Stem cell mobilising agent - plerixafor

Published: 12-Mar-2010
Regulatory Research & Development

Patients receiving a haematopoeietic stem cell transplant are also treated with an agent to...

Patients receiving a haematopoeietic stem cell - or bone marrow - transplant are also treated with an agent to mobilise the stem cells. This is typically granulocyte colony-stimulating factor (G-CSF) but there are large differences in response so new agents that have a more predictable effect would be extremely useful.

One such agent is Genzyme's plerixafor, acquired along with the company AnorMed.1 It acts at the CXCR-4 chemokine receptor, which has numerous signalling functions in the body, and also appears to be involved in stem cell mobilisation. The drug was originally designed as an anti-HIV agent, but was discontinued. However, it was also shown to increase peripheral leukocytes as it mobilised HSCs in the bone marrow by inhibiting the interaction between CXCR-4 and stromal cell-derived factor 1, and it is now being developed for this function.

In a Phase II study in autologous HSC transplantation in patients with relapsed or refractory Hodgkin lymphoma, 22 patients were given a combination of 10µg/kg G-CSF daily, plus 240µg/kg subcutaneous plerixafor 10-11 hours before apheresis.2 Results were compared with a historical control group who were given G-CSF alone. Two-thirds of the subjects met the primary endpoint of collecting at least 5x106 CD34+ cells/kg, and all bar one the minimum collection of 2 x106. This was a significant improvement over the historical control, and pharmacokinetics were similar to those seen in healthy volunteers.

Phase III trials have also been carried out. In one, 288 patients with non-Hodgkin's lymphoma undergoing autologous HSC transplantation were given 10µg/kg G-CSF for up to eight days, and then for four days from day four were also given 240µg/kg or placebo, with apheresis taking place daily from day five.3 The primary endpoint of 5x106 CD34+ cells/kg was met by 59% of those in the plerixafor group, and 20% of those given placebo.

As an add-on to this trial, patients on either arm who failed mobilisation were eligible for an open-label rescue protocol.4 These patients - 10 from the plerixafor arm and a further 52 from the placebo group - were given the same schedule again after a minimum seven-day rest period. Four of the pre-treated patients and 33 from the placebo group mobilised sufficient HSCs for transplantation. Common side-effects in the plerixafor group included mild gastro-intestinal effects and injection site reactions.

References

1. G. J. Bridger et al. J. Med. Chem. 1995, 38, 366

2. A. Cashen et al. Biol. Blood Marrow Transplant, 2008, 14, 1253

3. J. F. DiPersio et al. J. Clin. Oncol. 2009, 27, 4767

4. I. N. Micallef et al. Biol. Blood Marrow Transplant, 2009, 15, 1578

See more

You may also like

Research & Development

Quotient Sciences extends partnership for ultra-rare disease treatment

Read more
This collaboration reinforces Quotient Sciences’ commitment to supporting the manufacture of niche and smaller volume commercial products often overlooked by larger CDMOs

Trending Articles

  1. Echo MS+ integration enhances high-throughput mass spectrometry SCIEX expands compatibility with the ZenoTOF 8600 system to deliver greater sensitivity and automation capabilities
  2. You need to be a subscriber to read this article.
    Click here to find out more.
    Landmark NHS‑Galleri trial cuts Stage IV cancers and boosts early detection across England NHS‑Galleri trial shows multi‑cancer blood test sharply reduces late‑stage diagnoses and boosts early detection—possibly signalling a structural change in cancer drug demand, supply chain planning and long-term oncology production strategy
  3. CARBOGEN AMCIS and Celonic unveil integrated ADC development platform at World ADC London 2026 The strategic alliance combines biologics and highly potent payload capabilities to streamline ADC development and manufacturing
  4. Promega unveils cellular target engagement technology TarSeer BRETSA platform enables early-stage studies for difficult proteins in drug discovery
  5. IMed Group appoints Spencer Keegan as Talent Acquisition Manager IMed confirms expansion of business and implements new recruitment services for clients

Upcoming event

DCAT Week

23–26 March 2026 | Conference | New York, US See all

You may also like

Research & Development

Quotient Sciences extends partnership for ultra-rare disease treatment

This collaboration reinforces Quotient Sciences’ commitment to supporting the manufacture of niche and smaller volume commercial products often overlooked by larger CDMOs
Research & Development

OSE Immunotherapeutics receives positive IDMC review for Phase III ARTEMIA trial

OSE Immunotherapeutics SA has received a second positive recommendation from the Independent Data Monitoring Committee to continue its Phase III ARTEMIA trial of Tedopi in advanced non-small cell lung cancer
Research & Development

Vilnius University scientists reveal new role for CRISPR-Cas9 in bacterial immunity

The researchers report in Molecular Cell that the CRISPR-Cas9 protein plays a previously unknown role in selecting and integrating viral DNA fragments during bacterial immune memory formation
Research & Development

Lilly’s Orforglipron beats oral semaglutide in Phase III ACHIEVE-3 trial with superior weight loss

Head-to-head data published in The Lancet show Eli Lilly's oral GLP-1 candidate orforglipron delivered significantly greater HbA1c reductions and weight loss than oral semaglutide in adults with type 2 diabetes, supporting global regulatory submissions
Regulatory

CStone's sugemalimab approved by MHRA for Stage III NSCLC

The MHRA's decision marks the biopharma's second indication approved for the product in the UK, with a global commercial rollout for sugemalimab now actively underway
Regulatory

American Chemistry Council's statement to Trump: make chemistry in America again

The Council has released a statement ahead of the President's State of the Union Address, asking for policies that unlock domestic production and address unfair foreign practices
Regulatory

Johnson & Johnson files FDA application for IMAAVY in wAIHA

Johnson & Johnson has submitted an sBLA to the US Food and Drug Administration seeking approval of IMAAVY as the first-ever treatment for warm autoimmune haemolytic anaemia, supported by Phase II/III ENERGY trial data showing rapid and durable haemoglobin response
Subscribe now