Break­ing through

Up­beat re­ports on med­i­cal re­search highlight value of fed­eral, pri­vate fund­ing

Modern Healthcare - - Opinions -

It’s nice to read the news these days and come across some­thing even re­motely pos­i­tive. Amid talk of a dou­ble-dip re­ces­sion, stub­bornly high job­less­ness rates and the lat­est par­ti­san snip­ing in Wash­ing­ton and on the cam­paign trail, an op­ti­mistic story is most wel­come. In the past month, such en­cour­ag­ing news has come from the med­i­cal re­search front. Just last week, re­searchers based out of North­west­ern Univer­sity’s Fein­berg School of Medicine in Chicago re­ported that they had dis­cov­ered the com­mon mech­a­nism at work in all forms of amy­otrophic lat­eral sclero­sis, best known as ALS or Lou Gehrig’s dis­ease. In these pa­tients, the body’s mus­cles grad­u­ally de­te­ri­o­rate, lead­ing to dis­abil­ity, paral­y­sis and, ul­ti­mately, death. The dis­ease is es­ti­mated to af­fect about 350,000 pa­tients world­wide.

Ac­cord­ing to the re­search team, the ba­sis of the dis­or­der is a failed pro­tein re­cy­cling sys­tem in the neu­rons of the spinal cord and the brain. Nor­mal func­tion­ing of the neu­rons re­quires ef­fi­cient re­cy­cling of pro­teins crit­i­cal to cell de­vel­op­ment. In ALS pa­tients, be­cause the sys­tem is bro­ken, cells can’t stay healthy or re­pair them­selves, hav­ing a cas­cad­ing ef­fect.

With the dis­cov­ery of this root cause, sci­en­tists are now so many steps closer to de­vel­op­ing treat­ments for a dis­ease that has es­sen­tially been un­treat­able. The break­through also could lead to progress in other neu­rode­gen­er­a­tive dis­eases such as de­men­tia and Parkin­son’s dis­ease, says Dr. Teepu Sid­dique, a pro­fes­sor of neu­rol­ogy at North­west­ern who was the se­nior au­thor of the study pub­lished in the jour­nal Na­ture.

Mean­while, there was an­other re­port this month on a po­ten­tially promis­ing re­search out­come, this one in­volv­ing an ex­per­i­men­tal treat­ment for leukemia.

Sci­en­tists were suc­cess­ful in con­vert­ing the pa­tients’ own blood cells into “as­sas­sins” ge­net­i­cally trained to at­tack can­cer cells. To date, re­searchers have had lim­ited suc­cess in at­tempts to su­per­charge the body’s im­mune sys­tem to fight can­cer.

But ac­cord­ing to an As­so­ci­ated Press re­port, Dr. Carl June, a gene ther­apy ex­pert at the Univer­sity of Penn­syl­va­nia, and his team made changes to pre­vi­ous tech­niques, us­ing a novel method to de­liver new genes into blood cells as well as a sig­nal­ing mech­a­nism to or­der the cells to kill and mul­ti­ply.

“It worked great. We were sur­prised it worked as well as it did,” June said. “We’re just a year out now. We need to find out how long these re­mis­sions last.”

Other re­searchers are urg­ing cau­tion be­cause the study in­volved only three pa­tients. “Three’s bet­ter than one, but it’s not 100,” say Dr. Wal­ter Urba, a med­i­cal on­col­o­gist at Prov­i­dence Can­cer Cen­ter in Port­land, Ore., and an au­thor of an editorial on the re­search that ap­peared in the New Eng­land

Jour­nal of Medicine. What hap­pens long term is key, he says. Those two words—“long term”—are ap­pli­ca­ble to all re­search. It takes years of in­vest­ment, in time and es­pe­cially money, work­ing to­ward and hop­ing for that break­through. So as the de­bate in our na­tion’s cap­i­tal starts heat­ing up again with yet an­other dead­line ap­proach­ing to get some­thing set­tled con­cern­ing our fed­eral bud­get, the fu­ture of re­search fund­ing hangs in the bal­ance.

Med­i­cal con­di­tions from AIDS to can­cer to Alzheimer’s dis­ease all draw mil­lions of dol­lars through pri­vate fundrais­ing or­ga­ni­za­tions. And that pipe­line is vi­tal. Ac­cord­ing to a news re­port, one Chicagoarea foun­da­tion alone has raised $42 mil­lion in the past 30 years to fund ALS re­search, much of it go­ing to North­west­ern.

But when it comes to re­search, fed­eral fund­ing is the crit­i­cal spigot. And it’s es­sen­tial to the long-term pub­lic health of this na­tion that such spend­ing be spared the ax when Congress starts pin­point­ing bud­get lines to tar­get. This is money that truly de­liv­ers bang for the buck.

Let’s keep the good news flow­ing. DAVID MAY As­sis­tant Man­ag­ing

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