Disclosures for Ellis Neufeld, MD, PhD

1. Disclosures for Ellis Neufeld, MD, PhD In compliance with ACCME policy, ASH requires the following disclosures to the session audience: 51st ASH Annual Meeting ♦ New Orleans, LA

2. Aiming for high-quality basic and translational research training for hematology fellows Ellis J. Neufeld MD, PhD ellis.neufeld@childrens.harvard.edu Children’s Hospital Boston Dana Farber Cancer Institute Harvard Medical School

3. Lessons from Children’s/DFCI fellowship program • Start early • Offer highest quality opportunities, (not just divisional or departmental opportunities) • “Three years is not enough” • Scholarship oversight committee input must be both early and rigorous • No substitute for expert mentoring • Duty hours and high-quality lab training • What does it mean to train in “translational” research? Can a fellow really do this? • Recognize strengths and weaknesses. “What if basic science is not for me?”

4. Start Early. No Earlier • Expose prospective fellows to breadth of opportunity. Work research training choices into fellowship interviews. • Focus on accepted fellows before they matriculate. • If local, invite to lab meetings, work on projects • “Data blitz” for first year fellows • 4 dinner sessions, 12 speakers per session, 5 slides/5 minutes each, 5 minutes for questions. • Basic and clinical investigators, all ranks, includes other hospitals and universities • Advisors encourage first year fellows to use elective time to investigate labs/opportunities

5. Offer high-quality opportunities • If the best person at fellow’s topic of interest is across campus or across town, send the fellow. • Insist on highest level buy-in (chairs, deans) for high-quality research • Recruit best investigators (e.g. to data blitz) • Example 2009: new Program in Molecular Medicine at Children’s.

6. “Three years is not enough*” • This is the mantra of our program. • Two T-32 grants available in Division, plus two more in sister departments. Use Clinical/hospital/GME funds for year 1. • Three-year T-32 support covers year four (first year instructors). • We do NOT guarantee year five automatically. • K12 opportunities, other scholarships/fellowships cover many for year 4/5. • K08, K23 cover several more years mentored research training * Thanks to Sam Lux

7. Scholarship Oversight Committee input should be early and rigorous • Set high expectations for fellow • Include senior external member(s) with broad view of field • Review need for scholarly work product • Paper or manuscript • Grant application approved or favorable review • “project write-up” acceptable in some cases • Remind lab mentor of the need for this activity. • The scholarly work product might NOT be the main lab focus. Small clinical reports acceptable. • Meet often enough to catch problems with focus or large technical glitches

8. No substitute for expert mentoring • Mentor may or may not be the lab supervisor • Expertise in the relevant basic or translational field is essential. Expertise in the exact lab system is not essential. • Convene the mentors for all the fellows from time to time to review philosophy, best practices, challenges. • Mentors could serve on SOC or play complementary roles.

9. Strive for K awards or equivalent • Few if any fellows could apply for R grants during third year. • Development of data for K application is a reasonable fellow goal. • K12 need few preliminary data. Increasingly, K08 and K23 need a lot! • Foundation/philanthropic equivalents are just as good (NHF-Baxter hemophilia grants) • The biggest gift in K grants is protected time

10. Strive for K grants, continued • K08 most appropriate for basic research, • K23 can support any patient-oriented translational research • Training program should help with Career development plans for your K applicants • Faculty member or dean paying attention to CDP is invaluable for K applicants. Investment readily pays back

11. Duty-hour limitations and high quality research training • According to most recent ACGME decisions, lab hours count in duty hours • Terrible implications for post-call fellows in research settings. • May require rethinking of coverage schemes • Night-float weeks on call to reduce interruptions of research for most fellows most of the time • Inevitable move toward hospitalists • FACULTY don’t have duty hour limitations • All kinds of bad potential implications if clinical duty is pushed back to first faculty year (i.e. our research fellows’ fourth year) • Possible division of labor among more-clinical vs more-lab-based researcher • If fellowship gets LONGER because of the duty hour conundrum, what happens to research time?

12. What is “translational research training?” • This begs the question, “What is translational research?” • “Bench to bedside.” • New pathway discovered in the lab during a gene expression screen of MDS. • This leads to tests of pathway inhibitors in vitro. If successful, needs a clinical trial. • “Bedside to bench” • Fellow discovers an interesting patient with unknown genetic defect • Comes to lab to sequence candidate genes and learn pathophysiology.

13. Can an applicant train in translational research? • Poll • Yes • No

14. Training in translational research? • (Maybe) . Most translational researchers were trained to do something else • Traditional route A • Physician scientist trains in the lab, makes a discovery and learns how to translate it • Traditional route B • Physician investigator trains in clinical research methodology. • Collaborates with investigator A who has a new compound that needs testing • New route? • Formal training in methods for drug evaluation, IND submissions, statistics and methods for small scale phase I/2 trials, intellectual property issues, etc. Leads to masters degree • Harvard proposes such a pathway to complement traditional epidemiology/outcomes research for T1 researchers • (Neufeld skeptical)

15. Recognize strengths and weaknesses • “What if I don’t really like to _____________ • Write papers?” • Write grants or pay attention to lab budgets?” • Work in the lab, I like working with patients?” • “OK, fine.” Evaluate strengths, find training for the right alternative pathway • Regulatory/FDA • Clinical research? • Clinician pathway?

16. Warning! • It is not appropriate to say to a fellow who is bailing out of the lab, “OK, then you’re a clinical researcher, ” and unleash the fellow on the clinical research unit without training in clinical research • But this actually happens all the time!

17. “Real world” talk for second year fellows • Clinical and basic research leaders • Wine and cheese. • Last talk of a one week “consolidation course” September, year 2. • Cover some key concepts in aiming for academic career • Start up offers • Indirect costs • Protected time • K grants vs other pathways • Clinical vs. research pathways. (REALLY? 70%???)

18. DISCUSSION • Acknowledgments • Sam Lux • Stuart Orkin • David Nathan • David Williams • Tom Abshire