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New tools take time
June 2011
SHARING OPTIONS:
Happy days are here again. If not, it's up to us to
make it
so. I list here a few topics that I find interesting, where progress has been
quite good against early negative thinking.
ClinTrials.gov provides
transparency with clinical trials intentions and results. It's helpful to know
what has not worked.
Sharing the disappointing data is a very good thing. While
this got started only recently (September 2007), progress has been very good.
It's easy to
search on your favorite disease and see those studies that have
been completed and others in process. You can learn about the endpoints,
inclusion/exclusion criteria and much more. Take a look.
Molecular
imaging
of cells, organs, and mammals, based on a wide variety of principles, is making
a dramatic difference in discovery, preclinical and
clinical development. This
has moved from the esoteric to the routine. It's a validated approach. There
are some amazing new variations, such as cell
imaging on the fly with flow
cytometry and imaging of biopsied tissue with mass spectrometry. It's going to
get a lot better and more economical.
While we all looked at pond water
creatures swimming about on a microscope slide in high school biology, for most
of my scientific life, when cells
were to be seen, they were also to be dead,
fixed, stained and everything but stuffed. Now they can be seen very much alive
with specific components
labeled. The definition of a microscope has broadened
to an amazing array of high-performance devices. FRET and FRAP may sound like
cartoon characters
of old, but they are making biology history today.
Personalized
medicine
(at least patient segmentation into more narrowly defined classes) is gaining
ground with diagnostics that are prognostic of both
pharmacology and
toxicology. While approved companion diagnostics remain few, markers during
clinical research phases support the mechanism of action,
while demonstrating
efficacy and safety. Early success presages further change. Five years ago, there
was much skepticism, but that is slowly abating.
Related terms such as
"targeted" or "tailored" therapeutics are frequent additions to the lexicon and
likewise are real. Genomic characterization
of cancer biopsies is now an
available tool for personalized medicine. Given the long-understood phrase that
children are not small adults, pediatrics
remains a personalized medicine
challenge that is getting attention, but continues to suffer from both financial
and ethical realities. Being aware of
a problem is the first step to solving
it.
Malaria, HIV and TB:
Altruism is making progress here with open innovation among pharma,
academic
institutes and not-for-profit firms. For example, new approaches to an
infectious disease are being explored after a nearly 50-year hiatus in
the case
of tuberculosis. It is very gratifying to see the altruism of pharma sharing
libraries for this effort. More rapid diagnostics for TB are also
becoming
available, potentially saving many weeks of time. The Global Alliance for TB
Drug Development now includes the Global Health R&D Center of China.
Imagine trying that in 1970.
Rare diseases: Some
6,000 to 8,000 diseases have been too rare
to attract investment in
pharmacology. Thus, another consortium is assembling this year with the
unsurprising name of International Rare Disease Research
Consortium. A number
of national health organizations are signing on to participate, among them the
European Commission and the U.S. National Institutes of Health (NIH). The
challenges are daunting when it comes to return on R&D investment and
achieving
statistical power in clinical trials when the entire disease cohort
can be in hundreds, not millions. It therefore makes sense to plan to use
global
resources wisely and to share thoughts and data for the common good. I
suspect that the biology of these unusual cases may well help sharpen solutions
for more common diseases as well. Diseases thought to be common (such as cancer
and influenza) are becoming much more rare as subclassifications are
revealed.
Along the same lines, the NIH Chemical Genomics Center
(NCGC) has created a
database of approved and currently investigational drugs. This approaches 3,000
drugs approved in several countries. It
promises to stimulate the repurposing
of approved drugs for rare diseases, by providing sample compounds for
screening.
Peter Kissinger is
chairman emeritus of BASi, CEO of Prosolia in Indianapolis and a professor of
chemistry at Purdue University.
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