Gates Foun­da­tion to tackle sickle cell, HIV

The Korea Times - - HEALTH -

WASH­ING­TON (AFP) — The U.S. govern­ment and the Bill and Melinda Gates Foun­da­tion pledged Wed­nes­day to jointly in­vest $200 mil­lion over the next four years to achieve af­ford­able gene ther­apy-based cures for sickle cell dis­ease (SCD) and HIV.

The ad­min­is­tra­tion of Pres­i­dent Don­ald Trump an­nounced ear­lier this year its in­ten­tion to end the HIV epi­demic over the next decade and has also iden­ti­fied SCD, which dis­pro­por­tion­ately af­fects peo­ple of African de­scent, as a con­di­tion re­quir­ing greater at­ten­tion.

Gene ther­apy is a rel­a­tively new area of medicine de­signed to re­place faulty genes in the body that are re­spon­si­ble for a dis­or­der, and has been re­spon­si­ble for new treat­ments for blind­ness and cer­tain types of leukemia.

But the treat­ments are com­plex and costly, rul­ing them out as an op­tion for most of the world.

Fran­cis Collins, di­rec­tor of the Na­tional In­sti­tutes of Health, said the col­lab­o­ra­tion would fo­cus there­fore on “ac­cess, scal­a­bil­ity and af­ford­abil­ity” to make sure the even­tual treat­ments are avail­able glob­ally.

The NIH and Gates Foun­da­tion aim to achieve clin­i­cal tri­als in the United States and coun­tries in sub-Sa­ha­ran Africa within the next seven to 10 years.

Sickle cell dis­ease is a group of in­her­ited red blood cell dis­or­ders char­ac­ter­ized by the pres­ence of an ab­nor­mal protein in the red blood cells, caus­ing the feet and hands to swell, fa­tigue, jaun­dice, and episodic or chronic pain.

Over time the dis­ease can harm a pa­tients’ vi­tal or­gans, bones, joints and skin and it is cur­rently only cur­able via a blood and bone mar­row trans­plant, avail­able to only a tiny frac­tion of peo­ple who have the dis­ease.

When it comes to HIV, an­tiretro­vi­ral ther­apy (ART) are now able to re­duce pa­tients’ vi­ral load to the point that they are un­de­tectable and can­not be fur­ther trans­mit­ted.

But “a ma­jor goal is to find a cure, whereby life­long ART would not be re­quired,” said the NIH’s An­thony Fauci.

Though SCD is a ge­net­i­cally in­her­ited dis­ease, and HIV is ac­quired from in­fec­tion, genebased treat­ments are said to hold prom­ise for both, and “many of the tech­ni­cal chal­lenges for genebased cures are ex­pected to be com­mon to both dis­eases.” The goal for SCD is to achieve a genebased in­ter­ven­tion that ei­ther cor­rects the gene mu­ta­tion re­spon­si­ble or pro­motes fe­tal he­mo­glo­bin gene ex­pres­sion to achieve nor­mal he­mo­glo­bin func­tion.

For HIV, the pro­posed cure would in­volve tar­get­ing the reser­voir of provi­ral DNA that lurks in­side a small num­ber of cells even after many years of ART.

The NIH said that ap­prox­i­mately 95 per­cent of the 38 mil­lion peo­ple liv­ing with HIV glob­ally are in the de­vel­op­ing world, with 67 per­cent in sub-Sa­ha­ran Africa, half of whom are liv­ing un­treated. Around 1.1 mil­lion Amer­i­cans are af­fected

SCD af­fects ap­prox­i­mately 100,000 Amer­i­cans, ac­cord­ing to of­fi­cial fig­ures.


Bill and Melinda Gates are in­ter­viewed in Kirk­land, Wash­ing­ton, Feb. 1. The phi­lan­thropist cou­ple has teamed up with the U.S. govern­ment to more force­fully tackle HIV and sickle cell dis­ease.

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