science funding


What Does the Election Mean for Genomics? November 2016 with Nathan and Laura

While everyone is asking what will become of Obamacare, we ask our regular commentators, Nathan Pearson and Laura Hercher, specifically about genomics and medicine.

Nathan begins by saying that data scientists everywhere should be humbled. Does the failure to predict the election send out warnings about big data predictions in genomics?

Laura points out that Obamacare covers many of the new genetic tests which have become available in the past decade, such as screening tests for hereditary breast and ovarian cancer syndrome and lynch syndrome. Coverage for these tests is now up in the air.

"It is cruelly absurd to talk about the value to the human race of identifying the people with these syndromes if we don't then give them the ability to act on the information," she says.

No matter what happens to Obamacare, isn't there bipartisan support for genetic testing and for research funding? (See the passage yesterday of the 21st Century Cures Act.)

Both Nathan and Laura agree that genomic medicine will continue apace. However, they worry that under a Trump administration the less fortunate will become even more vulnerable and have less access to improvements in healthcare. They point to an area of testing that is already highly politicized: prenatal screening. Will women lose access to testing in an era that reverses gains made in women's reproductive rights?

We finish with a local election in the Florida Keyes where residents approved the use of Oxitec's genetically modified mosquitos. Fear, Laura points out, can quickly change suspicion into acceptance.

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.

Can You Name the World’s Largest Single Disease Research Charity?

Let’s take a break from the US and head over to the UK, home of the world’s largest single disease medical research charity.

Cancer Research UK (CRUK) raises five hundred million pounds a year for research and drug discovery into any and all of the two hundred plus types of cancer. The charity is extremely well integrated into U.K. culture, and uniquely English in that the donations are mostly small and come from all corners of society. A third of CRUK’s funding comes from donations averaging £10 or less.

Allan Jordan is head of chemistry for the drug discovery unit of CRUK. On today’s show he says that the democratic funding of the charity gives them a great deal of flexibility to do early stage drug discovery. Whereas a big pharma or biotech has to devote their resources to limited assets, or drugs, CRUK is able to spend more on basic biology research and follow the science into any type or cancer or multiple cancers.

There are very few conditions,” says Allan about his drug discovery unit in Manchester. "We don’t have to be specific about any particular disease area; we don’t have to be experts in one disease at the expense of all others. We can tap into that UK-wide expertise and network that can help us understand the biology.”

How is the charity working with the UK's national healthcare system? And does Allan hear the same kind of skepticism that we hear in the U.S. about precision medicine in oncology?

We’re Over Halfway There: Baylor's Richard Gibbs on Clinical Genetics

There’s a basic assumption in our field today that has been around for some time. We think of medicine as on a direct and even continuum with science. That discoveries in genomics, for example, will lead directly to breakthroughs in medicine. But the breakthroughs on the medical side have been much more rare to date than those coming from the study of biology and genomics.

Richard Gibbs is the Founder of the renowned Genome Sequencing Center at Baylor College of Medicine. He and his team were one of five worldwide sites contributing to the Human Genome Project (HGP). In today’s interview we find out what the sequencing pioneer has been up to since the days of the HGP and what his take is for how well genetic science is translating into clinical care.

In fact, Richard is willing to put a number on how far we’ve come.

“There’s a trajectory that began just about the time the Human Genome Project was being conceived through to this futuristic image of medical genomics where complete genomes are actually part of medical care,” he says. "That journey is not yet complete. We are somewhere between 50 and 90% there.”

Richard says that the HGP was actually a departure from what was typical in the field of human genetics. That it was a science project done more purely for the sake of science. Most of the history of human genetics research has been practical medical or clinical projects.

One of the areas where Richard’s team has made a big impact is in collaboration with the NHGRI's Center for Mendelian Diseases. The team is also participating heavily with the NIH’s Undiagnosed Disease Network. What is the difference between a Mendelian and a rare disease? What are the center’s solve rates for each of those areas?

We round out the discussion with a look at how Richard and his team get the 'best quality genomes' for their projects, an issue of utmost importance in the clinic.

Luke Timmerman on His New Biography of Lee Hood

There is tons of life science journalism. Our coffee tables and inboxes fill up each week with that quarterly or that daily. We sift through headlines and product advertisements to assess what’s going on in our industry. It’s our job to know. In this age of several-times-per-day newsletters and 24 hrs a day Twitter, we catch what we can.

And occasionally, we come across a carefully written piece or a well done interview, and we take a moment to realize with some awe the history that is being made in our industry.

Occasionally. Which is why a new book out by veteran biotech journalist and the guest of today’s show, Luke Timmerman, is such a rare treat.

Hood is a thrilling ride through the life of the visionary biologist, Lee Hood, told by someone who is not afraid to show the shiny and the not so shiny. From his boyhood in Montana to being chair of the biology department at Caltech where he oversaw the invention of the automated DNA sequencer, to being recruited to Seattle by Microsoft’s Bill Gates, Hood’s journey becomes the perfect vehicle for Timmerman to probe into the messy corners of science and put an intimate, human face on the history of biotech. Covering Hood’s move to the University of Washington as a young Seattle based reporter, Timmerman has known Lee Hood for several years. It's a full scale biography, efficiently and confidently written with an insider's perspective and access. Timmerman says it's an “unofficial biography,” meaning Hood was supportive of the project, but Timmerman had full freedom.

Playing historian has been somewhat of a fantasy for the long time journalist.

"There are things that are happening in the moment which a journalist can call people on, but you don’t really get the whole story. There’s only so much people can say and there are not a whole lot of documents that come available when you’re on deadline. But when you’re a biographer, and you have the luxury of time, and people have moved on, things become a lot less sensitive. People become more willing to talk, and a whole lot of documents become available through the public record.”

Who is this man, Lee Hood, and how has he impacted our industry? In the book, we read of the time when Hood holds a press conference to announce his team has done it—they’ve got an automated DNA sequencer. But, standing at perhaps the pinnacle of his career, Hood forgets to mention the "team" part. It’s a flaw that will go on to haunt what by any measure has been a remarkably successful career.

What impact has the subject made on the author? And what does Timmerman hope for the book?

To round out the interview, we get Timmerman’s thoughts on his new gig, the Timmerman Report, and the recent Sarepta decision by the FDA.

How Is the Brexit Impacting Genomics? with Clare Turnbull and Hadyn Parry

Today's guests have been separately on the program recently. And we've asked them, both Brits, to come back on for a discussion of the Brexit. Clare Turnbull is Clinical Lead for the 100K Genomes Project Cancer Program at Genomics England. Hadyn Parry is the CEO at Oxitec, a company based in Oxford which is already selling their genetically engineered mosquitos into Brazil to deal with viral diseases like Zika and Dengue Fever.

The first question we throw at them is whether they are still in shock over the outcome of the vote. Clare resides in London, and Hadyn in Oxford—both places that voted overwhelmingly to remain in the European Union. In fact, Hadyn says he doesn’t know a single person who voted “leave.”

Clare says Genomics England is funded through 2020, so in the short term, the 100K Genomes Project is secure. She also has a role in academia where most genomics research takes place. There, she says, they are being hit with an immediate impact. 10-15% of grant funding in the British system comes from the EU.

“People are on grants which are running. People have won grants that not yet been awarded. And people are looking to collaborate on grants in the medium term future. And one becomes a pretty unattractive collaborator on an international, pan-European grant when it’s uncertain whether or not we’ll be in the EU. The uncertainty is pretty immediate,” says Clare.

Hadyn says the uncertainty is certainly affecting business in the short term. But points out that in the longer term, the UK is solid. They have a great tradition of science, and they have four of the top ten universities in the world. And their entrepreneurial culture is strong. It’s much easier to do a startup biotech company in the UK than in the rest of Europe.

“The longer term, I’m quite comfortable with,” he says. "It’s all about the uncertainty in the short term. The sooner we move to understanding the rules of the game, the sooner the short term uncertainty can be removed.

For now, both see a country still in shock and denial.

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.

With 10K Genomes Sequenced, Genomics England in High Gear: Clare Turnbull, Clinical Lead

We’ve heard on the program over the past few years that genomic medicine will probably take off first in a country with a centralized health service. And when the U.K. announced their 100K Genomes Project at the end of 2012 with the creation of Genomics England in 2013, it was certainly a bold visionary move to do just that—to put the entire country on a progressive path toward precision medicine for all.

So with 10K genomes sequenced, how is the project going?

“We’re still early days in the program in delivering it,” says today’s guest, Clare Turnbull, Clinical Lead for the Cancer Program. "Because the National Health Service in England is a single health care provider, it is possible to leverage carrots and sticks to make sure things happen. This gives us a lot more opportunity to effect change than in a more disparate service such as in the U.S.”

What are those carrots and sticks? What new paradigm shifts must take place, and what are the biggest challenges?

Beginning with rare diseases and cancer in this first project, the overall goal, Clare says, is to bring next generation sequencing technologies "full scale in their entirety into our healthcare service, and build all the structures that are necessary to use these types of tests--in particular, whole genomes--as routine investigations in every patient in every hospital within our service.”

Clare says she is a fan of Mendelspod because we provide "a very American perspective" on the same challenges and opportunities.

April 2016 with Nathan and Laura: Big Money, More CRISPR Studies, Genomic Superheroes, and a Pot Chaser

This month we saw Big Money being infused into genomics and other life science research projects. There’s no question that science is big business, but do we see improved healthcare as a result?

Was the NIH too hasty in it’s ban on gene editing of human embryos?

Superheroes are lurking among us everywhere . . . or so the mainstream media would have us believe in their take on a new study from the Icahn School of Medicine.

Join us for our month-in-review program to hear what our regular commentators, Nathan Pearson and Laura Hercher, have to say about these questions. Stay tuned to the end for a pot chaser.



New to Mendelspod?

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

or skip signup