THE YEAR IN REVIEW

• MFRF Mission Update:  We announced 15 children having been dosed with experimental gene therapy, the touching story of one of the participating families, the coveted "See the Light" Award presentation, and new work with accelerated/low cost drug development programs.
• "Sweetness and Laughter" Entertainment:
• Bob Costas: 29 Emmy Award Winner shared his wit and wisdom while a premier Virtual Magic Show dazzled our audience

• 5 more nursing scholarships were awarded across our South Florida nursing college partnerships. 
• MFRF's team of experts conducted interactive virtual seminars for over 200 students, across multiple Nova Southeastern University and Florida International University campuses combining nursing care and screening education.

THE YEAR IN REVIEW

THE VIRTUAL GALA SERIES TRILOGY: For more details and videos:
While circumstances made it impossible to be together in person, the Mathew Forbes Romer Foundation pioneered a mini-series Gala celebration uniting our vast community of affected families, partners from industry, medicine, research and academia, and very generous supporters.

It was heartwarming and inspiring to have more than 300 smiling faces and participants on Zoom-- Laughing, Learning, and Loving...

RESEARCH:

• Conceptualized and grew the National Research Initiative with NTSAD to 64 projects, and over $4 million in grants leading to many promising therapies. This has generated over $20 million in funding from the NIH and similar institutions
• Supporting the University of Florida Powell Gene Therapy Center to help develop Gene Therapies for fatal genetic diseases of the brain
• Helped establish the Tay-Sachs Gene Therapy Consortium reinforcing pre-clinical and extraordinary efforts toward clinical trials
• Sponsors the Medical and Scientific Sessions at the annual NTSAD Family Conferences showcasing progress and opportunities

SCREENING:

• Hosted 23 South Florida Genetic Testing and Education Fairs
• Screened over 1,600 potential parents with as high as 50 percent detection, with JScreen and many other partners
• Programs range from prevention to now identifying opportunities for early intervention

NURSING:

• Funded more than 40 nursing scholarships with Florida Atlantic University, Florida International University, and Nova Southeastern University
• Helped conceive and implement nursing school training programs:
  • Classroom-based from caring to treating
  • Lab simulation of in-home/community based family care

THE SWEETNESS AND LAUGHTER GALA 2020

2020 Electronic Program:

MFRF has celebrated wonderful progress in gene therapy patient trials, education, and genetic testing for many affected children and families with fatal genetic diseases of the brain. Still, there is so much more to do, including earlier detection and expanding treatment availability.

MFRF has provided leadership and raised over $2.2 million of funding for projects spanning its mission and vision across three pillars: Research of Treatments and Cures, Education and Genetic Screening, and care through Nursing School Educational programs.

RESEARCH:

• Conceived of and growing the National Research Initiative to 61 projects, $3.9 million in grants and many promising therapies with NTSAD. This has led to over $20 million dollars in funding from the National Institutes of Health (NIH) and similar institutions.
• Helped establish the Tay-Sachs Gene Therapy Consortium reinforcing pre-clinical and extraordinary efforts toward clinical trials.
• Sponsors the Medical and Scientific Sessions at the annual NTSAD Family Conferences showcasing the progress and opportunities.

SCREENING:

• Hosted 21 South Florida Genetic Testing and Education Fairs.
• Screened over 1,550 potential parents with as high as 50 percent detection, with JScreen, Victor Center and many other partners.
• Transitioning from Prevention to now identifying opportunities for Intervention.

NURSING:

• Funded more than 35 nursing scholarships with Florida Atlantic University, Florida International University, and Nova Southeastern University.
• Helped conceive and implement nursing school training programs.
• Classroom based.
• Lab simulation of in-home/community based family care.
• From caring and maintaining quality of life to treating.

THE SWEETNESS AND LAUGHTER GALA 2019

We had an incredible Celebration of Gene Therapy for our “Sweetness and Laughter” Gala and “See the Light: Awards.

SEE THE LIGHT HONOREES:

“See the Light” Honoree Dr. Terence Flotte and the University of Massachusetts College of Medicine, Axovant Gene Therapies, and NTSAD Leadership.

“See the Light” Honoree Jeffrey Castelli and the other Amicus Therapeutics Leadership team members.

For the 20th anniversary celebration, we celebrated the accomplishments of the Foundation since its inception.. The Mathew Forbes Romer Foundation has raised over $2 million to fund projects spanning its mission with only five percent of the money raised going to administrative costs:

• Funded more than 30 nursing scholarships with Florida Atlantic University, Florida International University, and Nova Southeastern University; created nursing school training programs including simulating in-home family care.
• Hosted 20 South Florida Genetic Testing and Education Fairs; screened over 1,500 potential parents with as high as 50 percent detection. Produced the training video of the International Tay-Sachs Quality Control Laboratory.
• Conceived of and grew the National Research Initiative to 61 projects, $3.9 million in grants and many promising therapies with the National Tay-Sachs and Allied Diseases Association (NTSAD). This has led to over $20 million dollars in funding from the National Institutes of Health (NIH) and similar institutions.
• Helped establish the Tay-Sachs Gene Therapy Consortium to accelerate pre-clinical and clinical trials.
• Worked with FAU’s Brain Institute as a follow-on to the State Center for Rare and Genetic Neurological Diseases.
• Sponsored the Medical and Scientific Sessions at the annual NTSAD Family Conference.

* One of the highlights of our work includes that we now have a plan and are hopeful to be in clinical trials with children later this year

THE SWEETNESS AND LAUGHTER GALA

Saturday, March 10, 2018 Featuring the ILLUSIONISTS^TM – Live from Broadway^TM

TESTING AND EDUCATION WINNERS:

NURSING EDUCATION PROGRAM WINNERS:

RESEARCH WINNERS:

Almost $2 million has been raised by the Foundation since its inception in 1998… with only 5% of the money raised going to administrative costs. MFRF has provided leadership and funding for projects spanning its mission.

EDUCATION AND TESTING:
• Funding over 30 nursing scholarships with Nova Southeastern University, Florida Atlantic University and Florida International University.
• Creating nursing school training programs simulating in-home family care and focused on Pediatrics and Genetics.
• Helping re-establish the internationally renowned Lysosomal Storage Disease Clinical, Teaching, and Research Program at the University of Florida
• Hosting the South Florida Genetic Testing and Education Fairs
• Screened over 1,200 potential parents with as high as 50% detection.
• Partnering with JScreen and the Victor Center of Jack Nicklaus Children’s Hospital

RESEARCH:

• Conceived of and grew the National Research Initiative to 50 projects, $3.2 million in grants, and many promising therapies with National Tay-Sachs and Allied Diseases (NTSAD)
• Working with Florida Atlantic University Brain Institute as follow-on to State Center for Rare and Genetic Neurological Diseases
• Helped establish the Tay-Sachs Gene Therapy Consortium with a plan to get to clinical trials following successful results with mice, cats, and sheep and demonstrating critical dosage safety.
• Continues as the charter sponsor for the Medical and Scientific Sessions at the annual NTSAD Family Conference

SWEETNESS AND LAUGHTER GALA

Sunday, March 5, 2017 featuring Capitol Steps at the Kravis Center

SEE THE LIGHT AWARD HONOREES

Randy D. Blakely, PH.D

Joined Florida Atlantic University in 2016 as the founding Executive Director of the newly-formed FAU Brain Institute on the Jupiter campus. Some of his greatest achievements include mentoring over 180 future researchers and thought leaders from high school, to Post Doctorate, to faculty, in the approaches needed to translate basic science discoveries into models and medications to treat genetic brain disorders affecting children.

Rodney Murphey PH.D.

Dr. Rod Murphey is the Chairman of the FAU Department of Biological Sciences and Director of the Jupiter Life Science Initiative (JLSI). Dr. Murphey joined FAU as the original Director of the State Center for Rare and Genetic Neurological Diseases co-created with the Mathew Forbes Romer Foundation. MFRF supported two graduate students on Tay-Sachs and related diseases research in his lab.

Monica Gettleman

Monica Gettleman is a Director and Vice President of Family Services of  National Tay-Sachs & Allied Diseases Association (NTSAD).  With the Mathew Forbes Romer Foundation, she has collaborated on testing, educational and charitable events.

Over $1.9M has been raised by the Foundation since its inception with only 5% of the money raised going to administrative costs. MFRF has provided leadership and funding for projects spanning its mission.

EDUCATION AND TESTING:

• Funding over 25 nursing school scholarships with four South Florida academic institutions including Florida Atlantic University (FAU), Florida International University (FIU) and our newest partner Nova Southeastern University (NSU)

• Creating nursing school training programs of intensive care in the home and community with FIU and FAU and Pediatrics and Genetics training with NSU

• Accelerating the re-establishment of the internationally renowned Lysosomal Storage Disease Clinical, Teaching, and Research Program at the University of Florida

• Hosting the South Florida Genetic Testing and Education Fairs

o Screened over 1,000 potential parents with over 20% detection

o Partnered with the Victor Center and the Jack Nicklaus Children’s Hospital (Formerly Miami Children’s)

o Extended our partnership with JScreen at-home carrier screening plus research to improve testing techniques

RESEARCH:

• Conceived of and grew the National Research Initiative to nearly 50 projects, $3.1million in grants, and many promising therapies with National Tay-Sachs and Allied Diseases (NTSAD)

• Spawned National Institutes of Health (NIH) grants resulting in over $10 Million toward finding a cure

• Grew the Tay-Sachs Gene Therapy Consortium out of the Research Initiative. We have a new plan with the FDA that will lead to an Investigative New Drug (IND) Application and Clinical Trials over the next year

• Continues as the charter sponsor for the Medical and Scientific Sessions at the annual NTSAD Family Conference this year in Florida

$1.8 million has been raised by the Foundation since its inception with only 5% of the money raised going to administrative costs. MFRF has provided leadership and funding for projects spanning the theme of “Celebration of Screening” and its full mission of Education and Research.

Celebration of Screening:

• 2000:  Created training video for 40 labs worldwide with the Tay-Sachs International Quality Control Lab.
• 2003:  Creation of the South Florida Genetic Testing Education Fair with Genzyme Genetics and Baron Delivery on Demand. 100-200 people per year and >20% carrier detection rate.
• 2008:  Partnership with Victor Center expanding the testing panel and frequency of events.
• 2013:  Partnership with JScreen to explore in-home screening kits.
• 2015:  Launching the first combined screening program: Promoting in-person fairs and in-home options to maximize participation and coverage. Created Public Service Announcement that was shown Florida’s National Cinemedia’s Theater Network.

Education and Research:

• MFRF has funded over 22 nursing school scholarships with fourSouth Florida academic institutions to support genetics and pediatrics.
• In 2014, pioneered nursing school training of intensive care in the home and community with Florida International and Florida Atlantic Universities.
• Since 2003, MFRF with National Tay-Sachs and Allied Diseases, conceived of and grew the National Research Initiative to 42 projects, $3 million in grants, and many promising therapies.
• MFRF continues as the charter sponsor for the Medical and Scientific Sessions at the annual NTSAD Family Conference.
• In 2007, helped establish the International Tay-Sachs Gene Therapy Consortium which today is driving to clinical trials. Preparing for FDA review.

SWEETNESS AND LAUGHTER GALA

Sunday, February 22, 2015 featuring The 40th Anniversary Private Screening of Monty Python and the Holy Grail at Cinemark

SEE THE LIGHT AWARD HONOREES

Bernie Marcus – Honorary Chair

Bernie Marcus is chairman of the board of The Marcus Foundation, and co-founder of theThe Home Depot, Inc., the world’s largest home improvement retailer,His company revolutionized the home improvement business with its warehouse concept.

Mr. Marcus is passionate about his vision to provide screening options for everyone, wherever and whenever people want it, at the lowest price. The Marcus Foundation has provided important funding and leadership to JScreen, the Victor Center and related projects and partnerships with which the Mathew Forbes Romer Foundation has collaborated.

Dr. Michael Kaback

Michael Kaback, M.D. is both a pediatrician and medical geneticist whose research has focused primarily on hereditary neurodegenerative disorders in children. He conceived of and pioneered the first regional, national, and international programs for the prevention of Tay-Sachs disease.

Employing unprecedented approaches to community-wide genetic education,

voluntary genetic screening, and genetic counseling, Dr. Kaback directed the first program in the Baltimore-Washington D.C. area in 1971. Since then, 2 million tests have been performed and the incidence of this tragic condition has been reduced by more than 90% in the U.S., Canada, Europe, and Israel.

Lori Baron

Lori Baron, CEO of Baron Delivery on Demand/Messenger Service, helped pioneer the MFRF South Florida Screening and Education Fairs over 10 years ago by handling medical logistics, joining our Advisory Board, and providing critical funding.

Over $1.7 million has been raised by the Foundation since its inception with only 5% of the money raised going to administrative costs. MFRF has provided leadership and funding for projects represented by the theme for the Gala…Caring and Curing.

CARING:

Since 2003, MFRF has funded over 20 nursing school scholarships with four academic institutions in South Florida to promote care needs for our families.
This year we are pioneering nursing school training of intensive care in the home with Florida International and Florida Atlantic Universities.
Since 2003, we have grown the South Florida Genetic Testing programs having screened over 1,000 potential parents with over 20% detection rate.
Our screening partnership with the Victor Center throughout South Florida expanded to include the Miami Children’s Hospital Genetics Department and Brain Institute.
We are beginning to work with the JScreen program promoting and offering screening for many diseases on-line and from your home.
Together with the National Tay-Sachs and Allied Diseases Association (NTSAD) families and caregivers are benefitting from the “Parenting a Child with Life-Limiting Illness” Film.

CURING:

In 2002, MFRF in partnership with NTSAD, founded the Research Initiative.
41 projects and $2.9 million later in grant awards have led to over $10M of National Institutes of Health (NIH) grants.
This year the program received a record number of proposals from around the world.
MFRF continues as the charter sponsor for the Medical and Scientific Sessions at the annual NTSAD Family Conference.
In 2007, we helped establish the International Tay-Sachs Gene Therapy Consortium to drive to clinical trials.
Through gene therapy expertise from the University of Florida, we learned during pre-clinical animal treatments critical insight on proper dosing for safety and efficacy.

SWEETNESS AND LAUGHTER GALA

Sunday, March 2, 2014 featuring Capitol Steps at the Broward Center

SEE THE LIGHT AWARD HONOREES

Dr. Marlaine Smith

When it comes to caring, Dean Marlaine Smith, and her team at the Florida Atlantic University Christine E. Lynn College of Nursing, in Palm Beach County, have maintained a partnership with MFRF since 2003.  Together, we created the prototype for administering nursing scholarships focused on pediatrics and genetics. Conducted one of most well attended MFRF South Florida Testing and Education Fairs,  and are helping us to pioneer a new program moving the classroom into the home with clinical training where the students learn the needs of the patient and families while providing a much needed service.

Dr. Ora Strickland

Dr. Ora Strickland is Dean and Professor of the College of Nursing and Health Sciences at Florida International University in Miami/Dade County. She has led the launch and growth of the relationship with the Mathew Forbes Romer Foundation including the Pediatric Evaluation Lab, nursing scholarship programs, and  helping us to pioneer the new program that epitomizes the 24/7 in-home care needs of our families.

Dr. Thomas Conlon

The leader of the world- renowned Powell Gene Center Therapy Toxicology Team, at the University of Florida College of Medicine.

During our last Gala, we were celebrating the imminent plans for initiating clinical trials with children. Unfortunately, or fortunately, during the final phase of pre-clinical trials with monkeys, we discovered that too much of a good thing can be bad. Our gene therapy treatment was working too well, creating a harmful effect.

Dr. Conlon worked with the Tay-Sachs Gene Therapy Consortium to verify the findings and reproduce the toxic effects. This critical work is now the basis for redesigning a complete dosing experiment, a major step forward for gene therapy. The FDA is depending on this toxicology analysis for granting its future approvals and to support an Investigational New Drug (IND) application.

Highlights of prior years

Research + Education

• Co-Founded the National Research Initiative with the National Tay-Sachs and Allied Diseases Association (NTSAD)
• Helped raise over $2M of funding, for the NTSAD to support over 30 research projects; Funding complemented by over $7M of federal funding from the National Institutes of Health
• MFRF has developed solid partnerships with Genzyme, Tenet/West Boca Medical Center and most recently with the Victor Center for Jewish Genetic Diseases. with the Victor Center, we are not offering a screening panel with 18 tests — three times as may tests than when we began our journey.
• Over 15 nursing scholarships have been granted to students at South Florida colleges and universities. MFRF sponsored the pediatric evaluation training lab at Florida International University’s new nursing school building.
• MFRF partnered with the University of Miami Miller School of Medicine at the Miami Museum of Science to teach young people about DNA and how important it is to our health. The children participated in hands-on activities, including making a double helix out of candy.
• Significant progress is being made with filling the research pipeline for potential treatments and cures.  New South Florida talent has emerged.  For example, researchers with The State Center for Rare and Genetic Neurological Diseases (CRGND), co-created by Florida Atlantic University and MFRF, have developed novel imaging technologies
• Hoop Shoot – Dozens of kids participated in MFRF’s Hoop Shoot free throw competition in May.  The event provided some friendly, fun competition while raising awareness for genetic disease testing and research.
• The State Center for Rare and Genetic Neurological Diseases, created with Florida Atlantic University and MFRF, has helped mobilize 30 scientists around genetic and neurological diseases.  They are studying brain synaptic dysfunction in Tay-Sachs animal models.
• Drive Toward a Cure – Miguel Sena-Esteves, Ph.D. is the principal investigator of the Tay-Sachs Gene Therapy Consortium (TSGTC).  The Consortium consists of nine researchers from world-class institutions, including Massachusetts General Hospital, University of Cambridge in England, Boston College, and Auburn University.  The National insititutes of Health (NIH) has awarded a $3.5 million grant to the TSGTC to prepare for human clinical trials in three to four years.  This award was eagerly awaited by the NTSAD Research Initiative supporters, including MFRF, who raised nearly $600,000 to assemble the TSGTC, demonstrate treated mice can live to full life spans, and help maintain its research agenda while scientists worked to secure federal funding.Kevin Romer has been elected President of the NTSAD.  MFRF is the South Florida affiliate of NTSAD.  Kevin has been a Board of Directors member of the national association since 2000 – first as chair of the Quality Assurance Committee and then as chair of the Research Initiative Committee.
• On February 7th, MFRF celebrated our 10th anniversary with 175 friends and family at “An Evening of Sweetness & Laughter.”  The event raised more than $60,000 toward the mission of education and research for children’s genetic diseases of the brain.  Judy Levis Markhoff was the recipient of this special “See the Light” Award, for her generous support of the Foundation since its inception and her vision and commitment to its mission have been unwavering.  Past “See the Light” honorees were also recognized for their contributions and support.
• The Fifth Annual South Florida Genetic testing and Education Fairs were held at The Christine E. Lynn College of Nursing at Florida Atlantic University and Broward General Medical Center last fall with Genzyme Genetics.
• To mark Biosciences Month in Florida and DNA month nationally, MFRF held its second “Make Your Own DNA Necklace” program during the Sprint Children’s Fair in March.  Hundreds of children collected their own DNA (from their saliva), to make “cool” necklaces with the help of high school biotechnology students.
• MFRF further expanded support for the Neurogenetic Patient Registry, now tracking more than 500 children through The Miami Children’s Hospital (MCH) Brain Institute, and the Pediatric Genetics Residency & Fellowship with MCH in partnership with The University of Miami School of Medicine.
• MFRF strongly supports The Tay-Sachs Gene Therapy Consortium integrating four research institutions worldwide.  The team has announced an unprecedented goal of reaching clinical trials for Tay-Sachs disease in three to four years and has already demonstrated that treated mice can live full life spans (1).
• Through The State Center for Rare and Genetic Neurological Diseases (CRGND), co-created with Florida Atlantic University, researchers have pioneered the genetics behind synaptic malfunction in Tay-Sachs animal models. “As a result of the CRGND, 30 investigatiors have now crystallized around the subject of rare, genetic and neurological diseases,” accouding to FAU’s Biological Sciences Chariperson.
• Platinum Sponsor with Scripps Florida of the Inaugural Florida Biosciences Career & Education Expo including an educational curriculum and DVD, classroom presentations at area high schools and a new internship program.
• The MFRF Neurogenetics Data Registry to track clinical research patients at Miami Children’s Hospital Brain Institute.
• First-ever 5K Pick up the Pace Run/Walk for a Future Free of Genetic Neurological Diseases took place April 25, 2004 to increase awareness of these diseases. Almost 200 people participated in this event raising over $35,000. The event was a collaborative effort between the Mathew Forbes Romer Foundation and the National Tay-Sachs & Allied Diseases Association.
• The first NTSAD National Conference held in South Florida, featuring the Research and Medical Session, co-hosted and sponsored by the Mathew Forbes Romer Foundation. With Florida Atlantic University (FAU), with connections to Scripps Research Institute established the State Center for Rare and Genetic Neurological Diseases (CRGND) fostering collaboration dedicated to studying children’s genetic diseases,
• Schools of Nursing at FAU, Florida International University, Nova Southeastern University and Miami-Dade Community College, for pediatric nursing scholarships funded with a focus on caring for children’s genetic disorders.
• The Research MFRF funded in 2001 at the National Institutes of Health was published in the Journal of Human Molecular Genetics.
• Speaker at South Florida launch of the US legislation titled Rare Diseases Act of 2002 with Representative Mark Foley and the National Organization of Rare Disorders (ORD)
• Becomes a member of the Genetic Disease Foundation headquartered in New York.
• International Genetic Testing Training Videotape and user’s manual produced with Dr. Michael Kaback (who pioneered Tay-Sachs and Quality Control) and distributed to up to 40 labs.
• Boca Raton Community Hospital Education Seminar conducted in February focusing on education and importance of genetic testing for these rare diseases.

SWEETNESS AND LAUGHTER GALA 10th Anniversary Gala

10th Anniversary Celebration. Saturday February 2009, in Boca Raton, Florida.

SEE THE LIGHT AWARD HONOREES

Judy Levis Markhoff:

Featuring the 10th Anniversary Honoree Judy Levis Markhoff: Adolph and Rose Levis
Foundation major benefactor and Advisory Board Member of the Mathew Forbes Romer Foundation.

Please also view the video for the honorees dating back to 2000.