2015 CALL FOR RESEARCH PROJECT: CRYOSTEM COLLECTION Preface CryoStem is one of the projects selected in 2011 by the French National Research Agency (ANR) in relation to the “Cohorts” call for projects funded under the French government’s “National Investment Programme” (“Investissements d’avenir”). Launched in July 2012, the CryoStem cohort is a collection of biological samples, taken from allogeneic stem cell transplantation donor-recipients, through which it is hoped to achieve a greater understanding of Graft versus Host disease (GvHD), a severe complication arising from allogeneic transplantations, and whose characteristics are currently poorly understood. GvHD attacks human blood tissues and is the consequence of an excessive reaction of the host immune system occurring simultaneously to beneficial effects against blood disorders. It has major repercussions on 50-60% of patients receiving allogeneic transplantations as part of their cancer treatment. The CryoStem cohort has obtained all the necessary regulatory authorizations from the French Ministry for Higher Education and Research (MESR), the French Patient Protection Committee (CPP), the French Consultation Committee for Data Processing in Healthcare Research (CCTIRS), and the French Data Protection Commission (CNIL); a quality management system has also been introduced prior to obtaining ISO 9001 certification covering the entire network from the start of 2015. As of November 30, 2014 the collection comprises 1,930 patients and 830 donors, more than 60,000 biological samples, including more than 5,000 processed blood samples. For each blood sample collected, the derived products comprise blood plasma, dried white blood cell pellets, and viable mononuclear cells in Albumin/ DMSO (10%). The sampling schedule, spanning from the pre-transplantation period to one year posttransplantation, is indicated in the diagram below: D : Donor – R: Recipient D0 and R0 pre-HSCT Related.D Related Donor aGvHD : acute Graft-versus-Host Disease (<100days) a: sampling if aGvHD cGvHD : chronic Graft-versus-Host Disease (>100days) c: sampling if cGvHD a1 or c1 sampling at GvHD diagnosis a2 or c2 sampling at x days after GvHD diagnosis s: systematic sampling if no aGvHD and/or cGvHD at x days post-HSCT Call for research projects Collection access procedure Authorization to access the CryoStem collection is granted upon the approval of the CyroStem Scientific and Strategic Committee (CSS), following an evaluation by independent international experts. To ensure fairness of access, authorization shall not be granted to any team for two successive years, regardless of the subject of the call for projects. Please note that authorization through this call for projects only covers access to Collection samples; no funding will be awarded to any research applicants. Call for projects framework CryoStem CFP - 2014 Page 2 sur 11 This call for projects only covers biological research projects studying Graft versus Host Disease (GvHD) in humans Important information concerning access to samples Access to the Collection will be made possible as follows: Through a grant of €36,000 awarded by the Association Laurette Fugain Through a grant of €16,000 awarded by the Association Vaincre la Leucémie Through a grant of €19,000 awarded by the Association Cent pour Sang la Vie Through a grant of €15,000 awarded by the Association IRGHET (International Research Group on HEmatopoïetic cells Transplantation) Through the applicant’s own resources; pricing for providing samples will vary depending on the sample type and the status of the applicant (cf. Pricing list appendix) Results of research projects undertaken using Collection samples A scientific report will be requested at the conclusion of all research undertaken using CryoStem Collection samples. To promote the Collection with our partners, a review of each scientific project, around half a page in length, written in layman’s terms and aimed at the general public, will be requested from all research teams that enjoy access to the Collection; they may also be asked to communicate further with the general public. Applications Applications must be compiled by completing the standard application form, entirely in English, and must not exceed 10 pages, including the bibliography. Applications must be submitted by post before January 31th, 2015 (date as per postmark)to the following address : Projet CryoStem Institut Paoli-Calmettes – Centre de Thérapie Cellulaire 232 boulevard Sainte-Marguerite 13273 Marseille cedex 9 The results of the call for projects will be made public on April 1st, 2015. Terms and conditions governing the call for projects The projects put forward by the academic and/or commercial members in relation to this Call for Projects must abide by the General Terms attached to the application file. The applicants must acknowledge that they have read and accepted these General Terms and the appendices. CryoStem CFP - 2014 Page 3 sur 11 Application form Research theme: The biology of Graft versus Host Disease (GvHD) in humans Key selection criteria For projects of equal scientific value in this initial call for tenders, projects presented by French research and/or academic teams will be preferred. Particular attention will be paid to projects making use of proven available techniques that may accordingly be rapidly implemented. Applicants must present their project by completing the standard application form: 1. Summary (2 pages) 2. Layman’s summary for the general public (1 page) 3. Scientific proposal comprising a project overview with reference to the following points (7 pages): a. State of the art and research capabilities of the applicant(s) b. Objectives c. Strategy and methods d. Expected results e. Schedule f. Scientific and technical details regarding sample use g. Prospects and potential applications h. Publications arising from the project i. Proof of funding to access the Collection j. Proof of available research techniques to be implemented CryoStem CFP - 2014 Page 4 sur 11 1. Summary (2 pages) a. Administrative summary Project initiator Ms. Mr. Surname Name Job title(s) Date of birth Nationality Tel Fax E-mail Name and address of laboratory applicants Full details of research teams CryoStem CFP - 2014 Page 5 sur 11 Full details of research teams (cont.) List of five most prominent publications in the last five years (for all applicant teams) CryoStem CFP - 2014 Page 6 sur 11 b. Scientific summary Project title Project summary (500 words) Project length Type of samples requested D0 R0 a1/a2 c1/c2 s1/s2 Number of samples requested (please specify number of samples per period) CryoStem CFP - 2014 Page 7 sur 11 Rationale for the number and type of samples requested (statistical plan) Total funds required to access the samples requested (calculated in accordance with the Pricing List) Total funds required for the project Has a request been made for this project to be funded in any way by another organization? If so, please provide full details: Name and contact details of the organization responsible for collecting and handling the samples CryoStem CFP - 2014 Page 8 sur 11 2. Layman’s summary for the general public (in English, 1 page) CryoStem CFP - 2014 Page 9 sur 11 3. Scientific proposal (in English, 7 pages) Project overview with reference to the following points: a. State of the art and research capabilities of the applicant(s) b. Objectives c. Strategy and methods d. Expected results e. Schedule f. Scientific and technical details regarding sample use g. Prospects and potential applications h. Publications arising from the project i. Proof of funding to access the Collection j. Proof of available research techniques to be implemented CryoStem CFP - 2014 Page 10 sur 11 Appendix 1: Pricing List Pricing list covering the provision of samples from the CryoStem Collection† A: Academic project initiator and member of the CryoStem consortium B: Academic project initiator unaffiliated with the CryoStem consortium C: Non-academic / private / commercial project initiator By aliquot* Mononuclear cells in DMSO A B C €12.00 €15.00 €30.00 Dry precipitate €9.00 €11.25 €22.50 Plasma €8.00 €10.00 €20.00 Nitrogen storage (per year)1 €0.80 €1.00 €2.00 Removal from storage (flat rate)2 €5.40 €6.75 €13.50 Blood sample (flat rate) €1.70 €2.15 €4.25 By patient** Extraction of clinical data A B €12.00 €15.00 €30.00 €9.00 €11.25 €22.50 Selection and unpacking of aliquots By project Aliquot shipment A B C Based on the number of aliquots † Prices valid until December 31, 2015 * Single aliquot = 1 x 2ml cryotube 1 To be multiplied by the storage duration for each aliquot 2 Including extracting biological data ** C Unlimited number of aliquots for a single patient 3 Shipment in dry ice from CRB partners to the CryoStem platform, then onwards to the Project initiator CryoStem CFP - 2014 Page 11 sur 11