{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,25]],"date-time":"2026-01-25T01:23:18Z","timestamp":1769304198223,"version":"3.49.0"},"reference-count":38,"publisher":"American Society of Hematology","issue":"19","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2021,10,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>More than 20 years ago, clinical trials and federal grant support for sickle cell disease (SCD) research were not on par with support for other genetic diseases. Faced with the opportunity to spur research and advance treatments for SCD, and at the recommendation of advisors, the Doris Duke Charitable Foundation (DDCF) offered an SCD research funding opportunity starting in 2009 through its Innovations in Clinical Research Awards (ICRA) program. Twenty-eight new grants of $450\u2009000 for direct costs over 3 years and 7 renewals were awarded, for a total investment of $17 million. Only about half the research teams garnered follow-on funding directly related to their ICRA projects, but the financial return on the research investment was substantial (\u223c4 times the original $17 million or 300%). All but 1 of the ICRA investigative teams published original research reports that acknowledged DDCF as a source of funding; the median number of publications per team was 3. Major innovations in the diagnosis and treatment of SCD included but were not limited to a demonstration that genetic modification of BCL11A enhancer is a potentially important treatment modality, establishment that plerixafor mobilization is safe and effective for those with SCD, development and validation of a new diagnostic called SCD BioChip, and evidence that hydroxyurea treatment is safe and efficacious in African children. These outcomes show that relatively small research grants can have a substantial return on investment and result in significant advances for a disease such as SCD.<\/jats:p>","DOI":"10.1182\/bloodadvances.2021005709","type":"journal-article","created":{"date-parts":[[2021,9,27]],"date-time":"2021-09-27T16:35:12Z","timestamp":1632760512000},"page":"3855-3861","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":5,"title":["Impact of a grant program to spur advances in sickle cell disease research"],"prefix":"10.1182","volume":"5","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8294-5918","authenticated-orcid":false,"given":"Sindy N.","family":"Escobar Alvarez","sequence":"first","affiliation":[{"name":"Doris Duke Charitable Foundation, New York, NY; and"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elizabeth R.","family":"Myers","sequence":"additional","affiliation":[{"name":"Consultant, New York, NY"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"234","published-online":{"date-parts":[[2021,10,7]]},"reference":[{"issue":"2865","key":"2021100715241718500_B1","doi-asserted-by":"crossref","first-page":"543","DOI":"10.1126\/science.110.2865.543","article-title":"Sickle cell anemia, a molecular disease","volume":"110","author":"Pauling","year":"1949","journal-title":"Science."},{"issue":"2846","key":"2021100715241718500_B2","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1126\/science.110.2846.64","article-title":"The inheritance of sickle cell anemia","volume":"110","author":"Neel","year":"1949","journal-title":"Science."},{"issue":"4581","key":"2021100715241718500_B3","doi-asserted-by":"crossref","first-page":"326","DOI":"10.1038\/180326a0","article-title":"Gene mutations in human haemoglobin: the chemical difference between normal and sickle cell 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