Half a century ago, a mother whose baby daughter was diagnosed with a life-threatening genetic disorder decided to fight it. Doris Tulcin is that mother, and Cystic Fibrosis is the disease against which she went to war. First identified in 1938, Cystic Fibrosis affects roughly 30,000 children and adults in the United States and is therefore, like Rett, classified as a rare disease. In 2010 the Cystic Fibrosis Foundation generated donations in excess of $300 million, $120 million of which was raised from the general public. Now, Vertex Pharmaceutical’s VX-770, the first drug that treats not just symptoms but the underlying genetic cause of Cystic Fibrosis, has been fast-tracked through FDA approval.
Doris Tulcin has been a valued advisor to RSRT since our inception three years ago. The following in an excerpt from a recent
conversation between Mrs. Tulcin and RSRT Executive Director, Monica Coenraads.
MC: Congratulations! You must be thrilled with the early FDA approval---it’s been a long road and I know CF families are rejoicing today!
DT: It’s mind-blowing! VX-770 is for a small group of patients that have a rare mutation but will literally change the disease and these kids completely. And that’s what we are working on now for 90% of the patients with the major mutation. Things are moving really well.
MC: CFF provided substantial scientific, financial and clinical support for the development of VX-770, including the investment of $75 million. When I think about the vast amount of money CFF raises, with a patient population similar to Rett, I can’t help but ask myself, How have you done it? Are there specific decisions that you think have been pivotal?
DT: Well, you must remember that we have a long history; we’ve been in business since 1955. So it took over fifty years to get to where we are today. Going back in time, it is so hard to pinpoint any one thing. But we really focused on research, as you are doing.
MC: Of course, the scientific tools and knowledge that were available fifty years ago are so very different from research today. Everything has accelerated. Yet I know that getting to this point with Cystic Fibrosis has been a process with many components.
DT: From 1980 on is where we really took off with developing research programs with major universities, and by the 1990’s we became increasingly business-oriented and
focused on working with the biomedical community and pharmaceutical companies for drug development.
MC: As with Rett syndrome, the Cystic Fibrosis population is small. You know RSRT has a PSA that’s running in Times Square, the same screen that was used for the CFF. So 1.5 million people will see it every day for three months. How important do you feel it is to raise general public awareness, in terms of the ability to raise research funds?
DT: Name recognition is very important. The general public doesn’t have to understand the disease but they certainly have to recognize the name. You know, people will say “I’ve heard of Cystic Fibrosis”--- well, what do you think it is? They can’t describe it. They haven’t the vaguest idea, but they’ve heard of it.
MC: And you think that correlates to the amount of money you raise?
DT: I do. Our chapters raise roughly $100 million a year. But keep in mind that it took decades to get where we are today.
MC: For RSRT, at the three-year mark, most of our revenue comes directly from affected families and their networks. Do you think the same applies to you, or do you have people just spontaneously donating to CFF without any personal connection?
DT: I think now we do, yes. But what we started with and built on was indeed that personal commitment and involvement to develop networks of family and friends. The activism of those who are personally affected is crucial.
MC: How would you compare the current philanthropic landscape to what you’ve seen in past decades? These are difficult economic times for many.
DT: Yes, we are still in a bad economy now and that reflects on contributions, there’s no doubt. It’s tough, and there’s always competition and distraction.
MC: On a personal note, how is your daughter doing?
DT: Oh, thank God, she’s doing well. She’s a grandmother! My grandchildren have introduced me to Facebook, so we communicate in that way, because they’re all grown up and don’t have time for other things. But we do that too with the foundation, we’re on everything you can think of--- Facebook, Twitter, YouTube. Everything now is sent electronically. The website is being updated all the time, you can get every bit of information at any time off that website.
MC: Yes, the fact that electronic media is now ubiquitous worldwide has really changed the flow of information in important ways for families dealing with something like a rare disease. The ability to be in instant communication with others who really understand relieves some of the isolating strain of constant and intense caregiving, as well as keeping people informed of current developments. That families can so easily connect and learn from each other is vital and sustaining. The day-to-day struggles can be so overwhelming sometimes for Rett parents that there’s not always a lot of energy left for what I feel is truly going to change our children’s lives---the research. What are your thoughts?
DT: There is no doubt that you really have to find ways to stay focused, and I think that was one of our biggest successes. Care has been important but it is research that’s absolutely critical; it’s the only way to move forward.
MC: Research has always been my commitment, and I hope the work of RSRT will prosper as brilliantly as that of the CFF. Congratulations again on VX-770 and
thank you for your guidance and inspiration.
DT: Monica, I know how tremendously devoted you are to this fight. The Rett community is very blessed to have you.
MC: Thank you, Doris.