Dutch Group marks 1 year anniversary of D-Day fundraising

/Dutch Group marks 1 year anniversary of D-Day fundraising

Dutch Group marks 1 year anniversary of D-Day fundraising

By |2014-09-17T09:59:41+00:00August 1st, 2011|Fundraising, News|

Life Raft Group Research Team member, Dr. Brian Rubin and Life Raft Group’s Development Director, Christine Schaumburg, visited Amsterdam in June to recognize the one year anniversary of the launch of Project D-Day with Jeroen Pit and his colleagues who, combined, raised $2.1 Million for the D-Day project in a matter of weeks.

Dr. Rubin presented the latest D-Day project update, first describing the overall goal to leverage the success of tyrosine kinase inhibitors such as Gleevec, which is primarily cytostatic (cells divide but don’t die), to discover new gene/protein targets to cure GIST. He highlighted two issues:

  • Patients require lifelong therapy with Gleevec or other tyrosine kinase inhibitors
  • Patients develop acquired resistance to Gleevec (50% within two years of initiation of therapy)

Dr. Rubin described the four main components of the D-Day project:

1. Sequencing the GIST genome
2. Gene knockdown (RNAi) in GIST models
3. Drug screening
4. Gene target & drug validation

Results were the main focus of Dr. Rubin’s presentation in which he reported that sequencing is well underway and that the next phase will select the most interesting mutations and validate them on an independent set of GISTs to see if they are common to GIST.

Regarding gene knockdown, Rubin reported that population and individual RNA screens have been performed. Analysis is ongoing while validation has been initiated. In addition, new RNA libraries will be obtained to perform additional screens.

The drug screening portion of the progress report contained the following:

 The project is screening both compounds that are already developed for clinical use in humans and are even FDA-approved in some cases, but have not been evaluated in GIST along with the evaluation of a library of over 1,000 drug-like molecules that would require further development to become drugs.

 A substantial number of compounds have been evaluated and several KIT inhibitors have been identified and/or validated as well as several compounds that appear to exert activity against GIST without inhibiting KIT. The latter group is interesting because they could potentially be combined with KIT inhibitors such as Gleevec.

  • Chemical library screening will be initiated this summer.
  • Validation of compounds is ongoing.

The D-Day project was launched in 2010, following Jeroen Pit’s investigation into the best way to donate to GIST research and perhapsrubin 1yr dday_pub help save his own life. After consulting several leading GIST researchers, the patient decided that the Life Raft Group Research Team gave him the best chance for finding a cure. He raised 2 million dollars and challenged us to accelerate our efforts to keep him and other GIST patients resistant to Gleevec and Sutent alive. We added one million dollars and the result is the aptly named D-Day Project. Like that extraordinary day in 1944, when the Allied forces stormed the beaches of Normandy, our attack on GIST treatment resistance has reached an historic opportunity for success, focusing on the four key areas of sequencing, gene knockdown, drug screening and validation.

An additional $500,000 was raised to supplement the project in early 2011. A portion of this funding allowed for an additional scientist to work on drug screening. The Drug Screening Program will be expedited with two additional scientists added to the team thanks to another $150,000 grant from the Pit family.

Also during their visit to Amsterdam, Dr. Rubin and Christine Schaumburg had the great privilege of inducting Jeroen Pit into the Life Raft Group’s GIST Hall of Fame, which was created as a part of the LRG’s GIST 2010: A Decade of Difference Gala to honor the greatest contributions to the GIST community. Previous inductees include Dr. Brian Druker & Dr. George Demetri. The first patient to be inducted, Jeroen was recognized for his contributions to help find a cure for GIST patients like him and challenging GIST research.

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