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The Progeria Research Foundation, Inc.

 PO Box 3453
 Peabody, MA 01961
[P] (978) 535-2594
[F] (978) 535-5849
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 Printable Profile (Summary / Full)
EIN 04-3460220

LAST UPDATED: 07/08/2014
Organization DBA Progeria Research Foundation, PRF
Former Names --
Organization received a competitive grant from the Boston Foundation in the past five years No


Mission StatementMORE »

The Progeria Research Foundation’s (PRF) mission is to find treatments and the cure for Progeria and its aging-related disorders.  Progeria is a fatal, “rapid aging” disease that afflicts children, who die of heart disease at an average age of 13 years. PRF was founded in 1999 in response to the complete lack of resources and research on Progeria, and is the only organization in the world solely dedicated to discovering treatments and the cure for this devastating condition.

Mission Statement

The Progeria Research Foundation’s (PRF) mission is to find treatments and the cure for Progeria and its aging-related disorders.  Progeria is a fatal, “rapid aging” disease that afflicts children, who die of heart disease at an average age of 13 years. PRF was founded in 1999 in response to the complete lack of resources and research on Progeria, and is the only organization in the world solely dedicated to discovering treatments and the cure for this devastating condition.

FinancialsMORE »

Fiscal Year Jan 01, 2013 to Dec 31, 2013
Projected Income $2,823,000.00
Projected Expense $2,629,101.00

ProgramsMORE »

  • Clinical Drug Trials
  • PRF Diagnostics Testing Program
  • Public Awareness
  • The PRF Cell & Tissue Bank
  • The PRF Medical & Research Database

Revenue vs. Expense ($000s)

Expense Breakdown 2011 (%)

Expense Breakdown 2010 (%)

Expense Breakdown 2009 (%)

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


Mission Statement

The Progeria Research Foundation’s (PRF) mission is to find treatments and the cure for Progeria and its aging-related disorders.  Progeria is a fatal, “rapid aging” disease that afflicts children, who die of heart disease at an average age of 13 years. PRF was founded in 1999 in response to the complete lack of resources and research on Progeria, and is the only organization in the world solely dedicated to discovering treatments and the cure for this devastating condition.

Background Statement

The concept for PRF began in 1998 when Sam, the son of two Massachusetts physicians, Drs. Leslie Gordon and Scott Berns, was diagnosed with Progeria. They immediately collected as much information as they could find on the disease and discovered there was almost nothing available: no knowledge of its cause, no funding for Progeria research, no treatments and no organization advocating for children with Progeria. Unwilling to accept that there was nothing that could be done for their son and other children with Progeria, they gathered family, friends and colleagues and established PRF in 1999.  Dr. Gordon serves as Medical Director, Dr. Berns leads the Board of Directors and Audrey Gordon, Sam’s aunt, left her law practice to become PRF’s Executive Director. While PRF's origin was truly a family affair, thousands of international experts, dedicated volunteers and other supporters from around the world have since joined in to help advance its mission.

Developed with Dr. Gordon’s biomedical and clinical research insight and knowledge, and her steely determination, PRF’s essential programs provide the resources needed to advance the field of Progeria research and discover more of what Progeria can tell us about heart disease and aging. PRF’s comprehensive programs and services include: an International Registry, Diagnostic Testing, Cell &Tissue Bank, Medical & Research Database, Clinical Trials, Scientific Workshops, Research Grants and Public Awareness. Through PRF’s programs, we achieved extraordinary progress towards our mission, including:

·        The historic discovery of the Progeria gene in 2003, meeting our goal of finding the cause of Progeria

·        Publication of extensive treatment guidelines to improve the children’s quality of life

·        Awarding over $3.5 million in funding for research grants that produced important data in the search for a cure and opened new avenues for discovery in Progeria, heart disease and aging

·        The “Find the Other 150” campaign, a public awareness effort to identify all children with Progeria worldwide, brought the total known children to 100, an 85% increase since the campaign began in October 2009. These children can now receive unique help and specialized care from PRF.

·        A definitive connection was made between Progeria, heart disease and normal aging; thus, finding the cure for one of the rarest diseases on earth may help, not only these special children, but everyone!


Impact Statement

First treatment success. Studies on the genetic cause of Progeria led to finding a potential drug treatment. The 1st-ever clinical trial, funded and coordinated by PRF, tested a farnesyltransferase inhibitor (FTI) lonafarnib which blocks production of the disease-causing protein progerin that builds to toxic levels. Trial results published Sept. 2012 show every child improved in one or more areas, including the cardiovascular system – a particularly exciting finding as heart disease is the children’s ultimate cause of death. With the discovery that drugs can modify the disease, we look to a future cure with great optimism. (Lonafarnib is not yet FDA-approved, so children are now in a new “Triple Trial” that includes the FTI and 2 other drugs being tested.)

 Another promising drug identified.  Researchers published a PRF-funded study identifying a potential new drug treatment: rapamycin. This new study shows that the FDA-approved drug extended the lives of non-Progeria mouse models, decreased the amount of progerin by 50%, improved the cells’ abnormal shape and normalized and extended the lifespan of (PRF-supplied) Progeria cells. Unlike lonafarnib, which prevents progerin formation, rapamycin attacks progerin so cells remove it.

Record number of children identified.  Through the Find the Other 150 Campaign, PRF’s global awareness effort, PRF reached a milestone in 2012: 100 children with Progeria identified. Since the campaign launch in 2009 with just 54 known children, this is an 85% increase. The children and families are now connected to specialized medical care, social support and a chance to enroll in future drug trials.

 2013 goals include moving closer to a cure by:
1.    Continuing and expanding the Triple Trial to include up to 42 more children found after initial enrollment ended in 2010
2.    Beginning a trial to test rapamycin; and

3.    Increasing the number of children in PRF’s programs, so scientists can learn more about Progeria, heart disease and aging.


Needs Statement

Top needs include:
  • Funding the Triple Drug Trial to ensure enrolled children have uninterrupted access to a proven treatment, and additional children can enroll: $990,000
  • Launching the rapamycin trial to explore additional treatment options: $206,190
  • Continuing the global search for all children who have Progeria
  • Expanding staff to increase organizational capabilities: $200,000 
  • Fully funding two years of the PRF Diagnostics program, the entry point for all PRF programs: $161,241
There are new, encouraging findings from Progeria research. The first trial successfully tested a first-ever treatment that improved symptoms in the children, particularly mitigating premature aging in the cardiovascular system. New potential treatments were identified and are being tested in a Triple Drug Trial. With increasing numbers of children identified, PRF programs, beginning with Diagnostics, must expand to meet the needs of the children, their families, researchers and clinicians. Going forward, we must secure resources to maintain and grow the programs that bring us closer to even more effective treatments, and the cure. Our findings will directly benefit children with Progeria, and also aid in our understanding of heart disease and the normal aging process.

CEO Statement

There has never been a more encouraging time for children with Progeria and their families. Progeria studies moved from the lab to treating children in just nine years -  a prime example of successful translational research. This unheard of speed - from gene discovery, to preclinical research, through the first clinical trial which was sucessful, to now a 3-Drug Trial and the planned trial of a 4th drug rapamycin - is fantastic because the children’s need is so immediate, and was accomplished with a relatively small staff and few fiscal resources; however, PRF must obtain new and increased support due to a rapidly growing (known) population, soaring research interest and multi-drug trials that must continue. Several factors contributed to PRF’s rapid progress:

1. PRF co-founder Dr. Leslie Gordon is the mother of a child with Progeria and, as PRF’s Medical Director, provides oversight of PRF's research programs. When Dr. Gordon’s son was diagnosed, her reaction, as both a mother and a scientist, was that she could not accept that there was nothing that could be done for her child. Without hesitation, she left a planned career in pediatrics, and her determination has fueled PRF’s success.  No one in the world has more research and clinical knowledge and experience with Progeria than Dr. Gordon, and she has been recognized in such major media outlets as CNN, Time, the Wall Street Journal and The New York Times for her considerable expertise and accomplishments.

2. Before PRF, virtually no one knew what Progeria was. Now, through PRF’s outreach and educational efforts, millions know about the disease and the far-reaching implications for the aging population. Progeria is especially compelling: it involves children and the effects are clearly seen, as the children are easily identifiable by their unique appearance. Maintaining the disease in the public eye is vital to every aspect of PRF’s work, and spurs more children to be brought to PRF for diagnosis and care. Each year, more children are identified, more researchers apply to PRF for grants and patient cells to support their work, more scientists participate in PRF’s workshops and more progress is made toward the cure.

3. The now-established biological link between Progeria, heart disease and normal aging has stirred intense interest in Progeria research. As PRF searches for a cure, the data gathered may provide keys for treating millions with heart disease or other aging-related conditions.

Board Chair Statement

 My personal interest in co-founding PRF and serving as board chairman results from being the father of a child with Progeria. As a physician and a father, being told that there was nothing that could be done for my child – just “go home and enjoy the time you have left with him” - was unacceptable. My wife, Dr. Leslie Gordon, also a physician, agreed that there had to be something we could do. With the help of family, friends and colleagues, we founded PRF with the optimistic hope that there would be something that science could do for our son and the other children like him, and that we would spearhead this effort.  Indeed, there has been tremendous progress toward treatments and cure. In the 14 years of PRF’s existence, there have been the wonderful highs of discovery and the deep lows of children passing away before these discoveries could result in prolonging their lives. And through it all, we persist in our quest for the cure.

 We have a remarkable board of dedicated, passionate people who donate their talent, time and treasure in a variety of fields, including legal, medical, business and public relations. But even with an active board, we are facing the fall-out from a poor economy, a federal government that is not as financially able to invest in biomedical research as much as it has in the past, and the knowledge that Progeria is a very rare orphan diseases – all at a time when we need to raise even more money than ever, given the growth of every aspect of PRF’s work. We must continue our efforts to educate the world about Progeria and its biological link to heart disease and normal human aging, so that more people support PRF’s mission. The public is captivated by children with Progeria and we have experienced an exponential increase in scientific attention. The idea that finding the cure for Progeria could help millions with heart disease and the entire aging population may also attract the public’s attention. Thus, PRF will continue to focus its messaging on these facts, with the hope that revenue will increase, allowing more funding for clinical trials and other research projects that will continue to propel us forward toward the cure.   

Our successes in the past decade—the identification of the cause of Progeria, the ability to test for the disease and the discovery of the first treatment—are tremendous milestones in such a relatively short period of time.  But we remain in a race against time, as we strive to find even more effective treatments and, ultimately, the cure.

Geographic Area Served

Throughout the United States

PRF programs help 100 children with Progeria (22 in the US) in 37 countries speaking 25 languages. PRF has helped 50 others who have passed away since 1999, and about 100 children who didn’t have Progeria but for whom PRF answered important questions from their families and doctors seeking diagnosis and care advice.  PRF also reaches millions globally through its chapters, website, social media outlets, and media.  PRF has funded 45 researchers in 33 labs in 10 countries, given over 300 cell lines to 57 teams in 12 countries, and recently hosted 180 researchers from 18 countries at its workshop. 


Organization Categories

  1. Medical Research - Specifically Named Diseases Research
  2. Diseases Disorders & Medical Disciplines - Heart & Circulatory System Diseases & Disorders
  3. -

Independent research has been conducted on this organization's theory of change or on the effectiveness of this organization's program(s)



Clinical Drug Trials

Clinical Drug Trials bring children worldwide to Boston Children’s Hospital to test promising treatments. Before these trials, only symptomatic treatments existed, but none affected disease processes. The first trial’s results show that FTI treatment ameliorates some of Progeria’s damage, including lethal heart disease, proving that drugs can influence the disease process and potentially extend the children's lives. The Triple Drug Trial, to be completed in 2014, tests a combination of the FTI and two other drugs with similar modes of action. The first drug trial results are encouraging, and we hope that the Triple Trial will yield even better outcomes. In 2013 we will launch a Phase I "Quad Trial" to add everolimus, a form of rapamycin, which clears cells of toxic progerin. This 15-patient trial will determine drug dosage and address safety issues. It is a first step for the planned larger Phase 2 efficacy trial to test the 4 drugs together in up to 80 children.
Budget  $1,188,401
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases, Disorders & Medical Disciplines, General/Other
Population Served Children and Youth (0 - 19 years) Children and Youth (0 - 19 years) Children and Youth (0 - 19 years)
Program Short-Term Success 

An example of short-term success of PRF’s clinical drug trials program is that of PRF’s first clinical trial, which used a farnesyltransferase inhibitor, lonafarnib, a drug that prevents formation of the toxic protein, progerin, in affected children’s cells. The results were reported in the October 9, 2012 issue of The Proceedings of the National Academy of Sciences ( Of the 25 patients who completed this trial, every child showed improvement in one or more of four ways: gaining additional weight, better hearing, improved bone structure and/or, most importantly, increased flexibility of blood vessels.  This proves that the Progeria disease process can be positively influenced by drugs, a phenomenally successful outcome for the first trial and one that leaves the door wide open for even more successes with additional drugs.

Program Long-Term Success 

The long-term success of PRF’s clinical drug trials program will be that a cure for Progeria will be found. Because PRF funds and co-coordinates the clinical trials, PRF will be aware of the results of the trials and will track all of these children directly and through their local health care providers. The results of the trials, as were the results of the completed first trial, will be published in peer-reviewed scientific or medical journals. The peer review will ensure the integrity and validity of the results.

Program Success Monitored By 

PRF will determine the success of any clinical drug trial through careful acquisition of data through testing various parameters related to the hypothesized action of the drug or drugs tested. As noted above, results will be published in peer-reviewed scientific journals. Oversight of the clinical trials will be carried out by the Boston Children’s Hospital Institutional Review Board and the FDA, which will approve all clinical trials.


Examples of Program Success 

The success of the farnesyltransferase inhibitor clinical trial illustrates that pharmaceutical treatment can ameliorate some symptoms of Progeria. As noted above, all participants who completed the trial showed some improvement in symptoms. As quoted in CNN Health (September 25, 2012), the mother of Devin Scullion, a 16-year-old Progeria patient from Canada, who participated in the lonafarnib trial, noted that her son went from 23 lbs to 31 lbs during the two-year trial. She said, “His energy level is higher, it’s easier to get him up, his eating habits changed, he eats a lot more.” She further saw an improvement in his cardiovascular system, better movement and an increase in activity.


PRF Diagnostics Testing Program

PRF Diagnostics Testing Program offers genetic testing to confirm the diagnosis of Progeria in children. Once the gene was discovered in 2003, the first genetic test for Progeria was developed. In conjunction with clinical data, the test provides a definitive, scientific diagnosis. Earlier diagnosis, fewer misdiagnoses and earlier medical interventions make it possible to provide quality care for the children, and entry into potentially life-saving clinical trials.

A definitive diagnosis is the first step in enrolling children in PRF programs, where they and their families obtain early treatment recommendations and medical and emotional support to improve the children's quality of life during critical, formative years. The test also ensures that only definitively diagnosed Progeria patients are involved in PRF’s programs, maintaining the integrity of the biological materials, medical data and research. Since the gene discovery, 105 children were tested; 73 tested positive for the gene.
Budget  $78,536.00
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases, Disorders & Medical Disciplines, General/Other
Population Served Children and Youth (0 - 19 years) Children and Youth (0 - 19 years) Children and Youth (0 - 19 years)
Program Short-Term Success 

Once a child is brought to the attention of PRF Medical Director Dr. Leslie Gordon, the child’s medical record and photographs will be examined to determine if the child appears to have Progeria. Once so determined, the child is given a genetic test, either for the presence of the specific mutation in the LMNA gene or genetic sequencing to determine if another region in the LMNA gene or another gene that can cause Progeria or a progeroid condition is present. Success of the program is measured by identification of children with this genetic defect. The gene was discovered in 2003. By early 2013, 105 children were genetically tested for Progeria and 73 tested positive.


Program Long-Term Success 

 This program makes it possible for every child born with Progeria to be identified through genetic testing. This allows each child to be offered treatment and participation in clinical drug trials; such trials are expected to last into the foreseeable future. This process will identify every child born who has Progeria and will classify the specific type of Progeria: classic Progeria, in which the child has a truncated LMNA gene, or a Progeria that is from another part of the LMNA gene or another gene. The goal of the diagnostic program is to identify every child worldwide who has Progeria. Genetic testing is the door leading to other PRF services, including the Progeria Handbook, containing treatment guidelines and advice to families and medical personnel; emotional and social support through introductions to other Progeria families; and participation in the Cell and Tissue Bank that provides Progeria cell lines to researchers.


Program Success Monitored By 

 Program success is monitored by successfully identifying children with Progeria from the genetic tests, decreasing the average age at which diagnosis is made, increasing the participants in Progeria clinical drug trials and increasing the number of participants in PRF’s other, research-related programs ( International Registry, Cell & Tissue Bank, Medical & Research Database and Clinical Drug Trials). Children as young as four months of age have been tested by PRF and have been found to be positive for Progeria. Early testingallows children to enter PRF’s programs at an earlier age to manage some of the symptoms before or as they develop. When PRF was founded, there was no way to definitively diagnose a child with Progeria; diagnosis was based on physical examination. Four years later, the genetic defect responsible for Progeria was discovered and a genetic test for the abnormality was developed and implemented within a few months.


Examples of Program Success 

PRF’s public awareness efforts and the public’s fascination with the children and the disease have resulted in tremendous worldwide media coverage that has brought more children with possible Progeria symptoms to PRF. In 2000, just after PRF was established, the organization was aware of 30 children living with Progeria. At the current time, 100 children living with Progeria have been identified; our Cell & Tissue Bank has grown to 189 cell lines; our Medical & Research Database to 120 participants; 427 families, teachers and medical personnelhave received our Handbook; and the drug trial enrollment has grown from 28 in the first trial, to 45 in the current one, to a projected 80-85 in the next one. This across-the-board increase in program participation all starts with genetic testing. Thus, the success of the program is gauged by the ability to accurately diagnose children with Progeria.


Public Awareness

Pro-active public awareness efforts are needed to educate the public about Progeria and its connection to aging through PRF’s website, newsletters, educational materials and social and traditional media. In 2012, print, Internet and broadcast media coverage reached over 206 million people through nearly 400 pieces that aired worldwide, including on every major US TV and cable news network and major outlets in 9 other countries.  

In 2009, PRF launched a global awareness campaign, Find the Other 150, with pro bono support from health communications group GlobalHealthPR. This campaign is successfully searching for other children with Progeria so they can have access to appropriate care. Through media outreach and a special website ( with information in 20 languages, podcasts and photo galleries, the number of known children has jumped from 54 to 100! As worldwide awareness grows, so does the need for international PRF programs, requiring more services and funds.
Budget  $175,000
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases, Disorders & Medical Disciplines, General/Other
Population Served Children and Youth (0 - 19 years) Children and Youth (0 - 19 years) Children and Youth (0 - 19 years)
Program Short-Term Success 

 "Find the Other 150” ( has led to an astounding increase in children with Progeria known to PRF. In 2009, when the program began, PRF knew of 54 children in 30 countries. By February 2013, it was 100 children in 37 countries. Thus, PRF’s pro-active awareness efforts are successful. The media has also contributed to awareness success; PRF has participated in stories appearing in major media sources including CNN,The Wall Street Journal, The New York Times, ABC and CBS News and NPR. The HBO documentary, “Life According to Sam,” is the story of an extraordinary child with Progeria and his parents (PRF co-founders Drs. Leslie Gordon and Scott Berns), their search for a cure and their ability to live life to its fullest despite their difficult situation. The 90-minute documentary will air on HBO in October 2013, bringing awareness of Progeria and the need to find a cure to millions more, including those who know children with Progeria and will refer them to PRF.


Program Long-Term Success 

 PRF’s public awareness program includes publicity and the “Find the Other 150” campaign (, carried out by GlobalHealthPR on a pro bono basis. The Find the Other 150 campaign is a worldwide publicity program to help medical personnel around the world, along with parents and other family members of affected children, to identify and help children who have Progeria. The goal is to find every child in the world who has Progeria and to then offer the child entry into PRF’s research-related programs, including clinical trials. There are specific ways in which to treat these children, and both palliative and, in future, curative treatments will be offered. Worldwide publicity will mean that many people will have heard about Progeria and will understand the urgent need to find a cure and the need to locate each of the 1 in 4,000,000 to 1 in 8,000,000 children born with this syndrome.


Program Success Monitored By 

PRF frequently updates its statistics on the number of children with Progeria who are known to PRF and its programs and the number of countries that are home to these children. As we believe, based on incidence, that there are approximately 200 to 250 children worldwide at any given time who have Progeria, we will be successful when we can accrue as many of these children as possible to our programs and provide them with information about the disease, treatments and, in the future, a cure.

Examples of Program Success 

When the first clinical trial of a drug for children with Progeria was begun in 2007, the 26 enrollees comprised 75% of the world’s population known to PRF. By early 2013, after the 2009 start of the “Find the Other 150” campaign, the number of children known to PRF who have Progeria reached the 100 mark. Thus, PRF’s public awareness campaigns have been and continue to be successful. This is a remarkable achievement. Children from all over the world are being screened for acceptance into PRF’s clinical trials at Boston Children’s Hospital. Informational materials are available in 20 languages; there are podcasts, pictures of the children and other informative materials online. Supporters have organized eight PRF chapters in seven states and their publicity and fundraising events have raised public knowledge of and interest in the syndrome.

The PRF Cell & Tissue Bank

When PRF was founded, there was almost no Progeria research and almost no access to necessary biological materials, such as cells and tissue from affected children. Launched in 2002, the PRF Cell & Tissue Bank is now an essential resource, providing rare cell lines to scientists worldwide who explore the biology of Progeria, search for treatments and cures, and study biological links between Progeria and aging. There is no cost to the children or their families to participate, and all patient information is confidential.

PRF has 189 cell lines and tissue samples from children with Progeria and their families—an impressive number considering the rarity of the disease. In 2011, the bank was expanded to include induced pluripotent stem cells, derived from the children and their parents. These unique cells allow scientists to make Progeria stem cells and create (in the lab) Progeria blood vessel and heart cells for studying heart disease that leads to early death in Progeria.
Budget  $70,000
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases, Disorders & Medical Disciplines, General/Other
Population Served Children and Youth (0 - 19 years) Children and Youth (0 - 19 years) Children and Youth (0 - 19 years)
Program Short-Term Success 

 An important short-term success is the use of the bank’s cells and tissues for scientific research. This includes studies on the pathophysiology of the syndrome at a cellular level. To date, use of the Cell & Tissue Bank has been reported on in 34 scientific papers and the gene for Progeria was discovered using cells from this bank. The presence of the cell and tissue bank and its contents will lead to more scientific discoveries about Progeria: how a single genetic defect causes the entire system to malfunction. The newly derived induced pluripotent stem cells (iPSCs) can develop in the laboratory into nearly any type of cell for experimental purposes, particularly blood vessel and cardiac cells, the types of cells that show rapid aging in Progeria patients. Obtaining such cells from Progeria patients by biopsy would be difficult, would expose the children to infection and could cause other harms. Thus, with iPSCs, cell samples are available without further stressing the child.


Program Long-Term Success 

 PRF’s Cell & Tissue Bank is the largest repository of Progeria cell lines in the world. It contains biological materials from children affected by Progeria and their families. It also contains several lines of induced pluripotent stem cells (iPSCs) derived from mature cells from children with Progeria. iPSCs are reprogrammed to become immature stem cells that can create almost any cell type in the body, but these cells carry the Progeria genetic mutation. These materials are available to researchers to study the changes that occur in cells of patients with Progeria. The use of these biological materials allows researchers to learn more about the disease, and devise new treatments and potential cures. The ultimate changes expected are that Progeria’s biological effects will be prevented or the syndrome can be cured.


Program Success Monitored By 

The most obvious sign of success is that 57 research teams from 12 countries have received 322 cell lines, 61 DNA samples and 34 types of tissue! In addition, the cell bank has grown to house 192 cell lines from 173 individuals, including affected children worldwide, ages two months to 17 years, and their immediate relatives. The tissue and cell bank also has nine iPSC lines, derived from cells from children with Progeria. The requests to use the cell lines and the numbers of publications based on research from the cell lines are also proof of success. Development of concepts for treatments and/or prevention of symptoms of disease or prevention of manifestation of the disease in total through use of materials in the Cell & Tissue Bank would be considered proof of success of the bank itself, as would development of usable tissue from iPSC lines.


Examples of Program Success 

The answer to this is above: the number of cell lines housed in the Cell & Tissue Bank, the number of lines distributed, the numbers of peer-reviewed scientific publications derived from research on cells from the cell bank and the discoveries made from these cells all are examples of this program’s success.


The PRF Medical & Research Database

The PRF Medical & Research Database collects detailed medical data (e.g., medical records, radiology scans, weight and growth charts) on children with Progeria from around the world to learn more about the disease and how it progresses. Through thorough analysis of the data, PRF can devise new directions for studying Progeria, heart disease and aging. Dozens of scientific papers and important findings on disease progression have resulted from use of this database.

Information gathered from the Medical & Research Database was vital to the creation of a portable resource, The Progeria Handbook, published in 2010 by PRF to provide families, researchers and caregivers, such as physicians and teachers, ready access to information ranging from basic health facts and daily care recommendations, to detailed medical treatment guidelines. English, Spanish and Portuguese versions have been distributed to 427 families and caretakers. Additional translations are planned.
Budget  $98,000
Category  Diseases, Disorders & Medical Disciplines, General/Other Diseases, Disorders & Medical Disciplines, General/Other
Population Served Children and Youth (0 - 19 years) Children and Youth (0 - 19 years) Children and Youth (0 - 19 years)
Program Short-Term Success 

 The Progeria clinical drug trials’ primary clinical parameter of weight gain was determined by analyzing data from the database; Such pre-therapy measurements are vital to determining a drug’s success, and this program made it possible to do so. Progress is also measured by the number of scientific papers that used the Database. Since the 2003 gene discovery, the number of peer-reviewed scientific papers about Progeria has increased by 1,500%, with many citing the Medical & Research Database as a resource. Another measurable factor is the creation of treatment guidelines by PRF’s medical director and the Boston Children’s Hospital trial team: TheProgeria Handbook describes in detail treatment strategies for children with Progeria, helping the families and doctors to care for children with Progeria. Also, as information about children’s response to drugs to alleviate Progeria symptoms is added to the database, we can determine the long-term success of these treatments.


Program Long-Term Success 

 The PRF Medical & Research Database is a centralized collection of medical information from Progeria patients worldwide. The data are rigorously analyzed to help researchers and clinicians understand more about the pathophysiology of Progeria. This information has aided and will continue to aid in devising treatment guidelines and recommendations, all of which are included in PRF’s 100-page Progeria Handbook which, to date, has been distributed to 427 families, teachers and medical personnel throughout the world. PRF also uses the medical records data to understand more about the basis of disease in Progeria. This serves as a springboard for new research in Progeria and in the diseases of aging, such asheart disease, which afflict these children at a very young age.The ultimate goal of understanding the biology of Progeria is to identify a cure or cures for this currently incurable and fatal genetic disease. The Database is a vital component of attaining that goal.


Program Success Monitored By 

 Reports by the children’s health care providers, as well as test results from the clinical trial visits tell PRF which treatments are successful and which are not working. This information will be used to modify future versions of the Handbook. The program is monitored by the Institutional Review Board (IRB) approved by the Rhode Island Hospital and the Brown University Committees on the Protection of Human Subjects, providing required oversight.


Examples of Program Success 

 The database is used by clinicians and researchers to further advance basic science and progress toward treatments and cure, and by caretakers to help the children with their medical and social needs. Caretakers such as parents, teachers and social workers must address the sometimes complex and difficult social issues these children face, such as inability to sit in hard seats for long periods or carry heavy books (chair pads, an extra set of books for home and a buddy who will carry the child’s books in school are recommended). The collection of medical data has led to management recommendations, such as keeping the children well hydrated to prevent problems with their prematurely stiffened peripheral blood vessels. Because there are so few cases of Progeria, having a compendium of medical and other knowledge that can be tapped to provide the children a better quality of life has proven extremely successful.


CEO/Executive Director/Board Comments

Hutchinson-Gilford Progeria Syndrome (HGPS) results from a rare genetic mutation in the LMN Agene that occurs spontaneously at conception. Incidence is one in four million live births, so at any given time there are 200-250 children with Progeria living throughout the world. The condition’s occurrence is not affected by gender, race or ethnicity. At birth, the children usually appear normal, but in their early months, changes become evident: they have mid-facial cyanosis (blue coloration from lack of oxygenation), the nose becomes pointed with a narrow bridge, the lower jaw recedes and jaw size remains small, skin on the abdomen and thighs may thicken and they experience profound failure to thrive, with little to no weight gain and very slow growth. From ages 1 to 2 years, the children begin losing their hair, eventually becoming bald.They will lose much of their subcutaneous fat; veins, particularly in the scalp, will be prominently visible. Their joints will become stiff and difficult to move and bone density will decrease. Skin on the abdomen and upper thighs will become tight.Their primary teeth may erupt late and may not be shed. Secondary teeth may only partially erupt. They all develop progressive, severe cardiovascular disease, with strokes or heart attacks as early as ages 5 or 6. Despite all the physical symptoms, mental development is normal. Children die of cardiovascular disease between the ages of 6 and 20 years, with an average age of 13 at death.

PRF created a comprehensive network of research-related needs to promote advances in the field: a cell and tissue bank, diagnostic testing, international registry, grant funding and scientific workshops. Thus, PRF is firmly established as an international force driving Progeria research and education.

PRF’s success leads to challenges: although most of our programs are financially strong, clinical trials are increasingly expensive. As more children with Progeria are identified, more qualify for our clinical trials. These trials cost millions of dollars and costs are increasing. A major cost is airfare to bring children to our clinical trial site in at Children’s Hospital Boston. It is increasing and these delicate children must fly, sometimes from across the world, on direct flights. Other challenges include PRF’s increased research grants program. Awareness of the disease along with flat NIH budgets have increased applications for PRF research grants, and we have responded by increasing grant amounts and lengths. To handle increased program needs, we need more staff and more funds to pay them.



CEO/Executive Director Ms. Audrey Susan Gordon Esq.
CEO Term Start Mar 1999
CEO Email
CEO Experience Atty. Audrey Gordon is a graduate of Tufts University and Northeastern University School of Law, and is an attorney licensed to practice in Massachusetts since 1988 and Florida since 1990. She has 12 years of litigation experience in both states. As an attorney, she developed skills that have been useful in organizing, running and representing PRF, including public speaking, research, writing and corporate organization.

Working closely with the Board of Directors, officers, staff and volunteers, Ms. Gordon is responsible for ensuring The Progeria Research Foundation's financial growth, program development and day-to-day management. She helped spearhead the formation of PRF after her nephew was diagnosed with Progeria. Her vision for PRF is very personal and determined: "We will not stop until we find the cure."

Ms. Gordon has given numerous lectures about her work as leader of a rare disease research organization, including conferences sponsored by the Genetic Alliance, The Milken Institute, and the Whitehead Institute. She has also been recognized for her leadership skills through many awards, including the North of Boston Business and Professional Women of the Year award for non-profit organizations, the Mary Upton Ferrin Award for leadership in her community, and the Community Hero Award by Jewish Family Services . She is a member of the Peabody Chamber of Commerce and the Association of Fundraising Professionals, and serves on the Board of Advisors of Global Genes|RARE Project and the Rare Disease Report.
Co-CEO --
Co-CEO Term Start --
Co-CEO Email --
Co-CEO Experience --

Former CEOs and Terms

Name Start End
-- -- --

Senior Staff

Name Title Experience/Biography
Erin Bettencourt Clinical Trial Coordinator  Erin coordinates travel and lodging for families participating in the clinical trials and provides administrative support for the Executive Director and Medical Director. Before coming to PRF, Erin worked in administration in the Emergency Department at Massachusetts General Hospital.
Michelle Fino Director of Volunteer Activities

Ms. Fino is responsible for all volunteer recruitment, training and supervision, as well as oversight of all volunteer-driven activities including Miracle Makers, Coins to Cure Progeria, workplace events and in-house special events (gala held every 2 years, and annual road race and poker tournament). Working with volunteers to match their skills and interests to activities that will raise awareness and funds for PRF is a top priority for Michelle. Michelle is a graduate of Bridgewater State College and Simmons Graduate School of Management. As founder of the Newburyport Documentary Film Festival, a volunteer driven organization, along with her past experience as an Event and Communications Specialist at a local non-profit, Michelle has extensive knowledge and lots of enthusiasm for working with volunteers.


Lynne MacKenzie Donor and Social Media Specialist

Donor and Social Media Specialist:Handle all Donor activity from processing donations to thanking donors (includes general donors, Chapter event and fundraising participants, monthly donors, matching gifts, major gifts, PRF web store, etc.). Social Media Manager: Daily upkeep, growth and tracking of all social media channels (Facebook, Twitter, Pinterest, YouTube, Instagram, etc.) and online marketing of The Progeria Research Foundation.

Lynne is a 1988 graduate of Salem State College, where she majored in English/Art. After working many years as a Marketing Representative for A&M and Virgin Records, she shifted to administration as a Sales Assistant at Epic Records and Office Assistant at U
Gina Marie Ragonese Administrative Assistant

As Administrative Support Specialist and Workshop Coordinator for The Progeria Research Foundation, Gina handles a variety of administrative duties at the busy PRF office.   Her Project Management Masters Certificate from Boston University helps her keep the office running smoothly. 


Award Awarding Organization Year
Early Achievement Award for significant scientific or academic achievement/Leslie Gordon Brown Medical Alumni Association 2013
Community Hero/Audrey Gordon Jewish Family Services 2009
Charles O. Cooke, M.D., Distinguished Visiting Lecture/Leslie Gordon Brown University 2007
Working Mother of the Year/Leslie Gordon Working Mother Magazine 2006
Mary Upton Ferrin Award of leadership in community/Audrey Gordon Peabody Chamber of Commerce 2005
Woman of the Year for non-profit organizations/Audrey Gordon North of Boston Business and Professional Women 2004
Recognition for contribution to cloning of Progeria gene and Progeria research/Leslie Gordon Gerontological Society of America 2003
Outstanding Academic Achievement Award/Leslie Gordon Women in Medicine 1998


Affiliation Year
National Organization for Rare Disorders - Member 2009
AFP (Association of Fundraising Professionals) 2005
Chamber of Commerce 2004
-- --
-- --
Member of state association of nonprofits? Yes
Name of state association --

External Assessments and Accreditations

External Assessment or Accreditation Year
Charity Navigator - 4 Star Rating (2013) 2012
Better Business Bureau Wise Giving Alliance 2008


Academic collaborations include:
  • Brown University--provides Institutional Review Board (IRB) approval and staff support for three of PRF’s programs: Database, Diagnostics and Cell Bank. PRF partners with Boston Children’s Hospital, Dana-Farber Cancer Institute, Brigham and Women’s Hospital and NIH to conduct Progeria clinical drug trials.  
  • The National Human Genome Research Institute (NHGRI) of the NIH has been a full partner. Dr. Francis Collins (NIH director) has worked with Dr. Gordon since 2001, co-discovering the Progeria gene, creating a Progeria mouse model and identifying rapamycin as a drug of interest; PRF funds a National Cancer Institute researcher.
  • PRF partners with Rutgers University Cell and DNA Repository in New Jersey and Rhode Island Hospital for storage and distribution of cell lines for the PRF Cell & Tissue Bank. In 2010, in collaboration with The University of Toronto and Dr. William Stanford, Canada Research Chair in Stem Cell Bioengineering & Functional Genomics and co-Scientific Director of the Ontario Human iPS (Induced Pluripotent Stem) Cell Facility, PRF funded creation of a cutting-edge new research technology, expanding the PRF Cell & Tissue Bank to include Progeria iPSCs, which are used to create cells for research on heart disease in Progeria.

CEO/Executive Director/Board Comments

With the exception of the small staff, everyone involved with PRF, including its Board of Directors, committee members, corporate officers, chapter leaders, translators and those who organize special events throughout the world, generously volunteer their time, energy and talents to PRF so that less is spent on administrative costs and more on raising awareness and finding a cure for Progeria. There are approximately 100 people who volunteer regularly, an additional 200 who volunteer occasionally, and several hundred who have volunteered at least once.

In addition to the collaborations described previously, PRF has developed the following pro bono and collaborative partnerships to support research, maximize limited resources and move as quickly as possible toward a cure.
  • GlobalHealthPR is the largest organization dedicated exclusively to healthcare communications worldwide, with members in the US, Europe, Latin America and Asia. Its leader, Spectrum Communications based in Washington, DC, has been providing pro bono services to PRF since the 2003 Progeria gene discovery. In October 2009, PRF and GlobalHealthPR (again working pro bono) launched a tremendously successful and ongoing global awareness campaign called Find the Other 150, to search for unidentified children with Progeria worldwide. JCR Design of Foxboro, MA developed PRF’s logo, brochure, posters, events and continues to provide graphic design work on a pro bono basis. Others providing similar services include Brandu Marketing in Texas, and Regina Printing in New Jersey.
  • Matrix Financial completes all of PRF’s accounting and tax services on an unpaid basis.
  •  Weil, Gotshal and Manges, LLP, Sidley Austin, LLP, and Quinn Emmanuel, LLP law firms provide pro bono legal services.
Nevertheless, despite all these volunteered services, limitations on the ability to manage the foundation relate to the small staff, insufficient funds to support a larger staff, lack of an endowment and, most importantly, growth of the foundation and its services that could rapidly outstrip its ability to perform its significant functions. This puts PRF, an otherwise successful foundation, in the position of needing additional, new sources of funding. We would be particularly vulnerable if we lost some of the pro bono services we now receive or if we could not fund our clinical trials. Thus, we are looking for funding organizations or people willing to make contributions that can put the foundation on a more secure financial footing.

Foundation Comments


Staff Information

Number of Full Time Staff 5
Number of Part Time Staff 0
Number of Volunteers 101
Number of Contract Staff 2
Staff Retention Rate % 60%

Staff Demographics

Ethnicity African American/Black: 0
Asian American/Pacific Islander: 0
Caucasian: 5
Hispanic/Latino: 0
Native American/American Indian: 0
Other: 0
Other (if specified): --
Gender Female: 5
Male: 0
Not Specified 0

Plans & Policies

Organization has Fundraising Plan? Under Development
Organization has Strategic Plan? Yes
Years Strategic Plan Considers 5
Management Succession Plan Under Development
Business Continuity of Operations Plan No
Organization Policies And Procedures Under Development
Nondiscrimination Policy Yes
Whistle Blower Policy Yes
Document Destruction Policy Yes
Directors and Officers Insurance Policy Yes
State Charitable Solicitations Permit Yes
State Registration Yes

Risk Management Provisions


Reporting and Evaluations

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


Board Chair Mr. Scott D. Berns MD, MPH
Board Chair Company Affiliation Sr. VP Chapter Programs, March of Dimes, White Plains, NY
Board Chair Term Sept 2005 -
Board Co-Chair --
Board Co-Chair Company Affiliation --
Board Co-Chair Term -

Board Members

Name Company Affiliations Status
Carl Alviti CPA Partner at Matrix Financial, LLC, Wellesley, MA Voting
Karen Ballack Esq. Partner at Weil, Gotshal & Manges LLP, Silicon Valley, CA Voting
Scott D. Berns MD, MPH Sr. VP Chapter Programs, March of Dimes, White Plains, NY Voting
Sandra Bresnick Esq. Partner at Quinn Emanuel Urquhart & Sullivan, LLP, NY, NY Voting
Rabbi Harold Kushner International Writer and Public Speaker, Natick, MA Voting
Suzette Kushner MS Natick, MA Voting
John Marozzi President/CEO of Bell-Mark Sales Company, Pine Brook, NJ Voting
Kim Paratore Fundraiser, Wenham, MA Voting
John Seng CEO, Spectrum Communications, Washington, DC Voting

Constituent Board Members

Name Company Affiliations Status
-- -- --

Youth Board Members

Name Company Affiliations Status
-- -- --

Advisory Board Members

Name Company Affiliations Status
Monica Kleinman MD. -- Voting
George M. Martin MD -- Voting
Robert K. Morrison -- --
Robert Schwartzberg CFRE -- Voting

Board Demographics

Ethnicity African American/Black: 0
Asian American/Pacific Islander: 1
Caucasian: 8
Hispanic/Latino: 0
Native American/American Indian: 0
Other: 0
Other (if specified): --
Gender Female: 4
Male: 5
Not Specified 0

Board Information

Board Term Lengths 3
Board Term Limits 2
Board Meeting Attendance % 80%
Written Board Selection Criteria No
Written Conflict Of Interest Policy Yes
Percentage of Monetary Contributions 100%
Percentage of In-Kind Contributions 78%
Constituency Includes Client Representation Yes

Standing Committees

  • Advisory Board / Advisory Council
  • Development / Fund Development / Fund Raising / Grant Writing / Major Gifts
  • Public Policy/Advocacy
  • Scientific Advisory

CEO/Executive Director/Board Comments

PRF is an effective, well-run organization that relies on 5 staff members and is led by Attorney Audrey Gordon, Executive Director since its founding and aunt of Sam, who has Progeria. The governance of the corporation is vested in a national 9-member Board of Directors that serves without compensation and meets quarterly. Volunteer-driven Board committees include Public Awareness, Development and Medical Research. There is also a volunteer Medical Director and corporate officers. These highly experienced, multidisciplinary groups include professionals with years of experience in the legal, accounting, medical and business fields, as well as family members of children with Progeria, all of whom are passionately invested in PRF’s mission.
Staff include an Executive Director, Clinical Trial Coordinator, Director of Volunteer Activities, Donor and Communications Specialist and Administrative Assistant. Working with PRF are also a part-time grant writer and major gifts officer. In addition, PRF works with expert legal, public relations and graphic design professionals, all of whom provide pro bono services.

To achieve the best results in the shortest time, PRF strives to become part of local communities with the help of local organized groups. Thus, chapters were formed to help raise public awareness and conduct local fundraising events for PRF. There currently are 8 PRF chapters in 7 states, all run by families of children with Progeria. In addition, dozens of other volunteers around the globe raise awareness and funds in their communities for PRF.

Media coverage of PRF and the children has increased. The scientific community’s interest in Progeria has also grown at a phenomenal rate, with the potential to unlock keys to heart disease and aging. Having begun PRF at a time when virtually no one knew what the disease was and with no scientific support, it is a proud testament to the hard work of PRF and its support system. However, challenges still abound. They include increased program expenses due to the rise in interest and number of children in our programs. Research grants rose from 2-year grants at $50K/yr. to 3-year grants at up to $100K/year. NIH’s National Institute on Aging; National Heart, Lung and Blood Institute and the Office of Rare Diseases have provided support for PRF’s 7 scientific workshops. With NIH funding decreased, this funding is in jeopardy for the future. The 1st trial involved 28 children and cost nearly $2 million; we will now enroll 80 at a cost of $4 million, so that every child has a chance at a longer life.

Foundation Comments



Revenue vs. Expense ($000s)

Expense Breakdown 2011 (%)

Expense Breakdown 2010 (%)

Expense Breakdown 2009 (%)

Fiscal Year Jan 01, 2013 to Dec 31, 2013
Projected Income $2,823,000.00
Projected Expense $2,629,101.00
Form 990s

2011 Form 990

2010 Form 990

2009 Form 990

Audit Documents

2011 Audited Financials

2010 Audited Financials

2009 Audited Financials

IRS Letter of Exemption

IRS Letter of Determination

Prior Three Years Total Revenue and Expense Totals

Fiscal Year 2011 2010 2009
Total Revenue $2,028,547 $1,703,301 $1,569,511
Total Expenses $1,368,471 $1,241,034 $994,338

Prior Three Years Revenue Sources

Fiscal Year 2011 2010 2009
Foundation and
Corporation Contributions
-- -- --
Government Contributions $159,130 $351,844 $0
    Federal -- -- --
    State -- -- --
    Local -- -- --
    Unspecified $159,130 $351,844 --
Individual Contributions $1,399,770 $1,118,873 $1,131,357
Indirect Public Support -- -- --
Earned Revenue -- -- --
Investment Income, Net of Losses $24,329 $22,046 $41,115
Membership Dues -- -- --
Special Events $445,318 $210,538 $397,039
Revenue In-Kind -- -- --
Other -- -- --

Prior Three Years Expense Allocations

Fiscal Year 2011 2010 2009
Program Expense $1,170,685 $1,050,455 $830,159
Administration Expense $149,077 $140,049 $118,266
Fundraising Expense $48,709 $50,530 $45,913
Payments to Affiliates -- -- --
Total Revenue/Total Expenses 1.48 1.37 1.58
Program Expense/Total Expenses 86% 85% 83%
Fundraising Expense/Contributed Revenue 2% 3% 3%

Prior Three Years Assets and Liabilities

Fiscal Year 2011 2010 2009
Total Assets $3,562,853 $2,820,342 $2,411,538
Current Assets $3,368,343 $2,635,718 $2,234,686
Long-Term Liabilities $0 $0 $0
Current Liabilities $108,431 $25,996 $79,459
Total Net Assets $3,454,422 $2,794,346 $2,332,079

Prior Three Years Top Three Funding Sources

Fiscal Year 2011 2010 2009
1st (Source and Amount) -- --
-- --
-- --
2nd (Source and Amount) -- --
-- --
-- --
3rd (Source and Amount) -- --
-- --
-- --

Financial Planning

Endowment Value --
Spending Policy Income Only
Percentage(If selected) --
Credit Line No
Reserve Fund Yes
How many months does reserve cover? 20.00

Capital Campaign

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? Yes

Short Term Solvency

Fiscal Year 2011 2010 2009
Current Ratio: Current Assets/Current Liabilities 31.06 101.39 28.12

Long Term Solvency

Fiscal Year 2011 2010 2009
Long-term Liabilities/Total Assets 0% 0% 0%

CEO/Executive Director/Board Comments

The extraordinary progress PRF has enjoyed in its 13-year history, the dedicated Board, staff, researchers, clinicians, volunteers and donors, the courageous families and children with Progeria, and the attentive media have all created wonderful opportunities for PRF to grow and succeed.  We are continually working to recognize these champions, increase their involvement and cultivate new supporters as our financial needs grow and our race against time continues.

From the beginning, our greatest challenge has been educating people on what Progeria is, and getting the research community involved.  Thanks to worldwide media attention and social media, millions now recognize it, and we are finding children in remote areas around the globe. We address the need to raise awareness in many ways, including our “Find the Other 150” Campaign, frequent posts on our 6 social media accounts, PRF spokespeople interviews with major media outlets, press releases on major breakthroughs such as the 2012 discovery of the first-ever treatment, and the HBO documentary “Life According to Sam” coming out in October 2013. 

Progeria is one of the rarest diseases on earth; there are 200-250 children living with Progeria throughout the entire world.   Yet there is a proven biological connection between Progeria, heart disease and aging.  We must break down that barrier label of “rare disease” and effectively relay the message that finding a cure for this rare disease could help treat millions with heart disease, and impact the aging that affects us all. 

With its comprehensive programs including a cell and tissue bank, grant funding, international registry and diagnostics testing, PRF is hailed as a model for disease research organizations and a prime example of a successful translational research advocacy group, moving from creation, to gene discovery, to clinical trials to treatments in just 13 years.   And while helping this handful of children, the now-documented connection of Progeria to common heart disease and aging has tremendous universal implications.  

The progress – and thus our work - continues at an exponential rate.  Because of our work, researcher interest in the field is at an all-time high, and families have true hope that their children will live long, healthy lives. But our revenue must keep up with that pace as well, which is a challenge for every non-profit.  In 2013, we are increasing our staff to help meet this challenge, and are embarking on an international fundraising and awareness campaign related to the HBO documentary release.  


Foundation Comments

Financial summary data in charts and graphs are per the organization's audited financials.  Contributions from foundations and corporations are listed under individuals when the breakout was not available.


The Impact tab is a section on the Giving Common added in October 2013; as such the majority of nonprofits have not yet had the chance to complete this voluntary section. The purpose of the Impact section is to ask five deceptively simple questions that require reflection and promote communication about what really matters – results. The goal is to encourage strategic thinking about how a nonprofit will achieve its goals. The following Impact questions are being completed by nonprofits slowly, thoughtfully and at the right time for their respective organizations to ensure the most accurate information possible.

1. What is your organization aiming to accomplish?


2. What are your strategies for making this happen?


3. What are your organization’s capabilities for doing this?


4. How will your organization know if you are making progress?


5. What have and haven’t you accomplished so far?