00:00For about 14 years now I've been researching childhood cancers and then
00:10about six years after I had completed my PhD and started my own lab, my daughter
00:15Josephine was diagnosed with DIPG, which is diffuse intrinsic pontine glioma.
00:23When Josie was diagnosed we of course were shocked and devastated but also we
00:31were stunned by the lack of treatment options for the disease. That really hit
00:37home for me as a preclinical cancer researcher who was focused on developing
00:42strategies for kids with poor cancers that there could be a cancer that
00:45there's no effective treatments for, so it really encouraged me to start doing
00:49DIPG research. 90% of therapies that are used to treat cancers target proteins
00:57but yet in DIPG we've only really focused on sequencing the DNA of a cell.
01:02It's only thanks to the generosity of families that leave their child's tumor
01:07to our research after they tragically pass away that we've been able to get
01:10enough material to start to sequence the proteins. We're trying to break DIPG
01:17down into a more simple subcategory. In the first instance we're trying to
01:21separate each diagnosis into a particular subtype.
01:27We weren't able to subcategorize Josie's tumor into any particular subtype.
01:34Over time we were exploring drugs focused on her genomic changes. We discovered a new
01:39drug for DIPG called Taxelacib which targeted a mutation that was in Josie's
01:44tumor. That mutation is seen in about 30 to 40% of DIPGs. About seven months from
01:53diagnosis when Josie was in disease progression as it's called, we got
01:58approval from the pharmaceutical companies to get compassionate access to
02:03treat Josie and thankfully it stabilized the tumor for more than three months.
02:10We had a beautiful Christmas with our family and our friends but like all DIPG
02:15journeys, three months after the commencement of those two therapies the
02:18tumor was growing again. My goal was to try and use all the tools I had at my
02:24availability to try and buy more time. Run DIPG started when Josie was an
02:31inpatient at Sydney Children's Hospital and it's enabled us to have a platform
02:35to advocate for DIPG and to fundraise to do critical medical research in an
02:41attempt to increase survival of this devastating tumor.
02:47Once we understand how and why tumors are starting to respond to particular
02:52therapies then we can exploit them. We can make new drugs, we can design better
02:56treatment regimens, we can use therapies in a sequential way because we
03:01understand how a tumor will respond.
03:06It's a huge honor to be named the ASMR Medalist for 2024, knowing those that
03:13have gone before and I look forward to using the platform as best as possible
03:16to highlight the potential of new investment in health and medical
03:21research for Australia. There's a disease that takes the life of a child every two
03:27weeks in Australia but yet we don't have a single therapy for them. The leading
03:32cause of death from childhood cancer remains completely untreatable and I
03:37think that's completely unacceptable.
Comments