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Cystinosis Research Foundation

 18802 Bardeen Avenue
 Irvine, CA 92612
[P] (949) 223-7610
[F] (949) 756-5955
www.cystinosisresearch.org
[email protected]
Nancy Stack
FOUNDED: 2003
INCORPORATED: 2003
 Printable 1 Page Summary
 Printable Profile
Organization DBA --
Former Names --
Organization received a competitive grant from the community foundation in the past five years No
Employer Identification Number 32-0067668 00000

Summary


https://www.youtube.com/user/Natalieswish#p/a/u/0/oFNF-kNIU5E
https://www.youtube.com/watch?v=yPSFTlUvEGc

Mission StatementMORE »

CRF’s mission is to support bench, clinical and translational cystinosis research to find better treatments and a cure for cystinosis.
CRF is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease.


Mission Statement

CRF’s mission is to support bench, clinical and translational cystinosis research to find better treatments and a cure for cystinosis.
CRF is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease.



FinancialsMORE »

Fiscal Year 2017
Projected Expenses $2,747,313.00
Projected Revenue $2,993,000.00

ProgramsMORE »

  • Research Studies and International Research Symposium
  • Progress for Improved Treatment
  • Progress in our Quest for the Cure

Revenue vs. Expense ($000s)

Expense Breakdown 2015 (%)

Expense Breakdown 2014 (%)

Expense Breakdown (%)

No data available

For more details regarding the organization's financial information, select the financial tab and review available comments.


Overview


Mission Statement

CRF’s mission is to support bench, clinical and translational cystinosis research to find better treatments and a cure for cystinosis.
CRF is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease.



Background Statement

The Story of Natalie's Wish: On the eve of her 12th birthday, Natalie made a birthday wish that no child should ever have to make. Her wish, "to have my disease go away forever" was the catalyst for the formation of the Cystinosis Research Foundation. Her parents knew at that moment they needed to make every effort to make Natalie’s wish – and the wish of others with cystinosis – become a reality. Since that time, our extraordinary community of friends and family has helped make Natalie’s wish move closer to reality by donating more than $39.5 million for cystinosis research. The CRF is committed to find better treatments for cystinosis and to find a cure for this devastating disease. 

Impact Statement

The Cystinosis Research Foundation is playing an instrumental and leading role in the field of cystinosis research by awarding multi-year bench, clinical and translational research grants to doctors and scientists around the world. Since 2003, we have funded 151 multi-year research studies at world renowned institutions in 12 countries around the globe. We have targeted several types of cystinosis research including novel treatments, stem cell and gene therapy.
As a direct result of the stem cell research we have funded, the UCLA Institutional Review Board approved the first allogeneic stem cell treatment for cystinosis, clearing the way for a clinical trial using this potential cure. In addition, because of CRF-funded research a delayed-release form of cysteamine was approved by the FDA in 2013 and has greatly enhance the quality of life for our children and their families. Your support has made this possible – you have funded all of the research that has led to these revolutionary advancements in treatments. 

Needs Statement

It was only 14 years ago that the Cystinosis Research Foundation (CRF) was formed with the sole purpose of finding better treatments and a cure for cysitnosis.  We have aggressively pursued cutting-edge research by funding the best and the brightest scientists.  CRF is funding research for a potential new treatment for corneal cystinosis using nanotechnology and anticipates a clinical trial soon.  CRF-funded research discoveries has the  potential to help for other metabolic and neurodegenerative diseases including Huntington's, Batten's, and Parkinson's disease.  We have experienced tremendous growth as others join our efforts to find a cure for this devastating disease, but there is still much to be done.  All of the costs of the CRF are underwritten so that 100%  of your donation goes directly to fund cystinosis research.

CEO Statement

While there are only a small number of patients who suffer from any given “orphan” disease, knowledge discovered by studying one rare disease often leads to advancements in other rare diseases. In fact, today CRF-funded research is helping millions of other people around the world with more prevalent and well-known disorders and diseases such as Huntington’s disease as well as other rare lysosomal storage diseases, kidney diseases and corneal diseases. 


Board Chair Statement

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Other Ways to Donate/Volunteer

Donate online via website: https://www.cystinosisresearch.org/how-to-help/donate-online/
 
Mail a check to:
Cystinosis Research Foundation
Attn: Zoe Solsby
18802 Bardeen Ave
Irvine, Ca 92612 
 
Or call: (949) 223-7610 

Geographic Area Served

Internationally
Since 2003, the Cystinosis Research Foundation has funded 151 multi-year cystinosis research studies in 12 countries around the world.  To date, the CRF has funded and committed over $39 million to cystinosis research. 

Organization Categories

  1. Medical Research - Fund Raising & Fund Distribution
  2. Education - Research Institutes & Public Policy Analysis
  3. -

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Programs


Research Studies and International Research Symposium

Since 2003, the Cystinosis Research Foundation has funded and committed more than $39 million for 151 research studies and fellowships, in 12 countries, which makes the foundation the largest provider of grants for Cystinosis research in the world. CRF-funded researchers are making great progress in both better treatments and a cure for cystinosis, and their work is applicable to other more common diseases such as Huntington's disease and Batten's disease.
Budget  $1,600,000.00
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases & Disorders
Population Served Children and Youth (0 - 19 years) Adults Adults
Program Short-Term Success  Today, with the support of our friends and community we have significantly changed the course of cystinosis research. Our funding efforts have allowed talented doctors and researchers in the area of cystinosis to initiate novel research studies and to advance their research efforts.  In 2006, the CRF established the first Cystinosis Research Fellowship Program designed to support scientists and new researchers who have an interest in cystinosis research. The CRF also hosts an International Research Symposium to foster communication and collaboration among the community of cystinosis researchers.
Program Long-Term Success  There have been two very significant research advancements. The first being the development of a nanowafer for corneal cystinosis by Dr. Ghanshyam Acharya (Baylor College of Medicine) as a result of his collaboration with Dr. Jennifer Simpson (UCI). The second being the announcement that Dr. Stephanie Cherqui (UCSD) is approximately one year away from doing her first patient autologous stem cell transplant - a treatment which could potentially cure cystinosis.
Program Success Monitored By  Board of Directors, Scientific Review Board
Examples of Program Success  It was CRF funded doctors who discovered a delayed-release form of the critical, life-saving medication our children take. A few years ago, there was one medication available to treat cystinosis. It had to be taken every 6 hours of every day. But now, for patients in the U.S., a 12 hour delayed-release medication is available. That medication has led to better compliance and fewer side effects. Since its FDA approval in 2013, hundreds of children and adults with cystnosis are on the new medication - all because of our generous donors

Progress for Improved Treatment

CRF has issued 151 multi-year research grants in 12 countries and those researchers have published over 60 articles in prestigious journals. CRF "seed" money has been leveraged by 5 NIH grants totaling over $5.6 million to two CRF funded researchers. As a result of donor support, we have funded researchers whose work has exponentially increased the breadth and knowledge about cystinosis and as a result of that knowledge, new discoveries have been adchieved.
Budget  $21,707,800.00
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases & Disorders
Population Served Children and Youth (0 - 19 years) Adults
Program Short-Term Success  The research we fund gives us hope that a cure will be found. Hope that one day a life free of cystinosis - no medications, no pain, no hospital visits and blood draws, no worries about life expectancy - will be a reality. Hope lights our way!
Program Long-Term Success  CRF has been funding research that is focused on nanotechnology as a potential new treatment for corneal cystinosis, the painful eye condition that causes photophobia and sometimes blindness as our children reach adulthood. The current treatment for corneal cystinosis is rigorous and requires hourly dosing of medicated eye drops. The eye drops are painful to the eye and compliance is difficult. Dr. Jennifer Simpson at UCI and Dr. Ghanshyam Acharya at the Baylor College of Medicine, have been collaborating on research aimed at finding a better treatment. They have deployed a type of lens - a nanowafer - that can be loaded with medication to treat corneal cystinosis. The nanowafer is placed in the eye and as it dissolves, the medication slowly releases in the eye treating the eye for hours. If this treatment works, it will be a vast improvement over the current treatment. We are excited to report that CRF has obtained the license for the cysteamine-loaded wafer. Owning the license will allow us to drive the research forward with the ultimate goal of a new FDA approved treatment for corneal cystinosis. 
Program Success Monitored By  The Cystinosis Research Foundation's Scientific Review Board monitors progress on this program through six-month updates from the researchers.
Examples of Program Success  It was CRF funded doctors who discovered a delayed-release form of the critical, life-saving medication our children take. A few years ago, there was one medication available to treat cystinosis. It had to be taken every 6 hours of every day but today, for patients in the U.S., a 12 hour delayed-release medication is available. That medication has led to better compliance and fewer side effects. Since its FDA approval in 2013, hundreds of children and adults with cystnosis are on the new medication - all because of our generous donors. There have been two very significant research advancements since last year. The first being the development of a nanowafer for corneal cystinosis by Dr. Ghanshyam Acharya (Baylor College of Medicine) as a result of his collaboration with Dr. Jennifer Simpson (UCI). The second being the announcement that Dr. Stephanie Cherqui (UCSD) is approximately one year away from doing her first patient autologous stem cell transplant - a treatment which could potentially cure cystinosis.

Progress in our Quest for the Cure

We are very close to the first clinical trial for cystinosis using stem cell and gene therapy. Dr. Stephanie Cherqui has been working closely with the FDA for the past couple of years in order to file the IND and to move to a human clinical trial. Dr. Cherqui formed the Stem Cell and Gene Therapy Consortium that has initiated the design of an autologous stem cell transplant treatment to be used for cystinosis. The consortium includes experts in the field of nephrology, neurology, endocrinology, gastroenterology, ophthalmology, bone marrow transplantation and gene therapy.
Budget  $2,984,829.00
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases & Disorders
Population Served Children and Youth (0 - 19 years) Adults
Program Short-Term Success  By using stem cell therapy, Dr. Cherqui and her colleagues have shown a significant improvement in the functioning of the kidney, muscles, brain and eyes of mice with cystinosis.  They were able to decrease the cystine content in these organs by 80%, meaning the symptoms of cystinosis were dramatically reduced using this therapy technique.  This is the first such breakthrough in treatment and a potential cure for cystinosis.
Program Long-Term Success  Last year, these doctors presented us with a plan that will translate the basic research discoveries made in their lab to human clinical trials in patients with cystinosis. We firmly believe that their work, in collaboration with other CRF scientists, will lead to clinical trials to test stem cell therapy in patients with cystinosis within the next few years, bringing us one step closer to a cure for cystinosis.
Program Success Monitored By  Program Success is Monitored by CRF, CRF Board, and our Scientific Review Board.
Examples of Program Success  Dr. Cherqui's research findings were recently published in Blood Journal as the cover feature article.

Management


CEO/Executive Director Chair of the Board Nancy J. Stack
CEO Term Start Apr 2003
CEO Email [email protected]
CEO Experience --

Former CEOs and Terms

Name Start End
-- -- --

Senior Staff

Name Title Experience/Biography
Mrs. Zoe Solsby Vice President --

Awards

Award Awarding Organization Year
-- -- --

Affiliations

Affiliation Year
-- --

External Assessments and Accreditations

External Assessment or Accreditation Year
-- --

Collaborations

--

Staff Information

Number of Full Time Staff 1
Number of Part Time Staff 0
Number of Volunteers 25
Number of Contract Staff 1
Staff Retention Rate % --
Staff Professional Development No

Staff Demographics

Ethnicity African American/Black: 0
Asian American/Pacific Islander: 0
Caucasian: 1
Hispanic/Latino: 0
Native American/American Indian: 0
Other: --
Other (if specified): --
Gender Female: 2
Male: 0
Not Specified --

Plans & Policies

Organization has Fundraising Plan? --
Organization has Strategic Plan? --
Years Strategic Plan Considers --
Management Succession Plan Under Development
Organization Policies And Procedures Yes
Business Continuity of Operations Plan --

Risk Management Provisions

Nondiscrimination Policy Yes
Whistle Blower Policy Yes
Document Destruction Policy Yes
Directors and Officers Insurance Policy Yes

Reporting and Evaluations

Management Reports to Board? Yes
CEO Formal Evaluation and Frequency -- --
Senior Management Formal Evaluation and Frequency Yes Bi-Annually
Non Management Formal Evaluation and Frequency Yes --

Government Licenses

--

CEO Comments

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Foundation Comments

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Governance


Board Chair Chair Nancy J. Stack
Board Chair Company Affiliation Cystinosis Research Foundation
Board Chair Term -
Board Co-Chair Mr. Geoffrey L. Stack
Board Co-Chair Company Affiliation SARES•REGIS Group
Board Co-Chair Term -

Board Members

Name Company Affiliations Status
Mrs. Marcu Alexander Hearts for Hadley Voting
Mrs. Stephanie Cherqui PhD University of California, San Diego Voting
Mr. Bruce Crair Boingo Wireless, Inc. Voting
Mrs. Denice Flerchinger Tina's Hope For A Cure Voting
Mr. Thomas Gendron Woodward, Inc. Voting
Mrs. Traci Gendron Tanner's Time for a Cure Voting
Mr. John Hagestad SARES REGIS Group Voting
Mr. Michael Hayde Western National Group Voting
Dr. Stephen Jenkins MD Sam's Hope for a Cure Voting
Mrs. Erin Little Liv-A-Little Foundation Voting
Mr. John Manly Esq. Manly Stewart & Finaldi Voting
Mr. David Mossman Donahue Schriber Realty Group Voting
Mr. Robert Olson RD Olson Development Voting
Mr. Kevin Partington Cushman & Wakefield Voting
Mrs. Teresa Partington Jenna & Patrick's Foundation of Hope Voting
Mr. Donald Solsby Grandpoint Bank Voting
Mr. Geoffrey Stack SARES REGIS Group Voting
Mrs. Nancy J. Stack Cystinosis Research Foundation Voting
Mrs. Jody Strauss Gabbie's Wish Voting
Mr. Brian Sturgis 24 Hours for Hank Voting

Constituent Board Members

Name Company Affiliations Status
-- -- --

Youth Board Members

Name Company Affiliations Status
-- -- --

Additional Board Members and Affiliations

Name Company Affiliations Status
Ms. Corinne Antignac MD, PhD Lab. of Hereditary Kidney Diseases, Imagine Institute (FRANCE) Voting
Mr. Daniel Bichet MD, MSc Hopital du Sacre-Coeur de Montreal (CANADA) Voting
Mr. Sergio Catz PhD Scripps Research Institute Voting
Mrs. Stephanie Cherqui PhD University of California, San Diego Voting
Mr. Pierre Courtoy MD, PhD Louvain University Medical School; De Duve Institute (BELGIUM) NonVoting
Mr. Francesco Emma MD Bambino Gesu Children's Hospital (ITALY) Voting
Mr. Paul Grimm MD Stanford University School of Medicine Voting
Ms. Julie Ingelfinger MD New England Journal of Medicine; Harvard Medical School Voting
Mr. Martin Konrad MD University Children's Hospital (GERMANY) Voting

Board Demographics

Ethnicity African American/Black: 0
Asian American/Pacific Islander: 0
Caucasian: 20
Hispanic/Latino: 0
Native American/American Indian: 0
Other: --
Other (if specified): --
Gender Female: 8
Male: 12
Not Specified 0

Board Information

Board Term Lengths 3
Board Term Limits --
Board Meeting Attendance % 90%
Written Board Selection Criteria Yes
Written Conflict Of Interest Policy Yes
Percentage of Monetary Contributions 90%
Percentage of In-Kind Contributions 35%
Board Orientation --

CEO Comments

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Foundation Comments

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Standing Committees

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Financials


Revenue vs. Expense ($000s)

Expense Breakdown 2015 (%)

Expense Breakdown 2014 (%)

Expense Breakdown (%)

No data available

Fiscal Year Jan 01, 2017 to Dec 31, 2017
Projected Revenue $2,993,000.00
Projected Expenses $2,747,313.00
Form 990s

2016 Form 990

2015 Form 990

2014 CRF 2014 form 990 FINAL

2013 CRF 2013 form 990 FINAL

2012 Form 990

Audit Documents

2014 CRF 2014 Audited Financial Statement

2013 CRF 2013 Audited Financial Statement

2012 CRF 2012 Audited Financial Statement

IRS Letter of Exemption

IRS Letter of Determination

Prior Three Years Total Revenue and Expense Totals

Fiscal Year 2015 2014 --
Total Revenue $3,282,703 $3,439,384 --
Total Expenses $2,798,362 $3,103,620 --

Prior Three Years Revenue Sources

Revenue By Revenue Source
Fiscal Year 2015 2014 --
Foundation and
Corporation Contributions
$1,476,451 $1,698,868 --
Government Contributions $0 $0 --
    Federal -- -- --
    State -- -- --
    Local -- -- --
    Unspecified $0 $0 --
Individual Contributions -- -- --
Indirect Public Support $0 $0 --
Earned Revenue $0 $0 --
Investment Income, Net of Losses $22,758 $9,190 --
Membership Dues $0 $0 --
Special Events $1,736,676 $1,625,680 --
Revenue In-Kind $28,100 $53,000 --
Other $0 $0 --

Prior Three Years Expense Allocations

Expense By Type
Fiscal Year 2015 2014 --
Program Expense $2,221,488 $2,462,112 --
Administration Expense $111,359 $153,849 --
Fundraising Expense $465,515 $487,659 --
Payments to Affiliates $0 $0 --
Total Revenue/Total Expenses 1.17 1.11 --
Program Expense/Total Expenses 79% 79% --
Fundraising Expense/Contributed Revenue 14% 15% --

Prior Three Years Assets and Liabilities

Assets and Liabilities
Fiscal Year 2015 2014 --
Total Assets $4,779,733 $4,481,739 --
Current Assets $3,801,768 $3,426,545 --
Long-Term Liabilities $0 $0 --
Current Liabilities $2,195,760 $2,308,233 --
Total Net Assets $2,583,973 $2,173,506 --

Short Term Solvency

Fiscal Year 2015 2014 --
Current Ratio: Current Assets/Current Liabilities 1.73 1.48 --

Long Term Solvency

Fiscal Year 2015 2014 --
Long-term Liabilities/Total Assets 0% 0% --
Endowment Value --
Spending Policy --
Percentage(If selected) --
Are you currently in a Capital Campaign? No
Capital Campaign Purpose --
Campaign Goal --
Capital Campaign Dates -
Capital Campaign Raised-to-Date Amount --
Capital Campaign Anticipated in Next 5 Years? --

CEO Comments

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Foundation Comments

Summary financial data is per the Form 990s and consultation with the organization. Foundation/corporate and individual contributions are combined under Foundation and Corporation Contributions.

Documents


Other Documents

No Other Documents currently available.