(This is an excerpt of the Health Rounds newsletter, where we present latest medical studies on Tuesdays and Thursdays.)
By Nancy Lapid
(Reuters) -An experimental antibody drug being developed by Jasper Therapeutics in a small early-stage trial allowed children who needed stem cell transplants to skip highly toxic chemotherapy and radiation regimens that are typically required beforehand.
Normally before a stem cell transplant, diseased bone marrow must be cleared out in a process that can cause significant nausea, hair loss, and immunosuppression along with long-term issues including infertility and damage to the liver and kidneys.
Jasper’s briquilimab successfully eliminated patients’ stem cells without causing toxic side effects, researchers reported in Nature Medicine.
Briquilimab targets CD117, a protein found on blood-forming stem cells that controls their growth and development.
Researchers at Stanford Medicine in California tested the new drug in three children with Fanconi anemia, a rare genetic disorder. But the researchers say it could also benefit people with other genetic conditions requiring transplants.
Each patient received a single infusion of the antibody 12 days before they were scheduled to receive stem cells. No one’s immune system rejected the donated cells, and by 30 days after transplant, the healthy cells from the donors had almost completely taken over the patients’ marrow.
The researchers’ initial goal was to help patients reach 1% donor chimerism, meaning 1% of the bone marrow cells would come from the donor. Two years later, all three patients have close to 100% of their cells from their donors, far better than expected. All are doing well, according to the researchers.
A report of the trial notes that in all cases, the stem cells were donated by one of the patient’s parents.
Children typically cannot receive stem cell transplants from parents because their genes don’t match exactly. In these cases, researchers removed immune cells that can trigger the donor cells to attack the recipient from the donated bone marrow.
The researchers are conducting a mid-stage trial of the same protocol in additional children with Fanconi anemia. They also plan to test it in other genetic conditions.
Another team at Stanford Medicine is testing whether the antibody can help elderly cancer patients who can’t tolerate full doses of radiation or chemotherapy because they are fragile or have diseases in addition to cancer, according to the researchers.
OSTEOPOROSIS RISK FACTORS IN MEN WARRANT ATTENTION
Outreach to male patients with osteoporosis risk factors would likely reduce fracture-related illness and death in men, a new study suggests.
“Although (low bone density) is often considered a disease of women, a man at age 50 is actually more likely to die from complications of a fracture than of prostate cancer in his remaining lifetime,” said study leader Dr. Cathleen Colon-Emeric from Duke University School of Medicine in North Carolina.
“But unlike the clear guidelines for osteoporosis screening in women over age 65, there is no consensus about whether or not to screen men for osteoporosis before they have had a broken bone.”
For the study, nurses reviewed U.S. Veterans Affairs Health System electronic health records to identify 3,112 men ages 65 to 85 with at least one risk factor for fracture. These include arthritis, alcohol dependence, chronic lung disease, chronic liver disease, stroke, prostate cancer, smoking, diabetes, and use of certain medications.
In half the cases, the nurses notified the patients and coordinated their bone health testing and treatment remotely.
Osteoporosis screening rates in the bone health services group were 49%, compared to 2% in the usual care group, according to a report of the study published in JAMA Internal Medicine.
Just over half of those screened had osteoporosis or its precursor, osteopenia.
More than 90% of men in the bone health services group who needed osteoporosis treatment kept up with their medication plan, the researchers reported.
“We found that screening men over age 65 who have at least one additional risk factor identified a similar proportion of individuals who need osteoporosis treatment to prevent broken bones as does screening women over age 65,” Colon-Emeric said.
“The bone health model resulted in large improvements in screening, treatment, adherence rates compared to usual care,” she said. “Implementing this care model within health systems or large practices is likely to reduce hip and other devastating fractures for older adults at risk.”
ONCE-A-MONTH HIV PREP PILL BEING TESTED IN HUMANS
An experimental pill from Merck & Co for protecting against HIV infection would only need to be taken once a month, laboratory experiments suggest.
Currently available HIV pre-exposure prophylaxis (PrEP) pills must be taken daily. Longer-acting PrEP therapies are available but require injection by a healthcare provider.
The active ingredient in the new pill, dubbed MK-8527, belongs to a class of medications called nucleoside reverse transcriptase translocation inhibitors, which employ several mechanisms to block replication of viruses, including HIV.
The Merck pill is currently being tested in a mid-stage trial in humans, the researchers said.
“Monthly oral pre-exposure prophylaxis could provide a convenient option with the potential to improve adherence compared to daily HIV pre-exposure prophylaxis,” they wrote in a report of their laboratory and animal experiments in PLoS Biology. It could also reduce the barrier for some patients of accessing frequent healthcare visits to get long-acting injectable HIV PrEP regimens, they added.
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(Reporting by Nancy Lapid; Editing by Bill Berkrot)
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