A billionaire couple’s quest to cure the genetic disorder that claimed their son’s life | Inside Philanthropy

The Gilbert Family Foundation, established in 2015 by Rocket Companies founder Dan Gilbert and his wife Jennifer, was formed primarily to advance research into the genetic condition neurofibromatosis type one (NF1), a rare but serious genetic disorder affecting about 1 in 3,000 children. Like many major donors to medical research, the Gilberts are motivated by personal experience: their son Nick was born with the condition, which causes tumors to form in nerve tissue, causing a range of serious symptoms. Sadly, Nick passed away last year aged 26.

Since its founding, the Gilbert Family Foundation has been one of the leading philanthropic forces in NF1 research – and it doesn’t appear to be slowing down. The foundation recently announced its fourth major research funding effort, pledging $21 million to launch the NF1 Next Generation Models Initiative. Its three-year grants will go to 18 NF research teams at universities and institutions in the US and Europe.

Although neurofibromatosis occurs in about the same number of births as cystic fibrosis, another genetic disorder, it is much less well known. But its effects can be uniquely devastating and especially difficult to treat.

Treatment of NF is particularly challenging because the condition can result from any of 26 chromosomal mutations; as a result, the symptoms and manifestations of the condition vary widely, including tumors in different locations, deafness, blindness, and other effects. (NF1, usually diagnosed in childhood, is the most common of the three types of neurofibromatosis. Types 2 and 3, diagnosed later, are much rarer.) Treatment options, including medications and surgery, can help some patients, but there is still no cure for the progressive and potentially fatal condition.

The recently launched Next Generation NF1 Modeling Initiative follows three other research initiatives developed by the Gilbert Family Foundation over the past decade: the Vision Restoration Initiative, the Brain Tumor Initiative and the Gene Therapy Initiative. But this latest focus is particularly exciting, as it is using a new approach to drug testing with the potential to significantly improve and accelerate the search for treatments.

A new research approach fuels hope

Several research studies that the foundation’s new round of grants are supporting will test promising methods called organoid and assemblyoid technologies, in which researchers use a patient’s stem cells to create cultures of tissues and organs that look and respond like tissues and organs. body current. These methods can give researchers much more information than they can get by testing drugs on single cells. Researchers can use these lab-made tissues or organ cultures to model diseases and test new drug treatments, potentially speeding up the drug development process, said Laura Grannemann, executive director of the Gilbert Family Foundation.

Time is always of the essence in medical research. Traditionally, researchers and drug developers conduct early tests of potential new drug treatments in animals; if the tests suggest adequate levels of effectiveness and safety, then the research can progress to humans. But the many rules surrounding drug development involving humans have meant that the process can be painfully, if at all, slow.

“The promise of this new model is to accelerate that timeline as much as possible,” Grannemann said. “Researchers can use real tissue from a patient to test in healthy and unhealthy tissue to see the effect of a drug on tumors and healthy tissue.” Compared to traditional methods, such techniques allow researchers to test much more easily and quickly whether a drug is likely to be effective.

Researchers focusing on cancer and other diseases are also using organoid and assemblyoid technologies. This means that the NF-specific research that the Gilbert Family Foundation is supporting can be cross-pollinated to advance progress in other areas of study.

Through his parents’ philanthropy, the Gilberts’ late son, Nick, will remain connected to the search for cures for NF. In 2023, the Gilbert Family Foundation announced its lead funding role in a partnership with Henry Ford Health and Shirley Ryan AbilityLab to build a new medical and rehabilitation facility in Detroit. The foundation is contributing $375 million of the facility’s estimated $439 million cost. The center will house the first brick and mortar facility dedicated solely to NF1 research, the Nick Gilbert Neurofibromatosis Research Institute. Construction of this facility is scheduled to begin soon and is slated to open in 2027.

Scientists at the institute will also primarily use the organoid technology that Gilbert’s foundation is seeking to advance with its latest research initiative.

A massive footprint in Detroit

Dan Gilbert amassed a fortune as the founder of Rocket Mortgage (formerly Quicken Loans), one of the largest retail lenders in the U.S. Gilbert has also owned the NBA team Cleveland Cavaliers since 2005. He frequently appears on lists of America’s Most rich: Forbes estimates his current net worth at over $27 billion.

Despite its significant giving around NF1, the Gilbert Family Foundation is not solely dedicated to research into the condition. In fact, most of the foundation’s funding has been local and regional giving near Detroit, where the rocket companies are based. That includes a 10-year, $500 million commitment from the Gilbert Family Foundation and the Rocket Community Fund aimed at revitalizing Detroit neighborhoods — funding that included $15 million to eliminate property tax debt owed on 20,000 homeowners with low income.

That’s just one of Gilbert’s long list of philanthropic, business and investment commitments in his hometown that make the billionaire one of its most powerful and influential figures (if not THE most influential). They include support for the QLine, the downtown streetcar system, and most recently, support for BIPOC’s local artist/entrepreneurs, as my colleague Mike Scutari reported this spring. For years, Dan Gilbert’s prominence in Detroit has fueled debate over whether the megadonor has too much influence in the city where Politico once called him a “shadow mayor.”

But all the while, the Gilberts have also continued their long-standing efforts to advance neurofibromatosis research. In addition to the commitments I’ve already mentioned, the Gilberts have long supported NF research at other organizations, including the Children’s Tumor Foundation, Children’s National Health System, and the University of Alabama Birmingham. In 2017, they founded NF Forward, a sister non-profit organization that also funds advanced NF research. Through their family foundation and NF Forward, the Gilberts have given more than $125 million to NF research so far, and they don’t seem to have any intention of slowing down.