Controlling the Immune Response: Biologic Therapy Can Prevent Bone Marrow Transplant Complications


biologic therapy
Is biologic therapy the solution to preventing unexpected, negative side effects of bone marrow transplants?
Image source: Flickr CC user Zdenko Zivkovic

For decades, bone marrow transplants have successfully treated patients with blood-related cancers and genetic diseases. Despite how effective it can be as a cure, however, the procedure comes with its fair share of risks. The one most people are familiar with is, of course, outright failure of the tissue graft to take. The process to find a potential donor can be arduous, so an unsuccessful transplant where the grafted cells fail to thrive can be discouraging. But imagine the other side of that scenario: the case in which the donor cells thrive so well they end up attacking the host’s body.

Graft-versus-host disease (GvHD) is a condition in which the donor’s bone marrow cells reject the host’s body in an immunogenic reaction that can cause skin blisters, organ failure, and hemorrhaging. To make matters worse, acute cases in children have an 80% mortality rate.1 Despite the severity of the disease, there is a recourse for treatment. Patients with GvHD typically take steroids or immunosuppressants. The problem is that both of these options are imperfect. Steroids are only 30-50% effective and the immunosuppressants are used off-label and toxic. So while existing treatments may help patients with GvHD, they may also lead to more complications on top of those caused by the disease.

Stem Cell Biologic Therapy as a Method to Treat Cases of Severe GvHD

Given these circumstances, some companies focused their efforts on developing a biologic therapy for GvHD and after twenty years, they finally succeeded. Three years ago, Prochymal became the first drug approved for market authorization to treat acute graft-vs-host disease (GvHD) in children. The stem cell product is an especially attractive form of therapy because it can be used without type matching or immunosuppressing the patient. That means Prochymal formulations derived from a single donor can be given to several different recipients, increasing the percentage of people who can benefit.

Despite this, Prochymal isn’t intended for all people with GvHD. After all, it is an intravenous formulation. If steroids and immunosuppressants prove effective, there’s no need to subject patients to an invasive procedure. For people who are unresponsive to traditional steroids and immunosuppressants though, Prochymal can significantly raise survival rates—most notably in those with extremely severe cases of GvHD.

Companies Can Take Inspiration from Existing Stem Cell Drugs to Develop New Biologic Therapy Products

Not only was Prochymal the first biologic therapy approved to treat GvHD, it was also the first stem cell drug to ever receive market authorization. The field of stem cell therapies has long been viewed as one that’s failed to meet expectations, so Prochymal’s success injected new hope into the sector. Since 2012, twelve stem cell products have been received marketing approval worldwide to treat a variety of diseases.2 Hundreds more are currently in clinical trials. Does this mean the time for stem cell drugs has finally arrived?

The upward tick in stem cell products approvals and clinical trials isn’t the only indication this may be the case. A couple months ago, stem cell pioneer Ocata Therapeutics was acquired by Astellas Pharma, a major Japanese drug company.3 Many consider Ocata to stand at the forefront of stem cell-derived research, which is something that Astellas intends to capitalize upon and transform into therapies that can be brought to market. The Japanese company’s commitment to this aim could in fact re-energize the sector and encourage other firms to follow suit.

Considering the time and investment involved in developing stem cell biotherapeutics, however, it’s crucial for companies to select promising products. Considering once more Prochymal, its success grew out of the fact that it treated patients who didn’t respond to current treatment methods. Other life sciences firms can adopt similar strategies and focus on specific demographics within a patient group afflicted by a condition. Doing so will increase their chances of finding success and market advantage.

Biologic therapy based on stem cells also have another advantage: their treatment mechanism allows them to be a novel therapy for other conditions. Prochymal, for instance, is also undergoing clinical trials for irritable bowel disease, diabetes and the treatment of heart attacks. When creating stem cell-based biotherapeutics to address adverse transplant complications, R&D laboratories can select therapies that demonstrate versatile indications. Not only would this diversify their company’s product offerings, it would also protect their market positioning. Both of these factors are necessary to remain competitive.

The BIOVIA Biologics Solution is a complete suite of tools that supports life sciences companies in their R&D efforts to create innovative biologic therapy products. Whether derived from stem cells or other biological material, discovering and manufacturing new biotherapeutics requires the ability to process large volumes of data effectively and glean crucial insights from the information in order to make key decisions more efficiently. As an example, decision-makers can determine the developability and versatility of a potential therapeutic earlier in the R&D cycle, streamlining workflows by allowing companies to concentrate on only the most promising products sooner. If your life sciences company is interested in optimizing your biologics research, development and manufacturing processes, then please contact us today to learn more.

  1. “Prochymal – First Stem Cell Drug Approved,” May 22, 2012,
  2. “Stem Cell Market Global and Chinese Analysis for 2015-2018,” November 26, 2015,
  3. “Astellas Pharma to Acquire Ocata Therapeutics,” November 10, 2015,

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