Rare Leader: Melissa Chaikof, Board Chair and Acting Executive Director, Usher 1F Collaborative

The Basics
Name: Melissa Chaikof

Title: Board chair and acting executive director

Organization: Usher 1F Collaborative

Social Media Links:

Disease focus: Usher Syndrome is a genetic condition characterized by deafness at birth or progressive hearing loss and progressive vision loss. Symptoms include bilateral profound deafness from birth, vestibular (balance) problems from birth that can include poor head control as an infant, arching of back as an infant, late sitting, and late walking at age 18 months or later and then with poor balance for the first few years. Decreasing night vision begins in childhood to adolescence. Currently, there is no cure for Usher Syndrome. While cochlear implants have greatly ameliorated the most major impacts of deafness for those who receive them as babies, there is no cure for blindness. Early identification and educational programs provide support to those affected. Targeted research is needed to enable a cure.

Headquarters: Newton, Massachusetts

How did you become involved in rare disease: My older daughter, Rachel, was born in 1987. I knew very quickly that something was wrong with her hearing and was dismissed by the pediatrician as an overly anxious new mother who didn’t understand how newborns react to sound. But at two months old she was diagnosed. Our son was born with no issues. And then our third child, Jessica, was also born deaf. We knew at that point it was genetic. My initial foray into advocacy, before I knew we were dealing with a rare disease, was on the hearing loss side. Rachel was one of the first 200 children in the United States to get a cochlear implant as part of the clinical trials. We saw how well it worked with her and had Jessica implanted much younger. She was the youngest child in the United States at the time in 1996. That got me involved in hearing loss advocacy for families of children born deaf. That was my involvement until in 2006, when during a routine annual eye exam, the doctor saw the retinitis pigmentosa in Rachel’s eyes when she was 19. At that point, we did a reorganization of our efforts and got involved in the Usher syndrome community. We began to realize that even though there was lots of promising research for other types of Usher syndrome, because the research was gene-based, there was nothing for Usher 1F. I went to my husband and said, “Look, if we don’t do it, nobody’s going to do it.” Combined, we have the right backgrounds. That’s how it got started.

Previous career: Systems analyst and donor research analyst for a nonprofit fundraising firm.

Education: B.A. applied math University of Pennsylvania, M.S. in mathematical sciences The Johns Hopkins University

The Organization
Organization’s mission: To fund medical research to find a cure for the vision loss associated with Usher 1F.

Organization’s strategy: We’re very fortunate in that we have a great combination of the needed skills and backgrounds. My husband is not only the father of two daughters with Usher1F, he’s also an M.D. Ph.D. He’s chief of surgery at Beth Israel Deaconess Medical Center in Boston. He also has a busy research lab. When we decided to start the foundation, he pulled in two colleagues for the board. One he had done his Ph.D. with, Frank Gentile, but Frank had gone the biopharma venture capital route. He provided that expertise. And then our other original board member, Lijun Sun, is in the field of drug development. That gave us the knowledge that we needed to know what science to pursue and to form the connections. Then it was getting as many families involved as we could to do the day-to-day legwork of fundraising. We’ve been able to harness both of these needed areas for success.

Funding strategy: We have some very involved families. We keep expanding our network. We were in the first cohort of the Chan Zuckerberg Initiative Rare As One Project. That’s helped us tremendously. We’ve been able to hire a development associate and work with a professional fundraising consultant who is phenomenal. We’ve got a great team. I was fortunately able to leave my job and to focus on this full time. We’re in such a unique position with the scientists we have both on our board and with our researchers that we’ve been able to parlay our success and say to our donors, “Look how much we’ve accomplished in such a short amount of time. You’ve had such a major impact.” And they keep helping.

What’s changing at your organization in the next year: We just brought on a wonderful new development associate January 10^th. Bringing her on board is a huge help. We just received a third year of CZI funding. Because we have a bigger team now, we are planning three fundraisers. In terms of the research, we’ve been realizing some successes. We had our first published paper from our University of Maryland researcher about a potential treatment that he’s working to get a clinical trial for. One of our other researchers has developed a very promising gene therapy. Another great thing that was just launched in the past couple months is our natural history study. We partnered with Foundation Fighting Blindness for RUSH1F. We’ve got seven of 10 centers up and running. The next three should come on board imminently. COVID has slowed that down a little bit, but we have several patients already enrolled. That’s exciting because we knew that this was a critical piece before we could have a clinical trial.

Management Style
Management philosophy: I had never been a manager before, but I had been an employee. I knew that I was the most motivated when I was trusted and respected. That is a part of it—treat people well. Treat them as I want to be treated. Also nurture people’s strengths to get everybody involved using their skills in any way that they can contribute.

Guiding principles for running an effective organization: I would say that we work strategically. We have our goal outlined. In fact, we have our Ten to Cure initiative, which spells out exactly what we want to accomplish in the next 10 years. We keep our eyes on the prize. One of the core principles that we have used from the start is translational research. We don’t fund science just for the sake of science. We don’t have time for that. Everybody, my daughters, everybody, all of our kids, all of our adults, everybody’s in a race against time for a cure. We fund research with the goal of taking it from the bench to the clinic.

Best way to keep your organization relevant: First of all, sharing our research progress, keeping our families, keeping our donors in the loop, showing them the impact that they’ve had, and making sure that we’re funding research that’s moving forward and has great potential. Stay out there in front of our supporters and get the word out to new people so that we can keep doing what we’re doing.

Why people like working with you: Actions speak louder than words and I’m not telling other people what to do. I’m in there, every day, more than 40 hours a week. This is a labor of love for me. I don’t take a salary. They know that I’m giving it my all and that I’m not asking something of others that I’m not doing myself.

Mentor: I would have to say my first mentor was my mom because she raised me to the nth degree to not just accept, but question. That started on day one when we got the deafness diagnosis and the audiologist said “She’s never going to speak. This is the school you should send her to.” I didn’t like that. In the same way, when we got the retinitis pigmentosa diagnosis, we went to see a specialist and he said, “There won’t be a cure in her lifetime.” How can you say that about someone who’s 19? Shortly after we got the deafness diagnosis though—my father was an elementary school principal at a private school in Baltimore, and he happened to have a child in his school who had been born profoundly deaf in one ear with a moderate loss in her other ear. She was enrolled to start kindergarten. The summer before starting kindergarten she lost all of her residual hearing and her mom called my dad and said, “What should we do?” He said we’ll try it, and we’ll see how it goes and assess. As long as she’s feeling successful, we’ll keep going. P.S., the girl ended up going all the way through 12th grade in the school and graduating. When my older daughter was born, this girl was 11 years old, and my father connected me to her mother, Esther, and she became my mentor. Question the experts. They don’t necessarily know everything. And as the mom, you know what’s best for your family and for your child. Esther got me started on being assertive for my child and speaking up and not taking “no” for an answer. She guided me in the early years, and that got me set up on the path that we’ve been on of saying, “We can’t sit back and assume that others are going to do what’s needed.”

On the Job
What inspires you: The people I’ve come to know. It started out for my two daughters, but I’ve come to know so many adults with Usher 1F, families, children. And then also success. Every time we realize success, it’s very exciting and reinvigorating,

What makes you hopeful: The research progress that we’ve made from nothing to where we are now with at least one gene therapy that is very likely three to four years from being ready for testing in humans.

Best organization decision: To start the organization for sure. That would be the best one and the researchers we have chosen to support.

Hardest lesson learned: Don’t hesitate. I wish we had started the organization sooner. The hardest lesson learned is to get up and do it as soon as you can.

Toughest organization decision: Evaluating the research grants every year. We don’t have an unlimited pool of money and we have all these wonderful researchers. Deciding how do we allocate money? We don’t want to put small pools of funds into too many places so that it’s not enough for anyone to accomplish anything. We don’t want to leave any opportunities out, so how do we most effectively allocate the funds?

Biggest missed opportunity: One of the things that we really want to do is a high throughput drug screen. We have the zebrafish model for it. I had mentioned that to our board member who works in biopharma venture capital. He connected us with a company where that’s all they do. They test drugs in zebrafish.  We had the assay done and then they hit us with, “Okay, now that we know what’s involved, it’s going to be $1.3 million a year for two years. At that point, we also needed the natural history study, and we couldn’t afford both. We decided the natural history study was more critical. That drug screen is something we still really want to pursue. We had a company that was willing to donate to us a library of about 2,000 FDA-approved drugs for testing. That would be the quickest route to a treatment if we found a drug that was effective, that already had FDA approval. That’s something that we still want to accomplish.

Like best about the job: It’s meaningful. It’s fulfilling. I feel like I’m making a difference. When I worked in applied math, I worked for a company that did engineering consulting work for the U.S. Air Force. I spent two years on my last project, and I published this long paper about my work in-house. Then I went on maternity leave with my son. While I was out on maternity leave, my officemate called and said that they’re pulling the paper. They think there may be some classified information in it. It got pulled and nothing ever happened. It was two years of work for what? My husband is a surgeon and a researcher. When I first met him, I asked him, “Why did you go into medicine?” His answer was a very sincere: “To make a contribution to mankind.” I remember saying to him, “You get to go to work every day and feel like you’re making a difference.” I can’t think of anything else that I could be doing at this point that would be so meaningful for me, that would make me feel that I was doing invaluable work that makes a difference in other people’s lives, not just for my own daughters.

Like least about the job: The bureaucratic stuff and the accounting. We just had this stupid situation where we switched our payroll company and had to move our workers’ comp policy to the new company and make some other changes. Because they had to cancel and then reissue our workers’ comp policy a couple of times, they required an audit of the policy that was in existence from December 28, 2021 to January 1, 2022. Three days and gathering all the paperwork for that and submitting it took me at least half a day. That to me is such a waste of time. That’s the part I don’t like. Somebody has to do it and I do it, but I have all this stuff I’d rather be doing that’s really going to make a difference.

Pet peeve: Those who sit on the sidelines and don’t take a chance and jump in to make a difference.

First choice for a new career: I always tell my girls that I’m not going to retire until they’re cured. My next goal is retirement, but then again, I’m not the kind of person to sit back and do nothing. I love the nonprofit world. I worked in it for 14 years before doing this. In terms of a career, something in the nonprofit world is where I’ve found that I belong.

Personal Taste
Most influential book: Orphan: The Quest to Save Children with Rare Genetic Disorders by Philip Reilly

Favorite movie: Mary Poppins

Favorite music: I came of age in the ‘70s James Taylor, Carol King, Carly Simon

Favorite food: Salads and anything rich and chocolaty, like brownies

Guilty pleasure: Reading escapist novels, chick lit, or watching the Hallmark channel.

Favorite way to spend free time: I love to spend time with my family, reading, and working out.