Message from the Chair

Dear Friends,

It is indeed an honor to be elected as Chairman of the Board of Directors this year, the 50th anniversary of the discovery of Alpha-1.  This will be a busy year with lots of new, interesting activities and as always, unexpected opportunities.  We already had a great international science symposium and patient meeting in Barcelona and are committed to increased collaboration and support of Alpha-1 organizations worldwide.  We are also trying to do 50 awareness or fundraising activities, one in each of our 50 states.  The annual meeting in Washington should be a great celebration along with all of the usual catching up with friends and the newest research and advocacy efforts of the Foundation and Association.  We will be busy, much busier than usual, but I am sure at the end of the year we will look back and see we accomplished a lot and advanced our banner toward our goal of a cure for Alpha-1.

Consider how far we’ve come in 50 years and where we want to go, not in the next 50 years but in the next decade.  We know enough about the mechanics of Alpha-1 that we are now on the doorstep of some significant breakthroughs in better therapies and real fixes for our condition.  We have an infusion therapy which has helped many of us lead longer and more productive lives.  The near future will bring another option, inhaled therapy, as an alternative to weekly sticks with a needle and the one- to two-hour time needed to complete an infusion. We have a patient healthcare program, AlphaNet, which I think is one of the best in the world, helping patients manage a very difficult, debilitating condition.  We have a robust research program, run by knowledgeable professionals, supported by fundraising programs that generate $8 million a year and a for-profit subsidiary, The Alpha-1 Project (TAP), which is now investing money directly in biotech and pharmaceutical companies with the objective of commercializing a drug or therapy for a cure.  We have a voice in Washington and many states thanks to our advocacy efforts.  We have influenced many politicians and government agencies, helping them make decisions that benefit not only the Alpha-1 community but all rare diseases.  We can all be proud of our Foundation, which is recognized as a rare disease, nonprofit leader throughout the world.  All of this and much more has been accomplished by the concerted efforts of many people: our employees, our scientists, our doctors, our supporters and ourselves.

In the next decade, will we find a cure?  I believe we will.  Why?  Because we have so many dedicated people who are focused on our goal!  We are concentrating our directed research efforts through TAP on gene therapy and small molecules.  We have committed almost $1 million in a number of companies that are making great strides in these areas.  At the same time, we are continuing to support our basic research investigators ($3 million last year) and invest in the tools, such as iPS cells, that will facilitate all Alpha-1 studies.  On the patient front, we are working more closely with the Association and have signed a collaboration agreement which helps both organizations accomplish their goals more efficiently.  Our detection, Clinical Resource Center and Building Friends for a Cure programs are becoming increasingly more active.  Along with AlphaNet and Alpha-1 healthcare programs run by our infusion providers, we are providing our patients with the tools to improve their health and lead more active lives.  These activities, and others too numerous to mention, will lead to the therapies that will arrest the progression of both lung and liver decline and eventually to a cure for Alpha-1.  Then, although our work will not be done, we can really celebrate!

Sincerely,

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