Here’s a tale of two diseases that many people thing are alike, but are really different, in so many ways. Multiple Sclerosis (MS) and Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s Disease) has some similar symptoms, but far different causes (the differences are numerous).
And more importantly, the attitude of the scientific community and the public to them is different. MS gets lots of funding, with new tests and studies coming out all the time. There are a plethora of drugs for MS nowawdays – Betaseron, Copaxone, Avonex and others that work to modify the disease. While there is no cure for MS, patients can hold on for many years – and scientists are very hopeful they will be able to conquer the disease altogether.
The story for ALS is far different. ALS is an “orphan disease” – one that affects few people. In the U.S., orphan diseases are defined as those that affect fewer than 200,000 people. Interestingly, both MS and ALS have similar “infection rates” – about 2 in 100,000 in the U.S. annually – but there are a lot more people with MS than with ALS, because the former tend to survive for many years, even though they’re sick, while those with ALS generally die within five years of contracting the disease. Altogether, as many as one in 1,000 people can be expected to develop either MS or ALS.
One consequence of ALS’ status as an orphan disease is the lack of treatment available for it. Because there’s less of a market, there has been – at least in the past – less research into cures for ALS. In fact, there is currently only one treatment on the market for ALS, called Riluzole, which has a very limited effect on patients – extending their lives by a half year or less. It sounds heartless, but the Pharma business is just that – and the MS market is $15 billion annually, while the ALS market is only $3 billion. While more work has been done in the past decade, ALS is still a “mystery” disease, with not enough research being done to find a cure.
But despite its status as an orphan disease, ALS actually presents a business opportunity for Pharmaaceutical companies, says Eran Ovadia, CEO of Immunity Pharma. Ovadia is handling the development side of a treatment for ALS that is based on research by famed Israeli scientist Irun Cohen of the Weizmann Institute.
In 2004, while doing research on other diseases, Cohen made a chance discovery that may significantly increase the chances of survival for ALS patients;the IPL344 peptide that helps cells survive various toxic conditions. The peptide shows great promise in triggering a self-healing cascade to ultimately save the motor neurons that control voluntary movement. It activates signaling pathways that circumvent the disease’s destruction of cellular self-healing mechanisms – thus breaking the vicious circle typical to the disease and attenuating disease progression.
“It’s not a cure,” says Ovadia, “but it’s definitely a step in the right direction. The peptide significantly slows progression of the disease in its middle stages, enabling the patient to survive for months, or even years, longer - during which time, hopefully, a complementary treatment will be developed that will further help patients, thanks to the unprecedented research into ALS going on right now.”
Immunity Pharma has been conducting tests in mice that have the SOD1 gene, mutations of which have been clearly linked to ALS – and top experts in the field, including Professor Stanley Appel of Cornell and Profesor Robert Brown of the University of Massachusetts, were enthusiastic enough about Immunity Pharma’s research to join the company’s advisory board.
“I believe that we will be ready for a phase I study by the end of 2012,” says Ovadia, with an eye towards bringing a product to market within five years. It sounds like a long time, but considering how few options there have been for ALS sufferers until now, Immunity Pharma’s treatment promises to have a major impact on the lives of those affected. Of course, much of the future research depends on funding. “We have conducted tests on hundreds of mice, and they are expensive – with each ALS mouse costing almost $400,” Ovadia says.
But Ovadia, a businessman who has been in top management positions in several hi-tech and medical innovation companies, says that helping Immunity Pharma develop an ALS treatment could be just the thing for a smart investor. “The average chance of a drug reaching market starting a phase I study is one in five, and it’s one in three after a phase I study has been completed,” says Ovadia. “That’s far better odds than the chances for a tech startup, which has a 1 in 10 chance of making it businesswise.” Investing in Immunity Pharma will pay off not only for patients, says Ovadia – but for investors, too!