides.

From the scientist’s perspective, we think we have no choice but to be patient. The current reality dictates that pre-clinical studies and clinical trials together take many years or decades. We are taught that his reality and that to try to speed this up would inevitably mean taking shortcuts that compromise safety. Is that the only reality?

From a patient’s perspective, time is totally different. For many, time is not on their side and they cannot wait many years or decades. When I was first diagnosed with prostate cancer, everyone was grim based on the histology of the biopsy. Although I am doing great and in remission for almost 2 years now, I know that could change and it could come back. Especially in the first months after my diagnosis, my research found that there are essentially no treatments for advanced prostate cancer.  I don’t have that form of the disease at this point (and hopefully never will) but that is a disturbing reality that no treatments exist and that making new ones could take longer time than I might have.

Scientists can help patients understand the temporal nature of science is measured in years and why.

Patients can help teach scientists the urgency needed to move as fast as we safely can and to balance risk with reward.

High risk or life altering medical conditions justify higher risk forms of advancing clinical trials more rapidly.

If a hypothetical patient has only about 1 year to live or even 5 years to live, that patient’s perspective time is radically different than everyone else including his/her physicians and the scientists working on research that might provide a treatment or cure. Their perception of risk is also very understandably different.

When I was a kid I was a big Star Trek fan and I loved it when the Enterprise went into warp speed, seemingly defying the laws of physics to go faster than the speed of light and explore space.

I would ask is there some we can warp the timeline of developing cures without compromising safety?

What do you think?

We need to think about such reality challenging questions, particularly in light of the coming explosion in stem cell tourism.

 

3 Comments


  1. I think it is possible to quicken the process of bench top science to safe patient bed-side treatment. However, several modifications need to be made to the current established system. One modification would involve the way in which medical scientists go about their research. They should always keep in mind how their findings can be applied clinically so they don’t go down a path that does not have clinical relevance. I know this sounds straight forward but as a member of the stem cell science community your downstream vision can get blurry real quick by getting caught up in the day to day grind in the lab.

    I think changing a medical scientist’s perspective should be started at the student level – have training medical scientists take classes in both their graduate program and at the affiliated medical school. This would allow them to get a sense of current treatment, areas that are lacking, and what patients who are suffering have to endure on a day-to-day basis. Also, upper level medical scientists should attend seminars not just on basic science lectures but also medical lectures. This would educate and motivate them to have more clinically relevant research. This could also spark ideas for developing novel avenues of treatment by better understanding the patients predicaments.

    Another area that needs to be modified is the FDA’s tight restrictions on cinical trials. I think patients who have a poor quality of life and a short life expectancy should have the choice to sign themselves up for more risky clinical trials in order to speed up the process of finding safe and effective treatments.


  2. Perhaps parts of the process could be sped up, but this can’t be done at the expense of the validity of the experiment. .

    I think perhaps middle or late stage trials should have adittional funding allocated, in the form of interest-free loans, perhaps. This would help both the scientific purposes of the trial (by having a larger sample size) and make it avaiable to more people. Perhaps to the extent of jumping directly to phase III, after completing phase I, in particularily promising products? Supposedly biologicals have a lower failure rate than older drugs, so it might not be too financially crazy.

    That aside, here in Spain there is a practice called “compassive use of drugs”, which consists in providing people with otherwise untreatable conditions with experimental drugs, even if they are outside of a clinical trial. I think this is a good practice, but of course, it needs someone to provide the forementioned drugs (eg: lets say that a simmilar policy was adopted in the US: even assuming that medicare or somesuch was paying the procedure, how many requests for compassive use of, say Grnopc1 would Geron be able to satisfy? However, as the industry becomes more established and their production capability grows, perhaps this will become more viable…

    Two final notes: I think there’s pressure to speed up these things, in general, just like there was in the eighties with antiretroviral drugs. Back then said pressure resulted in the creation of the FDA fast track. Perhaps some other optimization of the process will result now (and hopefully one that does not sacrifice safety and effectivity too much)

    My final comment is rather a question, somewhat related to the topic at hand: there is much debate about the safety of iPS cells generated by the various methods, and the meaning of their differences with regular ESCs. So my question is: would, at present,be viable and/or justified to try to find out therisks of cells created by the various methods in a series of in vivo, proof-of-concept animal trials?


  3. As so often happens, asking the right question is the crucial first step to improvement, thank you.

    Thanks to both posters above for excellent comments.

    On a somewhat related note, my clinic experimentally treats conditions including pre- and post-implantation stem cell recipients, who report good to excellent results. Per Stephen Thom’s work http://ajpheart.physiology.org/content/290/4/H1378.full.pdf+html , Hyperbaric Oxygen produces an 800% increase in circulating CD34+ cells, and did so for a multiple myeloma patient (pre-extraction) and birth-injury CP child (post- cord blood SC implant at Duke University).

    The benefits for these patients occurred due to the unusual determination of both patients and team. Too many patients face outright discouragement, in spite of excellent record of positive results, and negligible adverse reactions, by professionals who have an obligation to learn before they speak.

    The tragedy is that, like the issues above, it turns out those who discourage such treatments are almost exclusively those who lack direct experience and knowledge. It is incumbent upon all responsible healthcare professionals to learn about a particular healthcare intervention before critiquing it positively or negatively. Far too many even highly-credentialed professionals proffer opinions that claim knowledge they do not have, and should be held accountable by patients and families.

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