Do We Really Need Another TV App?

I read an article today in Vulture, the culture and entertainment site from New York Magazine about how Quibi, the long-hyped, mobile-centric streaming platform devoted to short-form programming had raised $1.75 billion. Quibi, the brainchild of Jeffrey Katzenberg, the former Disney studio head and DreamWorks co-founder, had promised to reinvent television by streaming high-quality content in ten-minute-or-less chunks to “the TV in your pocket.” Given that  Quibi reportedly lost 90 percent of early users after their free trials expired, it seems like another example of a ton of money invested in an app that maybe people don't need. Which got me thinking about what a difference $1.75 billion would have made if invested in medical research. In particular, the impact almost 2 billion dollars would have on the research labs that are searching for treatments for patients with unmet medical needs.

To give you an idea of what a billion dollars in research looks like, Mass General Research Institute is home to the largest hospital-based research enterprise in the United States. It has a budget of $1 billion. Mass General ranks 1st among independent hospitals in the amount of annual funding received from NIH, and 10th among all institutions. Research at Mass General takes place in 30 departments, centers and institutes across the hospital. The research community includes more than 8,500 people and covers 1.3M square feet of research space in Boston, Cambridge and Charlestown. This community has 2,000 principal investigators and there are 1,200 postdoctoral fellows and 700 graduate students training in research at Mass General at any given time. Over one-third of the 3,000 physicians at Mass General are also conducting research. Now imagine what $1.75 billion looks like.

But my interest isn't in the big institutions themselves. My advocacy is for the promising research projects at specific individual labs, and the people behind the scenes with the big ideas. Nearly all academic researchers in the medical sciences rely on outside grants in order to pay salaries of the people in their labs doing the experiments, and to buy their equipment and supplies. The big institutions where they work do not fund them. But that pool of funding is shrinking, grant approval rates are dropping, and researchers are stuck spending more and more of their time and energy applying for grants. Many scientists are saying that the system is broken and the consequences could be disastrous. By some estimates, many top researchers spend 50 percent (or more!) of their time writing grants. Interdisciplinary research is less likely to get funding, meaning critical kinds of research don’t get done. Actually most of the researchers I know tell me that they spend up to 75 percent of their time fundraising. Philanthropic support is so important to keep these researchers going.

This is where the perspective of $1.75 billion dollars comes in. Imagine how transformative that money would be at say, $1 million per lab. That could be 1,750 different labs getting a million dollars (which is a lot believe it or not!). Researchers need to fund their own labs and this capital would help with supplies, equipment and added postdoctoral staff. And think of the labs and companies that are working on childhood and rare and orphan diseases. It is estimated that there are between 5,000 and 8,000 rare diseases and about 50 percent affect children. A rare disease in the United States is defined by the 1983 Orphan Drug Act as a condition that affects fewer than 200,000 people. One rare disease that I am most familiar is Fragile X syndrome, which has an estimated 90,000 patients worldwide. An example of a research lab that needs funding to address this unmet medical need is the laboratory of Guoping Feng, a renowned, National Academy of Sciences (NAS) researcher at the Broad and McGovern Institutes at MIT. I have spoken with Guoping and his team about his interest in starting a comprehensive Fragile X research program that would ultimately test gene therapy approaches using Fragile X mouse and monkey models as a potential cure for patients with Fragile X.  They are already making progress in another single gene neurodevelopmental disorder called SHANK3. But the initial research for Fragile X needs seed funding that is hard to find. To start developing the mouse and monkey models would require about a $1 million investment. Seems like such a small amount compared to the $1.75 billion invested in the Quibi mobile app.

Using the genome-editing system CRISPR, researchers at MIT have engineered macaque monkeys to express a gene mutation linked to autism and other neurodevelopmental disorders in humans. These monkeys show some behavioral traits and brain connectivity patterns similar to those seen in humans with these conditions. Mouse studies of autism and other brain disorders have yielded drug candidates that have been tested in clinical trials, but none of them have succeeded. Many pharmaceutical companies have given up on testing such drugs because of the poor track record so far. The new type of model, however, could help scientists to develop better treatment options for autism spectrum disorders. Feng wants to engineer a Fragile X monkey model because he believes Fragile X is one of the best test cases for gene editing across the autism spectrum. Think about the implications for the larger autism community! And ponder how many research projects could be funded with the amount of money Quibi's investors put in.

I am not naive to the fact that the backers of companies like Quibi are obviously investing in something they believe will become a huge success and provide a return on their investment. I totally get that. But...I also wonder if those investors realize that it doesn't take a huge amount of money to have huge impact in the research world. Big ideas are out there. And sometimes a scientist is working on one disease area but his/her ideas might apply to other areas...and that's where it's hard to get funding. I am on a mission to connect brilliant, passionate researchers with philanthropists who have the means to make a difference in millions of peoples lives. It's about the people. People like my friend Guoping Feng. You should meet him...you will then understand why I advocate for researchers like him. And you will realize how sincere he is about finding treatments and cures that may help you or me...or someone we love. Investing in his lab may not get you a 10x return, but you will feel good knowing that you could change the world...just not on a mobile app.

#gratitude #research #trust