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SILVER SPRING, Md., Oct. 2, 2015 /PRNewswire/ -- Some 250 people with Alpha-1 Antitrypsin Deficiency (Alpha-1), their caregivers and supporters filled the room at a public meeting held Sept. 29 by the U.S. Food and Drug Administration (FDA) to discuss Patient-Focused Drug Development for Alpha-1.

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Alpha-1 is a genetic condition that can lead to lung and liver disease. It is the most common known genetic risk factor for emphysema and is frequently referred to as "genetic COPD."

The FDA's Patient-Focused Drug Development initiative is intended to gather patients' perspectives on the impact of a condition on daily life and the available therapies to treat that condition. The Alpha-1 community is one of just 20 scheduled over five years for these FDA hearings.

Currently, there is no cure for Alpha-1. When a treatment that may cure Alpha-1 is developed, it will require the FDA's approval.

Developing new therapies or a cure requires testing medications in clinical trials. A majority of Alphas polled by the FDA said they were willing to take part in the trials if it would help find a cure.

"The Alpha-1 community is pleased to present a representative perception of the impact of Alpha-1 on our lives, to discuss therapeutic options, and to share our position regarding our participation in clinical trials," John Walsh, co-founder and CEO of the Alpha-1 Foundation, said at the Sept. 29 meeting. "We look forward to participating in the next phase of patient-focused drug development with the FDA because the end goal of drug development should be that the patient is engaged from day one through post-market. Nothing about us is without us."

In addition to the people who filled the room at the FDA headquarters, another 600 participated via a live webcast that offered a chance to comment or ask questions.

"What bothers me most is my loss of independence," said Charlotte Mattison, one of 12 panelists named by the FDA for the meeting. "Everything takes me longer, from doing the laundry to family functions. And oxygen equipment is often heavy and expensive."

Mattison was among many Alphas who initially got a very misinformed prognosis. A pulmonologist diagnosed her Alpha-1 lung disease and told her she had just two years to live. But today, at 71, Mattison has outlived that dire warning by many years.

Gordon Cadwgan, chair of the Alpha-1 Foundation, pointed to a major problem for Alphas: "Most of us, like me, have been forced to give up our jobs and go on disability."

Said another Alpha, "I miss being able to travel, mow the lawn, do the gardening, do routine maintenance on the house, and having to go to the hassle and expense of having others do this for me."

Although there is no cure for Alpha-1, there is a therapy to help slow the loss of lung function for Alphas with lung disease. However, there is no therapy for Alpha-1 related liver disease. When the disease is life threatening, a liver transplant is the only option. Liver-affected Alphas at the meeting stressed the importance of a realistic clinical trial design to help accelerate the review and approval of therapies currently in development and in trials in Australia and Europe.

Ross Pierce, MD, medical officer, Division of Hematology Clinical Review with the FDA, discussed the background of Alpha-1 and augmentation therapy for Alpha-1 lung disease. In his conclusions he echoed the opinions of many Alphas at the meeting: "Additional therapies need to be developed" to meet the needs of Alpha-1 patients, he said.

The FDA has opened a 60-day commentary period, ending Nov. 30, inviting public comments on Patient-Focused Drug Development for Alpha-1. Comments can be posted at http://1.usa.gov/1LU2Kbj.

About the Alpha-1 Foundation: The Alpha-1 Foundation is committed to finding a cure for Alpha-1 Antitrypsin Deficiency and to improving the lives of people affected by Alpha-1 worldwide. For more information, visit www.alpha1.org.

For information contact:
Bob Campbell
Director of Communications
Email 
305-567-9888 Ext. 230  

SOURCE Alpha-1 Foundation