Legislative News 3

Patient News 4

Philanthropy News 6

Medical News 10

Chapter News 12

Board News 14

Cooley’s Anemia FoundationL e a d i n g t h e F i g h t A g a i n s t T h a l a s s e m i a

CAF Partners with NYAS on Important Symposium

• Patients Speak to Rapt Legislators

• Protect Your Heart and Liver with T2* and Squid Testing • In Memoriam • TAG Conference • CDC Blood Safety and Surveillance Program

• Gala a Springtime Hit • Greek Community Opens Heart to CAF • OSIA Holds Dine-Around • CAF’s 2004 Cigar Night • DVD Benefits CAF • Elks Welcome CAF • Open Letter from CAF President

• CAF Brochures Available in Various Languages • TCRN Update • Medical Research Fellowships

• Chapters News • Chapter Contact List

• Joseph Sciame Honored • Tribute to Ralph Cazzetta • New Board Members

• Dr Cohen Honored by Board

contents

LifelineSeptember 2004

The Cooley's Anemia Foundation is collaborating with the prestigious New York Academy of Sciences (NYAS) to present the "Eighth Cooley's AnemiaSymposium" at the Hilton Hotel in the Walt Disney World Resort in Orlando, FL,March 17 - March 19, 2005.The goal of the symposium is thepresentation and dissemination of currentfindings and the setting of future directionsregarding basic and clinical research in thalassemia. The program will addressrecent advances in:

1. The natural history of thalassemia worldwide

2. Gene regulation and gene therapy3. Mechanisms of cell injury of iron

overload in thalassemia4. Development and therapy of iron

chelators

5. Stem cell transplantation6. New therapies for thalassemia7. The adult thalassemia patient8. Non-invasive iron measurements

The symposium will integrate basic science and clinical research so thatrepresentatives of both of these disciplines,as well as clinicians, can develop a mutualunderstanding of recent progress in thalassemia. Conference attendeesshould achieve an understanding of themolecular biology, pathophysiology,epidemiology, clinical care and psychosocialmanagement of thalassemic disorders. A combination of keynote addresses, state-of-the art lectures that provide an overview of progress in different aspects of thalassemia, oral presentation of recent findings by leading investigators,and poster presentations will provide

a comprehensive overview of progress in the understanding and treatment of thalassemia.

CAF and NYAS have collaborated on theseven previous Cooley's Anemia Symposia,beginning in 1964. As with previousconferences, the proceedings will bepublished as a volume in the series Annalsof the New York Academy of Sciences.

In related news, the Thalassemia ActionGroup (TAG), CAF’s patient-run supportgroup, is considering scheduling its 2005Patient/Parent Conference to coincide with the symposium, allowing patients and parents to partake in both of theseimportant events. Details will be announcedas they become available.

To date, major funding for the symposium is being provided by the National Heart,Lung & Blood Institute; the National Instituteof Diabetes and Digestive and KidneyDiseases; Novartis Pharmaceuticals; and Apotex.

For more information, contact Eva Chin at eva.chin@cooleysanemia.org.

National Executive Director’s Message

These messages can be enormously difficultfor me as I vacillate between evangelisticzeal and sober compassion. I try to alwayskeep in mind that you may be a sibling or parent of a youngster looking for goodinformation and a bit of hope. You may alsobe carrying the burden of loving someonewhose health is failing now and looking forstrength and understanding.

If I could tailor this message to youspecifically I would. Instead I ask each of you to understand our community at large, to understand that yours is not theonly story. There are patients whose healthis failing and patients who are havingbabies. There are patients declining as theyignore their pumps and patients getting the good news that their dedicated use of the pump has paid off.

statement of purpose

I remember an old television show thatbegan with a line something like: “There area million stories in this city.” That’s how I feel about our community. We all have ourown story. We all have our own issues.What we have in common though, is theneed for a collaborative effort and collective prayer.

There are great things before us in themedical world. Gene therapy is progressing,blood safety is improving, and the promiseof an oral chelator may finally be fulfilled.CAF has been at the forefront of all ofthese wonderful efforts. Our staff does a stellar job, but still a lot of what we accomplish comes from our volunteerranks…you and me.

So as I said at the top of this message, the evangelist in me is pacing back and

“T2*.” “SQUID.” “Ferritometer.”These strange terms – which you’ll findelsewhere in this issue – are familiar to those of us at CAF. They refer to variousinvestigational technologies that are beingused in research projects to non-invasivelymeasure iron concentrations in the heartand liver. These technologies are availablefree of charge to thalassemia patients at selected locations – and we urge patients to discuss the need for iron measurementswith their doctors and then makearrangements to take advantage of thesetechnologies. (See page 4.)

Having an alternative to an invasiveprocedure such as a biopsy is important forpeople with thalassemia. But it may

The Cooley’s Anemia Foundation, incorporated in 1954, is the only national,nonprofit health organization dedicated to serving patients afflicted with variousforms of thalassemia, most notably the major form of this genetic blood disease,Cooley’s anemia.

the foundation’s mission• Advancing the treatment and cure for this fatal blood disease.

• Enhancing the quality of life of patients.

• Educating the medical profession, thalassemia trait carriers and the public

about thalassemia/Cooley’s anemia.

President’s Message

Reproduction of material published in Lifeline for educational purposes is encouraged, provided it is accompanied by the following attribution: according to Lifeline, the national newsletter of the Cooley’s Anemia Foundation.2

EXECUTIVE COMMITTEE

Frank Somma PresidentAmy Celento 1st Vice PresidentRalph Colasanti TreasurerRobert Yamashita Secretary

Cammie Brandofino Diane KambleNunzio Cazzetta Richard MancinoPeter Chieco Gargi PahujaTerri DiFilippo Concetta ParadisoRobert Ficarra Elliott Vichinsky, MDJoseph Giammalvo Anthony J. Viola

Jayne Restivo National Executive DirectorCraig Butler Lifeline Editor

Frank Somma

Jayne Restivo

CAF : 129-09 26th Avenue, Suite 203, Flushing, NY 11354

(800) 522-7222 (718) 321-CURE (2873) (718) 321-3340 FAX www.cooleysanemia.org

be important to many more people, who will one day benefit from currentthalassemia research.

For example, people with hemochromatosis– an inherited disorder that is estimated to affect up to 1 in 200 Americans – also have iron overload issues. If the studies involving these new technologies areeventually approved for standard use,doctors will be able to better assess damage to the organs of people withhemochromatosis and to recommendtreatment to reverse or stem that damage.The same holds true for people with otherdiseases in which iron overload is a factor,such as African siderosis or sideroblasticanemia.

l e g i s l a t i v e n e w s

Appearing March 30 before an importantgovernment committee, representativesfrom CAF once again made a significantimpression upon important elected officialsas they requested continued governmentsupport in the fight against thalassemia.

Alicia Somma, 19, and Michael Giammalvo,13, who have thalassemia, addressed a meeting of the influential HouseSubcommittee on Labor, Health and Human Services and Education, chaired by Congressman Ralph Regula (R-OH).Congresswoman Rosa DeLauro (D-CT)introduced Alicia and Michael, describingthem as “brave and courageous.” Michael spoke about treatment forthalassemia, stating that “getting a bloodtransfusion (every two weeks) is not fun,but I have to do it to stay alive,” anddescribed the difficulty involved with nightlychelation treatments.

Alicia went into greater detail about theproblems associated with chelation therapyand spoke eloquently about a friend whohad recently passed away because of hisinability to maintain the difficult treatment.She urged the committee to recommendcontinued funding for programs that benefitpeople with thalassemia, adding that “we would not ask this of our government if it were not so important” and telling thecommittee that she and Michael werespeaking on behalf of their deceased friend“because he can’t be here to speak forhimself.”

The committee members, many of whom

were visibly moved by what they heard,responded positively to Michael and Alicia’stestimony.

“I really appreciated the opportunity to speak my mind,” Alicia said afterward. “I had been there before, but I was excitedto have this amazing experience again.I wish all of the patients could do this –

it’s great to be heard, and it makes you feelreally important. When all eyes are on youand you’re talking about the most importantthing in your life and you’re admitting thatyou need help, you may think that wouldmake you feel weak – but in fact you feelstrong. You know that you were able to askfor help for all of your peers, and it makesyou really excited.”

Although Alicia was nervous about speakingin public about her friend, she felt that it was important to do so. She alsomentioned that she and Michael receivedpositive feedback, and that CongresswomanDeLauro told them that their testimony hadbeen very effective. “She has always beenextremely supportive of CAF,” Alicia said of the congresswoman.

Among the thalassemia-related programsthat receive federal funding are theThalassemia Clinical Research Network(TCRN); the Centers for Disease Controland Prevention’s thalassemia initiatives; and several National Institutes of Healthresearch projects.

CAF thanks Michael and Alicia andcongratulates them on doing a splendid job.

3Cooley’s Anemia Foundation

Patients Speak to Rapt Legislators

Other areas of thalassemia research alsohave great potential for the population at large, including the landmark genetherapy work of Dr. Michel Sadelain and Dr.Stefano Rivella. While their studies focus onthe elimination of thalassemia, they haveimplications for many other diseases.

For decades, CAF has made medicalresearch a priority. We are proud that ourefforts, both direct and indirect, have bornefruit – and that the results of that researchhave a benefit that extends beyond our ownpatients to people around the world.

Jayne Restivo

For up-to-the-minute news about thalassemia-relatedissues, visit our website,www.cooleysanemia.org

forth and waving his arms, imploring you to volunteer. If you don’t know what to do,call us and we will introduce you to yourlocal chapter or describe the various areasof need.

The sober, compassionate Dad in me is holding your hand, praying and assuringyou that you have done everything possibleto maintain the health of your loved one andletting you know that in the end, what we allhave to cling to is our faith – our faith in God and our faith that tomorrow will be better than today through our efforts and our prayers.

May God bless you and your family.

Frank SommaAlicia Somma, Michael Giammalvo

4 Cooley’s Anemia Foundation

p a t i e n t n e w s

In MemoriamWe regretfully report the loss of

Cooley’s anemia patients

Todd BeckerLisa Cammilleri

Rosemarie Cavileri-WellmannRalph Cazzetta

Vivian Giordano LaGrecaNgoc "Tina" Tran

Evangeline Zeidman

and extend our sympathies to their friends and family.

The Cooley’s Anemia Foundation wants to take this opportunity to bring to yourattention some exciting new informationabout approaches to the management of iron overload and heart disease in thalassemia. New techniques areavailable to safely measure iron stores andevidence now shows that heart disease in people with thalassemia can beprevented and even reversed withimproved iron chelation.

Heart disease is the number one cause of death in thalassemia. Its prevention and treatment require a team approachbetween you, your doctor and a thalassemia center. Early detection of cardiac disease will result in preventingprogression if effective treatment is started. Even if cardiac disease existsand is severe, it can be reversed or stabilized. If a thalassemia patientalready has rhythm problems or heartfailure from iron overload, there are waysto slow down or stop the damage while the iron is being removed from your heart.(This information is not commonly known by physicians who are not intimatelyinvolved with the management of patientswith cardiac iron overload.)

As we all know, Desferal is very effective at removing iron from the liver. It is alsovery effective at preventing cardiac rhythmproblems and cardiac failure that can occurbecause of iron overload.

Understanding a patient’s total body ironburden is essential to optimal managementof his/her care. New technology, such as the SQUID (Superconducting QuantumInterference Device, which measures liveriron) can replace liver biopsy in determiningyour liver iron. The SQUID is as reliable

as a liver biopsy and without risk. An MRI using specialized equipment (T2*)is showing promise as a method to measure heart iron levels. Thistechnique, while still being studied, may be extremely helpful in determining a patient’s course of treatment.

All thalassemia patients should undergoregular evaluations for body iron stores and cardiac disease. An annualechocardiogram is a good screening testfor heart disease. The T2* may detectpatients at high risk for heart diseasebefore the echocardiogram showsabnormalities. The SQUID or liver biopsymeasures how much iron is in the totalbody and can predict who may develophormone, heart and other problems. The results of both of these studies willenable one’s physician to offer treatmentdesigned especially for each patient.Continuous Desferal therapy usuallyreverses early heart disease. Deferiproneplus Desferal may also be an importantadvance in improving heart disease and is now actively being studied. We recommend that you discuss this withyour physician.

T2* testing is available for patients with a diagnosis of thalassemia major or intermedia who are 7 years of age or older. For safety reasons, patients whohave pacemakers are not eligible for T2*measurements, but Port-a-Caths, otherintravenous catheters and surgical clips are safe and do not affect the image. Because the T2* must be done in a closedMRI machine, those patients who sufferfrom claustrophobia may not feelcomfortable with the testing procedure.

Protect Your Heart and Liver withT2* and SQUID Testing

If you are a thalassemia patient or a parentof a child with thalassemia and areinterested in having the T2* measurement,first get permission from your hematologistand then contact one of the below namedindividuals to schedule an appointment.

Kelly WalshChildren’s Hospital of Boston(617) 355-2797

Cathleen EnriquezChildren’s Hospital Los Angeles(323) 660-2450, Ext. 3426

To schedule a SQUID test, please contact:

Eileen Scott at CAF(Test conducted at Columbia PresbyterianMedical Center, NYC)(800) 522-7222, Ext. 207

Lisa CalvelliChildren’s Hospital, Oakland(510) 428-3429

5Cooley’s Anemia Foundation

Over 150 patients, family members and medical professionals from across the country gathered in Illinois for the 18th Annual Thalassemia Action Group(TAG) Patient/Family Conference. HeldMay 14-16 at the Wyndham Hotel in Itasca(just outside of Chicago), the Conferenceincluded a Pump/PJ party on Friday night;a series of lectures and workshops

on Saturday; and a fun-filled dinner on Saturday night.

Highlights of the Conference included a nurse-run psychosocial session and a panel on alternative iron chelators. Dr. Charles Peterson, Director, Division of Blood Diseases and Resources at the

The Centers for Disease Control and Prevention (CDC) has initiated a program to ensure that thalassemiapatients have access to appropriateinformation, treatment and preventionservices. CAF has been assisting the CDCin the development of the ThalassemiaPrevention Education and OutreachProgram. A main component of this initiativeis the Thalassemia Blood Safety andSurveillance Program.

By enrolling in the Thalassemia BloodSafety and Surveillance Program, everythalassemia patient will be tested for severalblood borne infections FREE OF CHARGE.The CDC will “bank” a small amount of theserum from the confidential blood sample.The formation of a serum bank will allow forrapid response to emerging blood bornethreats in the thalassemia community.

CDC Program Update

Blood collection for the Thalassemia BloodSafety and Surveillance Program began on July 15, 2004. Since then, three of thesix CDC Thalassemia Centers of Excellencehave collected and shipped over onehundred samples of blood to the CDC fortesting. The CDC is expecting to receiveadditional blood samples from the threeremaining sites within the next couple of weeks. Patients who have not yetenrolled in the program are encouraged todo so by both CAF and the CDC.

To enroll in the program, patients can visitany of the six Thalassemia Centers of Excellence. CAF urges individuals withthalassemia and their physicians to establisha relationship with one of these centers, asthe centers can provide specializedtreatment for thalassemia patients acrossnumerous medical disciplines. Contact CAF at (800) 522-7222 to locate a thalassemia treatment center near you.

CDC BloodSafety andSurveillanceProgramby Jacqueline Lofters

National Heart, Lung and Blood Institute,was a special guest speaker, whodiscussed the work of the ThalassemiaClinical Research Network.

Three individuals were given recognition by TAG as honorees: Patricia Giardina, MD, Chief, Division of Pediatric Hematology and Oncology,New York Presbyterian Hospital/WeillCornell Medical Center; Dru Foote, RN,MSN, CPNP, Thalassemia ComprehensiveClinic, Children’s Hospital Oakland; and Susan Winner, a thalassemia patientand a longtime member of TAG.

TAG President Gargi Pahuja cites the hope generated by the panel on upcomingprospects in alternative chelators as an important outcome of the Conference.

“This Conference was great,” Gargi says,“and we hope it inspired the patients in theChicago area to be more informed and getinvolved.” She also thanked the membersof the Chicago chapter, especially Theresa Tomiano and Bruce Rod, for theirtremendous efforts.

Plans for next year’s Conference are stillbeing finalized, but it is tentativelyscheduled to coincide with the CAF/NewYork Academy of Sciences Symposium inOrlando, March 17-19, 2005.

TAG Conference Full of Information and Hope

Jesal Kapasi, Huythong Nguyen, Bruce Rod, Gargi Pahuja

Dr. Charles Peterson

The silent auction at CAF’s annual Gala always has something for everyone.

Among the 150 items up for bidding at the 2004 Gala were:

Incredible Hulk Comic autographed by creator Stan Lee

Breton Legend Churchill cigarsMolyvos Restaurant gift certificate

Tiffany & Co. cheese setNY Knicks signed basketball

Custom fit dress shirts from Joseph’s ClothiersMets/Yankees Subway Series tickets

Willie Mays photograph

If you have an item that you would like to donate for a Gala auction, please contact

Jo Ann Barbella at (800) 522-7222 ext 202.

6 Cooley’s Anemia Foundation

p h i l a n t h r o p y n e w s

More than 300 people turned out for CAF's annual Springtime in New York Galaat Tavern on the Green on April 21.

Hosts Gianna and Chazz Palminteripresided over a lively evening that includeddancing, dining, a wonderful live auctionand a silent auction that featured more than150 items. Guests included Joe Piscopo,WKTU's John "Goumba Johnny" Sialiano,Tony Lo Bianco, Michael Amante, Daniel Rodriguez and many others. As usual, members of Order Sons of Italyin America (OSIA) joined us for thecelebration, as did representatives from theAmerican Hellenic Educational ProgressiveAssociation (AHEPA).

Gala a Springtime Hit

CAF is fortunate that many of its celebrityguests offered to perform during this year’sGala, resulting in such crowd-pleasinghighlights as Joe Piscopo’s Sinatra-inspired“Theme from New York, New York” andMichael Amante’s dramatic “Delilah.”

The live auction provided another highlight,courtesy of the Palminteris, who had offeredto auction off a dinner for six at their ownhome. Reacting to overwhelming audienceresponse, the hosts ended up auctioningtwo such dinners, as well as a third forfifteen guests.

The festive event was a tremendousfinancial success, grossing over $200,000,

which will be used to aid CAF in itsongoing fight against thalassemia.

“As always,” says CAF National ExecutiveDirector Jayne Restivo, “CAF wasfortunate to have Gianna and ChazzPalminteri as the guiding lights behind theGala. Their dedication to making this thebest event possible is the reason thatpeople keep coming back year after year.”

The 2005 Gala is in the planning stages now. Call Jo Ann Barbella at (800) 522-7222 ext 202 to make sureyou receive an invitation.

Gianna Palminteri

Jayne Restivo, Basil Mossaidis (AHEPA), Olga Mossaidis, Frank Somma Paul S. Polo (OSIA)

Chazz Palminteri entertains Gala guests

sile

nt auction

7Cooley’s Anemia Foundation

The New York State Grand Lodge of OSIA(Order Sons of Italy in America) held an enormously successful “Dine Around”event to benefit CAF on April 5. About 220 people attended the “CulinaryClassico 2004,” which took place at Leonard’s of Great Neck and featuredfood from 28 different restaurants.

Jenine Abruzzo, a thalassemia patient andthe recent mother of a baby girl, addressedthe crowd. She thanked them for OSIA’ssteadfast support and talked about how the money that OSIA members give toCAF has brought about so many changes – including the opportunity for people withthalassemia to now raise families of theirown.

WABC radio personality Curtis Sliwa alsospoke to the crowd, reminding them of theimportance of getting the word out aboutthalassemia.

As one of the groups most affected bythalassemia, the Greek Americancommunity has long been an important partof CAF. Thus, the Foundation wishes toacknowledge several important newrelationships that have been made withmembers of the Hellenic community.

Anna Vissi, the Greek music superstar,recently used the occasion of her U.S. concert tour to spread informationabout thalassemia and CAF. Ms. Vissi’spowerful voice, heard on more than 45albums, has made her a singing sensation in Greece. She has achieved multi-platinumsales success, while her critical acclaim canbe gauged by her Greek Music Awards forBest Female Singer, Best Interpretation and Biggest Airplay Song.

Ms. Vissi, well known Greek singer LambisLivieratos and many members of their banddemonstrated their dedication to CAF by donating a check for $40,000 to CAF.

In addition, acclaimed jewelry designer Doris Panos is donating sales of her originaldesign, “The Angel of Cure,” a sterlingsilver angel pin, to CAF. This beautiful pieceof jewelry made its debut at the May 26Anna Vissi concert in New York City.It sells for a donation of $100; for moreinformation, contact Jo Ann Barbella at(800) 522-7222 ext 202.

Doris Panos’ distinctive style and versatilecreations have been rewarded with

Greek Community Opens Heart to CAF

OSIA Holds Dine-Around

CAF thanks Rae Lanzilotta, New YorkState OSIA’s Cooley’s AnemiaChairperson, Co-Chairperson Constantino Sagonas, and the entirecommittee for their excellent work.

Rae Lanzilotta

“

numerous awards; in July 2001, she wasnamed Designer of the Year by theWomen’s Jewelry Association of America.

CAF is enormously grateful to Anna Vissiand Doris Panos for their generosity,” said CAF National Executive Director Jayne Restivo. “We appreciate theirkindness and feel fortunate to count themas allies in our fight against thalassemia.”

Ms. Restivo also acknowledges theimportant role played by Lisa Kontogiannisof Worldwide Simulcasting in connectingCAF with Doris Panos, Anna Vissi andLambis Livieratos. Ms. Kontogiannis joinedthe CAF Board of Directors in May,generously donating even more of her timeand talents to the cause of fightingthalassemia.

CAF President Frank Somma and NationalExecutive Director Jayne Restivo wereamong the CAF members that attended the82nd Annual Supreme Convention of theAmerican Hellenic Educational ProgressiveAssociation (AHEPA), held July 18-25 in Miami. More than 2000 AHEPA membersgathered for this important event, and theCAF contingent made contact with a greatmany of them.

“It was a wonderful opportunity for CAF toconnect with this community,” Ms. Restivosays, “and I was delighted to learn that so many members of AHEPA are already so knowledgeable about thalassemia.”

“We firmly believe that by working togetherwith organizations such as the Cooley'sAnemia Foundation that our joint efforts willlead to a cure for this terrible disease,” saysBasil N. Mossaidis, Executive Director ofAHEPA. “We look forward tostrengtheningour relationship with the Foundation."

Although CAF has always made outreachefforts to the Greek community, this recentsurge of activity promises to establishstrong and lasting new ties.

“There’s an old Greek blessing – ‘Upontouching sand, may it turn to gold,’” saysMs. Restivo. “CAF feels blessed to havemade these new, golden friendships.”

Anna Vissi

WKTU (103.5 FM in New York City)morning man, Goumba Johnny (JohnSialiano) has released his first comedyshow on DVD , with a portion of theproceeds to benefit the Cooley's AnemiaFoundation.

Goumba Johnny’s “Slice of ItalianComedy,” an all-Italian comedy special,was taped at Caroline’s on Broadway lastyear and includes a line-up of starcomedians such as Rich Francese, Angela LaGreca, Tony Daro, Vic DiBitetto,and “Sex and the City’s” Mario Cantone.

8 Cooley’s Anemia Foundation

p h i l a n t h r o p y n e w s ( c o n t ’ d )

Elegant surroundings, flavorful dining, thefinest cigars – all topped off with one of themost stunning views of New York City.That’s what guests at CAF’s Annual CigarNight at the exclusive Grand Havana Room(date TBA) can expect.

Located on the 39th floor of 666 FifthAvenue, the view from the Grand HavanaRoom is nothing short of spectacular. Its floor-to-ceiling windows offer a 360degree view of Manhattan, including thespires of St. Patrick’s Cathedral, the SonyBuilding, Trump Tower and Central Park.Add to this deep mahogany paneling andexquisite blue velvet curtains, not to mention the delicious menu created by chef Alberto Gomez, and you can seewhy this is one of New York’s most sought-after locations for private parties – and whythis setting helps to make CAF’s AnnualCigar Night such a tremendous success.

The 2004 Cigar Night will once again be chaired by CAF Board member FrankFusaro. Fusaro, recipient of thedistinguished Arthur J. Dixon Public ServiceAward, is the co-founder and President of Forum Personnel, one of New York’sleading recruitment service organizations,and is also a prominent Board member of the Columbus Citizens Foundation.

It's Almost Here: CAF's 2004 Cigar Night

Fusaro is fortunate to be joined in his effortsby Ron Purpora, the Garban-Intercapitalexecutive who served as last year’shonoree. Purpora has generouslyvolunteered to work with Fusaro this year to help make the 2004 Cigar Night thebiggest yet.

The Cigar Night is truly a one-of-a-kindexperience that should not be missed.Tickets and more information are availableby calling 631-863-0532.

Elks WelcomeCAF

Representatives from CAF, includingPresident Frank Somma, attended theannual convention of the New Jersey StateElks Association (NJSEA), held June 3-6 in the Wildwoods section of New Jersey.

“This is a very important part of ouroutreach and fund raising efforts,” saysMr. Somma. "The New Jersey Elks havebeen supportive of our efforts on behalf of people with thalassemia, and we wantedto thank them and to introduce CAF to those members who do not know aboutus or thalassemia."

More than 41,000 men and women belong to NJSEA, and they were wellrepresented at the annual Conference. “It was a pleasure attending theConference and getting to meet so manycaring, interested people,” Mr. Sommasays. “Even those who had never beforeheard of thalassemia took an immediateinterest in our work. They were visiblytouched and concerned when they heardabout the challenges that people with thalassemia face.”

The Benevolent and Protective Order of Elks of the United States of Americawas founded in 1868, and the New Jerseyassociation was founded in 1913. The Elks are dedicated to the ideals of charity and patriotism; last year, the NJ Elks donated $5,000 to CAF.

CAF thanks NJSEA Past NationalPresident Arthur Mayer and the membersof the Belmar Lodge for their efforts in securing this donation.

Comedy DVD Benefits CAFBonus featureson the DVD includeover 2 hours ofnever-before-seenfootage of the castand crew and an 8 page color insert with individual biographies and celebrityphotographs. To order the DVD call 866-4GOUMBA.

For more information, check outwww.goumbajohnny.com/goumbasoicdvd.htmor contact Robert Vinci Productions at 718-842-1606.

Frank Fusaro, Ron Purpora

9Cooley’s Anemia Foundation

Dear Friend:

Too many parents of children with Cooley’s anemia know the unendurableheartbreak of burying their sons and daughters. It’s something that noparent should ever go through, and we need your help to put an end to it.

Although I can thankfully say that my own daughter is alive and well (so far), despite her Cooley’s anemia, I have shared in the grief of manyother parents not so fortunate. I have added to the tears – tears beyondmeasuring – that burst forth when a child is taken so young and so full of promise.

But, like so many others affected by Cooley’s anemia, I refuse to give in to the tragedy. I know that there is tremendous reason for hope. I knowthat, even as I am writing this, respected scientists are doing importantwork in gene therapy that WILL one day lead to a cure.

I know that new drugs are in development, and that there is every reasonto believe that SOON there will be alternative therapies available thathold the promise of keeping patients alive until that cure is found.

I know that someday the blood transfusions every two weeks, the nightlydrug treatment that takes twelve hours, the constant monitoring of theheart and liver and worrying about transfusion-related infections like HIV or hepatitis – that all this will be a thing of the past.

The question isn’t “Will it happen?” The question is “When will ithappen?”

We don’t know the answer. But we do know that if you will help, it willhappen that much sooner.

Won’t you please aid all who suffer from Cooley’s anemia by returningthe enclosed form with a generous tax-deductible donation?

The silence in a home that was once filled with joy is beyond imagining.Please help us keep these homes filled with laughter.

Thank you.

Sincerely,

Frank SommaNational President

An Open Letter from the CAF President

10 Cooley’s Anemia Foundation

m e d i c a l n e w s

Funded by the National Heart, Lung andBlood Institute, the Thalassemia ClinicalResearch Network (TCRN) brings togethermedical professionals from the five majorthalassemia treatment centers in the United States and Canda to evaluatecurrent and future therapies for thetreatment of thalassemia.

Four multi-center TCRN clinical studies are now being conducted with thalassemiapatients in North America and the United Kingdom. These include:

Registry - In order to participate in otherTCRN studies, patients must first be

entered into the Registry. Enrollment in theRegistry began in June 2001, and over 800patients at 22 sites have been enrolled.

The Low Bone Mass Cross-SectionalStudy (LBMCS) - an observational studydesigned to estimate the prevalence of lowbone mass among patients withthalassemia and to evaluate various factorsin bone metabolism in hopes of identifyingpreventative strategies and effectivetreatment regimens.

Hepatitis C Clinical Trial - designed to evaluate the safety of peginterferon alfa-2a and ribavirin for the treatment of hepatitis C.

The Mitochondrial Ancillary Study - anancillary study to the Novartis ICL670 107Trial. The purpose of this study is toinvestigate the response of biomarkers ofoxidative stress-induced injury andmitochondrial function in beta-thalassemiapatients treated with ICL670 and Desferal(DFO). Effects of iron burden, inflammation

TCRN Update

Under the Thalassemia PreventionEducation and Outreach program,created as a collaborative agreementbetween CAF and the Centers for Disease Control and Prevention (CDC), a number of new pieces of valuable informational material have been created in a number of languages. This is part of theprogram’s efforts to reach out to currently underserved communities inwhich thalassemia is disproportionatelyrepresented.

CAF Brochures Availablein Various Languages

Following is a list of currently availablematerial. If you are interested in obtainingcopies for your own education or fordistribution to a wider audience, please contact Jacqueline Lofters at (800) 522-7222 ext 208 or j.lofters@cooleysanemia.org.

Have You Been Tested for BetaThalassemia Trait?Available in English, Cambodian, Chinese,Greek, Spanish or Vietnamese

Have You Been Tested for AlphaThalassemia Trait?Available in English, Cambodian, Chinese,Korean or Vietnamese

What is Hemoglobin E?Available in English, Cambodian, Chinese,or Vietnamese

What is Hemoglobin H?Available in English, Cambodian, Chinese,or Vietnamese

and antioxidant use will also be studied.

Future studies include a Cardiac L1/DFOStudy, a randomized, placebo-controlledstudy comparing DFO alone vs. DFO andL1 given in combination to patients who are at risk of heart failure. The study will be submitted to the Food and DrugAdministration (FDA) late this summer andit is hoped the study will start late in 2004.

Originally funded for 5 years in 2000, theTCRN now faces a process called grantrenewal. To continue this work, the TCRNmust be refunded as of July 2005. TheTCRN is funded by the National Heart Lungand Blood Institute (NHLBI) via acooperative agreement which allows manyinstitutions to perform researchcooperatively. The TCRN is currentlywriting the renewal application which willdetail the research to be pursued within theNetwork. Look for details about proposedresearch in future CAF newsletters.

11Cooley’s Anemia Foundation

The Cooley’s Anemia Foundation (CAF) has awarded 2004-2005 medical researchfellowships totaling $440,000 to elevenscientists conducting research intothalassemia or complications associatedwith thalassemia. CAF funds the two-yearfellowships at $40,000 per year.

Five of the eleven recipients are in thesecond year of their two-year fellowships;six are first year recipients.

“CAF recognizes the crucial importance of medical research to the thalassemiacommunity,” said CAF National ExecutiveDirector Jayne Restivo. “There can be no advance in cure, no steps toward a cure,without proper and adequate funding for research.”

Dr. Elliott Vichinsky, who chairs CAF’sMedical Research Fellowship ReviewCommittee, said that “the Committee feelsthat the quality of applicants this year wasexceptional. Each of the recipientscombines talent with vision, and I lookforward to seeing what each of theseindividuals produces in both the short and the long term. ”

CAF fellows who are beginning the secondyear of their two-year fellowships include:

Tapasree Goswami, PhD, of HarvardMedical School Children’s Hospital,researching Iron Overload in Cooley’sAnemia: Characterisation of Subcellular IronDeposition in Terms of Ultrastructure andMolecular Mechanisms of Regulation

Anping Han, MD, of Harvard-MIT Divisionof Health Sciences and Technology,researching Modification of the Severity of Cooley’s Anemia by Heme-RegulatedeIF-2a Kinase

Kitai Kim, PhD, of Whitehead Institute for Biomedical Research, researchingCombined Gene and Cell Therapy in a Mouse Model of Beta Thalassemia

Stefano Rivella, PhD, of Children’s BloodFoundation Laboratories, researching Gene Therapy for Cooley’s Anemia in a New Mouse Model

Jim Vadolas, PhD, of the MurdochChildrens Research Institute in Australia,researching Correction of Aberrant Splicingwith Antisense Oligonucleotides for theTherapy of Beta Thalassemia

CAF fellows beginning the first year of their two-year fellowships include:

Bogdan Dumitriu, MD, of Lerner ResearchInstitute, researching Role of theTranscription Factor Sox6 in Erythropoiesis

Xinfu Jiao, PhD, of Cell Biology & Neuroscience at Rutgers University,researching Stablization of beta-globin mRNA Introduced into Cells

Ashutosh Lal, MD, of Children’s HospitalOakland Research Institute, researchingReversal of Oxidative Damage from IronOverload with Alpha-Lipoic Acid

Xiaofeng Liu, PhD, of Children’s Hospitaland Research Center at Oakland,researching Manipulating Ferritin Pores for Improved Iron Chelation

Thipparat Suwanmanee, PhD, of UNC-Lineberger Comprehensive Cancer Center,researching Repair of Thalassemic beta-globin pre-Mrna by AntisenseLentiviral Vectors in Cell Culture and in Animal Model of Thalassemia

Suiping Zhou, MD/PhD, of Children’sHospital of Philadelphia, researching The Role of Alpha Hemoglobin StabilizingProtein (AHSP) in Beta Thalassemia

CAF congratulates all of its fellows andwishes them success in their endeavors.

Medical Research Fellowships

From Top: Xinfu Jiao, Jim Vadolas,

Tapasree Goswami and Kitai Kim

c h a p t e r n e w s

12 Cooley’s Anemia Foundation

California Chapter

The California chapter held its 2004 thalasemmiaconference on June 26th at Knott's Berry Farm.The panel included Dr. Thomas Coates, Susan Carson, RN and Bryce Imbler, MSW fromChildrens Hospital Los Angeles, as well as Dr. Keith Quirolo and Dru Foote, RN fromChildrens Hospital Oakland (CHO).

The conference was successful in bringingpatients together with panel experts, giving them the latest information during a dynamic roundtable forum. In addition, brochures on CAF,CHO, Dreamstreet and Make-A-Wish Foundationwere distributed. The patients, family and chaptermembers all had a wonderful time gettinginvolved in the activities and a beautiful quiltmade by chapter secretary Pat Saia was donated and raffled.

The California chapter thanks all its membersand panel participants for making this event a success. The chapter hopes even more canjoin us at the event next year.

Florida Chapter

The Florida chapter had a wonderful evening at its first Bunco night on April 24, 2004. A great time was had by all, and the chapterlooks forward to holding its next event.

The Florida chapter had a booth at the Health & Wellness Expo on September 11 and 12 at the University of North Florida. Many peoplestopped by the booth to find out aboutthalassemia and to pick up information.

Long Island Chapter

The Long Island chapter's annual Dinner Dance is set for November 20th at Leonard's of Great Neck. Honorees include Giorgio Meriggi, Antonio D'Arcangelo andPasquale Cervera. For more information, contact the chapter at (631) 421-4223.

Massachusetts Chapter

The Massachusetts chapter will hold its 17thAnnual Dinner Dance on October 29 at 7:00p.m. at Foreign Legion Post 440 in Newton. Call 617-332-5952 for information.

New Jersey Chapter

March 12 saw a crowd of enthusiastic CAFsupporters enjoying "A Night at the Races,"courtesy of the New Jersey chapter. The event,held at Brooklake Country Club in Florham Park,NJ, was a great success and a tremendousamount of fun. The chapter wishes to express itsthanks to the Westchester-Rockland chapter fortheir help in planning this wonderful evening.

The New Jersey chapter's Annual Golf Classicwas held Monday, May 17 at the MinisceongoGolf Club, 110 Pomona Road in Pomona, New York. This exciting day includes 18 holes of golf, brunch, a cocktail reception and anawards dinner. A wonderful time was had by all!

Queens Chapter

September 12th was the Queens chapter’s 2004Bowl-a-Thon at Babylon Lanes, sponsored onceagain by Verizon, which matches every dollarcontributed to the bowl-a-thon – a win-win for all.

The chapter’s 3rd Annual Golf Classic is scheduled for September 27 at North HillsCountry Club in Manhasset. Hosted by CityWideDemolition & Rubbish Removal, this is theperfect event for both avid golfers andbeginners. What could be better than a sunny fallday playing golf with friends and sharing goodfood and drink? A full day of golf is followed by an extravagant cocktail hour, awards dinner,prizes, and more fun.

The Queens chapter is the beneficiary of theColiseum Powerlifting Impact NYCChampionship, held October 2nd, 2004 at theColiseum Gym in Middle Village. Men andwomen from all weight classes and age groupswill compete in this NYC Championship.

Mark October 15 as the date for the Queenschapter’s 1st Annual Greek Dinner Dance – theChariot of Life Ball. Our honoree is James G.Kalpakis, Esq., Founder & Chairman of the GreekCommittee. This evening is presented in the truespirit of giving, or philanthropia, as might be saidin Greek. Join us at Jericho Terrace in Mineola

for a true Greek experience of fine food, Greekmusic and Greek dancing, with music providedby the Trojans of New York.

The Queens Chapter would like to announce its 2005 St. Valentine’s Day Dinner Dance. As our opening event for the year, we honor thegift of love. Share an evening of fine food anddancing with a loved one. The time and place willbe announced after Thanksgiving. We hope youcan make it this year.

For more information about Queens chapterevents, call 718-746-7677.

Staten Island Chapter

October 6 is the date for the Staten Islandchapter's Dinner/Fashion Show , starting at 7:30pm at the Hilton Garden Inn. The theme is "Oktoberfest," complete with beer and steinsand appropriate decorations. Many of thechapter's young patients will be modeling, and a production company will bring in designerclothes and professional models. There will beplenty of new fashions for fall as well as severalshearlings and "new age" handbags. Tickets are$45 per person and there will be raffles,including a 50/50. Ads are still being acceptedfor our souvenir journal. Call 718-761-5380 for information.

Suffolk Chapter

This year’s annual dinner dance, held June 18 atLeonard’s of Great Neck, was a tremendoussuccess, with a large crowd turning out to honor

New Jersey chapter’s Night at the Races

13Cooley’s Anemia Foundation

AtlantaTahseen Mahmood678-357-4021tmahmood@metlife.com

Buffalo (NY)Dennis Locurto716-834-8903

CaliforniaRobert Yamashita, PresidentChristine Giannamore, Coordinator800-601-2821ca_chapter@hotmail.com

Capital Area (DC, VA, MD)Carl Vitaliti301-989-8947cvitaliti@aol.com

Chicago AreaBruce Rod847-602-2616bruce@brucerod.com

ConnecticutPeter Chieco914-232-1808anemia@optonline.net

FloridaDr. Paul Pitel, PresidentLale Gerger, Executive Director(904) 613-0097cooleysflorida@msn.com

Long Island (NY)Joseph Giammalvo631-271-8577connie@rpcli.com

Chapters Contact List

MassachusettsRudi Viscomi617-332-5952

New JerseyMatt Stamateris973-893-2241njcaf@comcast.net

Queens (NY)Frank Zito718-746-7677cafqueens@verizon.net

Rochester (NY)Shirley Cammilleri585-482-5587

Staten Island (NY)Angela Padavano718-761-5380

Suffolk (NY)Edward Martella631-863-0532

TexasMateen Shah214-324-6147

Westchester/Rockland (NY)Peter Chieco914-232-1808anemia@optonline.net

Are you receiving duplicate copies of LIFELINE at your address?

If so, please let us know so that we may make the necessary corrections.

(800) 522-7222.

Thank you.

Hugo Bua and to pay their respects to Suffolkchapter member Ralph Cazzetta, who passedaway shortly before the event. We know thatRalph would have been touched by theoutpouring of love from all present.

The Suffolk chapter scheduled a benefit golfouting at Cherry Creek Golf Club onWednesday, September 8. There was a greatturn-out for this event, which is always a highlightof the fall season for golf fanatics who enjoy a great course and a great time.

The chapter's third annual Bike and Walk isscheduled for Saturday, October 2, starting at Atlantis Marine World Aquarium. Thismarvelous event includes a number ofentertainment options - a 62-mile bike ride, a 20-mile bike ride, a 4-mile bike ride or a 4-milewalk. Registration ranges from $10-$25 for an individual and from $20-$50 for a family,depending upon the event. More information,including online registration, is athttp://www.cooleysmilesforsmiles.com.Starbucks will be donating coffee and donuts forbreakfast and Applebee’s will be donating lunch.All participants will receive 4 complimentarytickets to Atlantis Marine World plus a t-shirt andwe will have a kiddie korner with rides for theyounger kids, as well as a magician.

The Marriott in Hauppauge is the site for thechapter's 2004 Fashion Show on Thursday,October 28. Come catch up on some of thelatest style while helping a good cause.

Mark your calendars for Tuesday, November 9.That's the date for this year's Beef-O-Rama, heldonce again at Pace's Steak House in PortJefferson. This wonderful event includes an openbar and features the best porterhouse steakdinner in New York.

Boxes for the Suffolk chapter's Super Bowlevent are sold from mid-November until mid-January. On Super Bowl Sunday, each personwho purchased a box may come to Pace’sSteakhouse in Hauppauge to watch the gameand enjoy a sumptuous buffet. Guests may be brought for an additional fee of $100.

For information about upcoming Suffolk chapterevents, call (631) 863-0532.

Westchester-Rockland Chapter

September 28 is the date for the Westchester-Rockland chapter’s Annual Golf Classic. Held at New York Country Club in NewHempstead, registration and a putting contestbegin at 10:30, with a shotgun start at 12:15.The day also includes brunch, a cocktailreception and an awards dinner, adding up to a wonderful way to spend a day. Cost for an individual golfer is $250. For moreinformation, contact the chapter at (914) 232-1808 or anemia@optonline.net.

Ralph A. Cazzetta, CAF Board memberand a former CAF National PatientServices Director, passed away on May 19, 2004. Ralph, who was alsoa thalassemia patient, was a belovedmember of the CAF community who wasinstrumental in the founding of both TAGand TIF. All of us at CAF are greatlysaddened by this loss but thankful to havebeen blessed through our association withthis wonderful human being. Whetherstarting the first high school blood drivesin his community or testifying beforeCongress on behalf of all withthalassemia, Ralph was a committed,passionate and loving individual.

Below are remarks of the first President of the Suffolk chapter, Tony Laurino,delivered in tribute to Ralph.

14 Cooley’s Anemia Foundation

Ralph Cazzetta, a member of the NationalBoard of Directors, a member of the Suffolk chapter’s Executive Committee, the first National Director of Patient Services and a committed member of theCooley’s Anemia Foundation, passed awayon May 19th, one day after his 39th birthday.

Ralph was an articulate, intelligent man who always saw the glass as half full. Beforehe even reached his teenage years, Ralph was testifying with his father, Nunzio,

b o a r d n e w s

Tribute

OSIA National President Joseph Sciame,who is also an important member of CAF’sBoard of Directors, was honored by theItalian city of Benevento and the culturalorganization Iside Nova on July 7. Over 3000 guests attended the fourthannual Premio A Prescindere ceremony,at which Sciame was recognized for hisachievements in promoting Italian cultureand higher education.

“I was honored to accept this internationalrecognition in the name of OSIA's manymembers. Once again, I was moved by thewarmth, cordiality, culture, artistic abilityand world of diplomacy in Italy," saysSciame.

CAF congratulates Mr. Sciame on this well-deserved tribute.

Joseph SciameHonored

before Congress. His personality, charismaand eloquent explanation of the problemsfaced by Cooley’s anemia patients helped the Foundation gain recognition in Washington and at the National Institutesof Health. Congresspeople and senatorscame to know and respect Ralph for his positive attitude in facing life.

CAF and the New York Academy of Sciences sponsored the Sixth Cooley’sAnemia Symposium on March 13-15, 1990.Ralph Cazzetta was invited to present at this symposium, the first time that a patient had been so invited to participatein one of the Cooley’s anemia symposia.Ralph ended his presentation with twostatements that are particularly apt, tellingthose present that "we must remember thatquantity of life does not guarantee quality of life" and that "we must treat the spirit as well as the body."

Ralph had so many talents, but the one that stands out above all others was hisability to feel the pain of others and to comfort them. Peter Chieco, a pastNational President, saw this in Ralph andquickly brought him to the National office as the Director of Patient Services.

to Ralph A. Cazzetta

Joseph Sciame (center) at ceremony

NYBC Ad

Ralph and patient

15

In his new role, Ralph solicited all knownpatients for TAG; made contacts and wasthe liaison with doctors around the countrywho treated Cooley’s anemia patients;advised patients and parents; and acted as the liaison with insurance companies. His communications skills, personality andcharisma made him the perfect person forthis job. Patients, parents and doctorscalled him at all hours of the day or night,for he gave everyone his office phone, cellphone and home phone numbers.

Ralph always talked about nursing. After a number of years at the National office,Ralph decided it was time to returnto school to obtain his R.N. Due to his longstays at Mercy Hospital for one problem or another, Ralph frequently was forced to miss classes. Ralph would then have to work that much harder to make up theclasses he missed while staying currentwith his other coursework. Most people,especially someone who had to undergothe frequent blood transfusions Ralph did,

would have given up and thrown in thetowel. Not Ralph. This only made him moredetermined to finish. After he received hisdegree and license, he began work at Mercy Hospital, a place he was familiarwith since he received his transfusionsthere every two weeks since he was 12 years old.

Working in ICU, Ralph brought cheer,comfort and empathy to patients andfamilies. He brought laughter with his witand funny red shoes. He gave patients thewill to keep going and, when that was notGod’s will, the comfort of going in peace.The love and respect his co-workers felt forRalph was clearly evidenced at his funeral:Some 80-100 co-workers from everydivision of the hospital - not just nurses and doctors, but also nursing assistants,food service workers, security guards,maintenance workers - gathered outsidethe church to form an honor guard as Ralphwas brought into St. Edward’s Church.

We will miss Ralph because he is notphysically present to laugh with, snap backa witty remark or comfort us. But we hadthe experience and the pleasureof knowing him.

It was a blessing to have shared his life,and I only wish that everyone could havebeen as blessed as I was.

Thank you, Ralph.

Ralph Cazzetta Ralph and his family

Non-Profit Org.

U.S. Postage

Paid

New York, NY

Permit No. 4814129 - 09 26th Avenue, Flushing, NY 11354

Address service requested

Dr. Alan Cohen, who stepped down as Chair of CAF’s Medical Advisory Board in December, 2003, received thePresident’s Award at the June 12 Board of Directors meeting. The President’sAward, given at the discretion of the CAF National President, recognizes an individual who has made a significantcontribution to CAF and the lives of thosewith thalassemia.

Dr. Cohen’s association with CAF goesback to 1980, when he received a CAF Medical Research Fellowship tostudy “Young Red Cell Transfusions inThalassemia Major.” Dr. Cohen later joined CAF’s Medical Advisory Board,becoming Chair in 1992. A graduate of Harvard University and the University of Pennsylvania School of Medicine,

Dr. CohenHonored atBoard Meeting

Dr. Cohen joined the Children’s Hospital of Philadelphia (CHOP) as an intern in 1972. He is now Physician-in-Chief at CHOP and Chairman of the Department of Pediatrics at the University of Pennsylvania School of Medicine.

Hundreds of thalassemia patients havebenefited from Dr. Cohen’s wisdom andwarmth, both in the United States andaround the world. CAF is grateful for hiscommitment to the cause of thalassemiaand is fortunate that he will continue to helpguide the Foundation as a member of its Board of Directors.

The following individuals were electedto the CAF Board of Directors at itsannual meeting on June 12th, 2004:

Charles DiSalvo

Lale Gerger

Diane Gulbenkian

Lisa Kontogiannis

Tahseen Mahmood

Christine Somma

Elliott Vichinsky, M.D.

Robert Yamashita

CAF welcomes these new additionsto the Board and looks forward to collaborating with them in thecoming years.

New BoardMembers

b o a r d n e w s ( c o n t ’ d )

Dr. Alan Cohen