The Hindu: Health » Medicine & Research
October 21, 2010
Lasker Award winner finds way to fight blindness
D. BALASUBRAMANIAN
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SAVING SIGHT: Patients with wet age-related macular degeneration are
now treated with Lucentis injections. Photo: S.R. Raghunathan
The HinduSAVING SIGHT: Patients with wet age-related macular
degeneration are now treated with Lucentis injections. Photo: S.R.
Raghunathan

Early October is the season for the announcement of the Nobel Prizes,
thought to be the highest recognition in the world for outstanding
achievement.

The Nobel Prize for Medicine went to Dr. Edwards, the IVF pioneer. At
about the same time also comes the announcement of the Lasker Awards,
equally prestigious awards given for outstanding biomedical research.
And it is generally believed that if one gets the Lasker, the Nobel
usually follows, and the other way around as well. Interestingly
however, Dr. Edwards got his Lasker in 2001, and had to wait for the
Nobel until now.

While the Nobel is named after Alfred Nobel, the Swedish scientist,
the Laskers are named after the philanthropic couple Albert and Mary
Woodward Lasker of the US, and have been given for the past 60 years.

This year's Lasker goes to four scientists, each of whom gets
$250,000. These are David Weatherall of Oxford, UK in recognition of
his decades of work in understanding and treating the inherited blood
disease thalassemia; Douglas Coleman retired from Jackson Labs, Maine,
USA) and Jeffrey Friedman (Rockefeller University, New York), who
share the prize for their work on the hormone called leptin which
controls our appetite (and shortfall in which is associated with
obesity); and the fourth one is Napoleone Ferrara (of biotech firm
Genentech in San Francisco, USA, who gets the Lasker for his 25 years
of work on angiogenesis, the process by which the body grows new blood
vessels.

It is Ferrara's work that we shall discuss in this article. A nice
summary is found in the Lasker website, from where I have taken
generous portions here.

Blood is a fluid that nourishes the body. It is a rich mixture of
proteins oxygen and other small molecules that make cells function,
grow and make more of themselves (proliferate).

This can be both good (which is what we want normally) and bad (when
we do not want certain cells to grow-as in cancer). Also, there are
certain tissues where we do not want blood vessels criss-crossing and
impede transparency (as in the eye).

Already, over 100 years ago, researchers found that blood vessels
proliferate before and during the growth of cancer cells into tumour.
This led Dr. Judah Folkman of Harvard (who passed away recently) to
suggest 40 years ago that if we restrict blood vessel growth or
angiogenesis to tumours, we could starve the tumour cells to death and
win over cancer. This provided the impetus to look for molecules or
factors that help the growth of blood vessels, or angiogenesis
promotion.

What then are these growth factors? By the early 1980s, two candidates
appeared on the scene, namely fibroblast growth factors a and b (aFGF,
bFGF). Fibroblasts are cells that act as strings that tie up together
to make connective tissues.

This was an exciting discovery, except for one thing. These growth
factors are stuck inside cells and do not come out; how then can they
help neighbouring cells grow, migrate and form blood vessels? A growth
factor needs to get out and trigger the process. So, back to the lab
again to look for diffusible growth factors.

It is here that Ferrara's sustained work becomes valuable. As a
post-doctoral fellow at the University of California, San Francisco,
he studied the pituitary gland cells which help in the development of
blood vessels.

While growing the pituitary gland cells in a flask, he decided to look
not just within the cells but in the medium or broth in which they
grow, the idea being lo look for factors that have come out of the
cells and can diffuse out to help angiogenesis.

When this “rich” medium was added to blood vessels, they proliferated.
He then went ahead and isolated various components of the medium and
found the molecule that fit the bill. It was named vascular
endothelial growth factor or VEGF. It is VEGF that helps grow the
cells that line blood vessels, the so called vascular endothelial
cells.

If VEGF is to induce endothelial cells to proliferate, the latter
should have the “lock” on their outer surface into which VEGF must fit
as the “key” and do its job. So, are there such “locks” or VEGF
receptors on cell surfaces? Ferrara's next step was to look for
isolate and study these putative receptors. He found them.

Then again, if one can find a way to block these locks or receptors
such that VEGF cannot fit in there, would such decoy molecules not
stop blood vessel proliferation and stop tumour growth?

Yes indeed, one can raise antibody molecules – proteins that fit the
receptors like a glove and block VEGF from getting there. Such an
antibody was his next discovery.

Thanks to this work, Genentech was able to produce and market this
antibody called Lucentis, a drug that helps control angiogenesis in
the retina of the eye. Patients with a form of blinding disease called
wet age-related macular degeneration (wet AMD) are now treated with
Lucentis injections to help preserve their sights.

Genentech also developed a similar antibody called Avastin to fight
cancer, using the same idea of blocking the ‘lock' on the surface of
cancer cells, rather than retinal cells. This too is out in the market
and used as an anticancer drug.

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