Growing Stronger Research Fund aims to accelerate medical research to improve quality of life for people with Achondroplasia, the most common form of dwarfism. Their goal is to eliminate the need for multiple orthopedic surgeries by developing drug therapies. They are currently funding four research strategies, including blocking expression of the mutant gene or disabling the mutant protein. Their next steps are to raise $600,000 over three years to support additional lab testing and advance promising strategies toward clinical trials and approval.

1. Twins
Our mission is to improve the quality
of life of little people through
supporting medical research
Presentation not contain medical advice. This presentation is for donors
www.growingstronger.org January, 2012

2. 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
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.
www.growingstronger.org

3. ACHONDROPLASIA
Genetic Mutation
80% of little people are born to average
height parents (random, rare) Short limbs forces
assistance for common tasks
Challenges
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
www.growingstronger.org

4. AWARENESS GROWING
6 seasons of TLC primetime
reality show ended in 2010
Current TLC reality show
started in 2009
Focused events, groups
www.growingstronger.org

5. 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
www.growingstronger.org

6. 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
www.growingstronger.org

7. Today
Potential
Achondroplasia
RESEARCH TO DRUG TIMELINE therapies
3-5 years 1-3 years 2-5 years
Publish
lab results
Lab IP capture Pre-clinicial
Discovery experiments
Companies trials
&
get interested
Live Mouse
Models
Drug Development
Stage Clinical
Lab results trials
Basic Research
and Theory
Non-profit funding: For-profit:
NIH, University, Foundations Companies, VCs
www.growingstronger.org

8. Today
Potential
Achondroplasia
RESEARCH TO DRUG TIMELINE therapies
3-5 years 1-3 years 2-5 years
We need to raise funds to
Publish
accelerate promising results
lab
Lab
research through theIPlab
Discovery experiments
capture Pre-clinicial
trials
Companies
&
and into the Live Mouseof drug get interested
hands
companies
Models
Drug Development
Stage Clinical
Lab results trials
Basic Research
and Theory
Non-profit funding: For-profit:
NIH, University, Foundations Companies, VCs
www.growingstronger.org

9. SUPPORTED LABS
www.growingstronger.org

10. 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
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
www.growingstronger.org

11. 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.
http://marchofdimes.com/downloads/2010AnnualReportFinancialSummary.pdf
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 http://www.lls.org/content/nationalcontent/pdf/2010annualreport
Focused on breast cancer. Raised $400M in 2010. Invested
$75M in research
http://ww5.komen.org/uploadedFiles/SGKFTC_FY10AnnualReport.pdf
www.growingstronger.org

12. RESEARCH STRATEGIES
FGFR3 Bone
FGFR3 Mutant Abnormal Growth
Mutant Expression Protein Signals Machinery
Gene (suppresses bone
growth) impaired
2. Disable the 3. Block/Reduce
1. Block expression of
Mutant effects of the
mutant gene
Protein mutant protein
4. Modify the
Growth
Machinery to
account for
We currently are supporting four FGFR3 defect
different strategies in parallel
www.growingstronger.org

13. 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.
siRNA is a popular strategy and has worked in
many conditions
www.growingstronger.org

14. 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
Strategy works on Achondroplasia cells
Currently being tested in mice
Currently funded. Will get more idea about
potential success next year
www.growingstronger.org

15. 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
www.growingstronger.org

16. 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
It is possible to leverage existing drugs developed
to treat other diseases for the treatment of
Achondroplasia (FGFR3 protein)
www.growingstronger.org

17. 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
www.growingstronger.org

18. 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
exactly FGFR3 is doing to cartilage cells.
With more understanding we will be able to
explore other pathways and discover new
potential therapies
www.growingstronger.org

19. UNDER DISCUSSION / EARLY STUDIES
Case Western University
Spain
France
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.
www.growingstronger.org

20. 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
www.growingstronger.org

21. Non-profit operated via
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
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
www.growingstronger.org

22. DONOR SHEET
Donate via Check or Wire
Payee: Growing Stronger Research Fund # 1038759
Mail to:
Fidelity® Charitable Gift Fund Please remember to include your name and return
P.O. Box 770001 address to receive a tax deduction receipt
Cincinnati, OH 45277-0053
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
www.growingstronger.org

23. CONTACT
For more information and how to donate please
contact:
Amer Haider, Munira Shamim
Invested parents
email: amerhhh@gmail.com, ms856@columbia.edu
cell: 408-234-0520, 408-828-3387
www.growingstronger.org