Sean Park­er finds a big role to play in can­cer R&D: The dis­rup­tive bil­lion­aire

In biotech, where every­thing is un­cer­tain, ex­ecs like to be as pre­dictable as pos­si­ble. Scripts are main­tained, nasty sur­pris­es are avoid­ed when­ev­er pos­si­ble and speed is fine, so long as you stick to your sched­ule and don’t look like you’re in too much of a hur­ry.

Forbes’ Matthew Her­p­er with Sean Park­er and Dr. Carl June

And then there’s Sean Park­er. Park­er had an easy role to play as the bil­lion­aire founder of a new can­cer R&D in­sti­tute. The trail­blaz­ers could get a fresh source of cash and a few years from now he could claim progress on the ever-pop­u­lar no­tion of find­ing a cure for can­cer.

Park­er, a cen­tral fig­ure in this gen­er­a­tion’s so­cial me­dia rev­o­lu­tion, clear­ly had a more dis­rup­tive plan in mind.

On Mon­day evening, the Uni­ver­si­ty of Penn­syl­va­nia and the Park­er In­sti­tute con­firmed the news that An­to­nio Re­gal­a­do at MIT Tech­nol­o­gy Re­view had bro­ken ear­li­er in the day: Park­er is bankrolling a sur­prise move by Penn’s leg­endary Carl June and some of his col­leagues to do the first gene edit­ing ex­per­i­ment in hu­mans us­ing CRISPR-Cas9, a new tech­nol­o­gy that has been her­ald­ed as a sim­ple, ef­fec­tive tool for slic­ing and dic­ing DNA.

They’re look­ing to use it on im­prov­ing adop­tive cell ther­a­pies for can­cer, one of the hottest fields in im­muno-on­col­o­gy re­search.

“The study, an open la­bel, Phase I tri­al which will ex­am­ine safe­ty and man­u­fac­tur­ing fea­si­bil­i­ty of au­tol­o­gous T cells en­gi­neered to ex­press NY-ESO-1 TCR and gene edit­ed to elim­i­nate en­doge­nous TCR and PD-1, will build on Penn’s progress in­ves­ti­gat­ing oth­er types of per­son­al­ized T cell ther­a­pies for can­cer, in­clud­ing both chimeric anti­gen re­cep­tor (CAR) T cells and NY-ESO-1 trans­genic T cells,” Penn said in a state­ment to END­POINTS. “The study is de­signed to test the hy­pothe­ses that check­point re­sis­tant T cells may be safe and have im­proved an­ti­tu­mor ac­tiv­i­ty. The pi­lot tri­al will en­roll pa­tients with mul­ti­ple myelo­ma, melanoma and sar­co­ma at Penn’s Abram­son Can­cer Cen­ter and two oth­er aca­d­e­m­ic cen­ters. Fund­ing for the tri­al has been pro­vid­ed by the Park­er In­sti­tute for Can­cer Im­munother­a­py.”

Part of the pur­pose of the study, not­ed Park­er’s in­sti­tute in a fol­lowup mes­sage, is to un­leash “the pow­er of the T-cell while si­mul­ta­ne­ous­ly re­duc­ing the chance for au­toim­mu­ni­ty.”

Penn’s team pre­sent­ed their study to an NIH safe­ty board Tues­day morn­ing, out­lin­ing plans to re­cruit 18 pa­tients: 6 pa­tients with myelo­ma, 6 with sar­co­ma and 6 with melanoma.

These pa­tients will be se­lect­ed by their ex­pres­sion of the anti­gen NY-ESO, with re­searchers at Penn, MD An­der­son and UC San Fran­cis­co all par­tic­i­pat­ing. Us­ing CRISPR, they plan to dis­rupt ex­pres­sion of en­doge­nous TCR and PD-1, which may in­crease the per­sis­tence of these cells, in­creas­ing the safe­ty and ef­fi­ca­cy of the treat­ment. Tar­get­ing PD-1, they be­lieve, should cre­ate “check­point” re­sis­tant T cells, im­prov­ing pro­lif­er­a­tion of the cells.

Ini­tial­ly, they ex­pect to try this on three pa­tients over 4 weeks, then check and see if there are any un­ex­pect­ed safe­ty is­sues. If there aren’t any, then they’ll go ahead with the rest of the pa­tients.

One of the great­est risks as­so­ci­at­ed with CAR-Ts has been cy­tokine re­lease syn­drome, or cy­tokine storm. The in­ves­ti­ga­tors not­ed that there’s a well-es­tab­lished pro­to­col for track­ing this threat and re­cruit­ing pa­tients that are less like­ly to be killed by it. And the re­searchers al­so want to see just how fea­si­ble it will be to man­u­fac­ture this CRISPR-edit­ed ther­a­py.

Pan­el mem­ber Lau­rie Zoloth pressed June and his col­leagues about the fi­nan­cial im­pli­ca­tions of the study. No­var­tis has played a ma­jor role in fund­ing Penn’s CAR-T work, adapt­ing cells with a chimeric anti­gen re­cep­tor to tar­get them against can­cer cells. But June not­ed that this isn’t a CAR, it’s a gene edit­ed cell which “has no re­la­tion­ship with No­var­tis.”

Pan­el mem­bers al­so ad­vised the Penn team to high­light the pi­o­neer­ing as­pect of the tri­al as the first ever use of CRISPR in hu­mans to any pa­tients be­fore they sign on. And at about noon they vot­ed unan­i­mous­ly – with one ab­sten­tion – to ap­prove the tri­al.

Com­ing up with a bet­ter, safer way to spur a T cell at­tack on can­cer has be­come one of the Holy Grails of biotech. But it wasn’t sup­posed to play out this way in gene edit­ing.

There are sev­er­al biotechs look­ing to use gene edit­ing tech in can­cer. Ed­i­tas, which took an ear­ly lead on CRISPR-Cas9, was al­so wide­ly billed as the one that would like­ly be the first to use it in hu­man stud­ies. And Cel­lec­tis has been us­ing TAL­ENS in an ef­fort to prove that its pre­co­cious CEO An­dre Chouli­ka has a bet­ter tech­nol­o­gy for the job.

Park­er, though, seems in­tent on tear­ing up a care­ful­ly arranged stage. From his start with Nap­ster, which got him in trou­ble with the feds even as it point­ed him down the road to Face­book fame and for­tune, Park­er en­joyed the dis­rup­tion — chang­ing the mu­sic in­dus­try and the way peo­ple com­mu­ni­cate in ever grow­ing net­works.

Now he’s found a way to adopt a sim­i­lar role in can­cer R&D. And every­one will be watch­ing to see what he comes up with next.

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Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

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