— Good ideas or not?

NOR­MAN SWAN is a doc­tor and multi-award win­ning pro­ducer and broad­caster on health is­sues.

Cosmos - - Contents -

Two hot new trends in medicine may be over­hyped – and over­funded.

THE PAST COU­PLE of decades have seen a ma­jor em­pha­sis on “trans­la­tional medicine”, the idea that promis­ing treat­ments need help to tran­si­tion more quickly from the lab bench to the bed­side. The big­gest ben­e­fi­cia­ries have been two big ideas: “per­son­alised medicine” – us­ing a per­son’s genome to of­fer tailor-made treat­ments – and “re­gen­er­a­tive medicine” – us­ing stem cells to re­pair their or­gans.

But are th­ese lofty ideas de­liv­er­ing on their prom­ise? In an opin­ion piece pub­lished in the Jour­nal of the Amer­i­can Med­i­cal As­so­ci­a­tion in Oc­to­ber 2016, three prom­i­nent re­searchers ar­gue they may not be. Epi­demi­ol­o­gists John Ioan­nides from Stan­ford Univer­sity and Michael Paneth from Michi­gan State Univer­sity, with phys­i­ol­o­gist Michael Joyner from the Mayo Clinic, say th­ese big ideas are soak­ing up re­sources at the ex­pense of oth­ers that may have a big­ger im­pact on health, such as find­ing new meth­ods to cur­tail smok­ing. They call for “a whole­sale re-eval­u­a­tion of the way for­ward in bio­med­i­cal re­search”.

Per­son­alised medicine was the prom­ise of the Hu­man Genome Project, com­pleted in 2003. The idea is that read­ing your genome, plus ac­cess to elec­tronic med­i­cal records to keep tabs on a per­son’s health, will de­liver tailor-made pre­ven­tion strate­gies. For in­stance, if you carry a high-risk breast cancer gene, the rec­om­men­da­tion would be to have a mas­tec­tomy. Per­son­alised medicine will also de­liver tailor-made ther­a­pies, usu­ally drugs, as well as “gene ther­apy” to cor­rect the faulty gene.

Re­gen­er­a­tive medicine, born in 1998 when re­searchers mastered the art of grow­ing hu­man em­bry­onic stem cells, promised a new era of “spare parts” for ail­ing bod­ies.

Ac­cord­ing to the crit­ics, about 60% of re­search fund­ing in the US is now fun­nelled into th­ese fields, with a huge up­swing in the num­ber of pa­pers pub­lished.

But does hav­ing your genome read re­ally help? Com­mon con­di­tions such as type 2 di­a­betes, de­pres­sion and heart dis­ease in­volve hun­dreds of dif­fer­ent genes. Pin­ning down a faulty gene that can be tar­geted with a drug has proved elu­sive.

Cancer has been an­other area of great prom­ise with lim­ited re­turns. The idea is that read­ing the DNA of a per­son’s tu­mour should re­veal its Achilles heel. There have been some stun­ning suc­cesses where rogue genes that drive a tu­mour’s growth or help it evade the im­mune sys­tem have been matched with drugs that can dis­able those genes. But so far, we’ve not seen ben­e­fits on a large scale.

Gene ther­apy has also been dis­ap­point­ing. Even sim­ple dis­or­ders, such as sickle cell anaemia that re­sults from a sin­gle wonky ver­sion of the haemoglobin gene, have not ben­e­fited from at­tempts to re­place the faulty gene.

There is also an­other down­side to per­son­alised medicine, say the au­thors of the JAMA ar­ti­cle: “The in­evitable over­diag­no­sis and overtreat­ment that fol­lows from more in­ten­sive mon­i­tor­ing”.

The au­thors also cast doubt on the likely im­pact of stem cell ther­apy and re­gen­er­a­tive medicine. Even some of the most promis­ing tri­als in heart dis­ease, they say, may be flawed. “So what?” you may ask. We know it takes decades to turn dis­cov­er­ies into treat­ments. We know fail­ure is par for the course. The crit­ics don’t deny that. But with so much com­pe­ti­tion for re­search grants, th­ese big ideas may be stran­gling other great ideas.

The so­lu­tion, say the crit­ics, is twopronged. On one hand, the fund­ing for­mula needs to be re­set, with a higher pro­por­tion go­ing to high-risk “blue sky” ideas. On the other, the mech­a­nisms to eval­u­ate the per­for­mance of big ideas need to be re­viewed. Re­ly­ing on the num­ber of stud­ies pub­lished is self-serv­ing, they say, be­cause pub­li­ca­tions tend to fol­low fads.

“Af­ter sev­eral decades of sub­stan­tial in­vest­ment, the fun­da­men­tal ques­tion is whether th­ese big ideas have im­proved qual­ity of life and life ex­pectancy, by how much, for how many, and for whom,” write Ioan­nides, Paneth and Joyner. “Th­ese are pub­lic dol­lars that should ben­e­fit the many, not the few.”

It is not an ar­gu­ment to stop fund­ing re­search in the ar­eas of per­son­alised and re­gen­er­a­tive medicine. Th­ese big ideas may yet de­liver. But per­haps it is time for other ideas to be given a place in the sun. The de­bate is worth hav­ing.

TH­ESE BIG IDEAS MAY BE STRAN­GLING OTHER GREAT IDEAS.

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