PRESS-NEWS.org - Press Release Distribution
PRESS RELEASES DISTRIBUTION

Financing innovation: proposal for novel adaptive platform trial fund offers new model for ALS drug development

Researchers propose new royalty-based investment model to bridge the ‘valley of death’ and accelerate drug development

2025-07-02
(Press-News.org) A team of researchers from the MIT Sloan School of Management, the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH), Questrom School of Business at Boston University, and QLS Advisors have introduced a new approach to funding clinical trials for amyotrophic lateral sclerosis (ALS) therapies. The study “Financing Drug Development via Adaptive Platform Trials,” published today in PLOS One, outlines a financing model that merges the efficiencies of adaptive platform trials — lower costs and shorter durations — with an innovative royalty-based investment structure designed to accelerate therapeutic development for ALS and other serious diseases.

ALS — also often called Lou Gehrig’s disease — is a progressive, neurodegenerative disease with no cure. Despite its devastating impact, the pace of new therapy development has remained sluggish — largely due to the high cost, duration, and risks associated with traditional clinical trials. This bottleneck has often discouraged conventional investors, leaving promising research to languish.

To tackle this challenge, the authors propose an investment fund that finances half the cost of an adaptive platform trial in exchange for future royalties from successful drugs that emerge from the trial. Adaptive platform trials allow multiple drug candidates to be tested simultaneously under a single master protocol and results are interpreted on a real-time basis to determine efficacy or futility. Drawing on data from the HEALEY ALS Platform Trial administered by the Healey & AMG Center for ALS and realistic assumptions, their simulated fund generated an expected return of 28%, with a 22% probability of total loss, which may be attractive to more risk-tolerant and impact-driven investors such as hedge funds, sovereign wealth funds, family offices, and philanthropists. Their findings suggest that generating returns more palatable for mainstream investors could be achieved by funding multiple platform trials simultaneously and by employing financial tools such as securitization—a method that bundles future income from assets like loans or royalties into investment products.

The study represents a multidisciplinary collaboration among physicians, clinical trial experts, and  financial engineers including Annette De Mattos, Kristin Drake, Merit E. Cudkowicz, Ricardo Ortiz, Meredith Hasenoehrl, Marianne Chase, Brittney Harkey, and Sabrina Paganoni of the Sean M. Healey & AMG Center for ALS at MGH; Eugene Sorets of Questrom School of Business at Boston University; Shomesh Chaudhuri and John Frishkopf of QLS Advisors; and Joonhyuk Cho and Andrew W. Lo of MIT. Their work bridges the gap between biomedicine and capital markets, laying a new path forward for how lifesaving therapies are financed and delivered.

“ALS clinical trials face significant hurdles—from high costs and long timelines to limited funding pools,” said Dr. Cudkowicz, Executive Director Mass General Brigham Neuroscience Institute and Director of the Healey & AMG Center for ALS. “Our platform trial model has already shown that we can test more therapies more efficiently. What’s still missing is sustainable financing. This novel approach could be a game-changer, enabling us to launch trials faster, include more promising therapies, and bring us closer to our shared goal: delivering effective treatments to people with ALS as quickly as possible.”

Dubbed a “Fund of Adaptive Royalties” (FAR), this model is uniquely positioned to transform how diseases like ALS are addressed, providing developers with access to shared infrastructure, centralized data analytics, and significantly reduced upfront capital requirements while offering investors a diversified, portfolio-based exposure to multiple drug candidates and the potential for high returns. While their study focused on ALS, the authors believe such a funding model could be applied to other disease areas as well, especially those with well-defined endpoints, where treatment success can be measured clearly and reliably, and few existing therapies.

“This financing framework addresses one of the core issues in biomedical innovation—bridging the valley of death between discovery and delivery,” said co-author Lo, MIT Charles E. and Susan T. Harris Professor and director of MIT Sloan’s Laboratory for Financial Engineering. “By aligning incentives between investors and developers and distributing risk across a portfolio of candidates, we can unlock new sources of capital for diseases that urgently need them.”

 

 

About MIT Sloan School of Management

The MIT Sloan School of Management is where smart, independent leaders come together to solve problems, create new organizations, and improve the world. Learn more at mitsloan.mit.edu.

 

About the Sean M. Healey & AMG Center for ALS at Mass General

At the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.

Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD, and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.

With many clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.

 

About the Boston University Questrom School of Business

Founded in 1913, the Boston University Questrom School of Business is a global top-tier academic research business school. Led by Allen Questrom Professor and Dean Susan Fournier, Questrom develops business leaders who create value for the world. Questrom redefines transformational business programs, strengthens partnerships with the business community, advances the impact of research on business, and manages the school as a high-performing enterprise committed to excellence with a service mindset. Comprising a renowned full-time faculty of 165 researchers, teaching faculty, and accomplished practitioners, Questrom generates insights to address today’s business challenges and prepares students with the tools they need to succeed from Day 1 in their professional lives. Questrom’s portfolio of academic programs is robust and includes a Top 20 undergraduate program of over 2,200 students; distinctive MBA offerings including 900 students in a full- and part-time MBA, the affordable Online MBA and specialty MBAs in social impact, health, and digital technology; several thriving specialized masters programs in areas including business analytics, mathematical finance, and management studies; and a rigorous PhD program. More than 50,000 Questrom alumni form a powerful global network of leaders driving value creation that changes the world. QUESTROM MEANS BUSINESS. For more information, visit bu.edu/questrom.

 

About QLS Advisors

QLS is an investment manager that is dedicated to improving outcomes for both patients and investors. We aim to achieve these goals by applying the tools of financial engineering, including modern portfolio theory, machine learning, and healthcare finance to our discretionary investment process. At QLS, we believe that quantitative and fundamental techniques are not mutually exclusive but can be combined to build enhanced portfolios in the healthcare space.

END


ELSE PRESS RELEASES FROM THIS DATE:

Disparities in treatment and referral after an opioid overdose among emergency department patients

2025-07-02
About The Study: In this study, Black patients were less likely to receive outpatient referrals for opioid use disorder. These findings underscore the need for targeted interventions to address racial disparities in emergency department care for opioid use disorder, particularly in enhancing referral processes.  Corresponding Author: To contact the corresponding author, Siri Shastry, MD, MS, email Siri.Shastry@mountsinai.org. To access the embargoed study: Visit our For The Media website at this link https://media.jamanetwork.com/ (doi:10.1001/jamanetworkopen.2025.18569) Editor’s Note: Please ...

Was Mars doomed to be a desert? Study proposes new explanation

2025-07-02
One of the great unsolved problems in modern planetary science is written on the surface of Mars. Mars has canyons that were carved by rivers, so it was once warm enough for liquid water. How—and why—did it become it a barren desert today? A study led by University of Chicago planetary scientist Edwin Kite puts forth a new explanation for why Mars never seems to stay balmy for long. Published July 2 in Nature, their model suggests that the periods of liquid water we see in the past were initiated by the sun brightening, and that conditions on Mars mean it ...

Study highlights major hurdles for multinational clinical trials in Europe

2025-07-02
Study Highlights Major Hurdles for Multinational Clinical Trials in Europe A new study by investigators from Europe, including the Netherlands and the United Kingdom (UK), has shed light on significant ethical, administrative, regulatory, and logistical (EARL) hurdles in delivering multinational randomized clinical trials. The research was the first to comprehensively quantify these barriers for an international platform trial and emphasizes the need for urgent improvements, particularly in preparing for future public health crises. Randomized controlled trials provide the highest level of evidence to inform medical practice. Yet, delivering such trials ...

Chemistry breakthrough has potential to make more effective cancer drugs with less harmful side effects

2025-07-02
Chemists have discovered for the first time a unique way to control and modify a type of compound widely used in medicines, including a drug used to treat breast cancer. The research, led by the University of Bristol and published today in the journal Nature, also found a new mechanism associated with the chemical reaction which enables the shape of the compound to be flipped from being right-handed to left-handed by simply adding a common agent in the chemical reaction. Study lead author Varinder Aggarwal, Professor of Synthetic Chemistry at the University of Bristol, said: “The findings change our understanding of the fundamental chemistry of this group of organic ...

Researchers identify new protein target to control chronic inflammation

2025-07-02
Chronic inflammation occurs when the immune system is stuck in attack-mode, sending cell after cell to defend and repair the body for months or even years. Diseases associated with chronic inflammation, like arthritis or cancer or autoimmune disorders, weigh heavily on human health—and experts anticipate their incidence is on the rise. A new study by investigators from Mass General Brigham identified a protein called WSTF that could be targeted to block chronic inflammation. Crucially, this strategy would not ...

Increasing contingency management incentives will help more patients recover from addiction

2025-07-02
Early recovery from drug addiction to opioids and stimulants is physically and mentally demanding, and a long road to recovery. “During the early stages of addiction recovery there is typically not much that is positive for patients,” shares behavioral health counselor Carla J. Rash, Ph.D. of UConn School of Medicine. “But Contingency Management is an effective, behavioral tool bringing some early-on positivity to a patient’s addiction recovery treatment plan until the positive benefits of their medication and body’s natural recovery kicks-in.” Rash adds, “Essentially, ...

Changes in the blood could protect against Alzheimer’s disease

2025-07-02
A study published in Cell Stem Cell reveals that some mutations in blood stem cells might help protect against late-onset Alzheimer’s disease. A team led by researchers at Baylor College of Medicine discovered that both a mouse model and people carrying blood stem cells with mutations in the gene TET2, but not in the gene DNMT3A, had a lower risk for developing Alzheimer’s disease. Their study proposes a mechanism that can protect against the disease and opens new avenues for potential strategies to control the emergence ...

New tool allows researchers to track assembly of cells’ protein-making machines

2025-07-02
Proteins are the infinitely varied chemicals that make cells work, and science has a pretty good idea how they are made. But a critical aspect underlying the machinery of protein manufacture has long been hidden inside a blobby cellular structure called the nucleolus. Now, a team of Princeton engineers have developed a technique to peer inside the nucleolus and reveal this hidden system of creation. Previous methods required researchers to break open the cell and destroy most of its structures, resulting in minimal access to the blob’s inner workings. By tracking the movement of RNA molecules inside the nucleolus using advanced imaging and genomics techniques, ...

New genetic marker linked to improved survival with immunotherapy in ovarian and other cancers 

2025-07-02
Ovarian clear cell carcinoma is difficult to treat, and treatment options are limited  Patients with specific PPP2R1A mutations in their tumors survived significantly longer after immunotherapy treatment  Targeting PPP2R1A may improve responses even further according to laboratory studies   PPP2R1A is an important predictive biomarker and possible treatment target for multiple cancer types, study found  HOUSTON, JULY 2, 2025 ― Patients with ovarian clear cell carcinoma (OCCC) whose tumors have specific mutations in the PPP2R1A gene were found ...

AI that thinks like us – and could help explain how we think

2025-07-02
Researchers at Helmholtz Munich have developed an artificial intelligence model that can simulate human behavior with remarkable accuracy. The language model, called Centaur, was trained on more than ten million decisions from psychological experiments – and makes decisions in ways that closely resemble those of real people. This opens new avenues for understanding human cognition and improving psychological theories. For decades, psychology has aspired to explain the full complexity of human thought. Yet traditional models could either offer a transparent ...

LAST 30 PRESS RELEASES:

Enhancing the “feel-good” factor of urban vegetation using AI and street view images

A single genetic mutation may have made humans more vulnerable to cancer than chimpanzees

Innovative nanocomposite hydrogel shows promise for cartilage regeneration in osteoarthritis treatment

2025 Guangci Laboratory Medicine Innovation and Development Conference

LabMed Discovery is included in the ICI World Journals database

LabMed Discovery is included in the China Open Access Journal (COAJ) database

Vaccination support program reduces pneumonia-related mortality by 25 percent among the elderly

Over decades, a healthy lifestyle outperforms metformin in preventing onset of Type 2 diabetes

Mental health disorders, malaria, and heart disease most affected by covid pandemic

Green transition will boost UK productivity

Billions voted in 2024, but major new report exposes cracks in global democracy

Researchers find “forever chemicals” impact the developing male brain

Quantum leap in precision sensing across technologies

Upgrading biocrude oil into sustainable aviation fuel using zeolite-supported iron-molybdenum carbide nanocatalysts

For effective science communication, ‘just the facts’ isn’t good enough

RT-EZ: A golden gate assembly toolkit for streamlined genetic engineering of rhodotorula toruloides

Stem Cell Reports announces five new early career editors

Support networks may be the missing link for college students who seek help for excessive drinking

The New England Journal of Medicine shines spotlight on forensic pathology

Scientists discover protein that helps lung cancer spread to the brain

Perceived social status tied to cardiovascular risks in women but not in men

Brain tumor growth patterns may help inform patient care management

This might be America's first campus tree inventory

Emoji use may impact relationship outcomes

Individual merit, not solidarity, prioritized by early childhood education policies

Preclinical study unlocks a mystery of rapid mouth healing

Extraterrestrial habitats: bioplastics for life beyond earth

U.S. military spending reductions could substantially lower energy consumption

Air pollution is linked to adverse birth outcomes in India

Using viral load tests to help predict mpox severity when skin lesions first appear

[Press-News.org] Financing innovation: proposal for novel adaptive platform trial fund offers new model for ALS drug development
Researchers propose new royalty-based investment model to bridge the ‘valley of death’ and accelerate drug development