Open Source Drug Development

ethics + policy

One lab’s reject may be another lab’s cure By Tania Rojas

S

hare and you’ll succeed. This is the motto for the open source software movement that started in the 1990s. It is characterized by the free sharing of software to a community of computer programmers who debug and update the software online. The result? High quality computer programs rivaling those of Microsoft and IBM. Fast-forward a decade and a half later, and the musings of the open source software movement have inspired the Tropical Diseases Initiative (TDI), a small group of scientists in the biotechnology industry, to create a web forum for scientists to collaborate in developing drugs. By open sourcing, or freely sharing drug research, TDI hopes

scientists across the world will work to create drugs for neglected tropical diseases. Is it possible to open source drug research and development? Sharing proprietary information may cause millions of dollars to be lost in labor and equipment costs. Furthermore, drug development, unlike software, can take up to 10 years and more than $800 million. Try making a drug for malaria in your friend’s garage!

The Need for Open Source Drug Development

There are strong incentives for open sourcing drug research. Western markets today are prohibitive to drug development for neglected diseases. Currently, 10% of global R&D is focused on 90% of the global health burden for neglected diseases. Patent incentives, heavy competition, and skyhigh clinical trial costs have deterred investors in the biopharmaceutical industry to fund research initiatives focused on developing treatments for neglected diseases that affect only developing nations. The first and foremost disincentive is the absence of pharmaceutical markets in most developing nations. In many cases, the general public cannot afford drugs from pharmaceutical providers. In Ethiopia, for example, where the mean household income of village dwellers is roughly $140 a year, only $36 annually can be afforded to pay for treatment costs. The low numbers of drugs to treat tuberculosis (TB) demonstrate an example of the pharmaceutical industry’s oversight of third-world R&D efforts. Only 22 active TB drugs are in development by pharmaceutical companies worldwide—“a startlingly low Photo by Marcin Tusinski figure for a disease with such heavy global Open source drug development would help provide treatments for those who need them the most. burden,” claims an article published in 2004

50 stanford scientific

by Pharmaprojects, a pharmaceutical R&D database. The World Health Organization declared TB a global emergency in 1993. The disease affects two billion people –one-third of the world’s population, and is the largest cause of death of any single infectious disease. For developing nations that cannot afford developing drugs for infectious diseases, open source drug research is a promising opportunity.

Open Sourcing Drug Research Currently, scientists can search through online molecular databases to find information on drug targets—molecules associated with a disease or physical condition—and drug leads –molecules that cause drug targets to behave a specific way, often inhibiting them. However, many drug compounds that are shelved by pharmaceutical companies are not accessible to the scientific community. Creating a public database that contains drug lead and target information not needed by scientists and companies is the first step in facilitating open source drug development. The most important feature of this database is that it should provide computer simulations of drug leads that can be successfully “docked” into the drug target. Certain drug leads, for example, may not fit as well with the drug target.

Artificial markets for pharmaceuticals can be created in countries that cannot afford them. These drugs will be assigned a low-score, and those with better-fitting structures, a higher score. After a lead search has been conducted, the database will return the highest scoring drug leads. This will increase the chance of getting potential “hits” - leads that affect the drug target the way researchers want it to. At the moment, screening a drug target with 2 million leads takes roughly two weeks. Ideally, the database would provide lead results instantly. While this would take enormous processing power, algorithms can be used to hasten the process. These algorithms would filter through candidate drug molecules that are compatible with drug leads, saving time and potentially millions in research.

Creating an Online Drug Development Forum The creation of an online forum would allow scientists to find, test, and optimize the best drug candidate. The website would contain compartmentalized projects overseen by volunteer scientists. Head scientists overseeing the drug identification and optimization process would be responsible for updating experiment results and data research. Along the way, project overseers could reward team members’ contributions online. The research conducted by each department could also be published in scientific journals, promoting their reputation across the scientific community. According to Steve Weber, Director of the Institute of International Studies at the University of California, Berkeley, the pharmaceutical industry would leverage from the open source model. By open sourcing a drug compound whose

layout design: Stephanie Le

ethics + policy

patent was about to expire (and loosing 80 – 90 percent in revenues to generic drug competition), a pharmaceutical company could benefit from open source innovation on the molecule that it would be better positioned to leverage.

The Future of Open Source Drug Development With open source drug development, pharmaceutical companies no longer shelve proprietary drug R&D for neglected diseases. Once the drug is approved, there are no proprietary licenses that disable it from being manufactured by other parties. As a result, pharmaceutical manufacturers will compete against each other to produce the drug, driving down its cost. Artificial markets for pharmaceuticals can be created in countries that cannot afford them. Open source drug development will be possible if c o m p a n i e s a r e looking to collaborate, not compete, in creating treatments that are desperately needed. Efficient search algorithms must also be developed to http://phil.cdc.gov/phil_images/20030811/8/HIL_4428_lores.jpg allow rapid testing Mycobacterium tuberculosis (TB). TB is the largest of open source drug cause of death of any single infectious disease. targets and leads. In Few pharmaceutical companies are actively addition, specialized developing TB treatments. communication technologies must be created to allow scientists to share their research findings and experiments online. Open source drug development is possible, but it is still in its beginning stages. With the help of researchers from institutions such as Stanford, it may start to take hold as a way to reduce global disparities in health. S Tania Rojas is a senior majoring in Human Biology and Science, Technology and Society. She is driven by several passions: biotechnology, science-fiction, and good karma. Her goal is to become a non-profit biotech entrepreneur that develops treatments for neglected diseases. To Learn More: The Institute of OneWorld Health The world’s first non-profit pharmaceutical company. http://www.oneworldhealth.org/ The Tropical Disease Initiative An organization dedicated to developing treatments for tropical diseases. http://www.tropicaldisease.org/ Low Hanging Fruit An open source database that shares drug/compound screens against parasitic organisms. http://www.ucsf.edu/mckerrow/fruit.html

volume iv 51