The Saga Continues: Ethan Perlstein, Indie Scientist, Part 3

Today Ethan Perlstein joins us on the program for a third time. His path as a ‘rogue scientist’ has become a bit of a case study here at Mendelspod as we look into alternate paths for scientists and for drug development.

When Ethan was first on the program, he’d just declared his independence, or break from the world of academia. He urged other scientists to do the same and to take to social media to upend a system that was creating the “postdocalypse.” And he didn’t just take a job working for big pharma. He wanted to carry on with the science that intrigued him: a new model of drug discovery.

The second time we featured Ethan he was set up at the QB3 incubator space in San Francisco as the Perlstein Lab, a hybrid of academic lab and regular biotech startup. He had secured some seed funding, and hired five scientists. It was then that he announced a new drug discovery platform using simple animal models (yeast, frutiflies, worms) that would provide proof of concept at a much lower price than what you see with traditional biotechs and pharma.

That was in 2014. Now, at the end of 2016 Ethan has some updates to share. Last month he announced a licensing and investment deal with the drug giant Novartis. He can boast of his first drug candidate, Per101. And he’s a graduate of the Silicon Valley’s business maker, the well known Y Combinator.

Ethan says the Novartis deal is more than just his first revenue generator, it validates the platform of using simple animal models. The deal also boosts what he calls his precision 'business development model.”

“People talk about precision medicine, but they don’t ever talk about precision business development. And that part of the discussion is critical, because you need to think about not only the science that gets the job done, but how the company practically gets put together.”

Working on rare diseases, Ethan is going directly to patients, albeit rich ones at first, with a low cost drug discovery method, creating a new business model that cuts past traditional funding partners and offers a somewhat more academic setting. Perlara, the newest name for Perlstein Lab, is a public benefit corporation.

What was Ethan's experience at the tech dominated Y Combinator? How has being a public benefit corp impacted his decisions this past year? And, come on, if he looks, smells, and acts like a regular old biotech entrepreneur, is he still a rogue scientist?

Here it is. The Indie Scientist, Part 3.

September 2016 with Nathan and Laura

There were many headlines this past week heralding the first three parent baby to be born. But in fact, as our commentators point out in today’s look back on last month’s genomics news, three parent babies have been around for some time. So why are couples going to Mexico for mitochondrial transfer today? Why is it not legal in the U.S.?

Nathan points out that every one of our ancestors back ten generations ago gave us a hundred times more DNA than the mitochondrial donor might give to a three parent baby. Yes, the donor is a parent, he says, but she’s also just a distant cousin. "This shouldn’t freak anybody out.”

Laura doesn’t like the term “three parent babies” at all:

"It’s like in the early days when we went around, ‘oh is that a test tube baby?’ This is a human being, a kid on this planet—you can’t call this boy a ‘three parent baby’. He has two parents. They are the people raising him.”

Our second story involves regulation as well. This month the FDA approved Sarepta’s drug to treat Duchenne muscular dystrophy. Here’s a new drug that could help a patient population desperately in need, and yet, most of the key opinion leaders in our industry are very disappointed with the FDA. Why? The answer comes down to whether the FDA should include more than "just the science” in their decisions.

A Maniacal Commitment to Science: Peering into Regeneron’s Genetics Center with Jeff Reid

Today we feature a pharma company that has been around for some time but recently getting more media coverage for the impressive scale of their new genetic center. Regeneron Pharmaceuticals, insiders joke, has been an overnight success that took 25 years.

One might think every big pharma company has their own genetic center for internal R & D. But today’s guest, Jeff Reid, Executive Director of Genome Informatics at the Regeneron Genetic Center (RGC), says that actually deep genetic research is often outsourced.

In just two years, the RGC has built an impressive sequencing lab and announced large partnerships with healthcare systems and academic centers that rival major government projects. One such collaboration with Geisinger Health System involves the sequencing of 100,000 genomes. Already, the RGC has sequenced over 100,000 exomes and has plans to sequence 500,000.

“What we’re doing is quite different,” says Jeff. "We are envisioned as a large scale academic genome center embedded in a pharma company."

Jeff says the strategy is to not only go wide with studies of large numbers of patients for the purpose of finding very rare variants, but to go deep as well. Big numbers can be distracting, he points out, saying that some times they get more insight off a small project, such as the treatment of children with a rare genetic disease.

“There are strategies all across the spectrum of project size,” he says.

Set up in an age when compute and data storage are no longer an issue, the RGC has become the first large scale genetic center to be entirely in the cloud. What is the major informatics challenge for Jeff and the center? And what does having such a large scale genome center mean about Regeneron and where we are today with genomic medicine?

It’s Not Really Bulls and Bears: John Carroll on His New Gig, the Brexit, and a New Metaphor for the Market

John Carroll has been the editor-in-chief at Fierce Biotech for thirteen years. Now he's moved to a new gig. Two weeks ago, he and a former colleague launched a new and independent life science media site, Endpoints.

In today’s interview, John says that he’s trying to do the impossible with Endpoints. He wants to turn the lights on and have an immediate audience. He thinks he can do it this time because he and his partner, Arsalan Arif, are bringing 35,000 social media followers with them. This gives them an instant audience and a new kind of leverage for building a new media site.

What is John hearing from drug developers about the Brexit? And what is the best metaphor for the market he’s ever come across?

Join us for a unique look at the landscape of life science media with one of the industry’s top journalists as he transitions to this new gig.

Bringing Home Some Diagnostics Gold: Brad Gray, NanoString Show How It’s Done

You hear it everywhere. And it’s getting old. That "diagnostics is a tough slog.” That it’s the “redheaded stepchild of healthcare.”

And today’s guest doesn’t disappoint, repeating both these phrases. But Brad Gray and NanoString can claim some big “slogging" success. They’re coming out on top in diagnostics through some clever business strategy built on a solid platform. Made CEO at just 33 years of age, Brad has taken NanoString public and overseen a successful expansion from the research to the clinical market.

In his interview, you’ll hear Brad lay out the three pronged approach at NanoString. Starting as a spinout from Lee Hood’s Institute for Systems Biology, the company began in the life science tools space with their nCounter platform. The machine proved a favorite for cancer researchers because of its ability to look at single molecules of nucleic acid. When the company pole-vaulted into the clinical space, rather than set up their own lab and do the testing themselves--such as diagnostics pioneers, Myriad Genetics and Genomic Health--NanoString opted for a decentralized model. They did just the kind of thing that makes the FDA happy. They created a “push button simple” platform with kits so that clinical labs could do the testing themselves in a highly reproducible fashion.

But that’s not all. Under Brad’s leadership, the company has made several major companion diagnostics deals with big pharma. An agreement with Merck announced earlier this year delivered NanoString an upfront payment of $12 million. That’s a nice boost for a diagnostics company slogging away at reimbursement.

“The Merck deal is especially exciting because it’s the first major molecular diagnostics partnership in the field of immuno-oncology,” says Brad. "And the scale of it makes it the largest companion diagnostics deal ever announced, in economic terms.”

And what of the reimbursement slog for theirs flagship Prosigna breast cancer assay? NanoString can now boast Medicare coverage in all 50 states.

That’s about as good as it gets in our industry.

Flipping Drug Development Upside Down: Niven Narain, BERG Health

The promise of rational drug design has driven pharma companies for years. The history of the industry has been one of trial and error, or “guess and check”, as scientists often say. Companies have screened thousands and thousands of compounds looking for one that might work—the proverbial needle in the haystack. With the arrival of molecular profiling and an explosion in understanding of basic biology, many in our field have hoped to make drug development more predictable: to start with the biology, and with the aid of new computing power, design a drug to work in a specific biological setting. But instead we've been making the haystack bigger. When will we see a tipping point where more drugs than not are derived through logic rather than luck? Though there have been great examples—Merck’s Truspot being the first and Novartis’ Gleevec perhaps the best known—rational drug design still remains a mostly unfulfilled promise.

BERG Heatlh, a Boston based pharma company, is working to change that with their AI platform and a new “back to biology” approach. Today we talk with BERG CEO, Niven Narain, who also is a co-founder of the company.

“We’re flipping the entire [drug development] model upside down,” Niven says. "We’re going to ask the patient biology what has gone wrong and generate as much of the omics data as we can from that patient. We then correlate that to their histories and health records in a population based way and compare it to healthy individuals. Out of that analysis we derive potential drug candidates and biomarkers.”

BERG has secured a considerable amount of press with the attention grabbing headline, “company using AI to cure cancer.” Backed by billionaire Carl Berg, the company can’t be faulted for thinking small. But are they doing anything different than other pharma companies? In today’s interview, Niven says they are looking at 15 trillion data points on a single sample. And the scientific approach is perhaps more robust that we’ve seen in the past. Rather than relying on the latest biomarker studies, the company takes a non-biased approach by letting the patient’s data create it’s own picture of the biology.

Why Drugs Are Priced So High and Diagnostics So Low

Pharma companies face escalated flack over high drug prices. Meanwhile the diagnostics industry toils away at comparative pennies to the dollar.

Today's guest, the irreverent and hilarious blogging radiologist, Saurabh Jha (@roguerad), offers an alternative view to the industry's knee jerk reaction to the drug pricing conversation. Saurabh's experience as a radiologist grappling with a medical culture of over diagnosis is on full display along with his typical biting sarcasm. (As background for the interview, we recommend his brilliantly titled blog, The War on Death). To those working to commercialize molecular diagnostics, Saurabh offers some bits of wisdom.

“I do think diagnostics should be more expensive, but I think they should be more expensive because they have some kind of expertise giving meaning to them. I’m very reluctant to simply giving people numbers. Numbers without context, by and large, is very dangerous,” he says.

One Less Voice on the BioBeat - Luke Timmerman Ends Stint with Xconomy

Monday mornings won’t be the same in biotech.

Luke Timmerman announced last week that he’ll no longer be writing his Monday morning BioBeat column or serving as National Biotech Editor at Xconomy.

Timmerman’s voice has stood out in our industry for consistency, optimism, pragmatism . . .  and great sports analogies.  


Source: Xconomy

Clinical Trials and Social Media with Craig Lipset, Pfizer

Podcast Sponsor: The Scientific Services Marketplace


Craig Lipset, Pfizer Bio and Contact Info

Bridging Listen (5:48) Bridging clinical research with eHealth

<img src="/images/speaker.jpg" alt=What happens when trial patients connect with each other online?"/> Listen (2:09) What happens when trial patients connect online?

How have patients changed Listen (4:01) How have patients changed?

What has Pfizer changed Listen (2:51) What has Pfizer changed?

Challenges with mobile health Listen (4:09) Challenges with mobile health?

How is Pfizer using social media Listen (4:58) How is Pfizer using social media?

Social media guidance Listen (9:44) Social media guidance

Pfizer and innovation Listen (1:28) Pfizer and innovation

Clinical trials in 5 years Listen (5:14) Clinical trials in 5 years

We talk with Craig Lipset of Pfizer about how social media is changing clinical trials. Craig has good support at Pfizer to launch innovative trial programs. At Pfizer he is bridging clinical research with eHealth technologies and exploring the opportunity to further inform and empower both patients and providers in the age of social media. Craig served as Vice President of Program Management at Adnexus Therapeutics and was part of the founding management team for Perceptive Informatics. He is the 2010 recipient of the DIA's Outstanding Service Award.

Start-up 'Helps Out' FDA: Brian Overstreet with

(Read our blog and watch a video about visiting Brian in Healdsburg, CA "Having Your Wine and Drinking It Too")

Podcast Sponsor:

Open Science Summit, Oct 22-23, Mountain View, CA Register Today


Brian Overstreet, President, Adverse Events, Inc. Bio and Contact Info

FDA site offers no easy way to research side effects Listen (5:39) FDA site offers no easy way to research side effects

What does RxFilter(TM) do? Listen (3:42) What does RxFilter do

Site free for patients Listen (4:21) Site free for patients

Why Healdsburg Listen (3:56) Why Healdsburg?

Living a dream Listen (0:47) Living a dream

Over 500,000 adverse events are reported for medications yearly to the FDA; estimated to be only 10% of all actual adverse events. If you experience a negative side effect taking a drug and want to report it, or if you are a drug developer and you want to research negative side effects, you can at in a section called Adverse Event Reporting System. There you will find tens of thousands of adverse events listed and connected to thousands of drugs. Or you can go to, a new website that provides accurate, real-time information on adverse drug events that are reported to the FDA. Using a unique data sourcing method, the site is a user-friendly, fully searchable, database of over 4,000 approved medications. Joining us today is the President of AdverseEvents, Brian Overstreet. Brian just launched his new company at Health 2.0 on Sept 26-27.

New to Mendelspod?

We advance life science research, connecting people and ideas.
Register here to receive our newsletter.

or skip signup