Efforts for a clinical trial are in process. The Nathan's Battle Foundation has an agreement with
Cornell University to attempt to initiate a clinical trial using gene transfer for CLN2. A feasibility
study performed by Cornell confirmed the viability of such a project and defined the necessary steps to
achieve a successful clinical trial. We have funded all the the work to date but lack the necessary
funding to continue the project. The development of this life saving therapy will stop without
additional funding. The project will cost over $2 million to complete and we will run out of funding to
support the project in a few months. If you know of anyone that can help please contact them or contact
me to inform them of our cause.
The following is some background information about what we have developed and who we work with:
The Neuronal Ceroid Lipofuscinoses Research Alliance (NCLRA) is a united group of foundations whose
purpose is to aid in the coordination of bringing potential therapies to clinical trials for the three
major forms of NCL (CLN1, CLN2, CLN3). With our united efforts and compassionate purpose, research has
rapidly advanced to a point that a therapy has evolved as a viable treatment. The increased knowledge of
the genetic defects underlying these diseases, coupled with advances in the field of gene transfer and
expression, provides an opportunity to utilize gene therapy strategies in order to treat these
disorders. Gene therapy using the Adeno-associated virus (AAV) has developed to be a promising therapy
for two of the major forms of Nueronal Ceroid Lipofuscinoses (CLN1, CLN2).
Some of the current documentation in organizing the trial is as follows:
The Business Plan outlines a strong business case to pursue a clinical trial on CLN2. Given that many
of the necessary components are in place to move forward with an AAV gene therapy clinical trial. From
having a fast tracking IND drug application, an available and consenting patient population, years of
cross correlating scientific research, a leading scientist, promising financial returns, and the NIH's
and FDA's guidance and support, a CLN1 and CLN2 gene therapy trial a promising and opportunistic
Cell Initiative Event Summary
Phil hosted a dinner at the Society for Neuroscience conference in San Diego
during November 01. The dinner was attended by some of the world's leading
stem scientists, biotech professionals, and NIH representatives for the purpose
of evaluating cell mediated therapy approaches for the NCLs. Several
proposals from attendees are being reviewed.
May 2000, "Clinical Trial Initiative" Conference Summary
This is a summary of the "Clinical Trial Initiative" May 2000 conference. The main objectives of this
conference were to generate pharmaceutical interest in pursuing a therapy for the NCLs, identify
partners in a CLN1/2 clinical trial effort, and to begin defining the next steps to move forward. The
summary contains: ConferenceOverview, Clinical Trial Role Definition, Next Steps, CLN1 research update,
CLN2 research update, Gene Therapy Research, FDA update, Business plan overview discussion, and Bio Tech
November 1999 "Action for Therapy" Conference Summary
This is the conference summary from the November 1999 "Action for Therapy" NCLRA conference at the NIH.
The purpose of this conference was to evaluate possible therapeutic approaches for the NCLs.
Non-For-Profit Therapy Development Model Presentation
This is a copy of the Rare Disease Non-For-Profit Therapy Development model presentation that was
discussed at the Society of Neuroscience conference in New Orleans during the Advocacy Group Breakfast
Business Plan Presentation (May 12, 2000)
This is a copy of the Business plan presentation that was given by Phil at the May conference, "Clinical
Business Plan Cover Letter
This is a copy of a non-specific cover letter for the Business Plan. This cover letter is sent with the
Business Plan to potential clinical trial partners. The intent is to determine the level of interest of
potential partners and evaluate attendees for the upcoming clinical trial business meeting. The letter
outlines the objectives and a tentative agenda.
May 2000 Conference Material (May 12, 2000)
This is a copy of the material that was handed out to each attendee. It contains the conference binder
cover page, agenda, objectives, attendee contact information.
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