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Biomarkers for autism
NEW YORK—Targeted toward clinical trials in 2019 is QBM-001, biotech accelerator Q BioMed’s autistic spectrum disorder (ASD) drug development program for non-verbal and minimally verbal autistic children. At the same time, Q BioMed also plans to validate key biomarkers and test for a possible companion diagnostic.
“At Q BioMed, we are tackling two obstacles: a lack of genetic information and a lack of diagnostic capabilities for our subgroup,” Robert Derham, Q BioMed's vice president of orphan products, tells DDNews.
“In conjunction with the clinical trial, we have planned to validate the biomarkers, which would allow for one or both to be used as a companion diagnostic,” says Derhanm, “where the test would need to be positive before someone could be treated with QBM-001, if QBM-001 were eventually approved.”
Although the nonverbal or very minimally verbal subgroup is definable by the lack of language development, there is no diagnostic tool to identify the children at risk at an early age, he explains. As such, Q BioMed has vetted diagnostic options over the past 10 months that could offer a way to diagnose children as young as two years old.
“What we have found from independent studies is that there are some overlaying commonalities of the nonverbal children, which lead to the augmentation of deleterious molecules in their bodies,” he says. “QBM-001 targets three different pathways that should ameliorate the buildup and reduce or hopefully normalize the situation so that the children can develop on a more typical path and develop speech.”
If QBM-001 reaches the market, “we will work on establishing diagnostic capabilities to hopefully diagnose infants as young as six months, because as with most disorders, the sooner treatment can be administered, the less chance for long-term damage,” Derham reports. “Independent studies on children that were nonverbal showed that past the age of 24 months, they have an accelerated loss of neurons in their cortex or speech region, and the assumption is, ‘if you don’t use it, you lose it.’”
“Fortunately, at that age the brain still has a lot of plasticity or ability to grow and regenerate, which is why we are aiming to find children as early as two years old for the trial,” he adds. “In addition, we plan on performing genome sequencing on all trial participants to further characterize our target group of children.”
In the meantime, Q BioMed continues to develop its intellectual property, including its patent-pending QBM-001 drug technology, which targets toddlers with pediatric developmental nonverbal disorder. An underlying commonality of this subgroup is elevated blood markers that lead to developmental delay, an autism diagnosis and eventual nonverbal or very minimally verbal capability for the rest of their lives, he says.
According to Derham, “This drug (QBM-001), acts as an allosteric regulator of faulty channels in the brain to potentially alleviate the condition and allow toddlers to actively develop language and speech and avoid life-long speech and intellectual disability of being non-verbal. QBM-001 also reduces inflammation in the brain, and by so doing, can reduce the amount of long-term nerve loss.”
In preparation for the clinical trial, Q BioMed has selected partnerships both with a contract and development manufacturing organization (CDMO) to start manufacturing, and a contract research organization (CRO) that specializes in autism spectrum disorders to finish preclinical studies, submit a pre-IND orphan drug designation filing and continue to secure its intellectual property through patent filings. The identities of the CDMO and CRO will be “announced shortly,” Derham tells DDNews.
“Autism spectrum disorders remain quite complex to diagnose and treat due to the varying differences or heterogeneity that abounds,” Derham says. “Treating autism might be similar to Richard Nixon waging war on cancer in the early 70s and expecting to eradicate it. We know there are thousands of different types of cancers, and similarly, the number of subsets within the autism spectrum will only continue to increase. However, hopefully the autism spectrum never proves to be as segmented as cancer.”
Many parents are “frustrated that a diagnosis comes so late with autism,” Derham notes. “Parents often have that sixth sense that something is not right with their child. And with autism, unless the newborn comes from a high-risk family with a history of autism, most pediatricians or child psychologists do not feel comfortable making a diagnosis of autism until the child is at least two years of age.”
Although more genetic causes are being found, not enough has been done in the past, Derham says. But that is changing, thanks to the Simons Foundation Autism Research Initiative, which funded and started Simons Foundation Powering Autism Research for Knowledge (SPARK), a landmark autism research project in the U.S, whose mission is to speed up research and advance our understanding of autism.
“One of the biggest challenges is identifying more unique subgroups within autism,” Derham says. “As the data accumulates, so do genetic commonalities that allow researchers to identify new subgroups under the autism umbrella. Those commonalities then allow for a diagnostic to be developed.”
Not all individuals who become nonverbal will benefit from QBM-001, Derham says. However, with validated biomarkers, testing from trained specialists and genetic testing, children who fall in this targeted population can be identified, and have a higher likelihood of responding to treatment.
“We are grateful for the feedback from clinicians, patients and their families and medical and scientific advisors,” said Denis Corin, CEO of Q BioMed. “All are excited about the roadmap for QBM-001 over the next six months and look forward to reporting on our progress.”