From academia to industry to advocacy, the National Hemophilia Foundation’s new president and CEO, Leonard Valentino, MD, has done it all. He spent more than 30 years treating people with bleeding disorders, and he founded the Hemophilia and Thrombophilia Center at Rush University Medical Center in Chicago, which he directed for more than two decades.
During a long academic career, Dr. Valentino led a basic science research laboratory investigating the molecular basis of joint disease in people with hemophilia, was involved in numerous clinical trials and published more than 150 manuscripts. More recently, he worked at Spark Therapeutics developing new therapies for bleeding disorders.
Dr. Valentino sat down with HemAware to discuss his past and what he sees for the future of NHF and the bleeding disorders community.
What drew you to benign hematology?
I’ve always wanted to be a physician. I was inspired on this path by a great-uncle who was Gen. Patton’s surgeon in World War I and by my cousin who was in medical school when I was in elementary school.
I was drawn to hematology because it’s a problem-solving field, one that is very knowledge-based. I also like numbers (I was a physics major in college) and am very analytical. Hematology is a lot of numbers and problem-solving.
Do you remember any patients in particular from your years as a clinician?
I remember a mother whose baby had just been diagnosed with severe hemophilia A. She told me the family was moving to a single-level home so they could accommodate the wheelchair she knew her son would one day need, because her father, who also had hemophilia, was in a wheelchair.
That started me thinking about the legacy of hemophilia patients and how I could play a role in changing that legacy to give her a different experience from her father’s. Today that baby is 25, healthy and not in a wheelchair.
How did the hemophilia treatment center at Rush change under your leadership?
When I came to Rush in 1990, there were four hemophilia patients. My partner had managed them from birth throughout their lives, and they had a very close relationship. In many ways, I say I “stole” those patients to create the treatment center.
Today, the center follows about 400 patients, including about 25 patients with inhibitors, and is one of the largest centers in the Midwest.
You no longer see patients. Do you miss that?
I miss the social and personal part of it, talking to people and understanding where they are in their life and their problems. But I don’t miss the ICU or emergency department.
Hemophilia is a family disease, so I had babies I cared for while I cared for their grandfather. I diagnosed women with bleeding disorders and then saw their mother or sister or father. I got used to seeing families together.
You were involved with the Twinning Program with the Haemophilia Foundation of Nigeria. What did you learn there, and how did it change you?
The World Federation of Hemophilia Twinning Program was life-changing. One of the main things I saw was the deprivation of care. It is night and day from what we have. It was hemophilia in the 1940s, which I can only imagine from reading in books. Children were crippled, living until their teens and then dying, having severe bleeding. There were no moderate or mild cases; everyone had severe disease because there were no lab facilities for testing.
While we brought supplies and factor, our goal there was to deliver education and advocate for care. The first time I gave a seminar, we expected 30 physicians but 300 came. It was truly amazing the hunger they had for knowledge.
Your research focused on gene therapy. Why?
I’d done a lot of work in inhibitors, but the holy grail has always been a cure. For years, my research focused on trying to understand why those who bleed in their joints develop arthritis and joint disease. We still don’t have that answer, but with the Human Genome Project and the era of genetic medicine, we started looking at the opportunity to intervene in the joint with gene therapy. I think gene therapy is a very exciting opportunity for the hemophilia community and for diseases in general, especially monogenic diseases.
The bleeding disorders community is vocal and active. How will you connect with its members to learn what they would like from NHF?
Communication with the constituency is vitally important for the success of any organization. I plan to work with the community to understand the needs and gaps in education, advocacy and research, and then attempt to fill those gaps with targeted programs.
What role should NHF play as the bleeding disorders community enters the gene therapy era?
Helping patients and healthcare professionals understand gene therapy is a role that NHF is particularly well-suited to fill. Choosing among different therapeutic options is also an area in which I believe NHF can make an impact for patients, families and healthcare professionals.
What challenges do you think will take center stage as you begin your role at NHF?
One area is the growing choice of therapies and how we help patients understand the nuances of therapies. Another is mental health issues. This is so important in bleeding disorders, given the many challenges patients face. With people with hemophilia living longer, we have to address age-related comorbidities. We never before had to deal with a 70-year-old with severe hemophilia who also has atrial fibrillation.
We also have to learn to deal with comorbidities after gene therapy. And, of course, research and education and advocacy go hand in hand.
NHF is uniquely positioned to drive a national research agenda as well as advocate for issues around payers and reimbursement, including defining the right outcomes for value-based reimbursement.
One challenge facing us is the sustainability of the care model. Fewer and fewer people are going into the field of benign hematology because oncology dominates. So the field is at a crossroads. We have to begin talking about hematology to kids early, before even high school or medical school. By the time they are residents or fellows, it’s too late.
Finally, we need more support. We can have as many ambitious goals as we want, but without the money to execute them, it’s useless. We need people’s support for that.