Be HEARD: A Rare Disease Science Challenge To Find Cures

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A challenge to try to bypass Big Pharma's failure to tackle rare diseases.

Curing or even finding treatments for rare diseases is hard. Not necessarily because these diseases are any more complex than more common ones. It has more to do with the fact that there is very little profit to be made in helping people with these diseases.

That is why a new initiative, the Rare Disease Science Challenge (which was discussed at this weekend’s Open Science Summit at the Computer History Museum in Mountain View) is so exciting. It is trying to collect a critical mass of nonprofits, scientists, and families dealing with these diseases and to offer them seed money and free services to start to find treatments and/or cures.

As I talked about in a recent blog, this model has worked well recently with cystic fibrosis. At first you might think cystic fibrosis is too common to need something like this. But it isn’t.

There are many ways to end up with cystic fibrosis, each of which might need a separate treatment or cure. A combination of nonprofits, scientists and families dealing with cystic fibrosis came together and brought Kalydeco to market. Even though it can only help ~4% of CF patients, it is a Godsend for these sufferers. Hopefully this new competition can spawn something similar for some other awful, rare diseases.

Dr. Kevin Lustig, President and CEO of Assay Depot

This is such an important project that I wanted to talk with one of its sponsors. Here is my interview with Kevin Lustig, CEO and President of Assay Depot:


Tell me more about the Rare Disease Science Challenge. What is it all about and why do we need it?

Assay Depot and Rare Genomics Institute are teaming up to sponsor the Rare Disease Science Challenge to accelerate rare disease research. Mainstream pharmaceutical companies have long ignored rare diseases. However, collectively rare diseases are not rare. The fact is that while a disease might be labeled as “rare,” the number of persons suffering from one of the thousands of rare diseases is estimated at 10% of the population, or approximately 30 million people in the United States and 350 million people globally.

For the majority (greater than 95%) of the 7,000 rare diseases there is no FDA approved therapy. Perhaps the cruelest twist of rare diseases is that so many of the patients are young children with over 30% of them dying before their 5th birthday!

There probably isn’t enough money in prizes to find a cure or even a treatment. What do you think is realistically possible with the competition?

The ultimate aim of this challenge is to push rare disease studies at any phase of drug development and bring awareness to the current broken system for diagnosing, treating and curing rare diseases. Although the prizes themselves cannot find a cure, they reduce the barrier toward a solution for many diseases.

Prizes for this challenge include $10,000 cash and greater than $400,000 of donated research services from 19 participating service providers; this is a great indicator that the life science industry wants to help treat rare disease. If we can improve the standard of care for just one rare disease, we will bring relief to thousands of people, very likely children, who currently suffer. We think that is a dream worth fighting for.

Let’s say I have a child with a rare disease but I am not a scientist. How can I tap into this money to help my child?

Families with a child afflicted with a rare disease will have the opportunity to work with a network of academic researchers and use donated research services to develop research proposals tailored to their child’s needs.

The Computer History Museum in Mountain View was the site of last weekend's Open Science Summit.

The Rare Disease Science Challenge isn’t the first competition you’ve been part of. What are some of the other competitions and have they yielded any results yet?

Our first Open Science Challenge was open to all Bay Area residents who wanted to take their research ideas to the next level. There were two citizen scientist winners of the challenge: Ryan Bethencourt and Derek Jacoby. Both received one-year memberships to the BioCurious community lab space in Sunnyvale as well as $5,000 to fund their research project.

Bethencourt is currently working on developing new ALS therapeutics, and Jacoby is seeking to find a probiotic method of improving uric acid clearance to combat gout.

Our second Open Science Challenge in the Tri-State (NY) area will be ending soon and we have ongoing high school science competitions in San Diego, Boston, and New York.

You participated in the Open Science Summit in Mountain View this past weekend. What is open science, and how is the work you do connected?

Assay Depot has always operated on a mission of “empowering scientists.” What that boils down to is continuously asking ourselves “does this help scientists?” We started learning about the Open Science movement and realized the question is much more far-reaching than we thought. For the unfamiliar, Open Science is based on the concept that science can be done by anyone and community collaboration is the key to innovation. Scientists, amateur or professional, were empowering themselves by having meet ups, organizing online discussion, pooling resources, and testing ideas outside of traditional laboratories. “Does this help scientists?” expanded beyond product design and became a question we asked of our community efforts.

You run an online site called Assay Depot. Tell me a bit about the site and what you hope to accomplish with it.

Not long ago, the barriers to conducting drug discovery research stood high. Today, thanks in part to the Internet and the advent of research marketplaces like Assay Depot, anyone with a laptop and access to funding can embark on the search for a cure.

Assay Depot is now the world's leading provider of outsourced scientific services. The company operates a network of online research marketplaces that dramatically streamline purchases between scientists and more than 7,000 global research vendors. Assay Depot is changing the way life science research is done, one researcher, one vendor, one pharmaceutical customer at a time.

If I am a high school teacher and I’d like to run an experiment but don’t have the facilities, is Assay Depot available to me? Or is it just for professional and academic scientists?

Yes, Assay Depot is empowering not only scientists and researchers, but also citizen scientists from any background, from artist to teacher to tinkerer. Assay Depot has recently helped start up a community lab in Carlsbad, CA, which should open early next year.

What did you do before starting the site? How did you come up with the idea?

I have spent most of the past 28 years either managing research groups or running my own experiments at the lab bench.

My co-founders Chris Petersen, Andy Martin and I started Assay Depot with the idea of enabling personalized cancer research. We quickly realized though that we could fill a much more valuable and unique niche by creating a cloud-based marketplace that connects scientists with research providers, and Assay Depot was born.

Prior to starting Assay Depot, I co-founded Kalypsys, a fully integrated drug discovery company that raised over $170 million in venture funding and put five drug candidates into human clinical trials in 2001.


Prior to Kalypsys, I directed lead discovery at Tularik, a highly successful biopharmaceutical company purchased by Amgen for more than $2 billion. I carried out postdoctoral work in Cell Biology at Harvard Medical School after receiving a PhD degree from the Department of Biochemistry & Biophysics at the University of California, San Francisco (UCSF).