Name: Lale’ Welsh
Organization: Neuromuscular Disease Foundation
Disease focus: GNE myopathy, or HIBM (Hereditary Inclusion Body Myopathy), is a rare inherited disease that causes severe muscle weakness in the legs and arms, usually leading to physical disability within five to twenty years of onset. Although some patients are able to live using just a cane for many years, in most patients the quadriceps muscles are spared. GNE Myopathy does not affect the vital organs, such as the heart, the lungs, and the ability to swallow. The disease is found in many populations worldwide: Europeans, South Americans, Africans, Middle Easterners; Arabs, Jews, Persians, and Asians of Japanese, Korean and Indian descent among others.
Headquarters: Beverly Hills, California
How did you become involved in rare disease: I started out in film and spent most of my career in Silicon Valley. I retired in 2006 and started my own foundation for the arts. In that process I learned so much about the challenges of running a nonprofit and how much more difficult it is to run a successful nonprofit than a for-profit business. Because of that, I ended up creating a niche for myself. People ask me to help them run their nonprofits. I parachute in, take a diagnostic look at their problems, set up a business plan, do fundraising, and get them on their way. This organization contacted me to come in. The fit was that I happened to speak Persian. The founding family at that time believed this was a Jewish Persian disease and they thought that was important. Since I joined we’ve learned it’s prevalent in many different demographics. This is my fourth year with this organization.
Previous career: Casting for film industry, and then marketing for Silicon Valley technology companies.
Education: BA in Communication (radio, television, and film), and a Master’s degree in Media from California State University, Fullerton
Organization’s mandate: To find cures and treatments for GNE myopathy.
Organization’s strategy: Our strategy is to expose the rareness of the disease by asking patients to self-advocate. Our second layer is to get scientists who previously worked in siloes to collaborate and share data. And the third layer of strategy is to partner with biopharma companies interested in the data we’ve collected and the funding that we can put towards taking something like gene therapy all the way to market.
Funding strategy: Unfortunately, with rare disease the only funding you can really get is from major donors related to the patient. And we’re working with foundations and biopharma for grants, but it’s slim pickings.
What’s changing at your organization in the next year: Gene therapy. We’ve made some incredible strides towards being able to have enough data to know confidently that we can move forward with gene therapy. We are moving forward with clinical trials in the next year.
Management philosophy: Until last year it was just me. Now we’ve hired a director of operations. My management philosophy is: when you are this short-staffed, and don’t have the benefit of a lot of project managers, bring in patients to come in and work on their own programs related to advocacy and focus on people who are more detail-oriented, to be self-driven, without management.
Guiding principles for running an effective organization: Apply the same business model that worked so well in Silicon Valley and that doesn’t work as well in the big, giant, 800-pound gorilla. Move like a gazelle and move quickly when you don’t have the kind of clout that you need. Then be able to maximize your impact by making smart, carefully thought-out decisions so you can act quickly when opportunity arises.
Best way to keep your organization relevant: To constantly make sure you are the squeaky wheel, constantly get out in front of anyone who will listen, and get patients and family members to advocate for themselves by reaching out to doctors and the public. Let them know that while this disease is rare, rare disease is very common. If we solve problems for this rare disease, it could end up being a solution for many rare diseases globally.
Why people like working for you: Keeping a sense of humor and keeping things in perspective is incredibly important. When you work in the nonprofit space, you tend to work with people who are underserved and therefore unhappy. Remembering to keep your sense of humor allows you to make healthier decisions that are not tied to wishful thinking, or magical thinking, but rather a solid strategy.
Mentor: In the Silicon Valley universe, it’s John Hegel, III, a partner at Deloitt. He’s a business and technology consultant. I learned a lot from him. In the nonprofit world it is Dan Pallotta, who started the San Francisco to LA Bike Ride for AIDS. He reminds people that the way we think about nonprofits is 100% wrong. Most people look at your overhead and things like that to determine if they should give. I’d argue you can have a 5 percent overhead and have a bake sale and raise $300, or you can have a 40 percent overhead and raise millions. When people think about what your end customer would prefer, they don’t care about the overhead or what it took to get you there. Amazon took seven years to make a dollar in profits. The nonprofit world is scrutinized in a way that keeps it from being successful because of deeply held beliefs that are not correct—shibboleths that no longer serve us well.
On the Job
What inspires you: The patients. Their resolve, their healthy attitude, their refusal to give up, and their willingness to be hopeful and to collaborate. It’s inspirational and humbling.
What makes you hopeful: Technology makes me hopeful. The rate at which technology is growing is exponential. A study that would have cost us a few million dollars ten years ago can now be done for a couple of hundred thousand dollars. It is going to add to the collective data we have, and when shared, which we are committed to doing, will exponentially increase the likelihood of a cure.
Best organization decision: Moving forward with gene therapy.
Hardest lesson learned: You can’t rely on a wealthy demographic to support you. At the end of the day, the only people who will support you are going to be stakeholders—the patients, their family members, and the scientist willing to work in that field.
Toughest organization decision: To move forward with gene therapy, because for the next five years, it will only help people in the trial itself. We have a saying that “time is muscle.” Some of the patients who need immediate help will not be able to get it this way until it goes to market.
Biggest missed opportunity: Not hiring professionals sooner—trying to make it an all-volunteer organization. Studies show that’s not prudent for big impact.
Like best about the job: I feel like I’m moving the needle in an otherwise unlikely scenario.
Like least about the job: Taking phone calls from frightened, rightfully angry patients and their family members who don’t feel enough is happening to solve their immediate problems.
Pet peeve: People who get involved in a cause that they don’t really care about or understand. They wear it like a gossamer dress to a party.
First choice for a new career: I’d like to start a biotech company.
Most influential book: The Power of Pull: How Small Moves, Smartly Made, Can Set Big Things in Motion, by John Hagel
Favorite movie: Sophie’s Choice
Favorite music: World music
Favorite food: Fruit
Guilty pleasure: Poker
Favorite way to spend free time: I have played 85,000 hands of poker on Zynga—not for money. I’m very proud of my 32 percent win rate.
March 8, 2018
Photo: Lale’ Welsh