Thalassemia Treatment Updates: Dr. Pankaj Malhotra
Transcript of Thalassemia Treatment Updates: Dr. Pankaj Malhotra
PANKAJ MALHOTRA, MDChandigarh, 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.
Novel treatmentsPankaj Malhotra, MD, FACP
Professor of Clinical Hematology
Dept. of Internal Medicine, PGIMER, Chandigarh, India
Normal production of globinchains
Hallmark of b thal
Produce b chains:1. Stem celltransplantation2. Gene therapy3. antisense molecules to silence mRNA splicing4. Induced pluripotent stem cells
Increase the g chain production:1. Drugs2. Gene therapy
Decrease a chain production
Gene therapy
1. Selection of virus2. Technique
1. How long will it work
2. Long term side
effects
1. Selection of virus2. Technique
1. Selection of virus2. Technique
1. How long will it work
2. Long term side
effects
iPSC
Caution!
Silence Hb F suppression
Increase the g chain production
1. Drugs2. Gene therapy
Silence Hb F suppression
Drugs for increasing Hb F
Other drugs for increasing Hb F
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