Are we on the brink of a Parkin­son’s break­through?

As cases soar, some spe­cial­ists be­lieve the key to a cure is within our grasp, finds Rosie Tay­lor

The Sunday Telegraph - - Features -

As you read this, we may have a cure for Parkin­son’s dis­ease, and just not know it. Sev­eral treat­ments on clin­i­cal trial could yet turn out to be a sav­ing grace for the mil­lions with the dis­ease, as well as the pop­u­la­tion at large. For, ac­cord­ing to new re­search, the de­gen­er­a­tive neu­ro­log­i­cal dis­ease is af­fect­ing more peo­ple than ever be­fore.

Last week, The Lancet re­vealed that the num­ber of peo­ple world­wide with Parkin­son’s had reached 6.1 mil­lion in 2016, al­most two and a half times the 1990 fig­ure of 2.5 mil­lion.

In the UK, Parkin­son’s af­fects around one in 350 peo­ple, but by 2025, the num­ber of Bri­tons liv­ing with the dis­ease will have in­creased by around 18 per cent to more than 168,000, ac­cord­ing to sep­a­rate fig­ures from the char­ity Parkin­son’s UK. This fig­ure is set to al­most dou­ble within 50 years, as the pop­u­la­tion ages and im­prove­ments in treat­ment mean more peo­ple sur­vive other dis­eases such as can­cer and heart dis­ease.

In the face of a grow­ing threat from the dis­ease – symp­toms of which in­clude tremors, mus­cle stiff­ness, prob­lems with bal­ance, as well as de­pres­sion – sci­en­tists have been work­ing flat out to find new treat­ments. At present, med­i­ca­tions merely re­duce the sever­ity of symp­toms. But, fi­nally, some think they are just five years away from un­lock­ing the key to a cure.

Bri­tain’s lead­ing spe­cial­ists be­lieve a break­through is fi­nally on the hori­zon, with sev­eral clin­i­cal tri­als ex­plor­ing po­ten­tial treat­ments launch­ing this year and in 2019.

Dr David Dex­ter, deputy di­rec­tor of re­search at Parkin­son’s UK, says: “It’s a pos­i­tive time for re­search be­cause we can see so much of the jig­saw com­ing to­gether.”

When it comes to in­vest­ment in re­search, Parkin­son’s has long been over­shad­owed by a fo­cus on other se­ri­ous con­di­tions, such as de­men­tia and can­cer. Bri­tish Parkin­son’s re­search char­i­ties re­ceived around £40 mil­lion in do­na­tions last year – less than half of the amount given to the coun­try’s main Alzheimer’s char­i­ties, and a tenth of that re­ceived by Can­cer Re­search UK.

But re­newed in­ter­est in find­ing a cure and cam­paign­ing from char­i­ties and celebri­ties such as the ac­tor Michael J Fox, who was just 29 when he was di­ag­nosed with early on­set Parkin­son’s in 1991, means there are fi­nally a host of hu­man tri­als of new treat­ments un­der way or about to start.

Sci­en­tists are op­ti­mistic that at least one treat­ment be­ing tested could hold the key to un­der­stand­ing what trig­gers the dis­ease – or prove to be a cure.

Parkin­son’s is a pro­gres­sive neu­rode­gen­er­a­tive dis­or­der where cells in the brain die off over time, lead­ing to a loss of the chem­i­cal dopamine, which is vi­tal for con­trol­ling move­ment. The main symp­toms – shak­ing or tremors of body parts when at rest, slow move­ment and stiff or in­flex­i­ble limbs – are of­ten paired with de­pres­sion, anx­i­ety, sleep prob­lems and loss of mem­ory or cog­ni­tive func­tion. Pa­tients are nor­mally di­ag­nosed when they are over the age of 50. Their health de­te­ri­o­rates over time and they can ul­ti­mately be­come se­verely dis­abled. While the con­di­tion does not di­rectly cause death, it makes the body vul­ner­a­ble to com­pli­ca­tions, such as prob­lems swal­low­ing, or in­fec­tions.

As there is cur­rently no cure, ex­ist­ing treat­ments are de­signed to help min­imise symp­toms. The drugs can have un­pleas­ant side ef­fects – in­clud­ing trig­ger­ing hal­lu­ci­na­tions or in­vol­un­tary move­ments – and lose ef­fec­tive­ness over time.

But Dr Dex­ter says the re­search world is pos­i­tive about the po­ten­tial of the new ther­a­pies. “Thirty years ago, we knew which cells died in Parkin­son’s, but we had no idea why. Now we’ve got to the point where we know so much more about the mech­a­nisms, how they work and how they in­ter­act with each other. The ex­cit­ing thing is that now we’re ac­tu­ally de­vel­op­ing the drugs to stop these mech­a­nisms. Within the next five or so years, we should know which ones are the key driv­ers in the dis­ease.

“Any one of those clin­i­cal tri­als test­ing one of those av­enues now may prove pos­i­tive,” he says. “But if they don’t, it will still add to our un­der­stand­ing.”

Dr Si­mon Stott, deputy di­rec­tor of re­search at the Cure Parkin­son’s Trust, ex­plains that in or­der to find a cure for the dis­ease, sci­en­tists must dis­cover how to stop its pro­gres­sion, re­ju­ve­nate any re­main­ing brain cells and re­place those which have died.

He says: “The bad news is that we don’t have any one sin­gle treat­ment on the hori­zon that will do all three things. But the good news – and the most ex­cit­ing part of Parkin­son’s re­search at the mo­ment – is that we have clin­i­cal tri­als on­go­ing for dif­fer­ent tech­niques deal­ing with each of these three com­po­nents.”

Dr Stott be­lieves a cure may be found us­ing a com­bi­na­tion of treat­ments for the three tar­gets. When it comes to stop­ping the dis­ease, sci­en­tists are fo­cused on halt­ing the spread of a “rogue” pro­tein called al­phasynu­clein. At least six tri­als in pa­tients are un­der way world­wide to ex­plore whether anti-pro­tein vac­cines could help, in­clud­ing some funded by the Michael J Fox Foun­da­tion.

In the UK, a £2.1 mil­lion trial funded by the Na­tional In­sti­tute of Health Re­search will start next year, study­ing whether a type of drug known as a cholinesterase in­hibitor (CHEI) can help to re­duce un­steadi­ness and im­prove con­cen­tra­tion.

The area of re­search which has caused the most ex­cite­ment is cell re­place­ment ther­apy (CRT) – where dopamine cells grown from stem cells are trans­planted into the brain. The treat­ment should help re­lieve the phys­i­cal symp­toms of Parkin­son’s.

Hu­man tri­als of CRT have been launched in the last few months in Ja­pan, the US and Aus­tralia, while a Euro­pean trial co-led by Cam­bridge Univer­sity and Lund Univer­sity, in Swe­den, is ex­pected to start next year.

Con­sul­tant neu­rol­o­gist Pro­fes­sor Roger Barker, who is lead­ing the Euro­pean project, says: “The idea be­hind cell re­place­ment ther­a­pies is to lit­er­ally trans­plant the cells into the brain to re­place those which have been lost. If the dopamine cell-based ther­a­pies work in the way we ex­pect, then I would be sur­prised if they’re not com­ing into clinic as a stan­dard ther­apy in five to 10 years’ time. While it’s not a cure, it would trans­form the treat­ment of Parkin­son’s.”

The mo­men­tum be­hind re­search has built up over the past two decades, with a “tsunami” of dis­cov­er­ies and devel­op­ments in re­cent years. “Within a few years,” says Dr Stott, “we will see ma­jor progress. ”

But he cau­tions pa­tients not to get their hopes up too early. “At this point, we want to keep pa­tients in­formed, but not to raise ex­pec­ta­tions too much. If the tri­als don’t work, they will still fur­ther our un­der­stand­ing.

“But if they do… it’s Christ­mas Day.”

‘It’s a pos­i­tive time for re­search be­cause we can see so much of the jig­saw com­ing to­gether’

Ma­jor progress: an MRI scan of the brain of a pa­tient with Parkin­son’s, which Michael J Fox, be­low, suf­fers from. Above right, Dr David Dex­ter, says the re­search world is feel­ing pos­i­tive

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