Growing Stronger Research Fund Overview Jan 2012 For Donors

Our mission is to improve the quality

of life of little people through

supporting medical research

Twins

January, 2012

Presentation not contain medical advice. This presentation is for donors

www.growingstronger.org

SUMMARY

� Achondroplasia is random and rare

� People with this condition are also known as

little people (LP) or dwarfs

� People with Achondroplasia typically go through 4-8


� People with Achondroplasia typically go through 4-8

surgeries to manage bone related complications

� Growing Stronger Research Fund 501(c)(3)

� Goal: Accelerate creation of a drugs to eliminate surgeries

� Method: Working with leading researchers

� Status: Currently funding two researchers. Planning to

expand to ten research sites.

ACHONDROPLASIA� Genetic Mutation

� 80% of little people are born to average

height parents (random, rare)

� Challenges

� Short stature (adult ht. ~4ft), spinal

Short limbs forces

assistance for common tasks


� Short stature (adult ht. ~4ft), spinal

compression, bowed legs, sleep apnea

� Social: Being different. Cannot perform

many daily tasks without assistance

from stools, extenders, adaptors

� GREAT NEWS

� Average intelligence

AWARENESS GROWING

� 6 seasons of TLC primetime

reality show ended in 2010

� Current TLC reality show

started in 2009


started in 2009

� Focused events, groups

AVAILABLE OPTIONS FOR MANAGING

ACHONDROPLASIA

� Lifelong Management of Orthopedic Issues

� up to 8 surgical procedures to reduce pressure on the

upper, middle and lower spine, open air pathways for

sleep apnea, straighten bowed legs

and make room for crowed teeth.


� Assistance Devices (ADs)

� Surgical Limb Lengthening

� Does not improve health. Painful and dangerous.

Only increases height nominally

EMERGING OPPORTUNITY

� Drug therapy on genes and related proteins makes

it possible to manage limited bone growth

� Responsible Mutant Gene FGFR3 identified in 1994

� 4p16.3. 90% of cases occur because of a point mutation in one copy.

G to A, nucleotide 1138


� Potentially reduce the impact of related orthopedic issues

� Improve quality of life with reduced pain

� Eliminate need for many assistance devices

Note: There is no cure to correct genetic mutation.

However, new therapies can significantly

improve the quality of life of little people

RESEARCH TO DRUG TIMELINE

2-5 years1-3 years

Publishlab results

3-5 years

Today

Potential

Achondroplasia

therapies


Drug Development Stage

Lab results

Discovery

lab resultsIP capture

Basic Researchand Theory

Clinical trials

Lab experiments

&Live MouseModels

Companiesget interested

Pre-clinicial trials

Non-profit funding:

NIH, University, Foundations

For-profit:

Companies, VCs

RESEARCH TO DRUG TIMELINE

2-5 years1-3 years

Publishlab results

3-5 years

We need to raise funds to

accelerate promising

Today

Potential

Achondroplasia

therapies


Drug Development Stage

Lab results

Discovery

lab resultsIP capture

Basic Researchand Theory

Clinical trials

Lab experiments

&Live MouseModels

Companiesget interested

Pre-clinicial trials

accelerate promising

research through the lab

and into the hands of drug

companies

Non-profit funding:

NIH, University, Foundations

For-profit:

Companies, VCs

SUPPORTED LABS


ROADMAP FOR RAPID PROGRESS

�Create scientific advisory board� Dr. Michael Ain, Associate Professor,

John Hopkins has agreed to join

�Conduct bi-annual peer advice and drive


�Conduct bi-annual peer advice and drive

research with a focused goal

�Create Relationships with Pharma� Create relationships with value added researchers to

accelerate export of discoveries to Pharmas and drug

development

ROADMAP FOR ORGANIZATION

DEVELOPMENT

Focused on dwarfism. With 10,000+ families in America we can raise $10M+/year. Our immediate goal is to raise $200K/year.

Growing Stronger is the first dedicated non-profit supporting research on dwarfism

Examples of existing non-profits contributing to science


Focused on healthy babies. Raised $187M in 2010. Spent $28M on research to prevent premature birth. Funded some Achondroplasia projects.

Raised $297M in 2010. Invested $72M in research which includes 103 new grants to researchers in academic institutions. Supported 347 research projects in the US, Canada and 9 other countries

Focused on breast cancer. Raised $400M in 2010. Invested $75M in research

http://www.lls.org/content/nationalcontent/pdf/2010annualreport

Examples of existing non-profits contributing to science

http://marchofdimes.com/downloads/2010AnnualReportFinancialSummary.pdf

http://ww5.komen.org/uploadedFiles/SGKFTC_FY10AnnualReport.pdf

RESEARCH STRATEGIES

FGFR3

Mutant

Gene

Expression

FGFR3

Mutant

Protein(suppresses bone

growth)

Bone

Growth

Machinery

impaired

Abnormal

Signals


� We currently are supporting four

different strategies in parallel

1. Block expression of

mutant gene

3. Block/Reduce

effects of the

mutant protein

2. Disable the

Mutant

Protein

4. Modify the

Growth

Machinery to

account for

FGFR3 defect

Strategy 1

� Block expression of mutant gene

� This is a potentially exciting option. Targets have

one normal copy of FGFR3 that can be rescued.

Recent developments in siRNA make this an

attractive solution for silencing the mutant copy.


attractive solution for silencing the mutant copy.

� siRNA is a popular strategy and has worked in

many conditions

Strategy 2a

�Disable Mutant Protein

� Very promising strategy used in Cancer

treatment.

�Add inhibitor that reduces the protective effect of

chaperone protein on mutant FGFR3


chaperone protein on mutant FGFR3

� Strategy works on Achondroplasia cells

� Currently being tested in mice

� Currently funded. Will get more idea about

potential success next year

Strategy 2b

�Disable Mutant Protein

� Small molecule inhibitor of FGFR3

�Analog of NF449

�Strategy works on improving growth in cells


� This approach has been used in other conditions

and it is approved

� Very new approach. Needs more basic research

� Potential research area for support

STRATEGY 3A

� Block/Reduce effects of the mutant protein

� New pathway identified that may be important to

bone growth effect of FGFR3 signals

� In April 2010 discovered new processing step in

FGFR3 protein


FGFR3 protein

� It is possible to leverage existing drugs developed

to treat other diseases for the treatment of

Achondroplasia (FGFR3 protein)

STRATEGY 3B

� Block/Reduce effects of the mutant protein

� Research done in Japan. Strategy looks promising

� Adding a protein (CNP) has shown progress in mice

� Drug companies focused on using CNP


� BioMarin - SF based. Very close to clinical trials

� Chugai Pharmaceuticals - Japanese firm. No update.

� Funded by BioMarin and Chugai

� Clinical Trials Announced Jan 2012

STRATEGY 4

�Modify the Growth Machinery to account

for FGFR3 defect

� Need to understand basic mechanisms for how

FGFR3 affects bone growth

This basic research will help understand what


� This basic research will help understand what

exactly FGFR3 is doing to cartilage cells.

� With more understanding we will be able to

explore other pathways and discover new

potential therapies

UNDER DISCUSSION / EARLY STUDIES

� Case Western University

� Spain

� France

Summary:


Summary:

Achondroplasia is an active area of research with several simultaneous efforts. We are on the edge of a breakthrough.

We need to accelerate lab testing to maintain momentum and reach the end goal – an approved therapy.

NEXT STEPS

�Raise $600K to enable lab testing� Based on years of research multiple strategies are ready to

enter lab testing. If we push we can get into drug development,

clinical trials and approval within next 2-6 years.

� Directly impacts kids under 16


�Use of Funds� Support mentored scientists in 2 labs for the next 3 years

� Will spend $200K/ year for the next 3 years

� $100K pays for a mentored scientist and supplies, maintenance of

mouse models of achondroplasia for one year

Current status: Raised $156K/year. Need to raise $200K/year by Feb 2012

FUNDRAISING

� Plan

� Approach friends and family donors then expand

� Logistics

� Donate to non-profit 501c3

� Funds will be delivered to the target labs on a bi-annual basis

Non-profit operated via


� Funds will be delivered to the target labs on a bi-annual basis

� Follow-up

� We will send a bi-annual report for the next 3 years

providing you with up-to-date status on supporting

current research

DONOR SHEET

Donate via Check or Wire

� Payee: Growing Stronger Research Fund # 1038759

Mail to:

Fidelity® Charitable Gift Fund

P.O. Box 770001

Cincinnati, OH 45277-0053

Please remember to include your name and return address to receive a tax deduction receipt


� If you prefer to wire funds please use the following info: JPMorgan Chase Bank, New York,

NY, ABA Number: 021000021 For credit to: National Financial Services LLC. Account:

066196-221 For benefit of: Fidelity® Charitable Gift Fund, Z– 97– 000442. For further credit

to: Growing Stronger Research Fund # 1038759

CONTACT

� For more information and how to donate please

contact:

Amer Haider, Munira Shamim

Invested parents


Invested parents

email: [email protected], [email protected]

cell: 408-234-0520, 408-828-3387

Health & Medicine

Growing Stronger Research Fund Overview Jan 2012 For Donors