Can We Con­quer All Dis­eases By The End Of The Cen­tu­ry? An In­ter­view with Chan Zucker­berg's Cori Bargmann.

As pres­i­dent of Chan Zucker­berg Sci­ences, Cori Bargmann’s job is “to build out the strat­e­gy and part­ner with acad­e­mia, biotech and en­gi­neer­ing” to help re­al­ize the vi­sion of the ini­tia­tive. End­points News is pleased to re­pub­lish the fol­low­ing in­ter­view for our au­di­ence. This ar­ti­cle orig­i­nal­ly ap­pears on Kaiser Health News, a na­tion­al health pol­i­cy news ser­vice that is part of the non­par­ti­san Hen­ry J. Kaiser Fam­i­ly Foun­da­tion.

The goal is lofty and ex­pan­sive: to cure, pre­vent or man­age all known dis­eases by the end of the cen­tu­ry.

Face­book Chief Ex­ec­u­tive Of­fi­cer Mark Zucker­berg and his wife, pe­di­a­tri­cian Priscil­la Chan, an­nounced last month that they planned to in­vest $3 bil­lion over 10 years to be­gin tack­ling every­thing from Alzheimer’s dis­ease to the Zi­ka virus.

The mas­sive un­der­tak­ing is part of the Chan Zucker­berg Ini­tia­tive, a wider ef­fort to fund in­no­va­tion and pro­mote equal­i­ty in health, ed­u­ca­tion and en­er­gy. The cou­ple es­tab­lished the ini­tia­tive last year af­ter the birth of their daugh­ter, Max­i­ma.

The first step in the dis­ease-bust­ing project, called Chan Zucker­berg Sci­ence, is a $600 mil­lion in­vest­ment in a new bio­med­ical re­search cen­ter in San Fran­cis­co called the Bio­hub — a col­lab­o­ra­tion among Stan­ford Uni­ver­si­ty, the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co and the Uni­ver­si­ty of Cal­i­for­nia, Berke­ley.

To learn more about Chan Zucker­berg Sci­ence, we talked to Cori Bargmann, a neu­ro­bi­ol­o­gist and pro­fes­sor at The Rock­e­feller Uni­ver­si­ty, who was named pres­i­dent of the or­ga­ni­za­tion. The con­ver­sa­tion was edit­ed for clar­i­ty and brevi­ty.

Q: How do you hope to ac­com­plish such a far-reach­ing goal?

Cori Bargmann (John Ab­bott/The Rock­e­feller Uni­ver­si­ty)

We have three pil­lars of how we plan to achieve that goal. The first is to bring sci­en­tists and en­gi­neers to­geth­er. Our sec­ond … is to build tools and tech­nolo­gies that will em­pow­er the en­tire sci­en­tif­ic com­mu­ni­ty to be more ef­fec­tive and suc­cess­ful. Our third pil­lar is to build a move­ment to in­crease sci­en­tif­ic fund­ing for re­search na­tion­wide and world­wide.

Q: Treat­ment, cures or even man­age­ment has proven to be elu­sive for some dis­eases, in­clud­ing Alzheimer’s and cer­tain can­cers. Is this project re­al­is­tic?

Eighty years ago, the very first an­tibi­ot­ic was de­vel­oped. Be­fore that point, the four ma­jor killers were all in­fec­tious dis­eases, none of which are in the top 10 any­more. Even in our life­times, we have seen so much progress. When Pres­i­dent [Dwight D.] Eisen­how­er had a heart at­tack, he was put on bed rest. Be­tween statins and blood pres­sure con­trol and then by­pass surgery, stents, valve re­place­ment and heart trans­plants, look what has hap­pened to car­dio­vas­cu­lar med­i­cine. Can­cer is go­ing through a rev­o­lu­tion right now. Chemother­a­py was de­vel­oped in the 1950s and 1960s. We are now see­ing the re­fine­ment of that in­to tar­get­ed ther­a­pies that are mak­ing a huge dent.

There are some re­al­ly dif­fi­cult prob­lems out there. But 80 years is a long time. And one of the things I like about the Chan Zucker­berg Ini­tia­tive is that long time­frame. … This long time­frame makes this [project] more re­al­is­tic than it sounds.

Q: What are the ben­e­fits and chal­lenges of bring­ing en­gi­neers and sci­en­tists to­geth­er?

Bio­med­ical sci­ence is in­creas­ing­ly com­pli­cat­ed. In or­der to re­al­ly make progress, we think there is val­ue to hav­ing physi­cians who have in­sights in­to dis­ease process­es, as well as sci­en­tists who can dig in to do bi­o­log­i­cal stud­ies to un­der­stand them. But in­creas­ing­ly, we al­so need com­pre­hen­sive col­lec­tion and analy­sis of the da­ta that we gath­er, and fast and ef­fi­cient dis­sem­i­na­tion of that knowl­edge. That is where en­gi­neer­ing comes in.

As Mark Zucker­berg says, “You can en­gi­neer any sys­tem to make it more ef­fi­cient.”

The chal­lenge of bring­ing sci­en­tists and en­gi­neers to­geth­er is that they come from dif­fer­ent train­ing and dif­fer­ent cul­tures. They don’t nec­es­sar­i­ly speak each oth­er’s lan­guage. What is spe­cial about the Bio­hub is that sci­en­tists and en­gi­neers are go­ing to be work­ing side-by-side in the same fa­cil­i­ty so that they are re­al­ly em­bed­ded in each oth­er’s cul­tures.

Q: How will the or­ga­ni­za­tion work with oth­er fun­ders and in­crease fund­ing for sci­en­tif­ic re­search?

It’s very im­por­tant to do some­thing new when you have new re­sources. The unique an­gle that we can bring is an em­pha­sis on en­gi­neer­ing and on tech­nol­o­gy. These are ar­eas that are not nec­es­sar­i­ly part of the tra­di­tion­al bio­med­ical en­ter­prise. We al­so have the abil­i­ty to be nim­ble and flex­i­ble. We can try new things and we can al­so change di­rec­tions very quick­ly com­pared to the way that a fed­er­al or­ga­ni­za­tion fund­ed by cit­i­zen tax­pay­ers can.

We are ab­solute­ly com­mit­ted to work­ing with oth­er or­ga­ni­za­tions through­out what we do. A great ex­am­ple of that is the cell at­las project. It’s a project to un­der­stand all the cells in the hu­man body. This a huge project and not some­thing that we could do on our own, and we don’t want to.

We are in a good po­si­tion to com­mu­ni­cate the ad­vances in sci­ence and the pos­si­bil­i­ties in sci­ence. We are in a good po­si­tion to start to make ad­vances that might en­cour­age oth­er phil­an­thropists to start their own re­search pro­grams. Set­ting a suc­cess­ful ex­am­ple, en­gag­ing the pub­lic and cit­i­zens, en­gag­ing politi­cians, and bring­ing for­ward the mes­sage that this is pos­si­ble, are the ways to build sci­ence.

Q: Is gov­ern­ment fund­ing prob­lem­at­ic for these types of projects?

The Na­tion­al In­sti­tutes of Health is a fan­tas­tic or­ga­ni­za­tion that does what it does re­al­ly, re­al­ly well. But it has a par­tic­u­lar way of do­ing that. For ex­am­ple, the Na­tion­al In­sti­tutes of Health is built around a se­ries of in­sti­tutes, each of which fo­cus­es on a spe­cif­ic or­gan sys­tem or a spe­cif­ic dis­ease. There’s the Na­tion­al Can­cer In­sti­tute, there’s the Na­tion­al Eye In­sti­tute. They can fo­cus and re­al­ly iden­ti­fy what are the prob­lems in can­cer right now, what are the prob­lems in the eye right now. What they are less like­ly to do is to think, “What are the tech­nolo­gies that we need that would dri­ve all of these projects for­ward?”

Q: Part of the goal is to de­vel­op tools that could help lead to dis­ease treat­ments or cures. What is an ex­am­ple of such a tool?

We are just get­ting start­ed on what our projects are. Stephen Quake, one of the co-pres­i­dents of the Bio­hub, de­vel­oped the blood test that al­lows you to test a fe­tus for Down syn­drome with­out am­nio­cen­te­sis. It’s a tool to ad­vance med­i­cine. It’s a tool to help preg­nant women and their chil­dren. What oth­er kind of blood tests can be de­vel­oped?

Joe De­Risi, the oth­er co-pres­i­dent of the Bio­hub, wants to de­vel­op tools and meth­ods to de­tect and re­spond to in­fec­tious agents, emerg­ing dis­eases, virus­es, bac­te­ria. You pick up the news­pa­per and you hear about the Ebo­la virus or the Zi­ka virus. Cre­at­ing bet­ter tools for know­ing where these or­gan­isms are, whom they are in­fect­ing and how they are spread­ing is an­oth­er ex­am­ple of how tools could have ef­fects on med­i­cine quick­ly.

Q: How will you pri­or­i­tize which chron­ic or in­fec­tious dis­eases to tar­get?

We are go­ing to pri­or­i­tize based on a com­bi­na­tion of un­met needs and op­por­tu­ni­ties. I am a neu­ro­sci­en­tist. I can tell you very eas­i­ly where there are un­met needs in brain dis­or­ders. There are a lot of brain dis­or­ders where we can’t do any­where near enough for the peo­ple who suf­fer from them. Not just things like Alzheimer’s, but seizure dis­or­ders or autism or schiz­o­phre­nia.

You put that to­geth­er with op­por­tu­ni­ties. The work in hu­man ge­net­ics for the first time has iden­ti­fied the ge­net­ic risk fac­tors that pre­dis­pose you to many of those brain dis­or­ders. We did not know in the past what the ge­net­ic fac­tor was that made you more like­ly to get schiz­o­phre­nia or autism. Now we do. There’s an op­por­tu­ni­ty to take that ge­net­ic knowl­edge, learn more about the un­der­ly­ing dis­or­ders and de­vel­op new ways to ap­proach those dis­or­ders.

Q: How does a project aimed at tack­ling all known dis­eases deal with the high­ly dy­nam­ic, con­stant­ly evolv­ing uni­verse of dis­eases?

In think­ing about broad ways to ad­dress the prob­lem, the Bio­hub work on in­fec­tious dis­ease is a great ex­am­ple. On the one hand, there are many, many in­fec­tious dis­eases. On the oth­er hand, vac­cines are a great way to pre­vent in­fec­tious dis­eases. One of the mis­sions in the Bio­hub, which will be led by bio­chemist Pe­ter Kim from Stan­ford, is to learn more about why vac­cines work and why they don’t work and how to de­vel­op vac­cines that work. That could be a very gen­er­al an­swer to a huge num­ber of ques­tions.

Q: How will you de­fine suc­cess?

We know we are suc­ceed­ing if we find peo­ple us­ing the tools and tech­nolo­gies we have de­vel­oped. We have to have good ideas. We have to have good peo­ple. We have to get them to work to­geth­er. I think this will work. There is an op­ti­mism in sci­ence, es­pe­cial­ly in younger sci­en­tists, about work­ing to­geth­er on chal­leng­ing prob­lems.

I am ex­cit­ed about de­vel­op­ing a new cul­ture of sci­en­tif­ic col­lab­o­ra­tion and of en­cour­ag­ing, mo­ti­vat­ing and re­ward­ing sci­en­tists for work­ing to­geth­er. I think it will make sci­ence more ef­fec­tive and it will make it more fun.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.