Thalassemia Treatment Updates: Dr. Pankaj Malhotra

Thalassemia Treatment Updates: Dr. Pankaj Malhotra

1. PANKAJ MALHOTRA, MD Chandigarh, India Associate Professor, Department of Internal Medicine Pgimer , Chandigarh, India Dr. Pankaj Malhotra received his medical degree from Punjab University and his training in internal medicine at PGIMER, Chandigarh, India. He has several invited and peer reviewed publications. Dr. Malhotra was awarded the CML Fellowship by International CML Foundation ( iCMLF ) to Knight Cancer Centre, Oregon Science and Health University, Portland, USA. Areas of Interest include leukemia, stem cell transplant and myeloma.

2. Novel treatments Pankaj Malhotra , MD, FACP Professor of Clinical Hematology Dept. of Internal Medicine, PGIMER, Chandigarh, India

3. Normal production of globin chains

4. Hallmark of b thal

5. Produce b chains: Stem cell transplantation 2. Gene therapy 3. antisense molecules to silence mRNA splicing 4. Induced pluripotent stem cells

6. Increase the g chain production: 1. Drugs 2. Gene therapy Decrease a chain production

7. Gene therapy

8. Selection of virus Technique How long will it work Long term side effects

9. Selection of virus Technique

10. Selection of virus Technique How long will it work Long term side effects

11. iPSC

12. Caution!

13. Silence Hb F suppression

14. Increase the g chain production 1. Drugs 2. Gene therapy Silence Hb F suppression

15. Drugs for increasing Hb F

16. Other drugs for increasing Hb F

17. Conclusions on novel (not so) treatments in thalassemia b globin production Gene therapy Antisense molecules to alter RNA splicing iPS cells Upregulation of gamma globin production Drugs ( HbF production) Upregulation of genes