Are open source principles the key to saving lives?

Alum Jaykumar Menon shares his organization's novel approach to creating scalable solutions in the pharma industry.

Can open-source principles and collaborative innovation hold the key to solving the world’s most intractable health challenges? Alum Jaykumar Menon (MTRL 08-10) and his two nonprofits, the Open Source Pharma Foundation and the Double-Fortified Salt Project, are leading a global coalition to discover and distribute ultra-low cost biotech innovations at a scale of billons.

By harnessing the power of real-time collaboration, knowledge from around the globe, and repurposing existing off-patent technologies, Jaykumar believes open-source principles hold the key to accelerating drug discovery and development rates and countering the growing trend of rising costs.

In this interview, Jaykumar shares how open-source principles are being applied to the pharmaceutical industry, why the industry presents the best opportunity to help the most people, and how this open-source paradigm is already bringing value to tens of millions of people in need.

What is the problem Open Source Pharma is trying to solve?

Medicines can be unaffordable, nonexistent in areas of need, and excessively time-consuming and costly to develop. As a result, billions of people in countries, both rich and poor, suffer or die.

For example, only 5% of human diseases have any FDA-approved therapy. When diseases are rare, or tropical, or can be cured rapidly, the current economic model often doesn’t create a sufficient financial incentive to address them.

Another noteworthy aspect of the pharmaceutical system is that development costs for a single drug, by leading estimates, exceed $2 billion. A famous observation, "Eroom's Law," shows that the number of dollars spent to develop a new, approved drug is increasing exponentially. In contrast, in industries like IT, we have seen exponential increases in productivity, as per Moore's Law, where the amount of processing power increases exponentially over time. So, while the pharma industry has achieved remarkable advancements and created medicines and vaccines that benefit us all, its current innovation model limits the ability to reach vast numbers of conditions and billions of people.

What is Open Source Pharma doing to address this issue?

Instead of trying to replicate the existing pharma system as we focus on low-commercial-value areas, which would be too expensive, we are creating a different paradigm inspired by the success of open source in the IT industry.

These principles include openness, particularly openness of IP, crowd-sourcing, tapping the global brain, and fostering real-time collaboration. We’ve been applying these principles to drug, medicine, and vaccine discovery along two levels – ecosystem and product development.

Firstly, at the ecosystem level, we’re advocating that this alternative approach is possible, and we have received significant support. Our co-founder, Bernard Munos, has been ranked by FIERCE Biotech as one of the 25 most influential people in pharma and authored an influential paper in Nature Reviews Drug Discovery on open-source pharma R&D. We have also been creating AI-based drug discovery tools, holding global conferences, running a Village Lab, and convening a Women Scientists’ Forum, enabling women scientists far from major cities to engage in computational drug discovery from home, and bringing modern and AI-based research methods to traditional medicine.

Secondly, we’re not just working at the conceptual level. We have made substantial ground in actively developing medicines and vaccines using open-source principles. Our projects include a two-cent pill for tuberculosis and a twenty-cent innate-immunity-based immunity booster to protect against many diseases simultaneously. This could save the lives of tens to hundreds of millions of elderly people and babies and curb the next pandemic.

How does the idea of open-source work in pharma?

In the short term, we are doing what you could call “lateral discovery.” We are focusing on repurposing existing generic medicines and vaccines that you can find at your local pharmacy for a few cents. Because these medicines are open IP, we can aggregate the world’s knowledge. This costs a fraction of what the classic model costs.

Longer term, we are looking toward novel discovery. It starts with early-stage computational discovery and crowdsourcing. This is followed by an experimental component, where a network of labs can collaborate and utilize available space and low-bono or pro-bono services. The last piece is generics manufacturing. We leverage existing generics manufacturers that operate for profit. They can produce medications for five cents per pill and sell them for eight cents, making them accessible, affordable, and market-based. You add all these things up, and you have an open-source system.

You’re also a prominent human rights advocate. Tell us about your background and what precipitated your move from international law to business.

Throughout my career as a human rights lawyer, I've represented a great many people. I've engaged in impactful work and achieved victories in challenging battles.

However, there was a moment that made me reflect on the broader impact of my efforts. After successfully freeing a man from death row, one of my co-counsels commented about the return on investment and cost-benefit analysis. They pointed out that it took fifteen lawyers fifteen years to free one person. It was a boorish thing to say, but it made me think. If my mission is to help millions to billions of people around the world, where could I spend a similar amount of effort and have the most impact?

Drawing on my interest in human rights, I thought maybe I could make the world my "clients." Both global health nonprofits I’ve founded – the Double-Fortified Salt Project and Open Source Pharma – were started with this "ROI” idea.

What made pharmaceuticals the best place to achieve the highest ROI?

I selected it because it had a lot of the infrastructure and reach already in place. Instead of starting from scratch, we followed a strategy I call "designing backward from scale." Usually, somebody who wants to help the world comes up with an idea and begins building, only to discover it’s hard to scale.

In the case of the Double-Fortified Salt Project, we knew that iodized salt reaches an astonishing five billion people a day. With that as our starting point, we could focus on tweaking the existing infrastructure to reach millions to billions of people quickly.

With Open Source Pharma, we looked at the generic drug industry, which reaches billions of people at low costs, and how, by repurposing existing medicines, we could bypass the early steps and focus on conducting clinical trials for new diseases.

You’ve mentioned your second nonprofit, the Double-Fortified Salt Project, a couple of times. Tell us about that initiative.

The Double-Fortified Salt Project focuses on addressing the world’s most widespread form of malnutrition, iron deficiency. It affects two billion people worldwide, particularly women and children, including, surprisingly, about 35% of women under 50 in the U.S.

To tackle this problem, I teamed up with individuals from the University of Toronto and other institutions to develop Double-Fortified Salt, which adds trace amounts of iron to iodized salt at as little as twenty-five cents per person per year.

The project has reached over 20 million people to date, with over a billion meals having incorporated Double-Fortified Salt.

Extensive research has been conducted, and one study showed that adoption resulted in a 15-23% reduction in iron deficiency within a year.

How are you thinking about continuing to drive innovation?

We are focused on accessibility and affordability. One of our key strategies is to work with existing generic medicines and vaccines that are already on the market and available in local drug stores. These medicines, which have been trusted and used for decades, can have low costs but can be repurposed to combat new diseases through polypharmacology – meaning that it allows us to identify how one medicine might apply to multiple targets and pathways.

We've developed a robust pipeline of candidates, some of which have reached Phase IIB or Phase III clinical trials. I’ll highlight two projects.

The first project involves repurposing Metformin, a widely used diabetes drug with a broad spectrum of immune effects, into a two-cent pill for patients suffering lung damage from tuberculosis. In partnership with the government of India, we've finished Phase IIB trials and are progressing toward a global trial.

The second project has gained significant media attention and is a revolutionary vaccine. It stands out in two ways: it is legally and economically open source, and it’s focused on a new scientific paradigm for vaccines. Traditional vaccines have primarily focused on the adaptive immune system, which takes time to develop the vaccine and for the body to react to its effects. However, we are targeting the innate immune system, which can provide immediate protection against many diseases that currently afflict the world and provide proactive protection for future pandemics.

On the nutrition side, we’d like to explore commercial channels further. Thus far, double-fortified salt has principally been distributed through government social safety net programs in India. And quadruple fortified salt, with iron, iodine, folic acid, and Vitamin B-12, to protect against anemia and neural tube defects is completing clinical trials.

How is the work funded?

Largely by people power and good ideas. When you can curb malnutrition by adding iron at a cost of 25 cents per person per year or heal the sick by developing instantly generic low-cost drugs at a thousandth the cost, the needed investment is trivial.

To date, the work has largely been funded by blue-chip grant-making institutions, from major philanthropy to national government, as well as by finance and tech executives. We will continue in that vein but may scale up and diversify into innovative finance, broad-based micro-funding, and double-bottom-line social ventures.

Any advice you’d like to share?

If you're trying to do social good, the problems are often very difficult, innately so. They're complex and systemic; it's difficult to measure outcomes; it's difficult to do one thing and achieve a result because it's so multi-causal. But we can change systems. And things can be done if you go full force and are creative and strategic about it. For example, ending world hunger sounds very hard, but curbing the world's most widespread form of malnutrition is doable, and we have done it.

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Jaykumar has been named one of America’s 50 Greatest Disruptors by Newsweek, and to the world’s Worthy 100, and has won the William Rogers Award, the highest honor for a Brown University alum, given to one graduate annually. His work has appeared in The New Yorker, Newsweek, The New York Times, Fast Company, the BBC, The Times of India, MIT Technology Review, and more.