Dr. Pankaj Malhotra is an Associate Professor of Internal Medicine at PGIMER in Chandigarh, India. He received his medical degree from Punjab University and training in internal medicine at PGIMER. His research interests include leukemia, stem cell transplant, and myeloma. He has published several invited and peer reviewed papers. The document discusses potential novel treatments for thalassemia including stem cell transplantation, gene therapy, antisense molecules to silence mRNA splicing, induced pluripotent stem cells, and drugs or gene therapy to increase gamma chain production or decrease alpha chain production. It notes challenges with gene therapy including virus selection, techniques, potential duration of effect, and long term side effects.

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

5. Hallmark of b thal

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

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

13. Gene therapy

18. 1. Selection of virus
2. Technique
1. How long
will it
work
2. Long term
side
effects

19. 1. Selection of virus
2. Technique

22. 1. Selection of virus
2. Technique
1. How long
will it
work
2. Long term
side
effects

25. iPSC

29. Caution!

30. Silence Hb F
suppression

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

37. Drugs for increasing Hb F

44. Other drugs for increasing Hb F

51. 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