A recent interview with NDF’s new Executive Director Geoffrey Gee by Global Genes.
Disease focus: GNE myopathy is a rare, inherited disease that causes progressive muscle weakness. The disease is caused by defects of the GNE gene. In this disorder the defect of the GNE enzyme disrupts the functioning of the sialic acid metabolic pathway and interferes with normal muscle function. GNE myopathy typically affects young adults. One of the first signs of the disease includes inability to lift the front of the foot (foot drop). This may cause toes to drag while walking. As the disease progresses, it usually leads to physical disability. GNE myopathy does not affect the muscles of the face or swallowing ability. It does not typically affect the heart or breathing muscles. GNE myopathy is a rare disease estimated to affect around 6 in 1,000,000 people. It is found in populations worldwide but is more common in certain populations. Like many other rare diseases, GNE myopathy is under diagnosed and many people with the disease remain undiagnosed.
Headquarters: Beverly Hills, California
How did you become involved in rare disease: It was through Lalé Welsh, who was then the CEO of the organization, and was stepping down. She was looking for somebody to replace her and found me after having spent seven years building the organization up. I don’t know if you know much about the disease, but it was said to primarily be a Persian Jewish disease, but it is actually worldwide in numerous communities. We have representation in nearly 50 countries around the world where there are both patients, doctors, and scientists who are involved as well as NDF patient advocates. It’s an international, global disease and very few of the people involved are Persian or Jewish.
Previous career: Practiced law in England and Australia, business and nonprofit executive
Education: Law degree Law Society’s College of Law
Organization’s mission: The mission is simple. The initial stage is to get to the clinical trial stage to find suitable treatment for those with the disease. The ultimate goal is a cure. Cure means different things to different people. But to me cure means the ability of people with the disease to live a normal life despite their physical challenges, and for young people, to get diagnosed through testing. As you probably know, there’s the Jewish genetic disease test that young couples can take if they’re going to get married and there’s often a rabbi or a Jewish doctor involved who will usually encourage them to go and get a test.
Organization’s strategy: The strategy is to promote the global aspects, the international aspects of the disease, to encourage more people to get involved both from a fundraising perspective and from a patient assistant perspective. That is the current short-term mission. The long-term goal is gene therapy and a cure.
Funding strategy: The general strategy is outreach to donors who might have family members with the disease. We do have a Los Angeles gala once a year. For the last two years, COVID affected us and there was no gala. Because I have some contacts in Beverly Hills and in the motor industry, I asked Beverly Hills Aston Martin if they would let us have a car for the event. They said “There’s an SUV called the DBX. We will allow you to auction it off to the highest bidder who will then get the reward of an Aston Martin experience for the weekend.” I would rate that as priceless. We are also having a casino night that we are calling Casino Royal.
What’s changing at your organization in the next year: My focus is the continued effort on research. We have an extraordinary group on the scientific advisory committee, experts in the field who are dedicated and devoted to finding a cure. We are continuing the effort with those 20 plus people. We have preclinical studies going on with mouse models and are using every contact we have to enlarge the global footprint of the organization.
To read the full interview on Global Genes website, click here.