ANGELIA BEARDS MEET PATIENT ADVOCATE NEW ALPHA-1 TREATMENT AND POTENTIAL CURE ON THE HORIZON? CREATING A NEW MODEL FOR ORGAN TRANSPLANTS

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1 Issue 31 June 2014 NEW ALPHA-1 TREATMENT AND POTENTIAL CURE ON THE HORIZON? CREATING A NEW MODEL FOR ORGAN TRANSPLANTS MEET PATIENT ADVOCATE ANGELIA BEARDS A PUBLICATION OF:

2 JUNE 2014 ISSUE NO. 31 TABLE OF CONTENTS 4 MEET PATIENT ADVOCATE ANGELIA BEARDS Angelia Beards is Coram s newest Patient Advocate. Read about how she is uniquely qualified for this position and why she says, This is who I am through and through. 7 NEW ALPHA-1 TREATMENT AND POTENTIAL CURE ON THE HORIZON? Dr. Ron Crystal is one of the leading researchers of Alpha-1 and originally developed the current Alpha-1 augmentation therapy in Discover what Dr. Crystal and his team are doing to take the next step in Alpha-1 treatment and a potential cure. 10 CREATING A NEW MODEL FOR ORGAN TRANSPLANTS Organ donors and their families are heroes. One donor s organs and tissue can save numerous lives, but the process to retrieve donated organs is very complicated. Read about how one organ procurement organization in Missouri has decided to create a new model for organ retrieval. The Alpha-1 Advocate, brought to you by Coram CVS/specialty infusion services, is a publication that is provided free of charge to the Alpha-1 community. Opinions expressed by contributing authors and sources are not necessarily those of the publisher. Information contained in this magazine is for educational purposes only and is not intended as a substitute for medical or billing advice. Do not use this information to diagnose or treat a health problem or disease without consulting a qualified physician. Consult a physician or appropriate clinical professional before starting or modifying any course of supplementation or treatment. Never disregard medical advice or delay in seeking it because of something you have read in this magazine Coram LLC. All rights reserved. No part of this publication be distributed, reprinted or photocopied without prior written permission of copyright owner. All service marks, trademarks and trade names presented or referred to in this magazine are the property of their respective owners.

3 Clinical Editor Lou Anne Epperson Managing Editor Allison Pizzuti Contributing Writer Valerie Hansen Graphic Design Wade Hiner We welcome your comments, stories, and suggestions. All correspondence, changes of address, and subscription inquiries should be sent to: Alpha-1 Advocate Coram CVS/specialty infusion services th Street, Suite 1500 Denver, CO alpha1source.com 2014 Coram LLC COR

4 PATIENT ADVOCATE ANGELIA BEARDS By Valerie Hansen Contributing Writer Coram s newest advocate for Alpha-1 patients, Angelia Beards, is uniquely qualified for the job. Not only does she have seven years of experience as a medical financial counselor, she is currently pursuing a degree in social work. I think what appealed to me most about Coram was the Patient Advocate position description, Angelia says. It is more of a case management role. As a financial counselor I dealt with the financial issues, insurance and such. I can bring that experience here, but now I m more involved with the care of the patient. During her first few months at Coram, Angelia focused on learning as much as possible about Alpha-1, so she could properly counsel her patients on both their financial needs and their therapy and medication needs. Alpha-1 is a complicated disease, and Angelia is still learning new things every day. However she is now fully capable of helping patients with their day-to-day questions. I recently had a patient who was having trouble keeping his treatment regimen straight. He was very sick. Together, we made a plan, and I told him, This is what you need to do, and I will follow up with you on Tuesday. I think giving him a structure and staying in contact really helped him. Angelia knew that this particular patient was getting close to his last days, and she did not waver from helping him walk through the steps he needed to take each day. For Angelia, one of the most important aspects of helping her patients along their journey is making sure they know they have a voice in their care. 4 MEET PATIENT ADVOCATE ANGELIA BEARDS

5 This is who I am through and through. - Angelia Beards I want them to know they have options. They absolutely can advocate for themselves and decide what s best for them, she says. Many times, patients will walk away from a doctor visit confused. They feel like, well, this is the doctor or the nurse and they know more than me. They don t understand that they have the right to ask questions about their medical concerns. Another aspect of the Patient Advocate job that is rewarding to Angelia is the ability to relieve some of her patients stress. She understands that many of her patients don t feel well physically. The last thing they need is to be concerned about administrative or insurance and billing issues. If I can make a patient s day better by doing the administrative piece, it makes me feel great. When I walk away at the end of the day, I think about the patient who didn t understand their benefits and I was able to explain it to them. Or I think about the patient who was having an issue with a bill, and I was able to connect them with someone who could better help them. Seeing patients who are struggling with illness demonstrate such resilience and such a positive attitude has brought Angelia a new appreciation for her own health and family. Their outlook on life is so enlightening, she says. I think that s what I have learned the most so far not to take for granted being able to breathe or having full use of my limbs. Angelia is looking forward to attending her first Alpha-1 Foundation Education Day. She is excited not only to learn more about Alpha-1 from the speakers, but to meet some of her patients and their families. With her new position as a Coram Patient Advocate, Angelia feels like she has found her calling. This is who I am through and through, she says. I am an advocate, a social worker. I want to make it better. ALPHA-1 ADVOCATE JUNE

6 NEW ALPHA-1 TREATMENT AND A POTENTIAL CURE ON THE HORIZON? By Valerie Hansen Contributing Writer Dr. Ron Crystal is one of the leading researchers in the area of Alpha-1 and COPD in the country. In fact, while he was the head of the National Heart, Lung and Blood Institute at the National Institutes of Health, Dr. Crystal and his team developed for the current Alpha-1 Augmentation Therapy (AAT). With this current form of augmentation therapy, the deficient Alpha-1 protein is purified from plasma. It is then sent into the body via an IV to bring the body s level of the protein back to normal. However, the added protein only lasts four or five days. Therefore, infusions are required on a weekly or bi-weekly basis. During the time Dr. Crystal and his team developed the intravenous therapy, they also developed an inhaled version of therapy. This particular therapy is currently in a clinical trial in Europe and Canada, and the trial is nearing completion. The company that plans to market the therapy, Kamada, Ltd., expects the complete trial information to be released at the American Thoracic Society meeting in May CURING ALPHA-1? Now, as chairman and professor of the Department of Genetic Medicine at Cornell University s Weill Medical College in New York, Dr. Crystal has been conducting research for a genetic cure for Alpha-1. The concept is not a temporary replacement of Alpha-1 Antitrypsin in the blood. Instead, it introduces a gene into the body that will produce the deficient protein. Gene therapy works by replacing a defective gene with a healthy one. In the case of Alpha-1, that would be a gene that produces the Alpha-1 Antitrypsin protein. Dr. Crystal and his team are currently testing on animals, and have received good results. 6 NEW ALPHA-1 TREATMENT AND POTENTIAL CURE ON THE HORIZON?

7 Because the Alpha-1 protein is widespread in the body, the team has been trying to determine how much of the protein needs to be produced by the gene to sustain normal levels. The strategy Dr. Crystal and his team have developed over the last several years is to inject a harmless virus called an adeno [gland]-associated virus containing the normal Alpha-1 gene into the pleura. (The pleura is the space between the lungs and the chest wall.) The virus delivers the protein-producing gene into the lung. Even though the deficiency actually starts in the liver, the Alpha-1 gene could be reproduced by any cells in the body. Dr. Crystal expects to begin clinical trials this year to see if the genetic cure will work on humans as well as it has worked on animals. The most important part of the testing was done first making sure that the genetic therapy is safe. The researchers have determined that it is. The next step is moving into human trials to find out how much of the genetically delivered protein is needed to correct the production of Alpha-1 Antitrypsin. The promising gene therapy is an outpatient, onetime process and is an actual cure for people with Alpha-1. Alpha-1 Antitrypsin Deficiency was discovered in Dr. Crystal and his team developed a treatment within 20 years of the discovery, and now may actually be on the brink of a cure. That is lightning fast in the research world and terrific news for the Alpha-1 community. ALPHA-1 ADVOCATE JUNE

8 Creating a New Model for Organ Transplants By Valerie Hansen Contributing Writer Organ donors and their families are heroes. One donor s organs and tissue can save numerous lives, including those of people with Alpha-1 who are waiting for lung or liver transplants. But retrieving organs from these donors is a complicated process involving teams of surgeons, lab testing equipment and many other factors. 8 CREATING A NEW MODEL FOR ORGAN TRANSPLANTS

9 Only a handful of hospitals in major cities across the country are equipped to do transplants. Therefore, surgical teams typically have to travel to the donor s location in order to retrieve the organs. In 2001, Mid-America Transplant Services, an organ procurement organization (OPO) that covers most of Missouri and a small part of Illinois and Arkansas, created a new model for organ retrieval. They opened a centralized facility specifically designed to access organs from donors. Equipped with all the necessary surgical and testing tools, as well as a trained staff, the center is easily accessible to the four major hospitals in St. Louis that handle transplants. There is no comparison between the disorganization and confusion of retrieving organs in a hospital setting and how it is done in our facility, says Dr. Maria Majella Doyle, author of a study on liver transplants recently published in the American Journal of Transplantation. It is difficult even in our big hospital [Barnes Jewish Hospital], let alone in a small community hospital that have performed one donor recovery in the last five years. The staff is working with up to four teams of surgeons, none of whom they know. They don t necessarily have the right instruments. The person on the operating table may often be a relative of someone who works in the hospital in these small towns. It s extremely stressful for the staff, who are not used to the recovery procedure. The process can become even more complicated if a trauma patient comes into the hospital in the middle of the retrieval. Small hospitals may only have one operating room available in the middle of the night and limited staff. The living patient is the priority, so the organ donor must be put on hold while the staff attends to the emergency. The implication of that, says Dr. Doyle, is that the five or six recipients waiting for the organs and the teams in place at the transplant hospital are left waiting three or four, even six hours, until the donor recovery team can do their work. Donated organs have a limited shelf life. For livers, the time period is six to ten hours, and hearts and lungs have even less time than that. To make the best use of the time they have, the facility has two operating rooms and all the necessary testing equipment. The staff there can really monitor the organ function, says Dr. Doyle. We can do all the things we need to do to maximize the organs. We transplant more of these organs as a result. The facility has increased the number of organ donors they receive from the region from 20% when they opened in 2001 to 92% of all organ donors and 98% of liver donors. According to statistics from the ALPHA-1 ADVOCATE JUNE

10 Organ Procurement and Transplantation Network (OPTN), 28,952 transplants were performed in 2013, and 121,832 people are currently on the waiting list. The national average of organ usage is 2.7 organs per donor, Dr. Doyle says. At our center, the average is 3.77 organs, so it is an entire organ higher. Not only has the center increased the number of organs retrieved for transplantation, but the organs are healthier, and the transportation time to the four hospitals is a matter of minutes. Also, the cost is less because the OPO is not paying the donor hospital charges. How OPOs Work Every area of the country has an independent OPO, and each OPO has its own arrangement with both retrieval hospitals and transplant hospitals. A few of these OPOs are following the Mid-America model of a centralized facility, including those in Michigan, Colorado and Pennsylvania. The benefit to having regional OPOs work independently is that they do not have to receive approval at a national level. While the United Network for Organ Sharing (UNOS) is the international organization for organ donation, it is not involved in directing each independent OPO. Our facility has reduced liver recovery costs by 35%, Dr. Doyle says. We are starting to look at all the different organs. We project the overall savings per donor is going to be close to 45%. That is because, although there is cost in running the facility, we don t pay hospital charges to retrieve organs. OPOs pay different hospital charges in different areas of the country. on life support back to St. Louis. When the organs have been removed, the body is sent to a location determined by the family, such as a funeral home. The donors and their families are treated with the utmost respect, Dr. Doyle says. Donor families are informed every step along the way. They are notified when the body arrives at the center. They are then told the results of the transplantation process including how many people their loved one has saved. Every summer, the Mid-America OPO hosts an event for donor families and recipients. We all hold candles up during a service and then people from the donor families are able to speak, Dr. Doyle says. Everybody mingles and talks to each other. All the transplant recipients are invited. It s really an amazing time to talk to the families and hear how proud they are that their loved one saved seven people s lives or five people or three people. It s a process that is very rewarding for the families, no matter how traumatic it was at the time. While the recipients never know the specifics of who donated the organ that saved their life, they are allowed to send a letter to the family through the OPO a couple of years after the transplant. Even though many areas of the country are not as fortunate as Mid-America to have four transplant hospitals within minutes of their centralized facility, more OPOs are looking at their model. The cost savings, increased efficiency, and number of organs used for transplants are worth the consideration. Dr. Doyle feels that their facility may be a model not just the U.S., but all over the world. The Families The top priority for every OPO is treating the donor family with respect and compassion. At the Mid- America facility, two members of the staff travel to the donor s hospital and transport the body 10 CREATING A NEW MODEL FOR ORGAN TRANSPLANTS

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12 Advocate Alpha-1 News and Information for the Alpha-1 Community The Alpha-1 Advocate is a publication of Coram CVS/specialty infusion services CORAM ( ) alpha1source.com youtube.com/coramhealthcare 2014 Coram LLC COR