Sci­en­tists hold out hope for re­gen­er­a­tive in­fu­sion via brain im­plant — de­spite fail­ing its first test in Parkin­son's

A re­gen­er­a­tive ap­proach to treat­ing Parkin­son’s dis­ease — fea­tur­ing an im­plant to pump a pro­tein di­rect­ly in­to pa­tients’ brains — floun­dered on its first clin­i­cal test, but that hasn’t stopped re­searchers in the UK from hold­ing out hope that it can even­tu­al­ly work by restor­ing dam­aged brain cells.

Cham­pi­oned by Parkin­son’s UK, the treat­ment works by boost­ing the lev­els of GDNF, or glial cell line de­rived neu­rotroph­ic fac­tor, a nat­u­ral­ly oc­cur­ring pro­tein thought to pro­tect cells and help them re­cov­er or re­grow. To re­ceive the pro­tein — which is too large to pen­e­trate the blood-brain bar­ri­er — pa­tients first have to un­der­go a ro­bot-as­sist­ed surgery to have four tubes placed in­to their brains, then get hooked to a ma­chine that in­fuse GDNF to pre­cise lo­ca­tions of the brain via a port on the side of the head.

Af­ter con­firm­ing safe­ty with six pa­tients, sci­en­tists re­cruit­ed 35 more pa­tients to see if in­fus­ing GDNF this way led to im­prove­ments in Parkin­son’s symp­toms, from mo­tor move­ments to dai­ly ac­tiv­i­ties.

The short an­swer is no: The “en­cour­ag­ing signs of im­prove­ments” among pa­tients in the drug arm did not con­sti­tute a sig­nif­i­cant dif­fer­ence from the place­bo group.

Alan Whone

But prin­ci­pal in­ves­ti­ga­tor Alan Whone was quick to of­fer po­ten­tial ex­pla­na­tions, name­ly the short dou­ble-blind tri­al time (nine months), the rel­a­tive­ly low dosage of GDNF, and the stage that pa­tients were al­ready at by the time they en­rolled.

His op­ti­mism is backed up by some ev­i­dence that GDNF had promis­ing ef­fects on brain cells al­ready rav­aged by the dis­ease — show­ing a 100% im­prove­ment in a key area of the brain af­fect­ed in the con­di­tion com­pared to place­bo, which didn’t in­duce any change.

“The spa­tial and rel­a­tive mag­ni­tude of the im­prove­ment in the brain scans is be­yond any­thing seen pre­vi­ous­ly in tri­als of sur­gi­cal­ly de­liv­ered growth-fac­tor treat­ments for Parkin­son’s,” Whone said in a state­ment. “This rep­re­sents some of the most com­pelling ev­i­dence yet that we may have a means to pos­si­bly reawak­en and re­store the dopamine brain cells that are grad­u­al­ly de­stroyed in Parkin­son’s.”

Fur­ther­more, a nine-month fol­low-up pe­ri­od dur­ing which the place­bo group al­so switched to GNDF saw all par­tic­i­pants demon­strat­ing “mod­er­ate to large im­prove­ment in symp­toms” com­pared to their own base­line scores, bol­ster­ing the hope that the ef­fects on symp­toms were just lag­ging be­hind im­prove­ment in brain cells.

Erich Mohr

All of this spells “ex­cit­ing signs of promise” for de­vice man­u­fac­tur­er Ren­ishaw as well as Med­Ge­n­e­sis, the biotech de­vel­op­ing GDNF and div­ing in­to an in­creas­ing­ly crowd­ed field with plen­ty of small­er play­ers tied up with big part­ners, tout­ing new tech­nolo­gies like pro­tein degra­da­tion to gene ther­a­py.

“We be­lieve this ex­per­i­men­tal Parkin­son’s Dis­ease Com­pos­ite Re­sponse (PD­CORE) may bet­ter cap­ture the full ef­fects of GDNF and we’re work­ing to get it sci­en­tif­i­cal­ly val­i­dat­ed so that it can be used in fu­ture tri­als,” CEO Erich Mohr said.

GDNF de­vice. Cred­it: Mint­Mo­tion for Pas­sion­ate Pro­duc­tions

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John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

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On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

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Abeliovich and Prevail, though, aren’t taking this one lying down.

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As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

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