How is Stanford ending toxic chemotherapy for stem cell transplants?
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How is Stanford ending toxic chemotherapy for stem cell transplants?

Stanford develops a novel antibody approach to replace toxic chemo and radiation in stem cell transplants, improving safety for vulnerable patients.

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By MoneyOval Bureau

3 min read

Image Credit: Stanford.edu
Image Credit: Stanford.edu

A groundbreaking development at Stanford Medicine is redefining how patients are prepared for stem cell transplants. Traditionally, patients undergo chemotherapy or radiation to clear diseased bone marrow before accepting donor cells.

These treatments, however, bring severe toxic side effects. Stanford’s new approach replaces such harsh methods with a novel antibody treatment.

The recent Phase 1 clinical trial used an antibody called briquilimab to selectively target and eliminate blood-forming stem cells.

This allowed transplant preparation without the need for toxic chemotherapy or radiation, marking a critical advance, especially for fragile patients with conditions like Fanconi anemia.

What is Stanford’s new method for stem cell transplantation?

Stanford’s team developed an antibody-based regimen targeting a protein called CD117, present on blood stem cells. Instead of blasting the bone marrow with radiation or chemotherapy, patients receive an infusion of briquilimab that clears out their stem cells safely.

This makes room for donor cells to engraft without exposing patients to harmful radiation or genotoxic drugs like busulfan.

The trial focused on three children with Fanconi anemia, a genetic disorder that normally makes traditional transplants highly risky.

Each child received a single dose of briquilimab followed by an infusion of stem cells donated by a parent.

The donated cells were enriched for blood-forming stem cells and depleted of immune cells that could cause transplant rejection or graft-versus-host disease.

Did you know?
Fanconi anemia patients typically develop life-threatening bone marrow failure by age 12, making early and safer transplants critical for survival.

How does the antibody-based treatment improve patient safety?

By avoiding chemotherapy and radiation, this method significantly reduces treatment-related toxicity. Fanconi anemia patients are particularly vulnerable to the damaging side effects of conventional transplant conditioning, making this gentler approach a major breakthrough.

Two years after treatment, all patients in the trial showed near-complete donor cell replacement in their bone marrow without graft rejection.

This success underscores both the antibody’s effectiveness in clearing host stem cells and the enriched donor graft’s compatibility.

Moreover, patients avoided many complications common with traditional stem cell transplant procedures.

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Stanford’s innovative transplant approach benefits fragile patients

This new regimen enables safer access to transplants for children with fragile health conditions. One notable success is 11-year-old Ryder Baker, who underwent this treatment in early 2022.

Before the transplant, Ryder suffered from severe fatigue and vulnerability to infections. Now, he is active and thriving, illustrating the transformative potential of the therapy.

Doctors note that patients and their families feel hopeful when they see a treatment option that alleviates the toxic burden of chemotherapy and radiation.

This approach also widens access by allowing half-matched donors, such as parents, to donate safely thanks to the specialized preparation of donor cells.

Advances expand donor options and reduce transplant toxicity

Historically, about 35-40% of patients needing transplants lacked fully matched donors. Researchers at Stanford progressively refined the transplant process by removing harmful donor T-cells and enriching blood-forming cells.

This innovation, combined with antibody conditioning, vastly expands the donor pool and lessens post-transplant complications.

Future research plans include applying this antibody technique to other genetic marrow failure diseases and possibly certain fragile cancer patients who cannot tolerate conventional transplant conditioning.

Stanford’s long-term goals are to revolutionize transplant safety and accessibility for many more patients.

The antibody regimen marks a paradigm shift in transplant care, a future where stem cell transplants can be performed safely without toxic chemo or radiation, giving patients a chance for improved quality of life and survival.

Do you believe antibody-based stem cell transplants will become the new standard?

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