From:          Jonathan Rothberg, PhD
                    Fred Aslan, MD

Date:           July 7, 2003

Subject:      Proposal to find new cures for orphan diseases, epidemics such as SARS, and
                    bio-terrorism such as Anthrax

Related links:    Drug Innovation 2000

• There are 2,000+ pharmaceutical compounds in clinical development that may be suitable for the treatment of orphan diseases and emerging epidemics such as SARS, or as counter-measures against bio-terrorism threats such as Anthrax- these compounds represent an untapped resource that can be of tremendous value to society
• Whereas there are multiple non-profit agencies with the methods and the resources in place to test the utility of these 2,000+ compounds for the purposes described above, these compounds are currently not made available for testing
• Protocols can be established to enable access and distribution of these 2,000+ compounds to testing agencies without compromising intellectual property and commercial value
• The Rothberg Institute (TRI) has established the proof-of-principle for this approach by finding new potential cures to treat tuberous sclerosis (TSC) from existing pharmaceutical compounds. Like TRI, there are many agencies eager to test clinical compounds in search for new applications
• This proposal represents a unique opportunity for pharmaceutical and biotech companies to pull together as industry through a cost-effective initiative to provide a societal benefit by addressing orphan diseases, emerging epidemics, and bio-terrorism

PREMISE: 2,000+ pharmaceutical compounds in clinical development have the potential of curing orphan diseases and emerging epidemics such as SARS, or to be used as counter-measures against bio-terrorism threats such as Anthrax. Pharmaceutical compounds act in many places within the human body, as well as in bacteria and viruses. These compounds are generally tested as cures for specific diseases. However, their full potential is almost never fully explored. Many of the 2,000+ compounds currently in clinical development have the potential of addressing orphan diseases, epidemics, and bio-terrorism but they are simply not tested for these purposes. What prohibits testing are not resources, but the availability of these compounds for testing. If these compounds were to be made available and could be distributed in a rapid and cost-effective fashion to non-profit agencies with resources for testing, many new cures for orphan diseases and emerging epidemics and counter-measures against bio-terrorism could be found. These 2,000+ drugs represent an untapped resource that can be of immense value to society.

SUPPORTING CASE-STUDY: It is common for pharmaceutical compounds to work across multiple diseases where they have never been tested before
Pfizer has recently demonstrated that pharmaceutical compounds can demonstrate utility in new areas if they are tested for it. Answering to public calls for the pharmaceutical industry to take a more active role in diseases impacting developing nations, Pfizer has recently discovered that their drug Zithromax, used for many years as a treatment for strep throat and other infections, may also be used to treat malaria. Despite killing 2.6 million people per year in developing countries, malaria has limited commercial value. Therefore, testing would probably not have been done if Pfizer had not decided to provide a social service

SUPPORTING CASE-STUDY: The Rothberg Institute (TRI) has found new potential cures for the orphan-disease tuberous sclerosis (TSC) by testing marketed pharmaceutical compounds
TRI, a non-profit organization with the mission of finding cures for TSC, has established the proof-of-principle for the approach of randomly testing pharmaceutical compounds to find new medical or bio-terrorism applications. The Institute has systematically tested approved pharmaceutical compounds and found one compound, currently used as anti-fungal agent, with the potential to treat TSC. Whereas TRI tested compounds currently in the market, there are an additional 2,000+ compounds in clinical development with the same potential but currently not available for testing. TRI, as well as other non-profit agencies, has the resources in place and is eager to test these compounds for new applications.

REASON TO SUPPORT: Instead of unfairly relying on the pharmaceutical industry to test compounds for new medical and bio-defense applications, these compounds could be made available to non-profit agencies so they can perform testing
There are many agencies that have the resources and techniques to perform testing to address orphan diseases, epidemics, and bio-terrorism. Society should not have to rely on the pharmaceutical industry for testing for two main reasons:

• Testing IS NOT a priority for pharmaceutical companies and they should not have to shoulder the financial burden associated with it
• Testing IS a priority for several non-profit agencies that have already secured resources to perform testing

PROPOSAL: Establish protocols that enable limited access and cost-effective distribution of these 2,000+ compounds in clinical development without compromising intellectual property and commercial value
The goal of the proposal is to have the 2,000+ clinical compounds available so that all orphan disease agencies could work towards curing their respective diseases. Compounds in clinical development are currently not accessible for testing. Pharmaceutical and Biotech companies should be willing to make these compounds available if protocols were established that would protect intellectual property and the commercial value of these compounds.

NEXT STEPS: Unite industry leaders, educate on societal benefits, agree on the vision, and establish protocols

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