Recently, Melissa Hogan sat down with the Rare in Common podcast to discuss navigating her son’s rare diagnosis. Listen
The Basics
Name: Melissa Hogan
Title: Founder and President
Organization: Project Alive
Disease focus: Hunter Syndrome, or mucopolysaccharidosis II (MPS II), is a rare genetic disease that is progressively debilitating, life-limiting and without a cure. It occurs almost exclusively in males and currently affects approximately 500 boys in the United States, and less than 2,000 worldwide. Individuals with Hunter Syndrome have a defect in a gene that normally causes the body to make an enzyme that is responsible for the breakdown of cellular waste. Without that necessary enzyme, the accumulation of these waste fragments in cells leads to progressive damage throughout the entire body. Most boys with Hunter Syndrome continue to develop physically and cognitively until between the ages 2 and 5, and then begin regressing. These boys gradually lose the ability to talk, walk, and eat. Most do not live to see their teen years. Approximately 20 percent of those affected by Hunter Syndrome do not suffer cognitive impairments or regression and may have normal intelligence. Currently, the only FDA-approved treatment on the market is an enzyme replacement therapy, which can stabilize many of the physical symptoms of Hunter Syndrome. However, as it cannot cross the blood-brain barrier, it is unable to prevent cognitive regression, meaning the child will continue to decline to an infantile state.
Headquarters: Nashville, Tennessee
How did you become involved in rare disease: In 2009, my son Case was diagnosed with Hunter Syndrome. He was only 2 at the time. I started writing on Caring Bridge, then I started writing a blog. At the time, I was sharing everything we were learning along the way. It began to be more and more read, and ended up being read regularly in more than 100 countries. We looked at the science that was happening and had been monitoring all of the research programs in Hunter Syndrome, especially in gene therapy. When we saw that, we decided it was something that we wanted to pursue and formed Project Alive.
Previous career: Healthcare corporate lawyer and strategy consultant to international law firms.
Education: B.A. in communication and political science from Georgetown College, and a J.D. from the University of Pittsburgh School of Law.
The Organization
Organization’s mandate: Our purpose is to cure Hunter syndrome through funding and designing research, advocating for efficient clinical trial design, and approval of treatments for Hunter syndrome. We are all about cures.
Organization’s strategy: We started by monitoring all of the research in Hunter Syndrome and still do, whether its academic, industry, small biotech. We have a program called RAMP, which is our Research Assistance and Monitoring Program. We offer our assistance in collaborating with them to help design their research programs, their clinical trials protocols, and then we also take supportive or leading roles in approvals or reimbursement objectives in order to get access to those therapies for our community.
Funding strategy: We have a multi-pronged funding strategy. We encourage and hold events across the country. We generate public campaigns, and actively seek media to generate unrelated donors. We’re known for our creative campaigns. We launched our first video campaign three years ago with a lot of celebrity support with the theme that kids want to be lots of things when they grow up, but our kids just want to be alive. We seek small unrestricted grants, and larger grants with an almost larger philanthropy model.
What’s changing at your organization in the next year: The biggest change, the most exciting change, is that we should be enrolling the gene therapy clinical trial that we’ve been funding the preclinical research for over the last five years. We signed a $1.4 million contract to produce the vector. Nationwide Children’s Hospital, with our support, has already received FDA approval of its IND to begin the trial.
Management Style
Management philosophy: I want integrity, hustle, and smarts in that order. I also run people through my filter, which is no shame, no fear, no pride. Will you do whatever it takes? Will you make the hard ask? Or are you just in it for the credit. If you are just in it for the credit, that’s not how we operate.
Guiding principles for running an effective organization: Always keep your eye on your top three key priorities. Everything from your daily tasks to your monthly goals to the achievements you are tracking should be able to key back to those priorities.
Best way to keep your organization relevant: To produce new and innovative content on a regular basis. By content, I mean things that are both helpful for public awareness, but also for your community. Videos, the blog, things in that way, and to engage our community in the generation of our content and what they are seeking from the organization.
Why people like working for you: I’m the first to show up and I’m the last to leave. I don’t take a salary, so people know I’m in it for pure motives. I assume people are going to be adults and I assume people are going to go big or go home. I give people the freedom to do that.
Mentor: Judge Alice Batchelder. She’s a judge on the U.S. Sixth Circuit Court of Appeals. I clerked for her after law school. She is one of the smartest, hardest working women I ever met.
On the Job
What inspires you: When I get an email from a newly-diagnosed family and they just want to dive into the work we are doing because they see momentum, the impact we are having, and they are thankful to have people who understand.
What makes you hopeful: The number of companies and research programs in Hunter syndrome right now is surprisingly broad. With that in mind, I have a living, breathing example of what successfully treated Hunter Syndrome can look like in my home. My son’s been in a clinical trial for over seven years. I get to watch every day what it’s like to have a child who might live despite Hunter syndrome.
Best organization decision: Hiring an administrative assistant and a publicist. There have been many times when I was sleeping just two to three hours a night. That wasn’t productive for me or my family. They do things I didn’t know how to do in half the time. I can use my time for our mission and tasks that I’m better used for.
Hardest lesson learned: You can’t please everyone about everything all of the time. Sometimes you just have to put your head down, follow your mission, and ignore all of the noise.
Toughest organization decision: It’s probably more smaller decisions, but it’s the perspective that we have to spend some money on needed or legally required infrastructure when I want every penny on research. It’s a hard thing to swallow, but necessary.
Biggest missed opportunity: I’d say the biggest missed opportunity was that we didn’t hire a publicist back in 2015 when we launched our first campaign. We had a great story to tell, and a compelling business case. We were so focused then on not spending money on anything but research that I think we missed a chance to multiply our revenue early on.
Like best about the job: The idea that this will have a lasting impact on our community and for newly diagnosed families. I remember the devastation of that first year like it was yesterday. To see that when new people are diagnosed that there is hope, that’s a good feeling.
Like least about the job: The flip side of that. That everything we are doing will not be in time to save a lot of the kids I love. That’s a hard pill to swallow.
Pet peeve: When my flagged emails rise above 200. I start to have heart palpitations.
First choice for a new career: A writer. I’m writing two books right now. I’m always writing music. I would love to spend a lot more time on that.
Personal Taste
Most influential book: A non-rare disease related book like Just Mercy: A Story of Justice and Redemption by Bryan Stevenson. We tend to think our world in rare disease is hard and full of injustice, but there’s a lot of injustice out there.
Favorite movie: The Breakfast Club, no question. I’m a child of the eighties.
Favorite music: Chris Stapleton or old school rap – like Run DMC.
Favorite food: Graeter’s Coconut Chip Ice Cream.
Guilty pleasure: Stealing weekends away with my husband.
Favorite way to spend free time: Writing while sitting on my back porch.
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