AVROBIO Gene Therapy Education

AVROBIO Gene Therapy Reference

02 Understanding Cell Therapy

Section 4

What is Hematopoietic Stem Cell Therapy?

Learning Goal

To learn about the different ways hematopoietic stem and progenitor cells can be used to treat disease.

Hematopoietic stem cell transplants (HSCTs) have been performed for more than 60 years1 and are used to treat diseases that affect the bone marrow, including:

  • Cancers such as leukemia, myeloma, and lymphoma2
  • Blood disorders such as aplastic anemia, beta-thalassemia, sickle-cell disease, and severe-combined immunodeficiency2
  • Enzymatic disorders such as Hurler syndrome and metachromatic leukodystrophy (MLD)3

The goal of an HSCT is to provide an individual with cells that can improve bone marrow function, or generate functional cells that can replace non-working cells.4

There are two types of HSCTs – autologous and allogeneic:

  • Allogeneic hematopoietic stem cell transplant (allogeneic HSCT or alloHSCT): a medical procedure in which HSPCs are obtained from one person (donor) and given to another person (recipient).5
  • Autologous hematopoietic stem cell transplant (autologous HSCT or autoHSCT): a medical procedure in which the person receives their own HSPCs that had been previously collected.6

The role of HSC transplantation is being explored in autoimmune diseases including systemic sclerosis and systemic lupus erythematosus and has already shown promising results in treating relapsing-remitting multiple sclerosis.4

HSCs are also being used in certain applications of gene therapy, such as autologous lentiviral gene therapy. HSCs are an ideal target for gene therapy because they are long-lived and introduction of a therapeutic gene to an HSC may lead to a persistent change in all of the blood cell types.7

Additional Interesting Fact

The first allogeneic HSCT procedure was performed in 1957.8

Key Learnings

Hematopoietic stem cell transplants (HSCTs) aim to provide individuals with cells that can improve bone marrow function or generate functional cells that can replace non-working cells.

HSCTs can be either allogeneic (giving cells obtained from one person to another) or autologous (returning cells to the individual they were collected from).

HSCs are particularly well-suited for gene therapy, including autologous lentiviral gene therapy, because they are long-lived and may induce a long-lasting change in all blood cell types.

Continue learning about gene therapy in the next section

Topic 3: Section 1

What is Gene Therapy?

To understand what gene therapy is and what it is designed to do.

Next Section ››

References

  1. Thomas ED, Lochte HL, WC Lu, Ferrebee JW. Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy. N Engl J Med. 1957;257(11):491-496. [PubMed]
  2. Stem cell information: Hematopoietic stem cells; 2016. U.S. Department of Health and Human Services, National Institutes of Health; 2016. https://stemcells.nih.gov/info/basics/stc-basics/#stc-I. Accessed May 6, 2022.
  3. Tan EY, Boelens JJ, Jones SA, et al. Hematopoietic stem cell transplantation in inborn errors of metabolism. Front Pediatr. 2019;7:433. [PubMed]
  4. Khaddour K, Hana CK, Mewawalla P. Hematopoietic stem cell transplantation. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2020. https://www.ncbi.nlm.nih.gov/books/NBK536951/. Accessed February 17, 2021.
  5. Bone marrow transplant. Allogeneic stem cell transplant. MayoClinic.com; 2019. https://www.mayoclinic.org/tests-procedures/allogeneic-stem-cell-transplant/pyc-20384863. Accessed February 17, 2021.
  6. Bone marrow transplant. Autologous stem cell transplant. MayoClinic.com; 2019. https://www.mayoclinic.org/tests-procedures/autologous-stem-cell-transplant/home/ovc-20384860. Accessed February 17, 2021.
  7. Morgan RA, Gray D, Lomova A, Kohn DB. Hematopoietic stem cell gene therapy – progress and lessons learned. Cell Stem Cell. 2017;21:574-590. [PubMed]
  8. Thomas ED, Lochte HL, Lu WC, Ferrebee JW. Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy. N Engl J Med. 1957;257:491-496. [PubMed]