Plans for stem cells tri­als for Parkin­son’s

● Suc­cess­ful tests on mon­keys pave the way for first hu­man pro­gramme

The Scotsman - - Around Scotland - By JOHN VON RADOWITZ

Scot­tish sci­en­tists yes­ter­day wel­comed a move that could see pa­tients re­cruited for the first clin­i­cal trial of a stem cell treat­ment for Parkin­son’s dis­ease be­fore the end of next year.

A team of Ja­panese sci­en­tists made the pre­dic­tion af­ter suc­cess­fully restor­ing nerve cells de­stroyed by a sim­i­lar con­di­tion in mon­keys.

The an­i­mals, suf­fer­ing an ar­ti­fi­cially in­duced ver­sion of the dis­ease, showed sig­nif­i­cant im­prove­ment two years af­ter hav­ing pre­cur­sor dopamine neu­rons, de­rived from hu­man stem cells, trans­planted into their brains.

In hu­mans, Parkin­son’s causes pro­gres­sive loss of the neu­rons, which re­lease the vi­tal nerve trans­mit­ter chem­i­cal dopamine nec­es­sary for con­trol­ling body move­ment. The sci­en­tists say they are now just a short step away from test­ing the treat­ment, based on lab­o­ra­tory-made stem cells called in­duced pluripo­tent stem (IPS) cells, in clin­i­cal tri­als.

They hope to start look­ing for suit­able pa­tients within the next 15 months.

Dr Tilo Ku­nath, a Parkin­son’s Uk-funded re­searcher at the Med­i­cal Re­search Council Cen­tre for Re­gen­er­a­tive Medicine at the Univer­sity of Ed­in­burgh, said: “This is ex­tremely promis­ing re­search demon­strat­ing that a safe and highly ef­fec­tive cell ther­apy for Parkin­son’s can be pro­duced in the lab.

“Such a ther­apy has the po­ten­tial to re­verse the symp­toms of Parkin­son’s in pa­tients by restor­ing their dopamine­pro­duc­ing neu­rons. The next stage will be to test these ther­a­pies in a first-in-hu­man clin­i­cal trial.

“As a side-note, the fact that the re­searchers have used in­duced pluripo­tent stem cells in­stead of hu­man em­bry­onic stem cells (HESCS) means that this ther­apy can be used in any coun­try world­wide.

“Some coun­tries, such as Ire­land and most of South Amer­ica, have banned the use of HESCS as a ther­apy.”

The new study in­volved 11 macaque mon­keys dis­play­ing Parkin­son’s-like symp­toms of tremors and im­paired bal­ance caused by a neu­ro­toxin.

Some of the mon­keys were given dopamine neu­ron pro­gen­i­tors grown from IPS stem cells de­rived both from hu­man Parkin­son’s pa­tients and healthy donors.

Once trans­planted the pre­cur­sor cells ma­tured and started to re­lease dopamine, re­sult­ing in the restora­tion of move­ment con­trol.

“Our re­search has shown that DA (dopamine) neu­rons made from IPS cells are just as good as DA neu­rons made from foetal mid-brain, said Prof Taka­hashi.

“Be­cause IPS cells are easy to ob­tain, we can stan­dard­ise them to only use the best IPS cells for ther­apy.”

Brain scans con­firmed that the cells were func­tion­ing as ex­pected and not trig­ger­ing a dam­ag­ing im­mune re­sponse.

A key find­ing from the re­search, pub­lished in the jour­nal Na­ture, was that no tu­mours had ap­peared in the mon­key’s brains – a recog­nised haz­ard of ex­per­i­men­tal stem cell ther­a­pies.

Each mon­key was given just un­der five mil­lion pro­gen­i­tor neu­rons. How­ever, the re­search showed that cell qual­ity was more im­por­tant than quan­tity.

Neu­ro­sur­geon Tet­suhiro Kikuchi, an­other mem­ber of the Ky­oto team, said: “Each an­i­mal re­ceived cells pre­pared from a dif­fer­ent IPS cell donor. We found the qual­ity of donor cells had a large ef­fect on the DA neu­ron sur­vival.”

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