Basic science and storytelling :

1. Basic science and storytelling: The experience of an Italianbiomedical charity • Annamaria Zaccheddu • Erice International School of ScienceJournalism

2. A big challenge for communicators • The narration of basic science is a big challenge, regardless of the specific topic (physics, climatology, biomedicine, etc.) • At the same time, anyorganizationisobliged to include fundamental science in itscommunicationstrategy • Whatdoesthismean for a biomedical charity as Fondazione Telethon?

3. Whowe are

4. Ourmission

5. Our story • USA, 1966: the comic actor Jerry Lewislaunches Telethon, a television marathon to raise money for the Muscular Distrophy Association (MDA) • Italy, 1990: a group of parents with children suffering from muscular dystrophy appeal to the Foreign Affair Minister Susanna Agnellito create Telethon also in Italy. The show is a big success and two years later the founders decide to extend the Telethon mission to all rare genetic diseases.

6. Rare diseasesare not – asfact– so rare • In Europe A disease is defined as rarewhen it affects less than 1 in 2000. There are more than 6000RDs. • On the whole, RDs may affect 30 million EU citizens. • 70% of RDs become manifest within the first 5 years of an individual’s life. • 80% of RDs are of genetic origin, and are often chronic and life-threatening. • For most of these diseases there is still no cure and many of them are still undiagnosed. Fondazione Telethonexists for thesepeople

7. How we operate

8. Telethon, number and facts* 75% spent in programs OVER 451M€ invested We support only top, internationally competitive projects, through highly selective peer review conducted by a prestigious international Committee ONLY THE BEST BASIC AND CLINICAL RESEARCH 475genetic diseases studied 10,222 scientific papers 2,570 Research Grants 20+ Therapeutic strategies at the clinical or pre-clinical stage 1,556 Funded scientists * Last update 30/06/2015

9. A big success of Telethonresearch Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a rare immunodeficiency (ADA-SCID) has just been approved by the European Commission. It has been developed by the scientists of the San Raffaele-Telethon Institute of Gene Therapy in collaboration with GlaxoSmithKline

10. Nextsteps for the future • Thanks to the strategicalliance with industries, the gene therapydeveloped by Telethonscientistscould be applied to othergeneticdiseases:

11. Basic vs. clinicalresearch

12. Differentcommunicationapproaches _ + CLINICAL RESEARCH “You invested more than 450 million euros in research and saved only 20 children?” “Why don’t you study my disease too?” Industries involvement Concrete results Expression of the mission (the cure) Emotional involvement Stimulating fundraising Difficult to explain Intermediate results (how and when?) Animal models False expectations within patients community Expression of the mission (many diseases) Contribution to the dissemination of scientific culture BASIC RESEARCH

13. The Telethon «researchladder» Over the years, our funding has taken us closer to patients, moving away from basic researchtowards pre-clinical and clinical research

14. Basic science, an essentialissue • An organizationaimingat the cure of rare geneticdiseasescan’taffordto ignorebasicresearch • There are manyreasonswhy an organization as FT has to continue supportbasic science and communicationstrategyhas to take itintoconsiderationtoo • Let’sseethree«good stories» wechose to communicatebasicresearch.

15. 1. Basic science is a powerful source of goodideas

16. Liviana’sgift (2012)

17. The same story, anotherperspective • Giovanni isone of the childrensuccessfullytreated with gene therapy: the first results of the clinical trial hasbeendescribed in Science in 2013 and received huge media coverage However, thismoving story – one of the mainsuccesses of Telethonresearch – can also be toldfrom anotherperspective: That of a scientist with a «crazy» idea

18. HIV, a killer to cure • The story is that of Luigi Naldini, an Italianscientist with arevolutionary idea: the possibility to use HIV to transfer correctversions of genes • The first reactionof the scientific community to Naldini’s “revolutionary” paper (Science, 1995) hasbeen: «Wonderful science but… you are notgoing to applyit to humans, are you?» • More than 20 yearslaternowlentiviralvectors are routinelyusedand in the meantimeallowedTelethonscientists to treatatleast 3 severe geneticdiseases So, are wesure that patients’ perspectives are the onlyoneswe can effectively use to communicatebasic science?

19. A new storytelling … capable of killingmillions of people, also veryfamous and rich A dreadful and mysterious virus… … working in the institutefounded by a virus fighter, Jonas Salk An Italianscientist «borrowed» by USA…

20. 2. Basic science is a strong ally of clinicalresearch

21. Researchisnot a one-way road • Basic science isveryhelpfulalso to clinicalresearch, becauseitprovides information aboutwhethertherapies work or don’t, and whyso. • Even in the presence of outstandingclinicalresultsweshouldn’tignoretheircorrelatedbasicstudies • Although more difficult to tell, these stories makepeopleunderstandbetterwhattranslationalresearchis (and whyittakes so much time to achieveresults)

22. An important source of knowledge • Starting in 2010, 8 children with a rare immunodeficiency have beensuccessfullytreatedthanks to gene therapy • Meanwhile, in the lab, Telethonscientistsstudied the «long-termdestiny» of the engineeredhematopoieticstemcells • Thisstudyprovideduseful information notonlyaboutthis specific rare disease, butalsoabouthematopoieticstemcelltransplantation (a very common treatment for otherdiseases, includingleukemia and otherbloodcancers) Although more difficult to communicate, thesecollateralresultsdeserve to be known by the lay public

23. 3. Basic science can be extremelysurprising

24. Unexpectedresults • A core message to be transmitted to donors, stakeholders and the public in general isthatsupportingbasic science meansinvesting in unexpectedopportunities • Narratingthese «unexpectedresults» couldmakebasic science more fascinatingto the lay public

25. From cancer to a very rare disease • A group of Telethon-fundedscientistsdiscoveredthat an oldanticancerdrugcould be effectiveagainst a rare geneticdisease – cavernoushemangioma–characterized by severe and unpredictablecerebralhemorrhages. • The drug, capable of arresting the production of new bloodvessels, showedeffectiveness in both the diseasemodels. Florence Griffith Leandro Castan Alberto Contador

26. The take home message • Communicating the results of clinicalresearch can be relativelyeasier, becausewe can relyupon “success stories,” that show the effectivenessof new treatments. Nonetheless, for an organizationlike FT iscrucial to talk about the possibilitiesoffered by basic science too. • We must show ourmainstakeholders(patientsand donors) that thanks totheirmoneyweare supportingbasic sciencewith greatpotential. • At the same time we have to clarify that research can be complex, expensive and time-consuming, and sometimesdoesnotlead to immediate results: dead-end roadsare part of the wholeprocess.

27. Neverforget the power of words

28. Thankyou!azaccheddu@telethon.it(+39) 06 44015402www.telethon.it