Seeking A CGD Cure By Making Transplants Safer

As reported in Biospace, the biotech company Jasper Therapeutics Inc. has announced the start of a Phase 1/2 clinical trial on a potential conditioning agent, JSP191, to facilitate hematopoietic cell transplants, their company’s core mission. While hematopoietic cell transplants are a known cure for chronic granulomatous disease (CGD), the conditioning agents needed to pull off the transplant have serious adverse consequences.
Now, Jasper Therapeutics researchers are putting forward a new conditioning agent, JSP191, into the start of a Phase 1/2 clinical trial, as a new safer agent in the conditioning regimen prior to the transplant.  They hope the new non-toxic agent could remedy the issues associated with the transplants, and help patients with CGD get on their way to a cure.

Chronic Granulomatous Disease

Chronic granulomatous disease (CGD) is a rare genetic immunodeficiency disease, that causes patients to become much sicker from common illnesses.  While those with the condition are otherwise healthy, the disorder causes the body to be more susceptible to fungi and bacteria, heightening risk of infection. Common illnesses include pneumonia, lung infections, skin infections, lymph node infections, liver infections, and gastrointestinal inflammation. It occurs when a type of white blood cell malfunctions, causing the cells to be unable to fend off infection. While there are antibiotics and interferon-gamma injections to kickstart the immune system, the main recommendation is to avoid infections.

Efficacy Issues With Hematopoietic Cell Transplants

While it might seem like a no-brainer to try to seek this once off cure for patients with CGD, such transplants are not easy to pull off. Hematopoietic cell transplants can have serious repercussions, since the conditioning agents needed to gather stem cells for the transplant is genotoxic, possibly causing genetic mutations. It also causes serious side effects and possible adverse effects such as veno-occulusive disease, infertility, and secondary malignancies. The resultant limited efficacy has motivated doctors to keep looking for better methods to carry out the transplant.

A Safer Solution

However, JSP191 is a humanized monoclonal antibody that can rid the bones of hematopoietic stem cells,  opening space for the altered cells to be grafted back in. Jasper Therapeutics hopes the safer conditioning agent can be used for other conditions that can be remedies with hematopoietic stem cell transplants, such as severe autoimmune disease, sickle cell disease and Fanconi anemia. It has so far been evaluated on 90 volunteers and patients, and the company will be testing it in two other clinical trials for rare condition.
Dr. Kevin N. Heller, Executive Vice President, Research and Development, of Jasper Therapeutics, said,
“We look forward to collaborating with NIAID on this Phase 1/2 clinical trial, which should provide important information about the potential of JSP191 as a safer and more effective conditioning agent for patients with CGD undergoing hematopoietic stem cell transplant. Through this clinical trial agreement with NIAID, as well as others with the National Institutes of Health and academic centers, we are continuing to develop JSP191 for additional pre-transplant conditioning regimens beyond severe combined immunodeficiency and acute myeloid leukemia/myelodysplastic syndromes, which have demonstrated safety and efficacy in early-stage clinical trials to date.”

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