Media Releases

U of T researchers discover new way to treat deadly childhood brain cancer

February 19, 2014

TORONTO, ON – Researchers from the Uni­ver­si­ty of Toronto’s Depart­ment of Lab­o­ra­to­ry Med­i­cine and Patho­bi­ol­o­gy (LMP) have dis­cov­ered a new way to effec­tive­ly tar­get a pre­vi­ous­ly dif­fi­cult-to-treat form of child­hood brain can­cer called ependy­mo­ma.

Pro­fes­sor Michael Tay­lor (MD,PhD, FRCSC), along with lead author Stephen Mack and co-authors includ­ing LMP Prof. Peter Dirks (MD, PhD), pub­lished the ground­break­ing find­ings in Nature on Feb­ru­ary 19, 2014. Their research showed that epi­ge­net­ics, the pack­ag­ing of DNA, is the main cause of this can­cer and can be tar­get­ed by FDA approved drugs.

DNA code is com­pa­ra­ble to a three-bil­lion let­ter long set of instruc­tions on how a cell should oper­ate. In this anal­o­gy, the major­i­ty of can­cers are caused by words that are mis­spelled, words that are added or delet­ed or entire book chap­ters that are added or delet­ed.

“When we look at ependy­mo­ma, we don’t find any mis­spelled words, delet­ed or dupli­cat­ed words or book chap­ters that are miss­ing. Instead, what we find is that the entire nov­el is writ­ten in the wrong font and the DNA is pack­aged improp­er­ly,” said Prof. Tay­lor. “While epi­ge­net­ics has been known to play a role in can­cer, this is the first time that epi­ge­net­ics is the prime dri­ver of can­cer.”

Receiv­ing a diag­no­sis of ependy­mo­ma, the third most com­mon type of child­hood brain can­cer, can be dev­as­tat­ing. While babies and tod­dlers are treat­ed with surgery and radi­a­tion ther­a­py, chemother­a­py has been inef­fec­tive and the can­cer often recurs.

Impres­sive­ly, there are already FDA approved drugs that can alter the cancer’s epi­ge­net­ics. “Usu­al­ly when you make a dis­cov­ery you have to make a new drug, but there are already drugs that change the font. In this case, there are drugs that are the Microsoft Word equiv­a­lent of select all and switch to Times New Roman,” said Prof. Tay­lor.

The team’s suc­cess relied heav­i­ly on their abil­i­ty to grow and study cells from these rare tumours. Once a patient’s tumour is removed from the oper­at­ing room, its cells are tak­en to Prof. Peter Dirks’s lab to be grown in spe­cial­ized con­di­tions. The process is so effi­cient that by the time a child has recov­ered from surgery, the team has test­ed the cells and will know whether a spe­cif­ic drug will work.

“I think that’s where this project has been so excit­ing. Dr. Tay­lor and Steve Mack made a foun­da­tion­al dis­cov­ery in this tumour, and we were able to bring our exper­tise togeth­er in grow­ing these live cells to see whether tar­get­ing this pack­ag­ing real­ly had any promise,” said Prof. Dirks.

The team is opti­mistic that this will pro­vide hope for chil­dren suf­fer­ing from ependy­mo­ma. Prof. Tay­lor explains, “We hope it will be an effec­tive treat­ment. One drug doesn’t usu­al­ly result in a cure, but there are mul­ti­ple drugs that attack the same mech­a­nism. Even if we can’t cure it, we could use the drug to keep it to a treat­able dis­ease like dia­betes.”

If the group receives fund­ing, it could take only three to five years to com­plete a clin­i­cal tri­al.


For more infor­ma­tion, con­tact:

Katie Bab­cock
Web and Com­mu­ni­ca­tions Coor­di­na­tor
The Depart­ment of Lab­o­ra­to­ry Med­i­cine and Patho­bi­ol­o­gy (LMP), Uni­ver­si­ty of Toron­to
Tel: 416–278-6568