The Australian non-profit organization FightMND has awarded Lauren Sciences a AU$1 million ($710,000 US) grant to further the biotechnology company’s development of a new treatment for amyotrophic lateral sclerosis (ALS).
Specifically, the New York-based biotechnology company will use the funds to advance LAUR-301, a medicine that aims to reverse the progressive neurological disorder, rather than just postpone its progression. No such treatment currently exists.
Lauren Sciences specializes in medicines for neurodegenerative disorders using its V-Smart drug delivery platform. LAUR-301 is a V-Smart nanomedicine that targets brain areas affected by motor neuron death in ALS.
The pre-clinical therapy is crafted to cross the blood-brain barrier (BBB) by non-invasive administration, then to selectively deliver agents into brain areas affected by ALS degeneration, according to the company website.
It’s customized with therapeutic agent glial cell derived neurotrophic factor (GDNF), a neurotrophic protein that cannot cross the barrier by itself, which has been shown to protect motor neurons and help preserve the neuromuscular junctions — the contact point between a motor nerve cell and a muscle fiber. It has improved outcomes of ALS animal models.
The grant project will study the therapeutic benefit of LAUR-301 in an ALS mouse model. The hope is that the U.S. Food and Drug Administration (FDA) ultimately will approve Lauren Sciences’ priority therapy.
”We commend FightMND for sharing our vision and commitment to LAUR-301,” Susan Rosenbaum, chairman and CEO of Lauren Sciences, said in a press release. “The FightMND award confirms LAUR-301’s success to date, ability to overcome the BBB [blood-brain barrier] delivery challenge, and value as a potentially life-saving therapeutic for all ALS patients.”
For its part, FightMND is excited about the potential treatment’s prospects.
”LAUR-301 has promise as an effective new treatment for ALS patients,” said Irwin Hollander, PhD, vice president of the organization’s research and development. “LAUR-301 will likely be a disease-modifying drug, unlike standard treatments that, in most patients, merely delay disease progression.”
ALS affects motor neurons in the brain and spinal cord, ultimately causing the loss of the ability to initiate and control muscle movement. According to the press release, more than 2,000 Australian residents live with ALS (also known as motor neuron disease). It also said that, based on population studies, some 5,600 U.S. residents are diagnosed with the disease annually.
The goal of FightMND is to find effective treatments and eventually a cure for the disease. The organization is the largest independent funding source of motor neuron disease in Australia.