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Hemophilia Headlines
The Hemophilia Foundation of Oregon
office: 10940 SW Barnes Rd. #129, Portland, OR 97225
phone: 503-297-7207
fax: 503-297-0127
email: [email protected]
web: www.hemophiliaoregon.org
SEPTEMBER 2013
Find Us on FacebookWant to know what is going on with the Hemophilia Foundation of Oregon?
Check out our facebook page Hemophilia Foundation of Oregon ‘like’ us and
you will be kept up to date on all the happenings.
Upcoming EventsSEPTEMBER:
September 12, 2013, 6:30pm, Family Support Group/Portland. At the Family
Fun Center in Wilsonville.
September 21 , 2013, 9:30am-3:00pm, Annual Meeting. At the Oregon Zoo
Conference Center, Portland.
September 21 , 2013, 3:00pm, Blood Brotherhood Meeting. At the Oregon
Zoo Conference Center, Portland.
NOVEMBER:
November 3, 2013, 5:30pm, Shooting for the Stars, Portland, OR. Auction
and Gala Affair. Location TBA.
November 8-9, 2013, Women’s Retreat, Portland, OR. Location TBA.
Virtual Walk -Easy as 1-2-3 ONE: Visit www.walkforhemophilia.com
TWO: Create a virtual character and choose a message
THREE: Select the OREGON Chapter, and help us with your support
Sharing the Virtual Walk on Facebook or Twitter earns our chapter
ONE additional walker. On the day of our chapter’s physical walk, one
virtual walker created counts as TWO walkers!
These are great opportunities to make an impact on our chapter’s ranking.
Please be sure to get your friends and family to participate. Together we
will help raise funds and awareness of bleeding disorders in Portland and
across the country!
Oregon Hemophilia Walk Recap
Thank you, to all that helped make the first Oregon
Hemophilia Walk such a resounding success! We were
greeted by close to 300 walkers, a beautiful day, and
the delights of both the White Rhino Marimba and
Pride of Portland Chorus. We are close to our goal
of $50,000 and we need just $10,000 more to reach a
match of an additional $10,000. Now is the time to
ask one more time your friends, families, and associates for one more give to the
walk. Help us make this match! Go to www.hemophilia.org/walk/ and click on OR.
You will find the donate button there. Checks can also come to the HFO office.
For more information about the 2013 Oregon Hemophilia Walk, contact Madonna
McGuire Smith at 503-297-7207 or e-mail to [email protected].
Hemophilia Foundation of Oregon
10940 SW Barnes Rd. #129
Portland, OR 97225
HFA Symposium Testimonial by Billy Conde Goldman
This was my first time attending the HFA Symposium. Born in 1964, Gemini
Dragon with severe hemophilia A. My entrance into the hemophilia community
and meeting others with hemophilia is a recent process which I am navigating
with curiosity, resistance and excitement.
I attended the COTT Town Meeting—an inspiring participatory forum for
communication and discussion. Passion and commitment and responsibility
(response ability) in action. Changes in health care and insurance were
discussed. A vigilance is needed to monitor these issues and COTT is
doing that work. Also discussed was creating a national living memorial
for people with hemophilia who have died of HIV/AIDS and hepatitis C.
This is important for the hemophilia community and for the country. Such
a memorial acknowledges those who have died and serves as a potent
reminder for the ongoing importance of the safety of the blood supply for
one and all. Long live COTT.
On one of the breaks between programs I was fortunate to meet Gary and Karen
Cross. Gary wrote Vial 023 which documents the life and death of his son Brad
who had hemophilia and the involvement of Gary and his family and colleagues
in the hemophilia/pharmaceutical company settlement. A powerful and moving
memoir and hystory of the tainted blood tragedy.
The symposium concluded for me with the Blood Brotherhood Rap Session.
This was the largest gathering of people with hemophilia I have experienced.
Everyone in the room had hemophilia. There were no doctors, nurses, social
workers or parents who were involved in the community but did not have a
bleeding disorder. Just people with hemophilia—from the Greek haima ‘blood’
and philia ‘love’. A convergence of blood friends. Knowing that each person
has lived with hemophilia provided a sense of community—together making
for centuries of experience. Much to share with each other and much to learn
from each other. I was reminded about the challenges presented by having just
hemophilia and no co-infections. While there have been functionally significant
advances in hemophilia treatment and care, physical, psychological and spiritual
challenges remain. Hemophilia impacts the whole being and attention needs to
be given to this full spectrum of needs. The Blood Brotherhood program is a gift
and a valuable resource. May it continue to thrive.
The HFA symposium was an awareness raising experience for me. Many thanks
to the Hemophilia Foundation of Oregon and the Hemophilia Federation of
America for financially supporting my presence.
Grief is the binding alloy of the armoring about the heart. … Opening the heart to the mind’s pain, we find space to explore mercifully. … We enter directly into our grief, encouraging it to reveal its deepest nature. So we discover what lies beyond a lifetime of holding: our healing and the finishing of our most subtly unfinished business. —Stephen Levine
Annual MeetingPlease join us for the 2013 Annual Meeting at the Oregon Zoo and Portland Children’s Museum
Saturday September 21, 2013 9:30-3:00 pm
We are thrilled to announce an Educational and Family Event. Join us for the Day!
There will be an adult program including information on using social networks to
develop better healthcare outcomes and information on how health care reform will
have an impact on your life. There will be daycare for children under the age of 4,
a children’s program for those aged 4-12, and a teen program for the 13-18 year
old group. Join us for a vibrant program, lunch, and a trip to the zoo. Look for an
active, fun-packed day. Bring the whole family. See you there. Questions contact
Marita or Madonna at 503-297-7207 or email to [email protected].
A ‘Trick ‘to the Immune System With Experimental Hemophilia B Gene Therapy
In July, researchers at The Children’s Hospital of Philadelphia (CHOP)
published the results of a study in which they used bioengineered decoys as
a ruse to foil the immune system. This allowed for the successful delivery of
gene therapy in mice with hemophilia B, or FIX deficiency. The lead author
of the study was Katherine High, MD, director of CHOP’s Center for Cellular
and Molecular Therapeutics.
High and her team used capsids, the protein shell that encapsulates a virus, as
the decoy. They also used adeno-associated viruses (AAVs) as delivery vehicles,
or vectors, to carry the actual genetic material that triggers the production of
factor IX. AAVs have recently become the “vector of choice” for researchers
because they can deliver the genetic material into living cells to sustain
therapeutic effect without causing disease. In addition, they can be targeted to
liver cells, which manufacture FIX.
However, one drawback in using injected AAVs is the body’s natural immune
response. In some cases, antibodies are released, neutralizing the AAVs. To
remedy that, investigators injected both empty capsids and genetically altered
AAVs into the mice. The empty capsids effectively drew antibodies to them and
away from the gene therapy.
“This decoy strategy could be individualized to patients and could greatly
expand the population of patients who may benefit from gene therapy,” said
High. “Right now, 30 to 60 percent of adult patients develop antibodies that
block the ability of an intravenously infused vector to reach the target cells
in the liver. This approach holds the promise of overcoming this roadblock--
pre-existing antibodies--and allowing successful intravenous gene therapy in
virtually all adult patients.”
This technique also proved effective in follow-up studies performed in rhesus
macaque monkeys. The therapy initiated higher levels of factor IX production,
with no adverse events reported. Although additional studies, including clinical
trials in humans, will be necessary before such a therapy becomes a reality, the
authors are encouraged by these preliminary findings.
“Our results, which held up over a
range of doses, suggest that in clinical
studies, it will be feasible to adjust the
ratio of empty capsids to gene vector
doses, depending on an individual’s
pre-existing level of neutralizing
antibodies. That means we could
personalize gene therapy to make
it more efficient for each patient,”
concluded High. “This work should
make it possible to bring effective
gene therapy to most adults with severe hemophilia B. Each patient would
receive a personalized final formulation that contains just the right amount
of empty capsid to neutralize any pre-existing antibody, and allow the
gene-expressing vector to reach the liver.”
The study, “Overcoming Pre-existing Humoral Immunity to AAV Using
Capsid Decoys,” was published in the July 2013 online issue of Science
Translational Medicine.
Source: ScienceDaily, July 17, 2013
Summer Camp 13Tapawingo Time-Travelers greeted 68
campers this year. We had a terrific week
visiting the Prehistoric, Egyptian, Wild West,
and 80’s time periods. Camp was peppered
with characters from these periods, a prehistoric
meal with no utensils available, and a wild west
gold rush. We want to thank all the parents
who entrusted their children to camp and a
hearty thank you to all the staff that created a
magical week. Contact the HFO office today
for more information. Call 503-297-7207 or
email [email protected]
HFO WebsiteWe hope you have had the opportunity to visit the new website www.hemophil-
iaoregon.org. Please take the time to find us. We also need you to register as
a member on the website. The information you submit helps us provide you with
information regarding upcoming events and programs. It also allows us to find
you when there are specialized programs and opportunities that might relate to
your specific needs. Please take the time today to register on the site, you will be
glad you did. Thank you.
Thank You Sponsors! We especially want to thank our benevolent Sponsors for their kind and
generous contributions to our notable life-saving cause. We at HFO could not
provide the high level of assistance and opportunity for our members without
you. Please find the full list of Sponsors who have contributed to the Hemophilia
Foundation Of Oregon on our website.
SILVER: Novo Nordisk BRONZE: CSL Behring, Hemophilia Center, Urban Waxx
