Rare Leader: Tim Rothwell, President, SDHB Pheo-Para Coalition

The Basics
Name: Tim Rothwell

Title: President

Organization: SDHB Pheo-Para Coalition

Social Media Links:

Disease focus: Pheochromocytomas and Paragangliomas are rare neuroendocrine tumors. A pheochromocytoma is a tumor arising from the adrenal glands. Those patients who present with an SDHB genetic germline mutation are associated with much higher rates of malignancy. Furthermore, there are no cures for this disease and the treatments are nonexistent and inadequate today. Surgical resection or removal of the pheochromocytoma is the most common and recommended treatment and should only be performed at centers experienced in the management of this rare disorder. Paragangliomas are closely related, though less, common tumors that originate in the ganglia of the sympathetic nervous system and are named based upon the primary anatomical site of origin. These tumors can be found anywhere in the body but are typically found in the head, neck, chest, abdomen, pelvis, and urinary bladder areas.

Headquarters: Doylestown, Pennsylvania

How did you become involved in rare disease: I became involved in this area because my youngest daughter has the disease. She also has the SDHB genetic mutation, as does my eldest daughter and my wife, which is why I started to focus on the SDHB affected patients. Paragangliomas come in two forms if you will: the non-genetic and the genetic. Those who have the genetic disposition, the SDHB germline mutation, unfortunately have a more a serious disease and uncertain outcome. In our family, my wife is the carrier, she is 67 and never had the disease. My oldest daughter is 36. She has not had the disease. And my youngest daughter is 32 and she has had the disease and has been operated on three times and had 12 tumors removed. The disease obviously can impact anyone. It does not discriminate, it affects young and old, people of all races, but in my view, it is particularly frustrating because there is no cure. I think you have seen this before with other rare diseases, but to see young people experience these kinds of symptoms early in their life, it is debilitating, and it is very difficult physically and emotionally for them to handle. In the case of pheochromocytomas, the outcome is much better for patients because pheochromocytomas, which arise on the adrenal gland, once diagnosed and surgically removed, result in 90 percent of these patients being fine. It is the paraganglioma patients that often face more significant challenges, because the paragangliomas can arise anywhere from the head and neck all the way down to the pelvic region. They can be found all over the body and they tend to multiply and metastasize, and this is what my daughter has. They can occur at any time. There is no cure. The only treatment that exists is an old chemotherapeutic regimen, which is CVD, but it does not work very well and at best delays disease progression. Radiation therapy has been used with mixed results as well. The best alternative patients have today is surgical debulking, but you can only surgically debulk up to a point in time because there is too much scar tissue. After that there is precious little you can do. That is my reason for becoming involved in the world of pheochromocytomas and paragangliomas. We need treatments that work for patients. I was one of the early board members of the Pheo Para Alliance before I co-founded the SDHB Pheo-Para Coalition. I have been very involved with these two organizations over the last 12 years with the goal of trying to find a treatment that potentially can work. That is where we are.

Previous career: Pharmaceutical executive for over 50 years in senior management positions and currently serves as chairman of a healthcare consulting firm.

Education: Bachelor of Arts in chemistry from Drew University and J.D. from Seton Hall University.

The Organization
Organization’s mission: The organization’s mission is to first educate healthcare professionals and patients on the SDHB genetic germline mutation, as well as its role and impact in this orphan disease. Pheochromocytomas and Paragangliomas are both neuroendocrine tumors and those patients who present with the SDHB gene, as I said before, are unfortunately associated with much higher rates of malignancy. The idea is to try and support research, which will ultimately have the impact of providing an effective treatment. Today, we don’t have any proven therapeutic or radiation therapies that cure or adequately treat this rare disease. We support research in the academic arena for the most part, and we’ve made some progress, but we have a long way to go.

Organization’s strategy: Our strategy, primarily, is to focus on the SDHB mutation form of the disease and to have an impact in two specific areas. The first area that we are focusing on, and an integral part of our strategy, is to support research at credible and experienced institutions where the research will hopefully advance our understanding the disease. From what I can tell in talking to healthcare professionals, we do not truly understand the disease or the pathways. You have to understand the disease before you can ultimately try and create effective treatments to address the disease. The second strategy, and an important one, is to increase awareness of this orphan disease both to healthcare professionals and patients alike. One of my greatest frustrations is the fact that we interact with patients as an advocacy group fairly frequently, but unfortunately, in many cases and during the time that we are interacting with them, it is too late. In other words, the disease is too advanced and there is little that can be done to help them. It is very tough because you are dealing with, in some instances, kids. I have seen kids four and five years old who have the disease and needed to have surgery to remove tumors and if we get to them early enough, they often have a good outcome.

Funding strategy: Our first priority is to try and secure a revenue stream that will allow us to execute on our agenda and achieve the stated mission that we just alluded to. We do this by conducting annual fundraisers, which are supported by corporate and individual sponsors. We are also keen to gain access to other potential investors. We have been successful with our annual galas in New Jersey for the last ten or 11 years with the exception of two years during the COVID crisis. Our first gala post COVID is coming up on April 28th, but it’s virtual. We have been able to get anywhere between $300,000 and $400,000 on a net basis through these galas. But most of our supporters who have been terrific are people from the pharmaceutical industry who I have been associated with over many years. I am 71 now. I have been in the pharmaceutical industry for 50 years. I started when I was 21 at Sandoz, which is now Novartis. Many of the supporters are my friends and associates who have supported me because they know me, or they worked for me, or I worked for them and are now retiring. Trying to find people who have an interest in supporting you, who know nothing about a rare disease that only affects about 2,000 to 3,000 people in the United States per year is hard. We partner with other organizations in academia to try and learn more about the disease, what the treatment options might be. We have sponsored research at Tufts Medical Center, Columbia University, University of Alabama, University of Florida, the Broad Institute of MIT and Harvard, University of Melbourne in Australia, as well as institutions in Holland and one in Germany. This has resulted in some positive results to date, but it is very early-stage and very basic research.

What’s changing at your organization in the next year: We are a volunteer organization of about a dozen people that are directly involved on a daily basis. We are very proud of some of the accomplishments to date, but what we identified last year is that we are an aging board. We have created a succession plan whereby we will add younger members to our board who will bring in fresh new ideas and perspectives. For example, we frankly did not get involved much in Facebook, Instagram, LinkedIn or Mailchimp previously, so we brought in a couple of additional young, knowledgeable, and experienced people to assist with running our marketing and social media sub-committees with the idea of them becoming board members after a transitional period. These two new people that we brought in at the end of the last year are expected to become full board members in 2022. We need people who have different backgrounds, and we need people who have different experiences. We need people who are, as I said before, younger, but we cannot just be reliant on the pharmaceutical industry because the pharmaceutical industry, as you well know, are not always the biggest supporters of orphan and rare diseases. We need to find other ways of trying to contribute to the funding strategy that I just mentioned. We need to change the organization to adapt to it and to try and get support for what we are doing by reaching out to others in different industries / businesses.

Management Style
Management philosophy: My management philosophy is pretty much the same as the philosophy that I have used for many years. I was the president and CEO—going back to 1986—of a U.S. pharmaceutical company. And I have been global head and U.S. head in a number of different pharmaceutical companies. My management philosophy is the same as my leadership philosophy. I have more of a participative leadership style with an emphasis on trying to set what I consider to be clear goals and objectives, a shared strategy, shared accountabilities, and responsibilities for a team. I am very much a team-oriented person. It is important to ensure that there exists good and open communication with all stakeholders, but you must give them the freedom to run. It is important to create an environment where they are able to improve upon some of the ideas that I might have, or others might have.

Guiding principles for running an effective organization: The main guiding principles I like to apply include (probably the most important being) diversity of ideas and thinking. As I said before, open and transparent communication, and a focus on team. Having said that, delegation, accountability, and trust are also important guiding principles in my mind. It is very important in this particular field, and I’m sure this is true of other rare diseases, to remain optimistic. We see many challenges and setbacks every day. We must maintain a positive outlook because we, at the end of the day, are an organization focused basically on hope.

Best way to keep your organization relevant: The best way is to stay current with the science, know it as it evolves, and support cutting edge research in this orphan field. Most importantly, be open to new ideas and approaches. It is interesting to note that some of the more innovative scientific approaches to this disease have come from a group at the University of Alabama, which is one of the largest oncology research centers in the U.S. I have been in the oncology field for 25 years. I always knew that the University of Alabama had a good football team, but I did not know they had one of the best cancer institutes. You have to be open minded to different organizations that are pitching you and who are suggesting that something may be worth pursuing. It is also important to communicate new information, share best practices with healthcare professionals, providers, and patients to help drive individuals to our website, which keeps us current and relevant.

Why people like working with you: I think I am a pretty good communicator, an honest guy, and trustworthy. I also have a lot of experience. I have 50 years of experience in the pharmaceutical sector, and I have learned many lessons over the years, which I think can be helpful in leading a non-profit organization such as this to achieve the desired results.

Mentor: Over the years, I have had many mentors. When my daughter was first diagnosed, I did not know how to spell the disease never mind identify who treats it. I started by talking to the smartest people I knew in the oncology space and asked them what they knew about this orphan disease and who has expertise in treating it. I quickly found out that there were no cures or treatments that work. I did identify through that networking approach about a dozen physicians and scientists who were either working in this area or treating patients and at least we’re familiar with it. It is a very small group. I would say that in the U.S., it is probably no more than 50 people if that. One of the individuals who has been my key mentor is Karel Pacak, who is at the NIH, and another is Steven Libutti, who is the head of the Cancer Institute of New Jersey. He spent many years at the NIH before going to Montefiore Einstein and then to the Cancer Institute of New Jersey. These people are leaders in the field that we are actively interacting with. They are cutting edge scientists and physicians, both of them. They treat patients as well as engage in research. Without a doubt, they have been terrific to work with and their support and mentorship is greatly appreciated. We are also grateful to many others that are active in this space.

On the Job
What inspires you: I am inspired by the volunteers on our board and our supporters, who share a passion for this orphan disease and are committed to our mission. I am also inspired by the scientists who are working in this space with little or no financial support, but they keep at it despite the challenges and frustrations they face every day. Dr. Pacak is a great example. He has seen his budget cut year over year and as you well know, you’re dependent upon your division director in what you will receive in terms of funding. And then finally the most important are the patients who are the unsung heroes. My daughter continues her fight, despite the obstacles she faces every day. She is pursuing a degree in clinical psychology, and she is doing so despite not knowing what her future holds. Thankfully, she has done as well as can be expected, but she still has some paragangliomas, albeit small, in her body. She is doing it because she said, “The medical treatment I got was terrific, but nobody thinks about providing us with any psychological help. I am going to get a Ph.D. in clinical psychology so I can help people who have a disease similar to mine or any other orphan disease.” She really wants to give back basically.

What makes you hopeful:  I am hopeful because more than 10 years ago when some of us first started this effort there was almost no research being conducted in this field. Now there are many institutions conducting research in this field and there are at least three organizations including our own that are supporting many of these efforts. One example of this is that we have been trying for over 10 years to develop an animal model that would potentially replicate the SDHB mutation that exists in humans and grow malignant tumors in animals. Those efforts failed until recently, but it looks like we are close to achieving this now, which would not have been possible without our support. It goes without saying that we are hopeful that we will develop better treatment options sooner versus later and/or a cure.

Best organization decision: The best organization decision was our internal honesty and recognition that we needed to identify some younger people who could take the lead of the organization in the next year or so. Over the last six months, we have spent more of our time communicating via a variety of social media platforms, which as I mentioned previously, many of the board directors and I are not familiar with. We are of course familiar with Facebook and LinkedIn, but not from the perspective that these young people are. It is having younger people involved, getting more involved in social media, and looking at different ways to fund the organization. We are a volunteer organization. None of us take any compensation. Any grant that we issue, 90 percent of the grant amount goes towards direct expenses ensuring that funds actually go to the research work itself. We only allow 10 percent for indirect costs. At the end of the day, all funds received by the organization are spent on research that can help ultimately better understand this disease.

Hardest lesson learned: Communicating the challenges and differences in outcomes associated with this disease. There are so few centers of excellence in the U.S. and around the world, and very few physicians that are expert in this orphan disease. When we have our first communication with the patient, the outcome is often unlikely to be very good. If we could get patients into the right hands sooner, they would have a better chance of an improved outcome. That is the hardest lesson to learn and live with.

Toughest organization decision: The toughest decision for me was leaving my full-time job as the Chairman and CEO of Sanofi U.S., which I did in 2009 after my daughter was diagnosed in 2007. Her first surgery was in 2008 and it became abundantly clear to me that I had enjoyed a great career. I continue on the board of a few biotech companies.  But the point is that it was time to get out of a full-time job and start to figure out how to try and help my daughter. The second was leaving the Pheo Para Alliance because I was one of the founders. After six years, I decided to shine the spotlight on the SDHB mutation, which is what our current organization is focused on. The Pheo Para Alliance is a great organization, but it’s broader in its scope. We believe we need to focus on the area where the patients have the greatest need, have the greatest potential malignancies, and the worst outcomes. That was my second toughest decision.

Biggest missed opportunity: Our biggest missed opportunity is our inability to get more funding for research. We have to work harder at trying to get funding, but it’s particularly challenging in orphan diseases, and even more challenging in a disease that affects only ~2,000 -3,000 people. If you find somebody who suffered from your disease who is a celebrity, it is different because they are able to use their platform and bring in other celebrities and other people who are willing to contribute. I am not saying that we don’t have celebrities who have the disease, but they are difficult to identify, and one has to remember that in many cases, the existence of this disease is often only found during an autopsy.  We must find new ways for fundraising and more effective ways of communicating to increase awareness in this regard.

Like best about the job: What I like best is being able to help patients, being able to help my daughter, and being able to network with healthcare professionals and put them in touch with one another so they can learn from one another. One of the challenges we have with some of these researchers is that they do not talk to one another because they are all about publications. They do not want to necessarily share their information until it’s public or published. I have been working on trying to get people to talk to one another. We have a small research advisory committee who are open to that approach.

Like least about the job: Our inability to help those in need. I mean, we have a long way to go to find an effective treatment and/or cure.

Pet peeve: Heavy traffic

First choice for a new career: I would be a medical doctor.

Personal Taste
Most influential book: Churchill: A Biography by Roy Jenkins

Favorite movie: The Green Mile

Favorite music: Queen

Favorite food: Fish and chips

Guilty pleasure: English chocolate

Favorite way to spend free time: We spend time at the New Jersey shore.