NHF

The Age of Gene Therapy: Why Plasma?

Laurie Kelley December 4, 2022

It’s happened: gene therapy is available, at least for hemophilia B patients in the US. The idea that hemophilia can be treated as a “cure,” or at least using one shot to cover your bleeding episodes for at least a few years, is tantalizing. And if not that, we still have recombinant products that have never touched human blood.

We just updated our factor chart here, which compares all the products available on the market for hemophilia A and B. You have lots of treatments to consider! But why are we still making plasma-derived products?

A few reasons. But first, understand where plasma-derived products come from.

Plasma-derived products come from human blood plasma. Plasma donors undergo strict screening for disease risk factors, and their plasma is tested for several viral diseases. Recombinant products are not derived from blood; they originate from genetically engineered mammalian cells containing the human gene for factor. Recombinants are produced in large bioreactors, with human and animal proteins used in the culture medium in first- and second-generation recombinant factor. Third-generation products contain no human or animal proteins in the culture medium or the final product. Fourth-generation products are made from human cell lines.

Although plasma-derived products potentially risk transmitting blood-borne viruses, all US factor products, whether plasma derived or recombinant, are considered safe by the FDA.

Why would anyone intentionally choose a plasma-derived product instead of recombinant? After all, recombinant factor is the product recommended by NHF’s MASAC. And now we have gene therapy for hemophilia B. Why inject anything other than the missing factor into your child?

For some people, it’s all about cost. Plasma-derived factor is less expensive than recombinant factor. And gene therapy is expected to cost about $3.5 million per shot. People who have high out-of-pocket expenses need safe products, but may choose less expensive ones. Sometimes, the decision depends on the type of bleeding disorder being treated. For example, intermediate purity factor VIII products contain factor VIII combined with VWF (the way it’s naturally found in the blood) and are useful in treating von Willebrand disease.

When choosing your factor therapy, always discuss options with your HTC! Use our factor product guide here, which now includes bispecific antibody products and gene therapy, and bring it with you, and ask what’s best for your loved one or you.

Go Away!

Laurie Kelley February 23, 2022

Spring is coming, and it feels like it, as more and more, the country is opening up. It’s wonderful to see that some of our community events are back in person! Read below to see which ones you might attend. Remember, we are not yet through the pandemic. Please be sure you are vaccinated, and be careful. I’m going to wait a bit more before attending, but remember, you can always attend virtually. Check it out!

Factor IX Families Welcome!

Register for the Coalition for Hemophilia B’s annual meeting in Orlando! May 19-22; travel grants available! Attend also virtually. Register here.

NHF’s 74th Annual Meeting

NHF is thrilled to announce that its 74th Annual Bleeding Disorders Conference (BDC) will take place from August 25 to 27, 2022, in Houston, Texas. After two years of uncertainty, NHF is excited to gather our community in person! You can also attend virtually. Read more here.

Saddle Up!

Texas is the place to be this year! HFA will hold its annual meeting in person in San Antonio, the first time since 2019. Register here!

Remembering Legendary Leaders

Laurie Kelley January 2, 2022

It’s been a sad couple of years, made sadder today by blogging at the first of the new year about the loss of two incredible leaders in our global hemophilia community, one in the US and one in Pakistan, gone in as many weeks. I’ll write tonight about the loss of Val Bias, the first leader in the hemophilia community I ever met, who made a lasting impression on me.

Val Bias and Laurie Kelley at an NHF Annual Meeting

Val Bias was an icon in the US hemophilia community, an unmistakable presence with his dazzling smile and hearty laugh, who possessed charm but purpose, intelligence but an everyman, relaxed posture, a polished speaker who could also sit back and listen deeply. His goal was ultimately to channel his own pain and suffering, of which there was much, into something for the greater good. He was relentless, and successful, in that goal.

I first met Val during my first National Hemophilia Meeting in 1992. I was the mother of a 5-year-old with hemophilia who had just written my first book, Raising a Child with Hemophilia. I knew about HIV and vaguely what was happening to our community, but … not really. Wrapped up in my own non-HIV world, trying to figure out my place in the community, I watched with horror as a room full of patients, parents and advocates began to rip one another apart over what was happening in the community. People were furious at NHF for their lack of leadership, for advising patients to keep taking their medicine. A patient-led group had formed and stood up, shouting and threatening. Suddenly, one clear, commanding voice boomed: “Everyone, sit down and calm down.” I looked around to see who had this voice, this presence, this leadership. It was a handsome man on the podium, who seemed to be the only one with a rational expression and the only one not afraid. Val Bias.

Val and Laurie in 1998

He was famous already in the community for being executive director of the Hemophilia Council of California, a lobbyist for NHF in Washington DC, and president of the board of the Hemophilia Foundation of Northern California. He was an active camp counselor, mentoring the boys with hemophilia. What amazed me most about him was his role in helping get the Ricky Ray Hemophilia Relief Fund Act of 1998 passed. This act allowed monetary compensation for those infected with HIV who have hemophilia.

Later, Val became the first African-American leader of NHF and one of the first patients to become a leader in NHF. His presence was galvanizing and he made impactful structural changes, which required great risks.

The tributes and condolences are pouring in on Facebook but this one, from our mutual friend and colleague, Dana Kuhn, a particularly close friend of Val’s, hit me the most:

“The pandemic of HIV called for a change in narrative of the national organization [NHF]. [A] group of 5 men realized it was their responsibility to teach men with HIV how to protect women from HIV infection and empower men to educate others to become leaders in their communities. These 5 men convinced NHF to create the Men’s Advocacy Network of the NHF (MANN) which brought altering changes to NHF. The change was strategically training and placing men with hemophilia in places of leadership. As a result, Val became chairman of the Board of Directors of the NHF, and Glenn Pierce became president of NHF. Many of us became directors on the NHF Board. Under new leadership the organization began to work through the decisions and responsibilities of the HIV pandemic in the hemophilia community. With the leadership of Val Bias, positive change was made. The community was unified to work on the Ricky Ray Hemophilia Relief Fund Act of 1995. New, improved and safer hemophilia products were coaxed into coming to market. The safety of the Nation’s blood supply became a priority especially through blood/plasma collection processes. Government agencies and Cabinet level committees accepted hemophilia consumer members to these positions turning a listening ear to their concerns and perspectives. NHF created initiatives under Val Bias and Donald Colburn challenging the raising of funds for research in gene therapy for a CURE. Under Val’s leadership ‘Chapter Development’ was made a priority and programs were implemented. Chapters became stronger and more universally developed. I could go on with much more.

Val with Chris and Jess Bombardier, and Laurie Kelley, in 2019, recognizing Save One Life; the last time they would see Val.

“However, how can I impress upon the bleeding disorders community the passion and compassion this leader had for his community and the awesome change he facilitated coming out of NHF’s challenges with the HIV pandemic. We need to honor this leader and the work he accomplished to help make this community better. The NHF Board and leadership, the Chapters, Youth Leadership Programs, Cure for the Disease programs, educational programs, relationships with donors would not be where they are today without his leadership.”

One of the hallmarks of a true leader is not what position they held, or how many accolades they receive, but what legacy they leave behind. Val was a transformational leader who changed our world, for the better. RIP Val; your work is done. Let those you mentored carry it forward.

Learn more about Val Bias in the movie “Bad Blood,” available on most streaming platforms.

Learn more about Val’s contributions in this article in HemAware.

NHF’s 62nd: Carnival, Concern, Unity

Laurie Kelley November 15, 2010

The theme of the 62nd Annual Meeting of NHF was “Marching Forward,” appropriate for a place like New Orleans, where you can expect a high school marching band to turn the corner of your street at any time. Situated at the Marriott on Canal Street, just a carnival bead’s throw from the Mississippi River, the event hosted about 3,000 community members, including physicians, nurses, social workers, nonprofit staff, NHF reps, pharmaceutical and homecare reps and of course, consumers!

My week started with a five-hour workshop on Novo Nordisk’s new HERO program, the largest psychosocial research project in hemophilia history. We gathered a select group of HTC and consumer representatives to discuss ideas to bring the results of HERO to the US, to share with our community: what can we learn from the results and what to do with the results? It was a lively and challenging task, but with a great group that meshed well, it was also fun!

Opening night was heralded with a marching band and carnival characters on enormous stilts, handing out strings of beads to all. NHF Chair Stephen Bender and father of a daughter with hemophilia gave a moving opening speech recalling his daughter’s birth, and how he became an advocate for better health care as a result of their harrowing experience. Executive director Val Bias gave a motivating speech about unity in the bleeding disorders community. He also unveiled a new campaign for women with bleeding disorders, called “Victory for Women.” This will replace Project Red Flag.

Later we walked through the exhibit hall, which contains booths by all the vendors who serve our community, and all had a Mardi Gras theme. There was face painting, games and great food.

There were many sessions on a variety of topics related to bleeding disorders. Women with bleeding disorders figured prominently as a topic. I also attended a very interesting town meeting by the Committee of Ten Thousand—COTT. COTT is often referred to as our third national organization. We have NHF, HFA and COTT. This certainly doesn’t refer to third place. Each of our national organizations has a vital role to play.

This two hour meeting had two topics: blood safety, prompted by the new movie Bad Blood; and privacy issues for patients. The first topic was kicked off by Corey Dubin, a person with hemophilia and an icon in our community. Next to him was Nathan, representing the Gay Men Health Crisis organization. This year 18 senators sought to repeal the FDA ban on blood donations from gay men in light of all the safety procedures and testing performed in the US. The ban has been in place for 17 years, started during the HIV contamination of the 1980s.

A lively discussion took place but what was unique and wonderful was the mutual admiration and respect of COTT and GMHC. Each respected each other’s view about blood safety and the right to donate blood, and whether that even was a right.

The second topic unleashed a firestorm of concern: patient privacy rights. Are patients being adequately informed of how their data is being used? One patient, a well known advocate, stated that patient data is gold. People in our industry make money off of patient data, good money. How is this safeguarded? Patients are signed up for clinical studies—are they well informed? With health care reform, and anticipated countermoves by the insurance industry, patient data is needed and desired to justify decisions to restrict access to care, access to products or to protect clinical treatments. Data is everything. But do patients know who has their data?

With representatives of several different and independent organizations and patient groups—and industry—in the audience, it became clear that each group had similar questions about ethics, funding, transparency, and patient protection of any organization collecting patient data. I explained how LA Kelley Communications collects patient data through our mailing list, and how fiercely we protect it.

For other groups, it’s not as clear what the data is to be used for ultimately. Will it be sold? There’s a market for it. A big discussion ensued about bringing various groups to the table to discuss privacy and to get some questions answered. COTT has acted as a watchdog for blood safety, and now may act as a watchdog for patient privacy— a topic more vital than ever in this era of health care reform. Patient Data equals power, power to discriminate, make policy, earn money, effect government policy… the possibilities are many. This data is our data—our child’s data, our carrier daughter’s data—and we need to protect it by knowing who has it and what they plan to do with it.

After the town meeting, some people went to social events. We had a special guest—Usha Parthasarathy from India, with whom I had just finished a 16-day tour of India. She was able to promote our Save One Life program to many people, and secured more sponsorships for the 300 children who are waiting for sponsors.

On Saturday morning I facilitated a symposium hosted and funded by Baxter Healthcare, titled “Paving Your Way, Protecting Your Future.” Guest speakers included Don Molter, social worker and career counselor at the Indiana Hemophilia and Thrombosis Center, and Dr. Art Wood, vice president of Patient Services Inc. Don stressed the importance of taking the SATs or ACT, applying for scholarships and to be open to education even later in life—especially in this economy, when many people need to rethink their career choices. He introduced the Baxter Education Advantage Scholarship, which helps people with funding for college and also vocational training. This is open to anyone, not just those on Baxter’s products.

Art’s presentation included salient highlights of the ACA, the new health reform act, and how this may impact families with bleeding disorders, and how patients can take action now to get informed and learn more. It will be absolutely vital for everyone to read their insurance policy and to read updates regarding the ACA and how it impacts individual policies.

We also had two community speakers: David shared a very touching story of having hemophilia, HIV, hepatitis, losing his thumb in an accident… and losing his wife of 24 years when the marriage was overwhelmed. With a deepening of his faith and assistance from his HTC, he successfully returned to work and now is remarried. Dwight and Kathleen are parents of eight children, four with hemophilia, and shared their trials in trying to stay insured. With the help of PSI, they were able to make it. Their handsome son Patrick was in the audience, admiring his parents.

The three day event closed with a final event, also sponsored by Baxter, at a warehouse where we walked by enormous plaster of Paris statues of King Kong, Pokemon, King Neptune, the Cowardly Lion and Yoda—all Mardi Gras characters. Also we saw floats being prepared for the next Mardi Gras. It was really amazing! At the end of the walk was a large indoor room to house about 2,000 people who feasted on New Orleans cuisine, dance to an excellent band, and have their fortunes read by Tarot card readers. I caught up with my friends from California—Laurel McDonnell, Paul and Linda Clement—and enjoyed the company of our very special community.
Please visit these websites to learn more:
www.cott1.org
www.uneedpsi.org
www.thereforyou.com
www.saveonelife.net

See you in New Orleans!

Laurie Kelley November 8, 2010


I am looking forward to arriving in New Orleans this week, to attend NHF’s 62nd annual meeting. I haven’t missed one in well over 15 years. There should be lots to talk about, with the new Health Care Reform bill passed this year, and the showing of the new documentary “Bad Blood.”

I’ll be facilitating a great symposium on Saturday morning, called “Build Your Tomorrow Today,” hosted by Baxter BioScience. The topic is a good one for anyone with a transitioning person with a bleeding disorder such as a teen, college kid, or even an adult who is looking for a new line of work. We will have two expert speakers: Art Wood of PSI, who will address the insurance issues that come with new phases of life. And Don Molter, a career counselor, Indiana Hemophilia & Thrombosis Center. We’ll also have community members give testimonials about their experiences in trying to change jobs or careers, and handling insurance,

Breakfast starts at 6:30 am and the program starts at 7 am sharp; see you there!

Great Book I Just Read
Genius on the Edge: The Bizarre Double Life of Dr. William Stewart Halsted by Dr. Gerald Imber

This is the amazing story of an American hero, the founder of modern surgery. Consider that just 100 years ago most doctors only needed a high school exam to get into “medical school,” and most had never even seen a patient by the time they graduated! It was more snake oil than science, and this at a time when Europe, particularly Germany, surged ahead in medical advances. In 1850 there was no anesthesia, no germ theory, no blood transfusions and few surgeries were even performed because the outcomes were usually disastrous. The best surgeon was usually the fastest. Patients who faced surgery often died of infection, gangrene or shock from blood loss.

Then along came Halsted, and changed all that. He studied in Europe, became a surgeon, and helped to create the leading edge medical school at Johns Hopkins University, the premiere hospital in America and one of the few that didn’t flunk the Carnegie Institute’s national assessments. He initiated the aseptic technique in America, which cut infections up to 90% or more following surgery. He insisted that everyone use latex gloves; he pioneered mastectomies, hernia repairs, and more. And even more, he trained men who became outstanding leaders and pioneers in their fields, including William Osler, William Henry Welch, Harvey Cushing (there are bios on each and I am going to read them! These men are like superheroes).

The book delves as much into his bizarre personality and personal life as much as his towering achievements. He was addicted to cocaine, and later morphine, which he apparently never kicked. He was fairly antisocial, and quite particular about his clothes and Italian shows–yet his house was cluttered like a hoarder! There is no doubt that his mark on history is great.

Whenever you or your loved one has surgery in the US, give thanks to William Halsted, whose high standards, complete dedication and genius, saved literally countless lives. I can’t wait to go to Johns Hopkins someday and see his portrait. I feel like I know him from this excellent book! Three stars.

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