Wednesday, June 19, 2013

International team submits IND application

On 18 June 2013, there was an announcement at the Princess Margaret Cancer Centre, Toronto. There was also a post on the California stem cell agency blog entitled: Clinical trial to thwart cancer stem cells may begin soon. An excerpt from the post:
The Prince Margaret Center announced the FDA filing, called an Investigational New Drug application (IND), at an event in Toronto recognizing the private donors. A press release about the announcement was picked up at this biotech news site.
The Principal Investigators present for the announcement were Dr. Tak Mak of the Princess Margaret Cancer Centre and Dr. Dennis Slamon of the University of California, Los Angeles (UCLA). What they have done is summarized in another excerpt from the blog post:
By working first to understand the various proteins that drive cells to divide, particularly in cancer, they were able to pinpoint an enzyme, that if blocked, could be the key to keeping cancer in check. They then discovered that this enzyme, called PLK4, can be derailed by a new drug they developed. In the lab, it has been shown to inhibit the growth of breast, ovarian, colorectal, lung, pancreatic and prostate cancer, as well as melanoma.

Information about the human PLK4 protein is available here.

Some background: In June 2008, it was announced that a partnership had been formed between Canada’s Cancer Stem Cell Consortium (CSCC) and the California Institute for Regenerative Medicine (CIRM) for international collaboration to advance cancer stem cell research. An excerpt from the CSCC's announcement:
It is proposed that one of the first initiatives to be launched by the CSCC will be a collaboration between Canadian and Californian scientists through CIRM's upcoming Disease Team Research Awards Competition, which will support multi-disciplinary teams of scientists in pursuit of therapies for specific diseases.The goal is to fund teams that will develop therapy or diagnostics for a particular disease or serious injury.Successful proposals will likely include a description of a path to an Investigational New Drug filing at the end of the four-to-five year grant.

Note the intent to file an IND by the end of the term of the grant.

The results of the Disease Team Research Awards Competition were announced on October 28, 2009. The award to Drs. Slamon and Mak is Grant number DR1-01477. See: Therapeutic Opportunities to Target Tumor Initiating Cells in Solid Tumors. As was stated in a page about Cancer Stem Cells on the website of the Canadian Institutes for Health Research (CIHR), this award was to one of two multi-disciplinary research teams co-led by Canadian and Californian scientists. The other team is co-led by Dr. John Dick of the University Health Network and Dr. Dennis Carson of the University of California, San Diego. My blog post (October 29, 2009) about the awards is entitled: Disease Team awards announced. The post ends with this Disclosure:
I'm a member of the Board of the CSCC, but also a staff member (emeritus) at the University Health Network. So, I was in conflict of interest, and was absent during all of the discussions, by the CSCC Board, about which Canadian applications should be considered for the Disease Team awards.


  1. There's an article in today's Globe & Mail about the FDA filing, and a tweet about it. The tweet:

    Beware the hype about #cancer breakthroughs & read the fine print by @picardonhealth via @GlobeLife #journalism
    — André Picard (@picardonhealth) June 21, 2013

  2. Another tweet about the FDA filing:

    MT @CAHOhospitals: Dr. Tak Mak defends #cancer "breakthrough" dismisses @picardonhealth view
    — André Picard (@picardonhealth) September 6, 2013

  3. Information about a clinical trial (NCT01954316) was received by on September 26, 2013. The information was provided by the University Health Network, Toronto. It's a trial of CFI-400945 fumarate. The trial has recently been approved to begin enrolling patients. See: With new multimillion-dollar grants, UCLA scientists take stem cell research to patients. Excerpts:

    "This clinical trial builds on Slamon's previous work, partially funded by CIRM, with Wainberg and Dr. Tak Mak, director of the Campbell Family Institute at the University Health Network in Toronto, aimed at developing a drug that targets those stem cells thought to initiate solid cancer tumors.
    The American–Canadian collaborative team will lead this first in-human Phase 1 trial testing their new therapy, which has received investigational new-drug approval from the U.S. Food and Drug Administration and Health Canada, Canada's therapeutic regulatory agency. The project has been approved to begin enrolling patients in both the U.S. and Canada".

  4. Another relevant article: Scientists bring stem cell science directly to patients, was published December 13, 2013. An excerpt, about the Disease Team Therapy Development III award funded by the California Institute of Regenerative Medicine (CIRM): "The Disease Team III grant to Dr. Dennis Slamon and Dr. Zev Wainberg and their US-Canadian collaborative team will support the first in human clinical trial scheduled to open in early 2014. The project builds on Dr. Slamon's previous work partially funded by CIRM to develop a drug that targets tumor initiating cells with UCLA's Dr. Zev Wainberg, assistant professor of hematology/oncology and Dr. Tak Mak, director, Campbell Family Institute of the University Health Network in Toronto, Canada".

    The amount of new funding from CIRM to Dennis Slamon was $6,924,317.