Aspen Neuroscience Receives $6.5M to Advance New Patient-specific Cell Therapy for Parkinson’s

Aspen Neuroscience

Aspen Neuroscience, a new biotech company, has raised $6.5 million to develop cell therapies for Parkinson’s disease using patients’ own cells.

The company was co-founded by renowned stem cell scientists Jeanne F. Loring, PhD, and Andres Bratt-Leal, PhD, and initially supported by Summit for Stem Cell, a non-profit organization that provides a variety of services for Parkinson’s patients.

Parkinson’s hallmark motor symptoms include tremor, slowness of movement (bradykinesia), stiffness (rigidity), uncontrollable movements (dyskinesia), and poor balance.

As the disease progresses, patients typically need to gradually increase their dopaminergic therapeutic dose for maximum benefit. Even after that they might sometimes experience reappearance or worsening of symptoms due to diminishing effects of dopaminergic therapy, known was “off” periods.

Importantly, dopaminergic therapy is delivered to areas of the brain other than the striatum, a key motor control region severely affected in Parkinson’s disease. Because of the therapy’s off-target behavior, patients also may experience side effects such as hallucinations or cognitive impairment.

Aspen wants to combine its expertise in stem cell biology, genomics and neurology and develop the first autologous (self) stem cell-based therapy for Parkinson’s disease.

In this type of cell therapy, a patient’s own cells (usually skin cells) are reprogrammed back into a stem cell-like state, which allows the development of an unlimited source of almost any type of human cell needed, including dopamine-producing neurons, which are those mainly affected by this disorder.

Because these cells are derived from patients, they do not carry the risk of being rejected once re-implanted, eliminating the need for immunosuppressive complementary therapies, which carry serious side effects such as infections and possibly limiting therapeutic potential.

In theory, replacing lost dopaminergic neurons with new stem cell-derived dopamine-producing ones could potentially ease or reverse motor symptoms associated with the disease.

Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinson’s disease,” Howard J. Federoff, MD, PhD, Aspen’s CEO, said in a press release.

“We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist,” Federoff said.

The company’s lead product (ANPD001) is undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinson’s disease. Aspen experts also are developing a gene-editing treatment (ANPD002) for familial forms of Parkinson’s, starting with the most common genetic variant in the GBA gene, which provides instructions to make the enzyme beta-glucocerebrosidase.

The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32, according to the press release.

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Brain Disorders Remain Largely Untreated, European Study Reveals

brain disorders

The majority of patients — eight of 10 — diagnosed with a brain disease for which there are available therapies remain untreated, according to data from the Value of Treatment study of the European Brain Council (EBC).

That finding and more were shared and discussed recently at the 4th Congress of the European Academy of Neurology (EAN), June 16-19 in Lisbon, Portugal.

The social and economic burdens associated with brain diseases are about €800 billion, or $921 billion, a year in Europe, and steadily increasing, with estimates that one-third of all European citizens — 179 million people — are living with a brain disorder that includes neurological or psychiatric conditions.

“Healthcare and welfare systems are often inadequately organized and have trouble keeping up with the rapid pace of medical advances,” Wolfgang H. Oertel, PhD, University of Marburg, Germany and one of the session chair’s at the conference, said in a press release.

Moreover, failure to correctly diagnose a disease only adds to the costs and the patient’s personal burden, with wasted time and resources.

“Early recognition, starting treatment as soon as possible and preventive measures would serve to minimize the risks or may even in some instances slow the progression of the disease,” Oertel added.

The Value of Treatment study is the result of two years of research with patient-centred questions at its core: What are the barriers to optimal treatment?; What are the financial costs for guaranteeing brain disroders patients with quality medical and psychosocial care?

The study, undertaken by the European Brain Council — an organization composed of several European professional medical societies and patient associations for brain disorders — evaluated clinical data to assess the benefits of individual treatments for specific patient groups.

It included case studies of nine brain disorders (Parkinson’s disease, Alzheimer’s disease, epilepsy, headache, multiple sclerosis, normal pressure hydrocephalus, restless legs syndrome, schizophrenia and stroke) from several patients across Europe.

Researchers evaluated patients’ outcomes and financial burdens when treated with the best therapeutics available, compared to standard treatment or no treatment.

The results showed that early interventions with the best available treatment were most cost-effective for the long term.

“We clearly saw that an early start to treatment and optimal care costs the least over the long term. Non-treatment is the most expensive variant for diseases such as restless legs syndrome or multiple sclerosis and epilepsy, which affect people from a young age,” said professor Maura Pugliatti at the University of Ferrara, Italy, also a chair at the session discussing the Value of Treatment study.

The Value of Treatment study also identified specific healthcare interventions and where they often fall short of what they set out to achieve. Examples illustrated how patients need to receive close monitoring of care instead of acute treatment alone, and how insufficient social support can affect patients’ and their relatives.

In the Parkinson’s disease Working Group, the Value of Treatment study addressed treatment gaps and unmet needs of Parkinson’s patients, namely delayed or inadequate diagnosis, no adequate treatment, and non-adherence to treatment.

Several recommendations were gleaned from the study’s results:

  • Better information and active involvement of patients and caregivers can help identify the best possible treatment and result in better treatment-adherence;
  • Improved communication and coordination processes can help establish integrated and multi-disciplinary care systems;
  • Each patient should have access to the best possible treatment and therapy at each stage of the disease;
  • The public needs to be better informed about the complexity of Parkinson’s disease and patient needs;
  • More investment in research is required.

“The ongoing economic and financial crises have seen an overall deterioration in access to neurological care in some cities and rural areas due to cutbacks or the introduction of excesses payable by patients. It often takes too long to come up with the right diagnosis and initiate personalized therapies — if at all,” Oertel said.

Early detection and access to the best therapeutic strategies available ensures better health outcomes and lower treatment costs.

“Measurable health gains are linked to early intervention such as better survival rates, fewer complications, lower incidence of disability, improved quality of life and, ultimately, lower treatment costs — all of that would be possible!” Oertel said.

The Value of Treatment calls for “suitable treatment from the occurrence of the first fit onwards — by a suitably qualified specialist. And this is where there are major — yet surmountable — treatment gaps to be filled. We have a humanitarian duty to bridge them to the best of our ability when it comes to neurological conditions,” Oertel concluded.

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Source: Parkinson's News Today