Nathan’s Battle Update MeetingNathan’s Battle Update Meeting“The Cure is within Reach”“The Cure is within Reach”

Perry Meridian High School AuditoriumPerry Meridian High School AuditoriumApril 22, 2001 6:30 p.m.April 22, 2001 6:30 p.m.

Anyone and everyone is welcomeAnyone and everyone is welcomeCome Learn How you Can HelpCome Learn How you Can Help

www.nathansbattle.com

Meeting Objectives and GoalsMeeting Objectives and Goals

Update community on accomplishments, successes and challenges

Continue to obtain help from community to solve our funding problem– Define specific ways people can help– Generate other ideas

AgendaAgenda

Background and Accomplishments Cornell Project Details The Problem to Solve Solutions

– Goal– Steps to follow

You can make a difference– Sample materials available

Current events

Background and Background and AccomplishmentsAccomplishments

Nov ‘99 “Action for Therapy” conference Identified potential gene therapy and met with FDA Developed Business Plan to market our disease May 2000 “Clinical Trial Initiative Conference” NBF is nationally recognized by the NIH as a research

leader October NIH conference for Gene Therapy Developed Non-For-Profit approach to develop therapies

and presented to world leader in gene therapy at Cornell

Background and Background and Accomplishments (cont.)Accomplishments (cont.)

NB accepts Cornell’s proposal to perform first phases of Clinical trial project

National media exposure from CBS (48 Hours and The Early Show…)

Cornell’s Feasibility Study returns promising data Received Devastating news on P.J. Negotiated agreement to begin development of the

“cure”/ a therapy. The formal project began 3/15/01 Developed work plan with milestones, tasks,

timeframes, and funding requirements. Raised over $400,000 to date

The Cornell Project DetailsThe Cornell Project Details

Batten Disease – Why gene therapy will work!!!

Formal project in progress with a major university to develop a therapy

Work plan with milestones and time lines Manufacturing and testing started 3/15/01 Clinical trial will begin between 18 and 30

months depending on funding

Project Work PlanProject Work Plan

Feb 2002 – 9 Months

9 Months

6 Months

3 Months

Milestones Min. Estimated Cost

Feasibility study and project planning $125,000

Drug development and testing procedures $300,000

Pre-clinical studies for distribution and expression $500,000

GMP vector process development $300,000

Clinical protocol development $500,000

Toxicology studies Previously included

FDA submission and approvals Previously included

Clinical trial initiation $500,000

Minimum Estimated Total Costs: $2,225,000

Project MilestonesProject Milestones

Project Financial OverviewProject Financial OverviewDate Mile Stones Cost NBF

BalanceNBF Balance After Stage

Need To Raise

October 2000 Feasibility Study $125,000 $440,000 $315,000 -

March-April 2001 Project Initiation $150,000 $315,000 $165,000 -

May 2001 Pre-clinical Studies $100,000 $165,000 $65,000 -

June 2001 ‘’ $100,000 $65,000 - $35,000

July 2001 Drug Production $100,000 - - $100,000

o o - - o

Pre-clinical Studies $100,000 - - $100,000

Protocol Development o - - o

Toxicology Studies o - - o

FDA Submission o - - o

Clinical Trial o - - o

$2,225,000 0 0 $1,785,000

The Problem to SolveThe Problem to Solve

Funding– Project is estimated to cost over $2 million– Cornell agreement of $100,000 per month to

keep the project moving. Cornell is taking a risk to proceed without funding secured

– Our funding runs out at the end of July

How to Solve Funding ProblemHow to Solve Funding Problem

Fund Raising Other Foundations Individual Philanthropists

Goal in Targeting Individual Goal in Targeting Individual PhilanthropistsPhilanthropists

Main goal is to receive funding to support a gene transfer clinical trial.– We need to get our project in front of

individuals who can help solve our problem– Network to identify relationships– Ask for money and you get advice, ask for

advice and you get money

Advantages for a PhilanthropistAdvantages for a Philanthropist A donor can make a difference and save children’s

lives with their financial contributions. A donor will be able to actually see the return on their investment as the project achieves milestones and ultimately saves lives.

Advancing medical science– Future of medicine– Cutting edge technologies

A donor can receive national media exposure for their generosity and good will

Involvement in a high profile, creditable project.

Steps to FollowSteps to Follow

Identify Philanthropic Individuals Research Individuals Interest Approach Philanthropic Individuals Follow-up - Closure

Identifying Philanthropist TargetsIdentifying Philanthropist Targets Library – many sources, Journals, indexes, guides… 990 Forms from NFP (IMA, ICM, Zoo) Internet Annual Reports of NFP Magazines – Time, People, Fortune, Indpls.

Monthly… Newspapers –Star, IBJ, New York Times… Network (ask everyone you know)

– Personal Contacts– Business Contacts– Fraternity /Sorority– High school

– Email

Researching InterestResearching Interest Research background to identify interest

– Children/ Family– Technology– Media attention– Spouse’s interest

Research/network to find relationship with individual (friend of a friend or to a relative of the person)

Customize material for individual and their interest– Materials are on the web site. They are electronic so

that you can customize– Example of targets for each letter type

Approaching the IndividualApproaching the Individual

Hand deliver or have material hand delivered

Mail to address and in a manner that will get the materials reviewed

Follow-up to Obtain ActionFollow-up to Obtain Action

Ask for advice Inquire on status or comments and try to get

action

You Can Make A DifferenceYou Can Make A Difference Challenge each of you You can make a difference Minimum time requirement (approximately 2

hours /week for 4 weeks) – Total of 8 hours needed– Identify 5-10 targets (week 1 - 3 hours)– Research (week 2 - 2 hours)– Approach (week 3 - 1 hour)– Follow-up (week 4 - 2 hours)

We can do it. We can accomplish what the expert told us was not possible.

Sample Materials Available Sample Materials Available

Life Saving Gene Therapy Project Overview* Nathan’s Story Sheet* Sample Wealthy Individual lists Sample Letters from Web site* Cornell Feasibility Cover Letter High Level Cornell Project Work Plan You Can Help! – Handout in the back

* Materials are on the web site

Nathan’s Battle InitiativesNathan’s Battle Initiatives

NIH grant mechanisms Foundations

– Individual’s– Large

Media Contacts – Oprah, Rosie, CNN, national publications

Upcoming Fund Raising Upcoming Fund Raising EventsEvents

Mark Schultz benefit concert - May 13, 2001, 7p.m.– Greenwood Christian Church 2045 Averitt Road;

Tickets available for $15 at Berean Christian Bookstore in the Target Mall in Greenwood, In.

Nathan's Battle Run/Walk - June 2, 2001– Sign-up on your way out

Nathan's Battle at the Ritz - June 22, 2001Tickets: David & Mary Salon, 1540 East 86th Street Indpls.;or by calling 883-2204 or 575-0283 Doors open at 6:30PM

Third Annual Golf Outing – Summer, 2001

SummarySummary We have a potential CURE! We now have a formal project in progress to develop

the CURE with Cornell University Funding needed fast

– Affects project vitality (project can stop)– Affects timeframes

WE NEED YOUR HELP!! (Nathan and P.J. need you)We have limited resources

Networking and personal connections to philanthropists and national media– Identify, Research, Approach, Follow-up– Oprah, Rosie, National Publications

Questions/Comments/SuggestionsQuestions/Comments/Suggestions

Thank YouThank You

“What was once a battle is now a WAR”