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Babu Mia։ Targeting PPM1D to Eliminate Malignant Stem Cells in Myeloproliferative Neoplasms
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Oct 15, 2026, 17:08

Babu Mia։ Targeting PPM1D to Eliminate Malignant Stem Cells in Myeloproliferative Neoplasms

Babu Mia, Assistant Professor at Icahn School of Medicine at Mount Sinai, shared a post on LinkedIn about a recent article he and his colleagues co-authored, published in Blood, adding:

”It’s my absolute pleasure and utmost honor to share our recent publication in the Blood Journals Portfolio of the American Society of Hematology, titled:

‘Molecular Consequences and Targeting of PPM1D Overexpression in Myeloproliferative Neoplasms,’ with me as first author!

Eliminating malignant stem cells while sparing healthy blood production is a major challenge in cancer therapy. By investigating PPM1D (a negative regulator of p53) across Myeloproliferative Neoplasms (MPNs), we found:

  1. Widespread Target: PPM1D is overexpressed in ~75% of MPN stem/progenitor cells, establishing it as a common therapeutic target across MPNs.
  2. How Cancer Stem Cells Evade Death: Developing patient-derived iPSC models and single-cell RNA-seq analysis revealed that PPM1D suppresses p53/DNA damage checkpoints while driving pro-survival pathways.
  3. Releasing Both Brakes on p53: Combining PPM1D and low-dose HDM2 inhibitors synergistically depletes malignant JAK2V617F+ progenitors in vitro and in vivo while sparing healthy stem cells.
  4. Clonal Replacement: Tapestri single-cell DNA and protein multi-omics showed dual blockade suppresses mutant clones and expands healthy wild-type cells.

I’m deeply grateful to Bridget Marcellino for her tremendous commitment to this work, and to all our brilliant co-authors and collaborators at the Icahn School of Medicine at Mount Sinai , Memorial Sloan Kettering Cancer Center, Mayo Clinic, and all other institutions.

Above all, thank you to the patients whose participation made this research possible, and to my family and friends for always cheering me on!

Thanks to Waters Biosciences (Formerly BD Biosciences) ( Dan MacDonald Luciano Martelotto Helen Smith, PhD Shreya Udani, Ph.D. ) for the help with BD Rhapsody platform, 10x Genomics ( Sedat Resad) Novogene America (caitlin c) GENEWIZ from Azenta Life Sciences ( David Geyer ) for the help in the process of single cell multi omics study.

We also used state of the art single cell DNA and immunophenotyping technique from Mission Bio Brian Kim !

We also used state of the art machine learning models for experimental data analysis including sequencing data!

We sincerely thanks The National Institutes of Health National Cancer Institute (NCI) NHLBI – Blood Research & Education for the funding!

To colleagues across academia, biotech, and pharma: If you are exploring p53 pathway reactivation, PPM1D therapeutics, or single-cell multi-omics in drug discovery, let’s talk!”

Title: Molecular Consequences and Targeting of PPM1D Overexpression in Myeloproliferative Neoplasms

Authors: Md. Babu Mia, Christoph Schaniel, Cara Clementelli, Sarah Elisabeth Haines, Malgorzata Olszewska, JoAnn Arandela, Sebastian Elghaity-Beckley, Sophie Klingborg, Zinedine Hoque, Ayman Mohammad, Shwetha Hara Sridhar, Brianna Kelly, Xiaoli Wang, Emmanuel Olivier, Alan H. Shih, Blake T. Langlais, Allison Cote, Noushin Farnoud, Raajit K. Rampal, Duke Butterfield, Amylou C. Dueck, Min Lu, Kristin G. Beaumont, Robert Sebra, John O. Mascarenhas, Eirini P. Papapetrou, Ronald Hoffman, Bridget K. Marcellino

Babu Mia։ Targeting PPM1D to Eliminate Malignant Stem Cells in Myeloproliferative Neoplasms

Stay updated on all scientific advances with Hemostasis Today.

Alan H. Shih Allison Cote Amylou C. Dueck Ayman Mohammad Babu Mia Blake T. Langlais Brianna Kelly Bridget K. Marcellino Cara Clementelli Christoph Schaniel Duke Butterfield Eirini P. Papapetrou Emmanuel Olivier Health Hematology Hemostasis Hemostasis News Hemostasis Today JoAnn Arandela John O Mascarenhas Kristin G Beaumont Malgorzata Olszewska Md. Babu Mia Medicine Min Lu myeloproliferative neoplasms Noushin Farnoud Raajit K. Rampal Robert Sebra Ronald Hoffman Sarah Elisabeth Haines Sebastian Elghaity-Beckley Shwetha Hara Sridhar Sophie Klingborg Xiaoli Wang Zinedine Hoque
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