Mi­cro­bio­me, in­flam­ma­tion and mu­ta­tions: US re­searchers win £60M in Can­cer Re­search UK grants to lead 'grand chal­lenges'

Can­cer Re­search UK is putting lead­ing US re­searchers as the faces of its lat­est Grand Chal­lenge — five-year re­search pro­grams that each draw from £20 mil­lion ($25.9 mil­lion) in fund­ing and an in­ter­na­tion­al group of sci­en­tists to tack­le some of the biggest ques­tions in can­cer.

Se­lect­ed from 134 ap­pli­ca­tions in the sec­ond in­stall­ment of the com­pe­ti­tion, the three projects will at­tempt to ma­nip­u­late the mi­cro­bio­me to fight bow­el can­cer, ex­plore the links be­tween chron­ic in­flam­ma­tion and can­cer, and un­der­stand why cer­tain ge­net­ic mu­ta­tions cause can­cer in some tis­sues but not oth­ers.

Sci­en­tists from Har­vard, UCSF and Brigham and Women’s Hos­pi­tal will lead the charge, co­or­di­nat­ing teams of 10 or 14 scat­tered be­tween the US, UK, Cana­da, The Nether­lands, Spain, and Is­rael.

Matthew Mey­er­son

“In­di­vid­u­al­ly, these re­search teams are among the best in the world in their re­spec­tive fields,” said Iain Foulkes, Can­cer Re­search UK’s ex­ec­u­tive di­rec­tor of re­search and in­no­va­tion, in a state­ment. “By bring­ing them to­geth­er across bor­ders, Grand Chal­lenge is en­abling these teams to think big­ger and es­tab­lish new and ex­cit­ing col­lab­o­ra­tions. The scale of the fund­ing re­flects the op­por­tu­ni­ty we see in har­ness­ing their abil­i­ty to un­der­stand and tack­le can­cer.”

Wendy Gar­rett

Matthew Mey­er­son at the Dana-Far­ber Can­cer In­sti­tute and Har­vard Med­ical School is co-prin­ci­pal in­ves­ti­ga­tor with Wendy Gar­rett at the Har­vard T.H. Chan School of Pub­lic Health on the mi­cro­bio­me project. By com­par­ing in de­tail a healthy mi­cro­bio­me with one as­so­ci­at­ed with can­cer, they hope to iden­ti­fy new in­ter­ven­tions to pre­vent and treat can­cer — whether by im­prov­ing re­sponse to ther­a­pies or be­ing fash­ioned in­to ther­a­pies of their own — that they will take in­to clin­i­cal tri­als.

Thea Tl­sty

In an­oth­er project, UCSF’s Thea Tl­sty wants to tune in­to what she calls “the oth­er side” of the con­ver­sa­tion be­tween can­cer cells and the cells sur­round­ing them, with the ul­ti­mate goal to “de­vise ex­cit­ing new ap­proach­es to treat­ment from re­pur­pos­ing every­day an­ti-in­flam­ma­to­ry drugs, to de­sign­ing cells that tar­get can­cer-pro­mot­ing tis­sues.”

Fi­nal­ly, Stephen Elledge — a pro­fes­sor at Har­vard Med­ical School and an in­ves­ti­ga­tor with the Howard Hugh­es Med­ical In­sti­tute — de­scribes his chal­lenge this way:

Stephen Elledge

We think the rea­son that spe­cif­ic ge­net­ic de­fects cause cer­tain types of can­cer comes down to the way dif­fer­ent cell types are ‘wired’, and whether the tis­sue sees it as a ‘GO’ sig­nal or not. We’re go­ing to de­con­struct what’s go­ing on by switch­ing can­cer genes on and off and track­ing the changes in nor­mal, healthy cells from dif­fer­ent or­gans. This will deep­en our un­der­stand­ing of the very na­ture of can­cer, and by us­ing cut­ting-edge tech­nolo­gies like organoids, we hope to find new tar­gets for can­cer treat­ments in fu­ture.

The Mark Foun­da­tion for Can­cer Re­search in New York is pro­vid­ing half of the grant — £10 mil­lion to this par­tic­u­lar project.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

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Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin and UBS in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.