Science as Social Enterprise

With the welcome attention to ‘neglected diseases’ such as malaria and tuberculosis associated with poverty, there is a tendency to forget the many health challenges such as heart disease, diabetes and cancer which wreak havoc in both worlds. These ‘shared diseases’ have dramatically different markets in both first and third world, and some thoughts about the disparity in options exposes some structural failings and innovation opportunities.

Leaving the development of diagnostics and treatments of such diseases and conditions to market forces, especially when intellectual property can be used to control entry of new players, will continue to render these debilitating but familiar illnesses a sad litany of social inequities. Are there ways that research-intensive solutions can be cooperatively generated that can grapple with this challenge?

More of the world dies of cancer than almost any other single disease. According to the WHO, cancer kills almost seven million people a year of whom about half are in the developing world. By comparison, AIDS-related conditions kill about three million people a year.

In the industrialized world, the availability of diagnostics and therapeutics for cancer, while often outrageously expensive, can lead to very favorable outcomes. In the poorer parts of the world, diagnostic technology, when available, is inadequate to the task, and few therapeutics are affordable. There are literally millions of people who die of cancer each year in the poorer parts of the world, many of whom could have had longer and more productive lives if the diagnostic and therapeutic options available to their rich neighbors were available to them.

With cancer, it is not the disease that is neglected, but the diseased.

Telomerase is an enzyme in human cells which repairs the ends of chromosomes, and is tightly regulated in normal and normally aging cells. However in tumor cells and tissues, telomerase is often wildly de-regulated, rendering such cells virtually immortal. As such, it has been viewed as the platform from which a key ‘silver bullet’ for cancer therapeutics, for diagnostics, for gerontology, even for production of stem cells, could evolve.

This was the focus of a fascinating press piece by Judy Foreman in 2003 called “Telomerase: a promising cancer drug stuck in patent hell?”

Cloning of telomerase and its ‘enclosure’ by patents was thus considered to be an enormous plum, and a single US-based company, Geron, made it its business to have exclusive control of this critical technology. The gene proved elusive however; when a tiny snippet of sequence of the suspected gene was found in a public database, a race was on. The gene was successfully cloned late 1997 by three groups: two US-based labs, including that of a Nobel Laureate, Tom Cech at University of Colorado, a National Science Medalist, Bob Weinberg at MIT and by CAMBIA Chief Scientist Andrzej Kilian, valiantly working almost alone and with few resources. It was almost a dead heat, but Cech’s work was published – and patents filed – just days before the others, albeit less completely and missing some of the key variants. Cech’s work, thought done at the University of Colorado, was exclusively licensed to Geron.

CAMBIA’s work was featured in 1997 on the cover of Human Molecular Genetics, and was widely hailed as a highly professional, thorough analysis. In 2005, after complex patent prosecution, two US patents were issued covering key aspects of the use of splice variants of the human telomerase gene associated with dozens of different tumor and cell types for diagnostics and therapeutics, and proteins based on this gene. But CAMBIA was not alone. Other patents have been continually issuing to Geron and to others, many deriving from the Cech filings. CAMBIA now has a Telomerase Patent Landscape, which outlines in some detail the situation regarding patents over this critical gene and enzyme.

In CAMBIA’s preBiOS business model, our hope would have been to license this technology both to Geron, which had been continuously cornering the market on telomerase-based strategies with massive patent acquisitions and filings, but also to a competitor of Geron to try to encourage market competition. Releasing the patents into the public domain was also considered, but we decided that doing so would have little effect on established practice, and would only entrench Geron’s monopoly.

Recognizing the ethical challenges, and the opportunities to use these patents to foment a serious public scrutiny of patent-based pharma strategy, in 2006 CAMBIA moved to make all its patent rights available for open source licensing. This project is now in its early planning stages.

We envision that in these open sourced patents could provide a publicly visible means of drawing attention to the opportunities of collective action on shared research priorities. Telomerase splice variants are not a “silver bullet”, but they are a part of a weapon that could be explicitly directed at cancer among the disadvantaged.

The CAMBIA telomerase patents may not be strong enough to stimulate a real movement by themselves, considering the hundreds of competing and even overlapping patents being granted and typically held by Geron.

Some pundits claim this monopoly over telomerase is essential for Geron to recruit sufficient capital to invest in new drugs or diagnostics. With such a clearly important target as telomerase, we feel that this assertion is baseless. Almost any drug company or diagnostics company wishing to address the cancer suffering market would jump at the chance to build proprietary solutions on an open platform of such importance. Indeed the monopoly has doubtless made Geron’s capitalization much easier. But that isn’t the point; the point is outcomes: plentiful, innovative and competitive.

Were we earlier – by as little as a week perhaps – then our patents would have been dominant and BiOS licensing could have been used to begin a dynamic commons, with a big stick to enforce it. Luckily, in some jurisdictions, we may yet secure the dominant rights, and an open source cancer program could gain traction. Are there alternatives? Is there anyone with resources sufficient to use this as a platform to encourage – to demand – a low cost, universally accessible response to human cancer? Can the creative minds of new communities of scientists use PatentLens navigation to circumvent monopolies and create options not primarily for personal gain, but for social good?