Multiple Sclerosis (MS) is an autoimmune disease of the central nervous system (CNS) affecting more than two million people worldwide with approximately 450,000 in the United States. MS is a life limiting disease which evolves over time as the immune system continues to attack and erode the brain and spinal cord.
MS is treated today with drugs that seek to reduce that self-attack. While there is benefit, most patients continue to progress and die from their disease. Significantly, regular bouts of incapacitating fatigue and ‘brain fog’ are untreated with current therapy.
Develop treatments neglected or shelved by other companies.
This was the devastating experience of a young woman physician scientist trainee – “N.D.” – working in the laboratory of Audacity founder David Housman. Diagnosed with MS in the middle of her training, N.D. suddenly deteriorated from working in the laboratory and hospital for 12-16 hours a day to not being able to walk a flight of stairs nor concentrate and stay awake for more than a few hours per day.
Look again at prior studies – what can we learn with today’s lens?
Sometimes discovery honors the most motivated. For her own health and with her training on hand, she began to look back at the vast scientific literature on MS. There were reports of the possible role of viruses in MS and some curiously favorable results in some patients given a drug designed to treat the retrovirus HIV; AZT, the oldest AIDS drug with a long history of being safe.
Always, discovery requires testing hypotheses. Given the various studies and theories about the possible trigger of MS by infection with the common Epstein Barr Virus, there was an opportunity to self-test a hypothesis; could AZT somehow act against EBV resulting in the sporadic favorable results seen in the prior studies?
Solve medical problems with practical solutions.
Volunteering to take AZT, relinquishing all other medications, resulted in recovery of N.D.’s normal work life and even return to normal athletics – within 6 months. After 3 years she has had reduction in her MS brain lesions and is nearly finished with her MD PhD training, including elucidating the mechanism by which AZT interferes with Epstein Barr Virus; a necessary understanding to support further proof of its potential as a primary treatment for MS.
Be diligent to make our products reliable – we aren’t in a race to be first, but we are in a race to help more patients
Of course, this study of n=1 is insufficient to allow widespread use of AZT in MS patients. A more rigorous study including comparisons of AZT vs. standard therapy in more MS patients must be conducted. Unfortunately for MS patients, AZT and other anti-HIV drugs are now generic priced and produced, which presents zero commercial interest for traditional pharmaceutical companies to prove the effectiveness of these drugs in MS.
Prioritize drug projects with great therapeutic potential vs profit potential
But, of course, the study should be done for the potential of nearly curative and least expensive therapy that could be prescribed for MS! This is how Audacity came to see an opportunity to exercise our public benefit pharmaceutical model. Because AZT, tenofovir and other closely related AIDS drugs have been in common clinical use and have outstanding safety profiles, Audacity proposes to initiate comparative studies in MS patients directly.
Bring patients and advocates into the lab, not just the lobby.
Clinical measures of fatigue in MS are well established, but Audacity will directly engage and partner with MS patients and organizations to co-develop and adapt a new measurement scale that patients can use to assess their disease experience.
Stand up for affordability
With the partnership and participation of MS patients, we see a future in which an effective treatment that impacts the most debilitating aspects of the disease will return these patients to their lives; and will reduce the need for expensive, relatively toxic and ineffective immune modulating therapies.
Our Stage 1 program will have enormous and immediate SOCIAL IMPACT returns and will mitigate risk in the development of a new better targeted and safe drug compound. A Stage 2 project to develop a new molecular entity, around which Audacity can establish patent protections, will allow Audacity to generate financial returns from licensing/sales of a proprietary drug to complement the social/medical impact of the Stage 1 project.