Health Rounds: Experimental cell therapy slows heart failure progression in Duchenne muscular dystrophy
Health Rounds: Experimental cell therapy slows heart failure progression in Duchenne muscular dystrophy Health Rounds is published on Tuesdays and Thursdays. Think your friend or colleague should know about us? Forward this newsletter to them. They can also subscribe here
By Nancy Lapid
July 30 (Reuters) - Hello Health Rounds readers! Today we feature a study of a cell therapy that should delight advocates for Duchenne muscular dystrophy patients, who have been battling for new treatments for years. We also report on a bladder-preserving drug for patients with advanced bladder cancer, and a new and improved way of monitoring Parkinson's disease medication levels.
Drug slows progression of Duchenne muscular dystrophy
An experimental cell therapy being developed by Capricor Therapeutics CAPR.O appears to preserve heart and muscle function in young patients with Duchenne muscular dystrophy, according to data from a late-stage trial.
The results represent a potentially important advance for the condition that primarily affects boys.
The data marks the first time a treatment has been shown to slow or halt not only the loss of upper limb function, but progression of heart failure in boys and young men with Duchenne, researchers said. Duchenne muscular dystrophy is an inherited disease that progressively weakens muscles throughout the body, with heart failure often leading to early death.
The disease is caused by a mutation on the X chromosome.
The treatment, deramiocel, is manufactured from cells taken from healthy hearts donated for transplantation but not usable for that purpose.
In the late-stage trial, 106 participants ages 10 to 22 with advanced disease received either deramiocel or a placebo intravenously every three months for one year.
The therapy slowed weakening of skeletal muscles by 54% and slowed heart dysfunction by 91%, the researchers reported in The Lancet.
“If young people maintain their upper limb function, it will help preserve their ability to do normal activities, such as feeding themselves. The heart benefits could reduce mortality,” study coauthor Dr. Craig McDonald of UC Davis Health said in a statement.
Some patients even experienced a slight improvement in heart function.
The researchers had previously found that infusions of so-called cardiosphere-derived cells regenerated damaged heart muscle after heart attacks. Later, they found these cells secrete packets of RNA molecules that help repair the body’s tissues.
The researchers said they plan to continue studying the therapeutic effects of deramiocel, including whether it can be used to treat other diseases.
Study leader Dr. Eduardo Marbán of Cedars-Sinai Health Sciences University, whose wife is Capricor's CEO, said the Phase 3 study was the first "to show that a cell therapy is effective against a genetic disease, or any type of heart disease.”
Immunotherapy can prevent bladder removal for cancer
A new immunotherapy helped many patients with an aggressive form of bladder cancer avoid surgical removal of their bladders in a late-stage trial.
The treatment kept many patients cancer-free for more than two years and caused mostly mild side effects, according to a report of the study in The Lancet Oncology.
The 115 patients in the study had high-risk bladder cancer that had returned despite standard treatment with bacillus Calmette-Guérin. Instead of surgery to remove the bladder – the usual next step – patients received cretostimogene grenadenorepvec, being developed by CG Oncology.
Two years later, 81% of patients had not needed surgery to remove their bladder, the researchers found.
The treatment produced a complete response, meaning no detectable signs of cancer, in 75% of the patients, with roughly 60% of those complete responses lasting beyond two years, the researchers said.
"Avoiding bladder removal can have a profound impact on daily living. Seeing patients remain cancer-free for years while keeping their bladder is exactly the outcome we've been hoping to achieve," study leader Dr. Mark Tyson of the Mayo Clinic in Scottsdale, Arizona said in a statement.
In one patient, the cancer has not returned for more than four years after completing treatment.
"For patients, this isn't just about treating cancer. It's about preserving quality of life," Tyson said.
Wearable patch helps fine-tune Parkinson’s medication dosing
A fingertip patch that continuously tracks Parkinson's disease medication levels through chemicals in sweat has the potential to greatly improve patients’ quality of life, new data suggest.
Prescribing the right dose of levodopa – the gold-standard drug for managing the loss of motor control caused by Parkinson’s disease — is very challenging. Too little leaves patients unable to move, while too much triggers severe, uncontrollable jerking movements.
Doctors currently rely on patients’ diaries to adjust their doses, but the approach is imprecise.
As reported by the research team in PNAS, the fingertip has a high density of sweat glands, and the patch contains an absorbent gel that acts like a sweat sponge to draw sweat out of the pores.
When levodopa in the patient’s sweat comes into contact with enzymes embedded in the patch, it triggers a chemical reaction, which in turn generates a small, measurable voltage. The voltage serves as an indicator of the patient’s levodopa level: lower voltage signals low levels, while higher voltage signals high levels.
In clinical tests involving both healthy volunteers and patients with Parkinson's, the patch tracked levodopa levels as accurately as standard laboratory blood tests, which take days to return results.
This technology lays the groundwork for a closed-loop system, where a levodopa monitoring patch could communicate with a pump to automatically deliver the precise doses of the drug right when the body needs it, the researchers said.
(Reporting by Nancy Lapid; Editing by Bill Berkrot)