Media Releases

Open-source partnership to uncover role of epigenetic regulation in Rett Syndrome

April 17, 2015

Toron­to, ON - The Struc­tur­al Genomics Con­sor­tium (SGC) at the Uni­ver­si­ty of Toron­to and the Ontario Brain Insti­tute (OBI) have entered into an “open-source” research part­ner­ship with two Toron­to-based hos­pi­tals to test tool com­pounds, called chem­i­cal probes, against epi­ge­net­ic pro­teins in research mod­els of Rett syn­drome.

The Hos­pi­tal for Sick Chil­dren (Sick­Kids) and Uni­ver­si­ty Health Net­work (UHN) will receive OBI fund­ing and com­pounds from SGC and will uti­lize their respec­tive exper­tise and dis­ease mod­els to advance our under­stand­ing of this dis­ease and iden­ti­fy poten­tial treat­ments.  Due to the open-source nature of the part­ner­ship, researchers have agreed explic­it­ly not to file for patents on any of the result­ing dis­cov­er­ies relat­ed to the col­lab­o­ra­tion.

The SGC’s tool com­pounds are the prod­uct of a glob­al pub­lic-pri­vate part­ner­ship that includes a net­work of researchers in hun­dreds of aca­d­e­m­ic insti­tu­tions and ten of the world’s lead­ing phar­ma­ceu­ti­cal com­pa­nies. By specif­i­cal­ly inhibit­ing a pro­tein in a dis­ease mod­el, the chem­i­cal probes can be used to val­i­date pro­teins as poten­tial drug tar­gets for that dis­ease.

“We are delight­ed to col­lab­o­rate with lead­ing Rett inves­ti­ga­tors in top Cana­di­an hos­pi­tals in order to learn more about the dis­ease and hope­ful­ly come up with new treat­ment strate­gies,” said Dr. Aled Edwards, Direc­tor and CEO at SGC.

This new part­ner­ship folds into one of OBI’s key research ini­tia­tives—the Province of Ontario Neu­rode­vel­op­men­tal Dis­or­ders (POND) Inte­grat­ed Dis­cov­ery Pro­gram. The mul­ti­dis­ci­pli­nary pro­gram brings togeth­er researchers, clin­i­cians, indus­try part­ners, and patients and their advo­cates to take a new approach to address­ing neu­rode­vel­op­men­tal dis­or­ders includ­ing autism spec­trum dis­or­der, obses­sive com­pul­sive dis­or­der (OCD), and atten­tion deficit hyper­ac­tiv­i­ty dis­or­der (ADHD).

“OBI is pleased to be a part of an excit­ing new part­ner­ship that fos­ters new col­lab­o­ra­tions among experts involved in the study and treat­ment of neu­rode­vel­op­men­tal dis­or­ders like Rett Syn­drome,” said Dr. Don­ald Stuss, Pres­i­dent and Sci­en­tif­ic Direc­tor, Ontario Brain Insti­tute. “We believe that this kind of coop­er­a­tion is pre­cise­ly what is need­ed to max­i­mize the val­ue of impor­tant resources and dri­ve dis­cov­ery in nov­el ways.”

The col­lab­o­ra­tion to test the epi­ge­net­ic chem­i­cal probes for Rett syn­drome is part of a larg­er SGC Open Source Tar­get Dis­cov­ery Part­ner­ship with clin­i­cal hos­pi­tals that will focus on a range of dis­eases from neu­ro­science to oncol­o­gy.

“This is a unique part­ner­ship that we are excit­ed to be a part of. By lever­ag­ing the exper­tise, resources and inno­va­tions through the lab of our con­tribut­ing researcher, Dr. James Eubanks, we hope to have progress against this dis­ease in a way that will have an impact for patients and fam­i­lies that are affect­ed by it,” said Dr. Don­ald Weaver, Direc­tor of the Toron­to West­ern Research Insti­tute, a mem­ber of UHN.

“Ontario is proud to sup­port and encour­age open source part­ner­ships that help accel­er­ate sci­en­tif­ic dis­cov­er­ies from the lab to the mar­ket­place and into patient treat­ments,” said Reza Mori­di, Ontario Min­is­ter of Research and Inno­va­tion. “These col­lab­o­ra­tions will help lead to bet­ter health out­comes for peo­ple in Ontario and around the world, and fur­ther Ontario’s capac­i­ty to com­pete in a com­pet­i­tive glob­al econ­o­my.”

In line with the SGC’s open access pol­i­cy, results, tools and knowl­edge from the col­lab­o­ra­tive projects will be open­ly shared with the wider research com­mu­ni­ties with­out restric­tions.

About Struc­tur­al Genomics Con­sor­tium (SGC)

The SGC accel­er­ates research in new areas of human biol­o­gy and drug dis­cov­ery by mak­ing all its research out­put avail­able to the sci­en­tif­ic com­mu­ni­ty with no strings attached, and by cre­at­ing an open col­lab­o­ra­tive net­work of sci­en­tists in hun­dreds of uni­ver­si­ties around the world and in ten glob­al phar­ma­ceu­ti­cal com­pa­nies.  SGC cur­rent­ly oper­ates from the Uni­ver­si­ty of Toron­to (Cana­da), Oxford Uni­ver­si­ty (UK) and UNICAMP (Brazil) and is fund­ed by Abb­vie, Bay­er, Boehringer Ingel­heim, Cana­da Foun­da­tion for Inno­va­tion, Eli Lil­ly Cana­da, Genome Cana­da, Glax­o­SmithK­line, Janssen, Mer­ck, Novar­tis, Ontario Min­istry of Research & Inno­va­tion, Pfiz­er, Take­da, and the Well­come Trust. For more infor­ma­tion about the SGC, please vis­it

About Ontario Brain Insti­tute (OBI)

The Ontario Brain Insti­tute is a provincially-funded, not-for-profit research cen­tre seek­ing to max­i­mize the impact of neu­ro­science and estab­lish Ontario as a world leader in brain research, com­mer­cial­iza­tion and care. We cre­ate con­ver­gent part­ner­ships between researchers, clin­i­cians, indus­try, patients, and their advo­cates to fos­ter dis­cov­ery and deliv­er inno­v­a­tive prod­ucts and ser­vices that improve the lives of those liv­ing with brain dis­or­ders. For more infor­ma­tion vis­it:

About Rett syn­drome

Rett Syn­drome is a rare genet­ic dis­or­der that affects neu­rode­vel­op­ment pri­mar­i­ly in girls. Chil­dren with Rett syn­drome spon­ta­neous­ly expe­ri­ence increas­ing prob­lems with mobil­i­ty, coor­di­na­tion, intel­lec­tu­al dis­abil­i­ty and dis­tinc­tive hand move­ments. Though most cas­es of Rett syn­drome are caused by a muta­tion in the methyl CpG bind­ing pro­tein 2 (MECP2) gene locat­ed on the X chro­mo­some, less than 1 per­cent of record­ed cas­es are hered­i­tary. Rett syn­drome is esti­mat­ed to affect one in every 10,000 live female births. There are cur­rent­ly no cures.

For more infor­ma­tion please con­tact:

SGC Con­tact:
Arij Al Chawaf, PhD
Strate­gic Alliances Man­ag­er
+1 (416) 854‑2745

OBI Con­tact:
Michelle Wil­son, MSc
Com­mu­ni­ca­tions Lead
+1 (647) 872‑1215